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he complications of the little boys genetic skin disease grew as he did. Tiny blisters had covered his back as a newborn. Then came the chronic skin wounds that extended from his buttocks down to his legs.

By June 2015, at age 7, the boy had lost nearly two-thirds of his skin due to an infection related to the genetic disorder junctional epidermolysis bullosa, which causes the skin to become extremely fragile. Theres no cure for the disease, and it is often fatal for kids. At the burn unit at Childrens Hospital in Bochum, Germany, doctors offered him constant morphine and bandaged much of his body, but nothing not even his fathers offer to donate his skin worked to heal his wounds.

We were absolutely sure we could do nothing for this kid, Dr. Tobias Rothoeft, a pediatrician with Childrens Hospital in Bochum, which is affiliated with Ruhr University. [We thought] that he would die.

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As a last-ditch effort, the boys father asked if there were any available experimental treatments. The German doctors reached out to Dr. Michele De Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia, to see if a transplant of genetically modified skin cells might be possible. De Luca knew the odds were against them such a transplant had only been performed twice in the past, and never on such a large portion of the body. But he said yes.

The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boys body by grafting on genetically modified cells taken from the boys healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases.

To cultivate replacement skin, the medical team took a biopsy the size of a matchbook from the boys healthy skin and sent it to De Lucas team in Italy. There, researchers cloned the skin cells and genetically modified them to have a healthy version of the gene LAMB3, responsible for making the protein laminin-332. They grew the corrected cultures into sheets, which they sent back to Germany. Then, over a series of three operations between October 2015 and January 2016, the surgical team attached the sheets on different parts of the boys body.

The gene-repaired skin took, and spread. Within just a month the wounds were islands within intact skin. The boy was sent home from the hospital in February 2016, and over the next 21 months, researchers said his skin healed normally. Unlike burn patients whose skin grafts arent created from genetically modified cells the boy wont need ointment for his skin and can regrow his hair.

And unlike simple grafts of skin from one body part to another, we had the opportunity to reproduce as much as those cells as we want, said plastic surgeon Dr. Tobias Hirsch, one of the studys authors. You can have double the whole body surface or even more. Thats a fantastic option for a surgeon to treat this child.

Dr. John Wagner, the director of the University of Minnesota Masonic Childrens Hospitals blood and marrow transplant program, told STAT the findings have extraordinary potential because, until now, the only stem cell transplants proven to work in humans was of hematopoietic stem cells those in blood and bone marrow.

Theyve proven that a stem cell is engraftable, Wagner said. In humans, what we have to demonstrate is that a parent cell is able to reproduce or self-renew, and differentiate into certain cell populations for that particular organ. This is the first indication that theres another stem cell population [beyond hematopoietic stem cells] thats able to do that.

The researchers said the aggressive treatment outlined in the study necessary in the case of the 7-year-old patient could eventually help other patients in less critical condition. One possibility, they noted in the paper, was to bank skin samples from infants with JEB before they develop symptoms. These could then be used to treat skin lesions as they develop rather than after they become life-threatening.

The treatment might be more effective in children, whose stem cells have higher renewal potential and who have less total skin to replace, than in adults, Mariaceleste Aragona and Cdric Blanpain, stem cell researchers with the Free University of Brussels, wrote in an accompanying commentary for Nature.

But De Luca said more research must be conducted to see if the methods could be applied beyond this specific genetic disease. His group is currently running a pair of clinical trials in Austria using genetically modified skin stem cells to treat another 12 patients with two different kinds of epidermolysis bullosa, including JEB.

For the 7-year-old boy, life has become more normal now that it ever was before, the researchers said. Hes off pain meds. While he has some small blisters in areas that didnt receive a transplant, they havent stopped him from going to school, playing soccer, or behaving like a healthy child.

The kid is doing quite well. If he gets bruises like small kids [do], they just heal as normal skin heals, Rothoeft said. Hes quite healthy.

Southern Correspondent

Max covers hospitals and health care.

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'Extraordinary' tale: Stem cells heal a young boy's lethal ...

Scientists reported Wednesday that they genetically modified stem cells to grow skin that they successfully grafted over nearly all of a child's body - a remarkable achievement that could revolutionize treatment of burn victims and people with skin diseases.

The research, published in the journal Nature, involved a 7-year-old boy who suffers from a genetic disease known as junctional epidermolysis bullosa (JEB) that makes skin so fragile that minor friction such as rubbing causes the skin to blister or come apart.

By the time the boy arrived at Children's Hospital of Ruhr-University in Germany in 2015, he was gravely ill. Doctors noted that he had "complete epidural loss" on about 60 percent of his body surface area, was in so much pain that he was on morphine, and fighting off a systemic staph infection. The doctors tried everything they could think of: Antibiotics, changing dressings, grafting skin donated by his father. But nothing worked, and they told his parents to prepare for the worst.

"We had a lot of problems in the first days keeping this kid alive," Tobias Hirsch, one of the treating physicians, recalled in a conference call with reporters this week.

Hirsch and his colleague Tobias Rothoeft began to scour the medical literature for anything that might help and came across an article describing a highly experimental procedure to genetically engineer skin cells. They contacted the author, Michele De Luca, of the Center for Regenerative Medicine University of Modena and Reggio Emilia in Italy. De Luca flew out right away.

Using a technique he had used only twice before and even then only on small parts of the body, De Luca harvested cells from a four-square-centimeter patch of skin on an unaffected part of the boy's body and brought them into the lab. There, he genetically modified them so that they no longer contained the mutated form of a gene known to cause the disease and grew the cells into patches of genetically modified epidermis. They discovered, the researchers reported, that "the human epidermis is sustained by a limited number of long-lived stem cells which are able to extensively self-renew."

In three surgeries, the child's doctors took that lab-grown skin and used it to cover nearly 80 percent of the boy's body - mostly on the limbs and on his back, which had suffered the most damage. The procedure was permitted under a "compassionate use" exception that allows researchers under certain dire circumstances to make a treatment available even though it is not approved by regulators for general use. Then, over the course of the next eight months while the child was in the intensive care unit, they watched and waited.

The boy's recovery was stunning.

The regenerated epidermis "firmly adhered to the underlying dermis," the researchers reported. Hair follicles grew out of some areas. And even bumps and bruises healed normally. Unlike traditional skin grafts that require ointment once or twice a day to remain functional, the boy's new skin was fine with the normal amount of washing and moisturizing.

"The epidermis looks basically normal. There is no big difference," De Luca said. He said he expects the skin to last "basically the life of the patient."

In an analysis accompanying the main article in Nature, Mariacelest Aragona and Cedric Blanpain wrote that this therapy appears to be one of the few examples of truly effective stem-cell therapies. The study "demonstrates the feasibility and safety of replacing the entire epidermis using combined stem-cell and gene therapy," and also provides important insights into how different types of cells work together to help our skin renews itself.

They said there are still many other lingering questions, including whether such procedures might work better in children than adults and whether there would be longer-term adverse consequences, such as the development of cancer.

There are also many challenges to translating this research to treating wounds sustained in fires or other violent ways. In the skin disease that was treated in the boy, the epidermis is damaged but the layer beneath it, the dermis, is intact. The dermis is what the researchers called an ideal receiving bed for the lab-grown skin. But if deeper layers of the skin are burned or torn off, it's possible that the artificial skin would not adhere as well.

"No matter how you prepare, it's a bad situation," De Luca said. For the time being, he says he's continuing to study the procedure in two clinical trials that involve genetic diseases.

Meanwhile, Hirsch and Rothoeft report that the boy is continuing to do well and is not on any medication for the first time in many years. Doctors are carefully monitoring the child for any signs that there may be some cells that were not corrected and that the disease may re-emerge, but right now that does not appear to be happening in the transplanted areas. However, the child does have some blistering in about 2 to 3 percent of his body in non-grafted areas and they are considering whether to replace that skin as well.

But for now, they are giving the boy time to be a boy, Rothoeft said: "The kid is now back to school and plays soccer and spends other days with the children."

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Genetically modified skin grown from stem cells saved a 7 ...

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(CNN) - For the first time, doctors were able to treat a child who had a life-threatening rare genetic skin disease through a transplant of skin grown using genetically modified stem cells.

The grafts replaced 80% of the boy's skin.

The skin of his arms, legs, back and flanks, and some of the skin on his stomach, neck and face was missing or severely affected due to epidermolysis bullosa.

The compassionate-use experimental treatment is detailed in a case study published in the journal Nature on Wednesday.

Skin as fragile as a butterfly's wings -- that's how children with epidermolysis bullosa are described and why they're often called butterfly children.

The disease, of which there are five major types and at least 31 subtypes, is incurable. People with the condition have a defect in the protein-forming genes necessary for skin regeneration.

About 500,000 people worldwide are affected by forms of the disease. More than 40% of patients die before reaching adolescence.

Their skin can blister and erode due to something as simple as bumping into something or even the light friction of clothing, according to an email from Dr. Jouni Uitto, a professor and chairman of the Department of Dermatology and Cutaneous Biology at the Sidney Kimmel Medical College in Philadelphia. Uitto was not involved with this study.

Epidermolysis bullosa makes the skin incredibly susceptible to infections, and in the case of 7-year-old Hassan, whose treatment was detailed in Nature, those infections can be life-threatening.

A week after he was born in Syria, Hassan had a blister on his back, his father said through an interpreter in an interview provided by the hospital in Germany where the boy was treated.

Hassan's last name, as well as the first names of his family members, are not being disclosed to protect the privacy of the family.

In his first few weeks of life, Hassan was immediately diagnosed with epidermolysis bullosa, and their doctor in Syria told Hassan's family that there was no cure or therapy.

Over the years, their efforts to find help for their son's disease led the family to the Muenster University hospital in Germany in 2015, when Hassan was 7. His condition worsened, and he struggled with severe sepsis and a high fever. He weighed just over 37 pounds.

They didn't think he would make it, and doctors at Muenster decided in summer 2015 to transfer Hassan to the Ruhr-Universitt Bochum's University Hospitals, including the burn center -- one of the oldest in the country.

By the time Hassan arrived at Bochum, he had lost two-thirds of his surface skin.

"We had a lot of problems in first days just keeping him alive," said Dr. Tobias Rothoeft, consultant at the University Children's Hospital at Katholisches Klinikum Bochum.

Doctors tried to promote healing by changing his dressings and treating him with antibiotics, as well as putting him on an aggressive nutrition schedule, but nothing helped. They even tried transplanting skin from Hassan's father.

"By that time, he had lost 60% of his epidermis, the upper skin layer, and had 60% open wounds all over his body," said Dr. Maximilian Kueckelhaus of the Department of Plastic Surgery at Bochum's Burn Center.

Every approach failed, so the doctors prepared Hassan's family for what end-of-life care would entail. But the parents pleaded, asking the doctors to consult studies and research for experimental treatments that might help.

They found Dr. Michele De Luca at the University of Modena's Center for Regenerative Medicine in Italy. His publications described an experimental treatment transplanting genetically modified epidermal stem cells that healed small, non-life-threatening wounds in adults.

The medical team reached out to De Luca, asking whether he could help them replicate the procedure on a larger scale to help Hassan, and he agreed. De Luca told Hassan's parents that he believed there was a 50% chance of the treatment being successful.

They were more than willing to accept the risk, to do anything to help their son have a chance at a normal life.

Hassan "was in severe pain and was asking a lot of questions: 'Why do I suffer from this disease? Why do I have to live this life? All children can run around and play. Why am I not allowed to play soccer?' I couldn't answer these questions," his father said. "It was a tough decision for us, but we wanted to try for Hassan."

To obtain the skin's stem cells, the doctors took a small biopsy -- only accounting for 1 square inches -- from an unaffected part of Hassan's skin. The stem cells were processed by De Luca in Italy. A healthy version of the gene that is normally defective in epidermolysis bullosa patients was added to the cells, along with retroviral vectors: virus particles that assist the gene transfer.

This genetic transfer would essentially "correct" the cells.

The single cells were grown and cultivated on plastic and fibrin substrate, which is used to treat large skin burns, to form a large piece of epidermis. This method enabled the researchers to grow as much skin as they needed. The whole process took three to four weeks, Kueckelhaus said.

Once the sheets were ready, they were transferred from Italy to Germany and transplanted onto the well-cleaned wounds right away during two surgeries. The first procedure in October 2015 applied the sheets to Hassan's arms and legs. The second surgery, in November, grafted the sheets to Hassan's entire back and the other affected areas.

Hassan began to improve immediately. The researchers noticed that the grafts were not rejected; they bound to all of the areas they were transplanted.

"For everyone that was involved, taking off the bandages and seeing for the first time that this is working out, that the transplants are actually attached to the patient and growing skin, that's an incredible moment," Kueckelhaus said.

Hassan was discharged from the hospital in February 2016.

After steady followups over 21 months, the researchers found that Hassan's new skin healed normally, didn't blister anymore, and was resistant to stress. It was even growing hair. Unlike some skin graft patients, he doesn't require any ointment to keep his skin smooth and hydrated. And like any growing kid, he bruises and recovers normally.

They also learned that only a few stem cells contribute to the long-term maintenance of the epidermis, shedding light on cellular hierarchy in this regard.

"The investigators removed of small piece of patient's skin, isolated cells with stem cell potential for growth, introduced a normal copy of the mutated gene to the cells, propagated a large number of these cells in culture and then grafted them back to the skin," Uitto said. "This concept is not new, but what is remarkable here is that they were able to change essentially the entire skin of the patient with normal cells."

Hassan's family is currently living in Germany. Hassan, now 9, is able to go to school and play sports, but he maintains a schedule of frequent monitoring at the hospital to ensure that the initial success of the treatment continues. The area of his skin that was not treated sometimes shows small blisters, and if it worsens, he may receive transplants there as well.

"Seeing him 18 months after the initial surgery with an intact skin is incredible because he has been in the ICU for so long," Kueckelhaus said. "He had bandages all over his body except his hands, feet and face. He was on extremely strong pain medication. So the quality of life was really, really bad for him. Seeing him play soccer, play sports, play with other kids, that is just amazing because that's something he couldn't do before."

"It felt like a dream for us," the boy's father said. "Hassan feels like a normal person now. He plays. He's being active. He loves life."

Everything points to a good long-term outcome for Hassan.

The researchers will continue to monitor him for complications. Sometimes, genetic modifications can cause malignancies in cells.

"That is of course one thing we really have to be aware of," Kueckelhaus said. "However, analyzing the integration profile of that gene into the boy's DNA, which we did, we saw that it's mostly in areas that don't cause too much concern about developing malignancies."

Epidermolysis bullosa patients can be at a very high risk of developing skin cancer simply because of the disease. Because Hassan now has intact skin and intact DNA, this risk might even decrease, but that will have to be proved through follow-up, Kueckelhaus said.

Given that this was one successful outcome for one patient, the experimental treatment can't be applied for other patients just yet. De Luca is conducting clinical trials using the treatment.

"This is one case with a distinct type of EB, and further studies will show whether this approach is applicable to other forms of EB as well," Uitto said. "It should be noted that in some severe forms of EB, the patients also suffer from fragility of the gastrointestinal and vesico-urinary tract, and some forms are associated with the development of muscular dystrophy. Obviously, gene therapy of the skin cannot correct them, and these issues have to be addressed in further studies."

Hassan's treatment also cost hundreds of thousands of dollars. Although the process could be optimized, doctors would still have to individually grow transplants for each patient, which could get very expensive.

But for patients' families, epidermolysis bullosa is already expensive.

"Standard maintenance treatment of patients with EB, including daily bandaging, antibiotics and special moisturizer, as well as frequent hospitalizations, can be extremely costly, and gene correction as described in this paper may well be cost-effective over the lifetime of these patients," Uitto noted.

Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association of America, has a 10-year-old daughter, Rafi, with recessive dystrophic EB. Between January and August, $751,1778 for wound/burn dressings was charged to Kopelan's insurance company, he says. That doesn't account for drugs or hospital visits and surgeries.

Kopelan's nonprofit sends free supplies and bandages to families. The nonprofit can provide its employees with insurance that covers the medical equipment, but that isn't the case for everyone impacted by the condition, he said.

Kopelan is hopeful about the results of the study. The baths and bandage changes that are necessary for epidermolysis bullosa patients to stave off life-threatening infections can last hours and feel torturous.

"Do you remember the last time you got a paper cut and put Purell on it? It burned, right? Now think of 60% of body being an open wound, and opioids don't really work for this kind of pain," Kopelan wrote in an email. "This is what make EB kids and adults the strongest people on Earth."

The study "confirms our hopes that gene therapy is potentially the most efficacious path forward to providing a significant treatment option for those with epidermolysis bullosa," Kopelan said. "While it's important to remember that this is only one patient and more work needs to be done to demonstrate how effective this gene therapy platform may prove to be, I am very enthused."

"I wish that all children with the same disease could be treated in this way," Hassan's father said.

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Scientists replace skin using genetically modified stem cells

If you or a loved one will be having a bone marrow transplant or donating stem cells, what does it entail? What are the different types of bone marrow transplants and what is the experience like for both the donor and recipient?

A bone marrow transplant is a procedure in which when special cells (called stem cells) are removed from the bone marrow or peripheral blood, filtered and given back either to the same person or to another person.

Since we now derive most stem cells needed from the blood rather than the bone marrow, a bone marrow transplant is now more commonly referred to as stem cell transplant.

Bone marrow is found in larger bones in the body such as the pelvic bones. This bone marrow is the manufacturing site for stem cells. Stem cells are "pluripotential" meaning that the cells are the precursor cells which can evolve into the different types of blood cells, such as white blood cells, red blood cells, and platelets.

If something is wrong with the bone marrow or the production of blood cells is decreased, a person can become very ill or die. In conditions such as aplastic anemia, the bone marrow stops producing blood cells needed for the body. In diseases such as leukemia, the bone marrow produces abnormal blood cells.

The purpose of a bone marrow transplant is thus to replace cells not being produced or replace unhealthy stem cells with healthy ones.

This can be used to treat or even cure the disease.

In addition for leukemias, lymphomas, and aplastic anemia, stem cell transplants are being evaluated for many disorders, ranging from solid tumors to other non-malignant disorders of the bone marrow, to multiple sclerosis.

There are two primary types of bone marrow transplants, autologous and allogeneic transplants.

The Greek prefix "auto" means "self." In an autologous transplant, the donor is the person who will also receive the transplant. This procedure, also known as a "rescue transplant" involves removing your stem cells and freezing them. You then receive high dose chemotherapy followed by infusion of the thawed out frozen stem cells. It may be used to treat leukemias, lymphomas, or multiple myeloma.

The Greek prefix "allo" means "different" or "other." In an allogeneic bone marrow transplant, the donor is another person who has a genetic tissue type similar to the person needing the transplant. Because tissue types are inherited, similar to hair color or eye color, it is more likely that you will find a suitable donor in a family member, especially a sibling. Unfortunately, this occurs only 25 to 30 percent of the time.

If a family member does not match the recipient, the National Marrow Donor Program Registry database can be searched for an unrelated individual whose tissue type is a close match. It is more likely that a donor who comes from the same racial or ethnic group as the recipient will have the same tissue traits.

Learn more about finding a donor for a stem cell transplant.

Bone marrow cells can be obtained in three primary ways. These include:

The majority of stem cell transplants are done using PBSC collected by apheresis (peripheral blood stem cell transplants.) This method appears to provide better results for both the donor and recipient. There still may be situations in which a traditional bone marrow harvest is done.

Donating stem cells or bone marrow is fairly easy. In most cases, a donation is made using circulating stem cells (PBSC) collected by apheresis. First, the donor receives injections of a medication for several days that causes stem cells to move out of the bone marrow and into the blood. For the stem cell collection, the donor is connected to a machine by a needle inserted in the vein (like for donating blood.) Blood is taken from the vein, filtered by the machine to collect the stem cells, then returned back to the donor through a needle in the other arm. There is almost no need for a recovery time with this procedure.

If stem cells are collected by bone marrow harvest (much less likely,) the donor will go to the operating room and while asleep under anesthesia, a needle will be inserted into either the hip or the breastbone to take out some bone marrow. After awakening, there may be some pain where the needle was inserted.

A bone marrow transplant can be a very challenging procedure for the recipient.

The first step is usually receiving high doses of chemotherapy and/or radiation to eliminate whatever bone marrow is present. For example, with leukemia, it is first important to remove all of the abnormal bone marrow cells.

Once a person's original bone marrow is destroyed, the new stem cells are injected intravenously, similar to a blood transfusion. The stem cells then find their way to the bone and start to grow and produce more cells (called engraftment.)

There are many potential complications. The most critical time is usually when the bone marrow is destroyed so that few blood cells remain. Destruction of the bone marrow results in greatly reduced numbers of all of the types of blood cells (pancytopenia.) Without white blood cells there is a serious risk of infection, and infection precautions are used in the hospital (isolation.) Low levels of red blood cells (anemia) often require blood transfusions while waiting for the new stem cells to begin growing. Low levels of platelets (thrombocytopenia) in the blood can lead to internal bleeding.

A common complication affecting 40 to 80 percent of recipients is graft versus host disease. This occurs when white blood cells (T cells) in the donated cells (graft) attack tissues in the recipient (the host,) and can be life-threatening.

An alternative approach referred to as a non-myeloablative bone marrow transplant or "mini-bone marrow transplant" is somewhat different. In this procedure, lower doses of chemotherapy are given that do not completely wipe out or "ablate" the bone marrow as in a typical bone marrow transplant. This approach may be used for someone who is older or otherwise might not tolerate the traditional procedure. In this case, the transplant works differently to treat the disease as well. Instead of replacing the bone marrow, the donated marrow can attack cancerous cells left in the body in a process referred to as "graft versus malignancy."

If you'd like to become a volunteer donor, the process is straightforward and simple. Anyone between the ages of 18 and 60 and in good health can become a donor. There is a form to fill out and a blood sample to give; you can find all the information you need at the National Marrow Donor Program Web site. You can join a donor drive in your area or go to a local Donor Center to have the blood test done.

When a person volunteers to be a donor, his or her particular blood tissue traits, as determined by a special blood test (histocompatibility antigen test,) are recorded in the Registry. This "tissue typing" is different from a person's A, B, or O blood type. The Registry record also contains contact information for the donor, should a tissue type match be made.

Bone marrow transplants can be either autologous (from yourself) or allogeneic (from another person.) Stem cells are obtained either from peripheral blood, a bone marrow harvest or from cord blood that is saved at birth.

For a donor, the process is relatively easy. For the recipient, it can be a long and difficult process, especially when high doses of chemotherapy are needed to eliminate bone marrow. Complications are common and can include infections, bleeding, and graft versus host disease among others.

That said, bone marrow transplants can treat and even cure some diseases which had previously been almost uniformly fatal. While finding a donor was more challenging in the past, the National Marrow Donor Program has expanded such that many people without a compatible family member are now able to have a bone marrow/stem cell transplant.

Sources:

American Society of Clinical Oncology. Cancer.Net. What is a Stem Cell Transplant (Bone Marrow Transplant)? Updated 01/16. http://www.cancer.net/navigating-cancer-care/how-cancer-treated/bone-marrowstem-cell-transplantation/what-stem-cell-transplant-bone-marrow-transplant

U.S. National Library of Medicine. MedlinePlus. Bone Marrow Transplant. Updated 10/03/17. https://medlineplus.gov/ency/article/003009.htm

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bone marrow/stem cell transplant - verywell.com

The following was written withProf. Gerold Riempp, a professor of information systems who was diagnosed with Parkinsons disease 16 years ago at age 36. He is co-founder of a charitable organization in Germany that supports the development of therapies that aim to cure PD.

The idea behind cell replacement therapy(CRT) for PD is pretty simple: lack of mobility in PD is the result of the dysfunction and death of a specific kind of cell in the midbrain. While there are a few other things that go wrong in PD, the progressive loss of motor skills is the biggest problem most diagnosed face. Since we are reasonably sure that this lack of mobility results from the impairment and death of dopamine producing cells in an area of the midbrain called the substantia nigra,why not try to replace those cells?

A group of iPS cells grown from human skin tissue at Osaka University

Replacing those cells is one of three core problems that each person diagnosed with PD needs to address. They are:

1. Keeping remaining cells healthyOnce diagnosed, most people have already lost production of 50-80% of dopamine in their midbrain. The problem then is to stop further disease progression by figuring out how to get rid of everything that might be harming the remaining 20-50% of cells while giving their body everything it needs to keep those cells alive and active.

2. Clearing clogged cellsOf those 50-80% of non-dopamine producing cells, a portion are still alive, they are just not doing their job, producing dopamine. This impairment is a result of a range of interrelated factors that harm the cells and eventually lead to their death. Most researchers believe the problem can be boiled down to the clumping of a misfolded protein called alpha-synuclein. Many different methods are being tried in labs around the world to clear these clumps and stop more from accumulating. But this might only be part of the story since a wide variety of other factors also lead to cell death.

3. Replacing dead cellsThen we come to what to do about all of those dead cells. A couple of different options are being considered to get the brain tostimulate the production of new neurons orreplace the function of dead ones. However, the most promising therapy being developed is stem cell therapy, now commonly referred to as cell replacement therapy. It works by placing new dopamine producing neurons into the part of the brain where the dead neurons used to release dopamine.

If a patient manages to address problems one and two they might have no need for CRT. The reason for this is that he or she can likely rescue a considerable portion of the damaged but still living cells and thereby bring dopamine production back to a level that allows for normal movement. CRT will generally be for people who have had PD for a longer time and whose remaining healthy cells plus the rescued ones together are not capable of providing enough dopamine.

The late 80s and 90s saw a number of CRT trials for Parkinsons disease with mixed results. But we nowhave a much better understanding of what kind of cells to use, how to culture and store those cells, how to implant them, and who this therapy would be best for.

We also now have iPS cells (induced pluripotent stem cells). Discovered in 2006, these are cells that have been chemically reprogrammed, usually from adult skin tissue, back into pluripotent stem cells. (Pluripotent means they are capable of becoming almost any cell in the body). Using these cells for transplantation has two major advantages. One, it eliminates the need for potentially harmful immuno-suppressors. Two, it has none of the ethical issues that come with using fetal stem cells. But iPS cells are much more expensive and technically difficult to produce.

Despite all the progress made, cell replacement therapy is still very controversial and fraught with all sorts of technical issues. Luckily, CRT for PD is one of the only fields of medical science where the top labs around the world are cooperating with each other. An international consortium of labs has come together under a name that sounds like it was ripped out of a Marvel comic, the GForce-PD. Each lab in the GForce-PD aims to bring CRT for PD to clinical trial within the next few years.

Infographic made by PhD neuroscientist Kayleen Schreiber at kayleenschreiber.com

The GForce-PD

New York City Run by Dr. Lorenz Studer out of the Rockefeller research labs in New York City. Dr. Studer pioneered many of the reprogramming techniques being used around the world to convert pluripotent stem cells into dopamine producing neurons. His lab wasrecently announced to be part of a huge funding initiative from Bayer Pharmaceuticals to help speed up development of CRT. Studers lab is aiming to start transplantation of embryonic stem cells in human trials in early 2018.

Kyoto, Japan Dr. Jun Takahashis lab in Kyoto is working on producing several iPS lines for the Japanese population. One advantage they have is the relative homogeneity of Japanese people allows them to use a dozen or so iPS lines for almost everyone in the country. The lab recently made headlines with results from monkey trials that showed human iPS cells graft safely, with no signs of malignant growth, two years after transplantation.

Cambridge, England Dr. Roger Barkers lab has been working on cell replacement therapy for Parkinsons disease for a number of years through the Transeuro project. His lab is pushing forward with more embryonic stem cell transplantations expected to begin in 2020. They also work very closely with the team in Sweden.

Lund, Sweden The lab in Lund has been working on CRT for PD since the 80s and has been part of a number of human trials. The lab is now run by Dr. Malin Parmar whose team has also pioneered many of the techniques used in direct programming that will one day allow researchers to skip the stem cell phase all together and produce dopamine cells directly in the brain.

San Diego, California The team is moving rapidly towards iPS cell transplantation under Dr. Jeanne Loring at the Scripps research center. They are the only lab that uses patients own cells for transplantation. Another unique feature of this lab is that it has been a community funded initiative under theSummit For Stem Cellsfoundation.

(Dr. Roger Barker talking about CRT for PD)

Though there is a lot of excitement building around cell replacement therapy, we need to proceed carefully. The field has potential for setbacks from some of the less rigorous trials being conducted in places like Australia and China where regulatory standards are more lax. Researchers in these areas are already going ahead with trials that do not meet the standards set by the GForce-PD. These have the potential to put a black-eye on all cell replacement therapies.

Also, producing pure batches of dopamine neurons is still a highly technical process that only a few labs in the world are capable of doing safely and effectively. Thankfully a few other labs around the world are joining the efforts of the GForce-PD, such as Dr. Tilo Kunaths lab in Edinburgh, which is working on techniques to better differentiate and characterize the cell lines used for transplantation.

(The pictures above show human embryonic stem cells being differentiated into dopamine cells at days 2, 4 and 7. Courtesy of Dr. Tilo Kunaths lab at the University of Edinburgh)

The Future of Cell Replacement Therapy

These therapies being developed for Parkinsons disease will, in essence, be version 1.0 of CRT. Clinical trials are set to begin next year and the therapy is expected to be widely available to people diagnosed with Parkinsons disease within the next 5-10 years.

Version 2.0 will be CRISPR-modified, disease resistant grafts, with genetic switches to modulate dopamine production and graft size.

Version 3.0 will make use of an emerging field called in vivo direct programming where viruses are inserted into the brain and transform other existing cells into dopamine producing cells.

(Edit: Credit to Dr. Tilo Kunath for correcting versions 2.0 and 3.0)

Dopamine neurons grown from iPS cells at 40 times magnification, from the Gladstone Institute

CRT for PD is one of the most exciting areas of research on the planet. It is a powerful demonstration of the progress we as a species have made in our attempt to gain mastery over the forces of biology.It has the potential to improve the lives of the millions living with PD, and the millions yet to be diagnosed. Once the transplanted cells have connected with their surroundings and start delivering dopamine to the right places, it should allow patients to gradually reduce their medication. Being able to move normally and not deal with the side effects of all the drugs and other therapies is what PD patients around the world are dreaming of.

Click here for more information on the future of cell replacement therapy for Parkinsons disease and the work of the GForce-PD.

And if you want to be part of bringing CRT to the clinic you can do so by supporting organizations like Summit For Stem Cells.

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Cell Replacement Therapy For Parkinsons Disease And The ...

Stem cells (magenta with green nuclei) encapsulated in nanogel (yellow) for heart repair. Credit: MolGraphics.com

Cardiac stem cell therapy is a promising treatment for damaged hearts. However, researchers are still working on two major issues with the therapy how to keep the stem cells in place and how to prevent rejection when the stem cells are not from the patients own body. A new approach from NC State researcher Ke Cheng and a team of international collaborators may solve both of these problems.

The heart is a powerful muscle. Thats great if youre running a marathon, but if youre trying to inject stem cells into the heart with the hopes that theyll stay put, its a problem. One of the major drawbacks of cardiac stem cell therapy is simply that the cells do not stick to the injured heart tissue.

Enter a thermosensitive nanogel that is liquid at room temperature but becomes a thick, sticky gel as it warms. Cheng, associate professor of molecular biomedical sciences at NC States College of Veterinary Medicine and associate professor in the NC State/UNC Joint Department of Biomedical Engineering, partnered with chemical engineers from the University of Adelaide and cardiac specialists from the University of Zhengzhou and UNC-Chapel Hill to test the nanogel as a delivery mechanism for cardiac stem cells.

The nanogel, poly(N-isopropylacrylamine-co-acrylic acid), or P(NIPAM-AA) for short, had another property that made it appealing for use: in its thickened state it had porous openings large enough for a stem cells healing factors to escape, but not large enough for immune cells to enter. And it could be adjusted to slowly degrade over time, giving stem cells enough time to repair a damaged heart before dissolving away.

Autologous stem cells grown from a patients own cells are ideal to use in therapies, but that isnt always practical, Cheng says. For one thing, growing the cells takes time, which a patient may not have. For another, the heart cells themselves may be affected by disease, so stem cells taken from that source would not be useful.

Thats why were working on allogeneic stem cell therapies, but whenever you introduce cells from an outside source into the body, the immune system will attack them. The nanogel delivery method keeps the cells in place, protects them from the bodys immune response, and allows the regenerative factors released by the stem cells to reach the heart.

Cheng and his collaborators tested the nanogel delivery system in mice and pigs with hearts damaged by a heart attack. Without the nanogel, only about one percent of injected stem cells stayed in the heart. With the gel, up to 15 percent of the stem cells stayed put. They also found that in both animal models heart function improved three to four weeks after treatment. Mice showed a greater improvement than pigs, but in both models heart function was maintained and did not decrease.

We are pleased with these results, Cheng says. The nanogel is a safe, cost-effective way to deliver the cells directly to the affected area, and the large animal (pig) data is promising, which may lead to a human clinical trial in the future.

Chengs work appears in the journal ACS Nano, and was supported by the NIH and by NC States Chancellors Faculty Excellence Program and Chancellors Innovation Fund.

Continued here:
Stuck on You: Nanogel Capsule Helps Cardiac Stem Cells ...

Stem cell therapy is a two-step process. First, the patients blood cells are destroyed by chemotherapy, radiation therapy or immunosuppression. This conditioning process also eradicates any cancer cells that survived first-line treatment. Second, the patient receives stem cells harvested from a donors bone marrow or peripheral blood (circulating blood). While this can be an effective cure, it can cause graft-versus-host disease (GVHD) in up to 50% of patients. GVHD is more likely to develop in patients who have received a peripheral blood transplant and can kill 15%-20% of patients.

Two types of GVHD can develop, acute and chronic, and patients may develop either one, both or neither type. GVHD is less likely to occur and symptoms are milder if the donor cells closely match those of the patient. Acute GVHD can develop within 100 days of a transplant. The first step of stem cell therapy can cause tissue damage, and bacteria from the gut can escape into the bloodstream. This primes the patients antigen-presenting cells (cells that activate the immune response), which subsequently encourage donor T cells to proliferate and attack the patients tissues. Symptoms include vomiting, diarrhea, skin rashes, nausea, vomiting and liver problems. This can be resolved relatively quickly in one third of patients using immunosuppressive treatments, but some patients can progress to chronic GVHD.

The biological mechanisms responsible for chronic GVHD are not completely understood, but scientists believe that other immune system cells from the donor (B cells and macrophages) are stimulated and damage the patients tissues. Symptoms include dry eyes, mouth sores, muscle weakness, fatigue and joint problems.

Unfortunately, development of effective treatments for GVHD is not keeping up with the increasing number of GVHD patients or with advances in understanding this disease. At present, standard treatments include corticosteroids and drugs that reduce IL-2, an immune system chemical that helps T-cells multiply and diversify. These treatments have various side effects including suppressing the patients immune system, thereby increasing risk of infection.

One challenge stalling drug research is that a small degree of graft-versus-host response must occur for successful stem cell therapy: donor cells will destroy any cancer cells that remain after the first stage of therapy. This challenge is discussed in a recent article in Science Health.Although several treatments have been trialed, success is variable and often targets only acute GVHD or chronic GVHD. Biomarkers have also been detected that may help identify individuals at risk of developing severe GVHD, information that may aid the development of personalized treatment strategies. Drugs that have been approved for other diseases, but not for GVHD, show promise and include ibrutinib for chronic GVHD (approved for specific blood cancers) and ruxolitinib for acute GVHD (approved for bone marrow disorders).

The impact of stem cell therapy must not be underestimated: up to 50% of recipients will develop GVHD. Unfortunately, some individuals will develop chronic GVHD, a condition that is just as difficult to survive as cancer. This highlights the importance of developing continued care strategies for individuals receiving stem cell therapy as a final defence against cancer.

Written byNatasha Tetlow, PhD

Reference: Cohen J. A stem cell transplant helped beat back a young doctors cancer. Now, its assaulting his body. Science Health. 2017. Available at: DOI: 10.1126/science.aan7079

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Stem Cell Therapy: A Lethal Cure - Medical News Bulletin

On Saturday morning, a convoy of vintage Ford Broncos carrying some very precious cargo made a stop at La Caadas Descanso Gardens.

En route from Childrens Hospital Los Angeles, the motorcade was led by a golden 1971 Bronco with Thousand Oaks resident Tyler Kelly at the helm. Tucked safely into a car seat in the back was 2-year-old Pierce Kelly, known by family and friends as Fierce Pierce, still recovering from a July 21 bone marrow transplant.

At the La Caada home of relatives Donna and Dave McLaughlin, Pierce will recuperate under the watchful eye of mom Aubrey. For 100 days following the procedure, he must reside within a 30-minute drive of Childrens Hospital for monitoring.

Saturdays 13-mile drive was just one portion of a tumultuous journey the Kellys have been on since April 7, when Pierce was diagnosed with acute myeloid leukemia. His chance of surviving the devastating illness with treatment alone was only 50%, according to mom Aubrey. But there was one hope if the Kellys could find a bone marrow donor, Pierces odds would improve by at least another 15%.

We were at the mercy of whoever had registered, Aubrey Kelly recalled.

Among the nearly 13.5 million Americans already listed as donors on the Be the Match Marrow Registry, there were no donors close enough to be a match with Pierce.

Raquel Edpao, a community outreach specialist for Be the Match, said on any given day there are 14,000 people like the Kellys, searching registries for a bone marrow match. Its her job to help educate people how simple it is to join the registry and to donate if called.

Potential donors register online at bethematch.org, then receive and turn in a cheek swab. After that, theyre contacted if they are a potential match for someone. Edpao estimates about one out of every 430 registrants will be asked to donate.

There are so many misconceptions about donating, she said, invoking myths about spinal drilling, painful extractions and missed days at work. Its usually as simple as donating blood.

In about 20% of cases donors are asked to undergo a marrow extraction, a 45-minute outpatient procedure involving a general anesthetic.

Luckily for the Kellys, a search of donors worldwide returned a single donor in France whose human leukocyte antigen (HLA) protein was a 10-out-of-10 match with Pierces. While the marrow was shipped, the 2-year-old underwent chemotherapy to destroy most of his damaged stem cells in preparation for the donation.

Its a fine balance of leaving him with enough cells to receive the new ones, but not so many that the new cells dont have enough room to grow, Aubrey Kelly said, explaining how her sons blood type switched from A positive, his own type, to the donors O negative.

Pierces recovery from the transplant requires a sterile environment that means he cannot stay with siblings Sierra, 4, and 6-month-old Harper. Donna McLaughlin, a cousin of Aubrey Kellys dad, said she and husband Dave were happy to offer their home in La Caadas Paradise Valley neighborhood for his recovery.

Ive worked for the past week cleaning my house its never been so clean, she said of her preparation for Pierce and Aubreys 57-day visit. Im being paranoid, I know, but he is going to be OK on my watch.

Knowing he would have to return to Thousand Oaks to take care of Pierces sisters, Tyler Kelly wanted to ensure his sons trip from the hospital would be a special one. The Bronco the same vehicle his mother drove to the hospital in 1981 so he could be delivered, and the same one he and Aubrey have used to get to the delivery room in time for the birth of their own three children seemed a fitting conveyance.

We wanted to continue the tradition, he said.

Hoping to assemble a retinue for the drive, Tyler Kelly reached out to enthusiast club SoCal Broncos and classicbroncos.com. Several people responded, including Agoura Hills Bronco owner Dan Bennett, for whom the cause was personal. About 10 years ago he saved a life by donating his own bone marrow.

To be able to go in and help play an intrinsic role in saving someones life is a really special thing, Bennett said. I think everybody should do it.

sara.cardine@latimes.com

Twitter: @SaraCardine

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Convoy from Children's Hospital to La Caada carries precious cargo a 2-year-old bone marrow recipient - Los Angeles Times

The Oncology Institute of Hope Innovation welcomes Dr. Homsi to its team of specialists.

Downey, CA (PRWEB) September 08, 2017

The Oncology Institute of Hope Innovation welcomes Dr. Homsi to its team of specialists.

Dr. Yaser Homsi is a passionate and knowledgeable Hematologist that received his Medical education from the University of Aleppo in Aleppo, Syria. After graduation, Homsi moved to Indianapolis, Indiana where he completed his internship, residency and fellowship at the Indiana School of Medicine. Homsi's fellowship training included: Blood and Bone Marrow Stem Cell Transplant, Hematology and Oncology.

Dr. Homsi has completed multiple medical researches including Cellular Therapy and Hematopoietic Stem Cell Transplantation for Cancer and has published papers in various disciplines of Oncology including the Role of Angiogesis in Cancer and The Outcome of the combination of Tacrolimus, Sirolimus and ATF.

Dr. Homsi is Fluent in Arabic.

Professional Memberships:

American Society of Clinical OncologyAmerican Society of HematologyPatient Philosophy:

Dr. Homsi believes in treating each of his patients as individuals as he aids his patients and their families through their treatment plan. He believes strongly in communication and strives to clearly educate all of his patients. He and his staff make every effort to give the best treatment and care possible.

For the original version on PRWeb visit: http://www.prweb.com/releases/2017/09/prweb14669766.htm

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Dr. Yaser Homsi Joins The Oncology Institute of Hope and Innovation - Benzinga

I can see him in his glass-fronted Cambridge office from the foosball table in the light-filled central atrium. Hes standing there talking to a visitor and seems to be finishing up. This entire side of the third floor in MITs new Media Lab building is partitioned with glass, and professor Hugh Herr and his colleagues and whatever madness theyre up to in their offices and the open, gadget-filled, lower-floor lab are on display. Several people, myself included, are peering down, hoping to see a bit of magic.

Months ago, when I e-mailed Herr to propose writing an article about him, I told him about my rare bone cancer and resulting partial paralysis below the waist as a way to explain my interest in his work. Though I didnt tell him this, I also harbored a secret wish that he could help me. People write to Herr, a 52-year-old engineer and biophysicist, daily about his inspiring example. Theyve heard him promise an end to disability. They have conditions that medicine cant fix and futures they cant stand to consider. Theyre wishing for his intervention, wanting of hope. Crossing his threshold, Im the lucky one. Im here.

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Herr welcomes me into his office, a clean, well-ordered space. Theres a round glass table with a laptop on it, a handful of hard office chairs, and a pair of prosthetic legs Herr designed that are arranged like statuary behind us, one in either corner. Above us on a wall looms a large mounted photograph of another pair of prosthetics. These are hand-carved from solid ash, with vines and flowers and six-inch heels. The real-life legs were famously worn by a friend of Herrs, the amputee track-and-field athlete and actress Aimee Mullins.

I have hobbled into Herrs office with a dented $20 stock metal cane on one side and a foot-lifting Blue Rocker brace on the other. (The dent is from my recently firing the cane at the wall.) I had imagined Herr noticing the cane and asking more about my story to see how he could fix me, like he has fixed so many others. The moment I realize that the meeting Id imagined isnt the meeting were going to haveIm here as a reporter, not a friend or patient, after allI start to stammer. Herr deftly resets the conversation by suggesting we look at his computer.

On it are the PowerPoint slides of his next big project, a breathtaking $100 million, five-year proposal focused on paralysis, depression, amputation, epilepsy, and Parkinsons disease. The work will be funneled through Herrs new brainchild, MITs Center for Extreme Bionics, a team of faculty and researchers assembled in 2014 that he codirects. After exploring various interventions for each condition, Herr and his colleagues will apply to the FDA to conduct human trials. One to-be-explored intervention in the brain might, with the right molecular knobs turned, augment empathy. If we increase human empathy by 30 percent, would we still have war? Herr asks. We may not.

As he continues with the presentation hes been giving to technologists, engineers, health researchers, and potential donorslast December alone, he keynoted in Dubai, Istanbul, and Las Vegaseach revolutionary intervention he mentions yields a boyish grin and a look that affirms: Yes, you heard that right. In a talk I hear him give a few weeks later, hell dare to characterize incurable paralysis as low-hanging fruit. In his outspoken willingness to fix everything, even things that some argue should be left alone, he knows how he sounds. If half the audience is frightened and the other half is intrigued, I know Ive done a good job, he says.

Herr on a 5.12 route on Arizonas Mount Lemmon in 1986. (Beth Wald/Aurora)

Herr calmly ticks off one condition after another. He shows me an animation of an innovative surgery that will restore an amputees lost proprioception, giving a person the ability to feel and control a prosthetic as if it were their own limb. In another slide, of a paralyzed man in a bulky walk-assisting exoskeleton suit, he asks me to imagine a futuristic treatment that uses light to control cells in muscle tissue. Then he presents a video clip of a rat with a severed spinal cord dragging around its paralyzed hind legs.

Having dragged my mostly unresponsive left leg around for two years, I think I know something about the rodents life. In the next clip, however, that rat, just 90 days later, is walking on all fours. A team at the MIT center led by Herrs colleague Robert Langer successfully regrew the rats spinal cord by implanting a dissolvable scaffold seeded with neural stem cells. In Herrs world, the limbless can be whole again, the paralyzed can walk. Making the extraordinary seem ordinary is maybe the whole point.

Herr himself is proof positive. Trim, fit, and handsome, he is the showpiece for the Center for Extreme Bionics. Im kind of what theyre selling, he says. The fuss over Herr has been building for decades but reached new levels in 2014, courtesy of his TED Talk, which has now been viewed in excess of 7.3 million times. In it, Herr describes the horrific 1982 winter climbing accident in New Hampshires White Mountains during which he suffered severe frostbite, leading to the amputation of both legs below the knee. Then 17, Herr was told hed never climb again. Instead, he rebuilt himself almost immediately, willfully reshaping his artificial legs and realizing that he wasnt handicapped, the technology was.

By hacking his prosthetic devices for his vertical world, he was able to quickly return to climbing, becoming the first athletedecades before Oscar Pistoriusto blur the line between para and not. His accomplishments landed him on the cover of Outside a year after his accident, something that sticks with him not because of the many accolades other climbers bestowed on him, or even the controversy it reignited around the tragic death of one of his rescuers, but because of the questions the article raised about how far Herr would be able to go. I was a sad case. I was going to end up in this machine shop, disabled, Herr recalls of the piece, pausing to let the perceived insult ripen in his mind. Yeah, its a real sad story.

The triumphant, fully realized man in the TED Talk is a marvel. His outrage at the unnecessary suffering from disability is fiercely personal. What first-time viewers like me invariably fixate on is the way Herr gracefully owns the stage. Hes wearing pants that end above the knee, revealing shimmering high-tech silver and black prosthetics. Herr is focused on what hes saying, not what his artificial legs are doing. The crime of physical impairment is that it often steals from a persons sense of self. If you didnt look below his knees, youd never guess that Herr is missing half of each leg. He walks through the world the way we all would hope to.

He has effectively ended his disability, or at least the perception of it, just as he said he would. Inspired by his accident, he earned a masters degree in mechanical engineering at MIT in 1993, followed by a Ph.D. at Harvard in biophysics. Ever since, Herr has produced a string of breakthrough products, starting with a computer-controlled artificial knee in 2003. In 2004, he created the biomechatronics group at MIT, a now 40-person R&D lab drawing on the fields of biology, mechanics, and electronics to restore function to those whove lost it. Three years later, the team produced a powered ankle-foot prosthesis that allows an amputee to walk with speed and effort comparable to those with biological legs. Called the emPower, the apparatus weighs a few pounds and houses 12 sensors, three computers, tensioning springs, and muscle-tendon actuators. The ankle system is manufactured by a private company Herr started called BionX.

Last year, Herr advanced another of his labs goals, to improve human performance beyond what nature intends by creating a brace-like exoskeleton device that reduces the metabolic cost of walking. The implications for people who want to get places fasteror perhaps a soldier trying to conserve energy on a long marchare vast.

In the near future, Herr and his colleagues at the MIT center are committed to, among other things, reversing paralysis. Herrs goal is to develop a synthetic spinal cord thataids the damaged original. A prosthesis, in other words.

In his office, Herr draws up his pant leg and rolls down a silicone sleeve to show me a newly developed fabric that lines the socket of his prosthetic and cushions the problematic intersection between the biological stump and the man-made limb. The exquisitely comfortable fitdigitally derived, he explains, but highly personalis something he delights over with a savoring gush.

With our first meeting nearing its end, I grow distracted thinking about the wounded few Herr has smiled upon. In 2014, he worked on a bionic prosthetic for the dancer Adrianne Haslet-Davis, who lost her left leg in the Boston Marathon bombing. Currently, hes working with Hari Budha Magar, a double-amputee former Gurkha soldier who plans to climb Mount Everest in 2018, and also Jim Ewing, an old New Hampshire climbing buddy. Ewing was climbing a wall on vacation in the Cayman Islands in 2014 when he fell with his teen daughter on belay. She couldnt brake the rope, and he plummeted some 60 feet, shattering his pelvis and left foot on impact.

The dancer, the Gurkha, the climber, and Herr himself are examples of what he often describes as the millions of humans who might appear broken but are not. Haslet-Davis, on a bionic limb embedded with dance intelligence, brilliantly performed the rumba again, and Ewing underwent a pioneering amputation procedure developed by Herrs biomechatronics team in partnership with MIT colleague and surgeon Matthew Carty, who performed the operation at Brigham and Womens Faulkner Hospital, to prepare Ewing for an advanced prosthesis. Magar will be outfitted with short prosthetics to reduce leg drag and sophisticated crutches for speed as he attempts Everest history.

The stories Herr tells, the future he sees, the beautifully functioning artificial limb before meits all I can do not to show him my atrophied left leg and ask for his godlike intervention to fix what I know is broken. But I dont, not yet.

When I wrote Herr to tell him about my interest in his work, I summarized my case history. I explained how in the summer of 2014, I found myself with increasingly debilitating nerve and lower-back pain. When I finally got an MRI, I learned that I had an extremely rare bone cancer called chordoma that had spread from my lower lumbar vertebrae into my right hip flexor. Radiation and a difficult multi-stage surgery successfully removed the softball-size tumor, but months later, possibly due to a loss of blood to the spinal cord, Id yet to regain sensation or strength in my hips and legs. The doctors didnt know if it was permanent, but the prognosis didnt look good.

Jim Ewing and his robotic prosthetic. (Boston Globe/Getty)Aimee Mullins. (Lynn Johnson/Getty)Mountaineer Hari Budha Magar. (Himalayan Ski Trek)

Id expected a rapid, maybe even exceptional recovery. I am an athlete and adventurer who has had the good fortune to do a lot of cool stuff over the years. Id become a whitewater guide, climbed Grand Teton, raced the hill climb at Mount Washington on foot and by bike, and mountain-biked half the 3,000-mile-plus Great Divide route. I expected to complete the other half someday.

Id progressed from a walker to a cane, from a recumbent tricycle to a pedal-assist e-bike. Then my nerve regeneration halted. In May 2015, after the surgery, Id contacted Boston neurologist Bill David for muscle and nerve testing. An avid cyclist and kindredspirit, hed hopefully stuck needles into my skin every six months to chart my recovery. Late last year, he confirmed what I had already sensed. Short of a miracle, Id gone about as far as I could. I really wish that we had met on a mountain or river as opposed to a medical clinic, David said.

Id negotiated several stages of recovery, but the one I feared most was right nowat the end, my future fixed. Ive been coming to grips with who I am as an incomplete paraplegic and figuring out how to make the best version of this new person, I wroteto Herr.

Id imagined a stirring epilogue to our encounters, a moment perhaps when a radical trial arose and a crazy volunteer was needed. To be closer to the person I once was, I would try anythinginjected viruses, exoskeletal suits, implants. When I got together with a close friend for lunch, I told her how the story with Herr was progressing, and how the limbs he created were so advanced that Id read about people wanting them even though their leg complications didnt medically require amputation. She listened carefully. Let me ask you something, she said. Would you, um, get your legs cut off?

Exactly when in his childhood Hugh Herr decided to become the worlds best climber is impossible to pinpoint, but the goal was nurtured during family road trips across the West. He and his older brothers climbed, fished, and hiked in the American and Canadian Rockies, whetting the youthful Herrs appetite for adventure. The Shawangunk Mountains in New York were a four-and-a-half-hour drive from the Herrs home in Lancaster, Pennsylvania. The Gunks were an emerging mecca in the seventies, and Herr quickly established himself as a prodigy, climbing this stuff when I was 11 that only adults had done, and at 15 that no one else had done, he says.

When he and Jeff Batzer, a friend from Lancaster, drove to New Hampshires Mount Washington in January 1982 for a weekend ice-climbing outing, it wasnt to do anything audacious. Theyd attempt a classic route in Huntingtons Ravine, and maybe, depending on the weather and avalanche conditions, summit Mount Washington before racing down for the 12-hour drive home. Herr was a 17-year-old junior in high school, his friend Batzer, 20.

The decision to tack on the summit of Washington turned out to be a tragic mistake. They left a sleeping bag and bivy sack behind to reduce weight but encountered howling winds and blizzard conditions near the top, and they ended up losing their way, mistakenly descending into a different valley from where theyd come.

After four days trekking through a storm in deep snow and below-freezing temperatures to find their way out, Herr was no longer able to walk. Early on in the odyssey, he had punched through a frozen streambed into shin-deep water, soaking his boots and pants, and was suffering from severe frostbite. In Second Ascent, a biography by Alison Osius, Herr said that he had reconciled himself to death when a backcountry snowshoer saw some of Batzers tracks and followed them to a makeshift shelter the two were bivouacked in. The climbers were evacuated to a nearby hospital in Littleton, where doctors treated both for hypothermia and frostbite. Herrs legs were in terrible shape. At the hospital, he learned that doctors might not be able to save them and that a member of his search party, a 28-year-old climbing-school instructor named Albert Dow, had been killed in an avalanche. Two months later, doctors amputated Herrs legs four inches below the knee. Batzers fingers on his right hand were amputated, along with his left foot and the toes on his right foot.

I asked my doctor after the amputation what Id be able to do with my new body, Herr recalls. The doctor said, What do you want to do? I said I wanted to drive a car, ride my bike, and climb. The doctor said youll be able to drive a car, but with hand controls. He said I would not be able to ride a bike or return to climbing.

Herr did all of the above within a year. He worked closely with his prosthetist on one pair of artificial legs after another and tinkered on his own in the machine shop of a vocational school hed begun attending in 1981. He soon figured out that he could hack his artificial limbs to suit the requirements of particular climbing routes. He built limbs that extended or shortened his stature; he carved out feet with wedge ends to slice into crevices. He began to knock off routes that he hadnt been able to do previously, including leading an ascent of Vandals at Skytop, the first 5.13 on the East Coast. It ignited a new controversy: that his adaptations were a form of cheating. Herr likes to tell audiences that he invited his affronted rivals to chop off their own legs.

Some people were bitter and angry about the accident, says Jim Ewing, a summer roommate of Herrs in the 1980s, and with Hugh coming back and climbing so well, they started making up excuses, saying things like, He can stand on a dime, his feet dont get sore, he doesnt have calf fatigue. Id just look at these people and think, By God, you havent seen this guy crawl to the toilet in the middle of the night because he doesnt have his legs on. He is handicapped; it is a handicap. People had no idea.

The 1982 rescue. (Jim Cole/AP)Herr in the hospital. (Jim Cole/AP)Herr in 1984. (Peter Lewis)

While there was a lot of media attention about Herrs accident, he kept private the struggles and self-doubt he faced after he lost his legs. When he returned to New Hampshire to climb again 18 months later, the unease from locals over Dows death and Herrs resurgence was palpable.

The harsh early views of Herr didnt soon go away. When I asked him what he thought when the American Alpine Club last year honored him at a celebratory awards evening in Denver, he said he was stunned. They had named him a new inductee of the Hall of Mountaineering Excellence for lasting contributions on and off the mountain. It shocked me, he said. The initial story line of the accident was that these young, irresponsible, incompetent climbers caused the death of an experienced, beloved local climber. That narrative went on for a very long time. So for two decades at least, I wouldnt even expect the American Alpine Club to invite me to be in the audience.

When Herr talks about Albert Dow, who he never met, its with the fondness of a friend. That was Albert! he recounts about Dows insistence that he go looking for Herr and Batzer because hed want someone to do the same for him. Last year, Herr told a Reddit audience that he strives to honor Dow. I hate the idea that his death somehow enabled me to live so I could do good work, he says. What I like is that his kindness and who he wasand his sacrificeinspired me to work really hard.

In 1985, Herr free-climbed New Hampshires exceptionally steep and unprotected Stage Fright, with his friend Jim Surette on belay. It was a significant and life-threatening milestone, and afterward Herr had a dream that set his new path. He describes a nightmare in which Surette, bunking on a neighboring couch, throws off his covers to reveal mangled, bloody, amputated legs. We both go Aaah! in the dream, says Herr, but then I turn to Jimmy and say, Dont worry, Jimmy, its just a dream. Im the one without legs. Prior to that, in all my dreams I would be running and jumping, and I would have my biological legs. It was the first time my brain recognized my new state.

Some mightve interpreted the nightmare with melancholy, an attempt to come to terms with a sorrowful lifelong condition. Herr saw it as a beautiful vision.

The auditorium is full at the Princeton, New Jersey, headquarters of the Robert Wood Johnson Foundation, all 150 in attendance looking stage left as Herr introduces an image of himself in a New Hampshire hospital room decades earlier. What do you see? he asks.

It is Herr in the moments after his legs have been amputated. The 17-year-old is gazing down at a white sheet and the outline of his stumps. The audience is riveted.

What do you see? he asks again. I see a new beginning, he declares. I see beauty.

Herr, who prefers to use the term unusual instead of handicapped or disabled, often says that he wouldnt want his biological legs back. He loves the legs he started building after the accident and has steadily improved upon for the past several decades.

His meteoric rise in academia is almost as improbable as his comeback to elite climbing. I actually graduated from high school not being able to take 10 percent of 100, he says. I had no idea what a percent was. His older brothers were all in construction. He understood that the family trade was unavailable to him, so he shut himself away and applied the same obsessive focus to science that hed once reserved for climbing. He read everything he could find and enrolled at the local college, Millersville University.

Wed watch all these films of animals locomoting to try to learn about motion, says Don Eidam, his first adviser at Millersville and an unapologetic superfan who writes a newsletter about Herr. Hed put all these ideas on my blackboard, and the chalk would literally be disintegrating. Hed call me at midnight with an idea. Ive never met anyone so committed or intense.

In 1991, Herr became the first student from Millersville to be accepted at MIT. The academic degrees, innovations, and honors have since overflowed. He is the holder or coholder of over 100 patents. The powered prosthesis he developed for ankle-foot amputees was the product of a special mind with a special motivation. By copying the behavior of a biologically intact leg, Herr and his biomechatronics lab were able to create a breakthrough replacement. In 2011, Time crowned him the leader of the bionic age. Last year he won Europes top prize for inventors, the prestigious Princess of Asturias Award.

In Hughs mind, he has not successfully innovated until people are able to benefit from his innovation, says Tyler Clites, a Harvard-MIT student who has worked in Herrs lab for six years. He has said to me, Look, Tyler, Ive invented hundreds of times, but Ive only ever innovated twice. The two items, his prosthetic knee and the ankle-foot, are the only ones commercially available to others.

The idea of an endlessly upgradable human is something Herr feels in his bones. I believe in the near future, in a decade or two, when you walk down the streets of Boston, youll routinely see people wearing bionic systems, Herr told ABC News in a 2016 interview. In 100 years, he thinks the human form will be unrecognizable. The inference is that the abnormal will be normal, beauty rethought and reborn. Unusual people like Herr will have come home.

At a small luncheon after his talk in New Jersey, the organizers ask me to say a few words about my condition. I give a five-minute recap of my struggles with cancer, the spinal-cord complication, and my up-and-down recovery. It is my first time speaking publicly about my situation. As I do, I sneak a glance or two at Herr. I wonder what he thinks hearing me tell my story. He is sitting immediately to my right, raking through a towering salad.

There is no clear signal from him, but I leave feeling that Ive pulled ever so slightly into his orbit. I am also beginning to understand the weight he bears of being a savior. A friend who saw his impassioned SXSW talk in 2015 told me how she raced up to thank him afterward, only to encounter a different guy. He was polite but aloof. She was put off, but I think I understand. The man has to set boundaries. He cant save everybody.

You might say that Herrs the sort of disrupter the research world needs, or you might say hes overpromising. One spinal-cord-injury scientist I spoke with wasnt so sure that a bold tech solution is the answer in a field long focused on the biology of nerve regeneration.

Nicholas Negroponte, the cofounder and former director of the MIT Media Lab, says Herrs sense of humor helps him handle any negative commentary. Its particularlyimportant when you do and say risky things, some of which invite harsh criticism, he says. You smile and keep going, because you know youre right.

A week after his talk in New Jersey, Herr and I meet up at a seafood restaurant near his MIT office. I arrive 30 minutes early, wanting to get situated. Having lived with my disability for some time now, I understand that I cant just sweep in like I used to. Herr, to my surprise, given his packed schedule, arrives ten minutes early.

Bomb survivor Adrianne Haslet-Davis. (Michael Dwyer/AP)

Herr told me earlier that he rarely pushes himself on climbs anymore. He proudly mentioned his two preteen, homeschooled daughters, who are avid hikers and spend almost every weekend with Herrs former wife, Patricia Ellis Herr, in the White Mountains happily exhausting themselves. They long ago summited Mount Washington and have high-pointed in 46 of the 50 states.

Herr and I talk at length about some of the people he has worked with and why. The Haslet-Davis project took a group from his biomechatronics lab 200 days to create the prosthetic, counting down to the 2014 TED Talk. She said she wanted to dance again. I really related, he says. He told himself, Im an MIT professor, I have resources. The timeline was tight enough that there was a TED Talk plan A (with her) and plan B (without). As everyone knows who has watched the video, Herrs team hit its deadline. Haslet-Davis unforgettably danced again, and there wasnt a dry eye because of it.

But as incredible as the moment was, its a source of frustration that the prosthetic cant be permanently handed over to Haslet-Davis. While Herr would love to give it to her, its a prototype that would cost millions to reproduce. As for Herrs climbing buddy Jim Ewing, thats a similarly uncertain situation. Months after Ewing had his foot amputated, he was fitted with a newly designed ankle-foot prosthetic that responds to his brain waves and allows him to feel his appendage. It is also a prototype that Ewing will eventually have to return.

Haslet-Davis and Ewing understood that they were part of a research project and wouldnt be able to keep the prototypes. Meanwhile, Herrs knee and ankle prosthetics, which cost tens of thousands of dollars, arent yet widely covered by insurance and remain too expensive for most who have a need for them. Herr has been in discussions with insurers to try and change that. According to Amputee Coalition of America estimates, there are 185,000 new lowerextremity amputations annually in the U.S. By contrast, there are only 1,700 emPower ankles in circulation right now. About half of them are worn by vets, paid for through reimbursements covered by the Department of Veterans Affairs.

Herrs work is important and coming from a good place, says Alisha Sarang-Sieminski, an associate professor of bioengineering at the Massachusetts-based Franklin W. Olin College of Engineering, a school involved in numerous projects related to lower-cost accessibility design. But people have different needs for different contexts. Also, so much of the high tech is really not accessible to very many people financially. Should people keep building them? Definitely. Should we also explore basic solutions? Yes.

Still, Ewings pioneering amputation is a huge success for Herrs group, the Brigham and Womens surgical team, and, most notably, Ewing. When I visited him at a climbing gym near Portland, Maine, he was planning a trip back to the Cayman Islands. For Ewing, the amputation has reduced the acute pain he used to feel in his biological foot and dramatically changed his outlook. He says that after his accident, he contemplated suicide. Being alive isnt enough, he says. Breathing isnt enough. I had to do something. Hugh understood my motivation probably better than I did.

Herr hadnt seen Ewing for years when he got an e-mail from him asking for advice about his foot. He was in a bad place, says Herr. Also, I really felt for his daughter. I know guilt so well, that poor girl.

Ewing says that the way hed set up the ropes is to blame for his daughters inability to brake the fall. Though she has returned to climbing at the gym and bouldering, she wasnt interested in rope climbing in the accidents aftermath, and Ewing worried that hed ruined the sporta passion theyd shared for yearsfor her.

Meanwhile, the gift Herr has given Ewing is exceptional. It might be the first time Herr is not the most technologically advanced lower-limb amputee. Herr often describes himself and others facing disabilities as astronauts testing new life-enabling technologies. As for his own legs, Herr wants to go even further but would need to leave the U.S. to undergo the operation he has in mind. Id love to do it, he says, without revealing any details about the procedure. Im just weighing the risk. I definitely dont want to go backwards.

In the short term, hes using a newly designed set of titanium legs and pushing forward on his work, noting hoped-for funding this year from the military to show we can synthetically take over a paralyzed limb. Herr then asks about my rehabilitation experience. This is finally my chance, I think, to ask if theres anything he can do for me.

I tell him that I identify with amputees and often wonder how some people without legs are more adept than some of us with them. Every time I watch a person with artificial legs walking, I selfishly wonder, Why not me? Why not us? Herr says they have some good ideas but acknowledges that the field has been way more successful in the amputation arena than with spinal-cord injuries. Its hard, he says.

While Herr has complete autonomy selecting projects in his lab, his interventions are rare, and they dont happen unless the time and circumstances are right. Often, people ask for help and I dont have the resources or the solution, he says. Exceptions like Haslet-Davis and Ewing come from feeling deeply about it and being in the position to make it happen.

I realize talking to Herr that its not my story thats weak, its the technology. Id incorrectly understood his comment about an imminent cure. Paralysis is lowhanging fruit in that its a condition they can impact in ten to twenty years instead of fifty. There are no toys to play with in Herrs lab closet. Not yet.

Before Herr and I wrap up our last visit, I ask what hed do if he were at an impasse. Its clear, at least to me, that Im talking about myself. Being a scientist, he focuses on process. He says he throws everything and anything at a problem. He visualizes each idea as a rock and starts turning them over. He mentions an acquaintance who came to see him earlier in the day who was struggling with depression. Herr started in, imagining at hyperspeed all the places the person might go and hadnt yet. Acupuncture? No? Meditation? No? Are you running? No? What medications have you tried? One? One! Theres like 20 antidepressants! Go, go, go! he says he wanted to plead. He chuckles at his overexuberance, but his belief is real. This can be solved!

When I say goodbye to Herr and watch him bound down from the upper level of the restaurant to the rain-drenched sidewalk, Im struck by a malaise. Maybe its the rain. Maybe its the opportunity lost. Maybe its the way he flipped a switch on his emPower ankle and raced effortlessly into the street. But then I think about Herr turning over one rock at a time and the span of possibilities he presented to help with depression. Im not out of options. There are hundreds of researchers working on a paralysis cure, and I immediately think of a world map I saw recently on a website with dozens of bright red circles representing centers of innovation. I can hear the words of my neurologist, who on my last visit leaned in with something else when he said goodbye. Keep moving, he urged. Theres even a clinic in New Hampshire I heard about where theyve produced exceptional walking recoveries using a robotic gait trainer available nowhere else in the U.S.

I begin to wonder, was Herrs story about his depressed acquaintance allegorical? An on-the-spot intervention? Had I just been, ever so lightly, smiled upon, too?

Longtime Outside contributor Todd Balf is the author of The Last River. Guido Vitti is anOutsidecontributing photographer.

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The $100 Million Plan to End Paralysis - Outside Magazine

GUANGZHOU, China, Sept. 5, 2017 /PRNewswire/ -- Chinese Grand Fan Group formally signed the agreement to acquire the French CICABEL brand on September 4th. Grand Fan Group is openly optimistic about CICABEL's technology and development prospects, while the investment into the French brand represents the first step in the execution of the strategy behind the group's entry into the skin care market. The signing ceremony took place in France.

Santinov is a 130-year-old French traditional pharmaceutical manufacturer founded in 1887. Santinov created and launched the CICABEL Mask, a three-step revitalizing and hydration face mask set using stem cells as the principal component, following years of research and development on the back of strong technological competence. At variance with traditional skin care products, the set is expected to become a disruptor and transform the public's expectations from the beauty industry.

A Grand Fan Group executive said "By adopting the management and operations model commonly deployed by international brands, we put in place partnerships with several leading international beauty and health brands based on our own brand, achieving a diversified brand scenario as well as access to advanced technology R&D. These moves will serve to offer more and better choices to consumers."

With the enhancement of the general public's awareness of skin care, traditional skin care products no longer meet the basic expectations and needs of consumers. Brands with an ill-defined image or a hodge-podge of seemingly unrelated products, uneven quality, inadequate supervision and other issues have led the industry to be subject to a high level of criticism. To add insult to injury, most traditional skin care products actually do little for the skin. In line with accepted biotechnology and medical standards, the CICABEL Mask is expected to reverse the perception.

Through the activation of skin stem cells, the mask provides nutrition that penetrates deep into the dermis and promotes the regeneration of new cells, delivering an in-depthreplenishment effect. Put in another way, CICABEL uses the body's own multifunctional cells to achieve a new level of skin beauty. The CICABEL Mask from France is expectedto become the "Terminator" of traditional masks available in the market.

CICABEL will formally go on sale in China soon, with plans for roll outs in several global markets shortly thereafter.

Contact: +86-400-639-1958, rel="nofollow">hantao@1958difo.com

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China-based Grand Fan Group acquires leading French skincare brand - Markets Insider

Press Release

Researchers at Ohio State University Wexner Medical Center uncovered a novel pathway for hair follicular regeneration. Palm tocotrienol complex (EVNol SupraBio) is shown to induce hair follicle growth via protein expression of epidermal E-cadherin dependent beta-catenin - the key signaling molecule for inducing pluripotent stem cells in the adult skin.

In this study (1), male mice with mutated leptin receptor were applied with either 5mg/cm2 palm tocotrienol rich fraction (TRF) (ie. EVNol SupraBio - bioenhanced palm tocotrienol complex, supplied by ExcelVite) or placebo on shaved dorsal skin thrice per week for 21 days and the evaluation of hair growth was recorded by the color of dorsal skin. The mechanism of palm TRF-induced hair growth, the dependency on the loss of E-cadherin and the activation of beta-catenin for hair follicle formation were examined by quantification of gene expressions, immunoprecipitation and immunoblots.

When compared to placebo, palm TRF treated group showed significantly increased number of anagen (ie. cycle of growth) hair follicles, increased fetal characteristics of hair follicular development in the adult skin, increased epidermal keratinocyte proliferation, significant decreased E-cadherin expression that was associated with high translocation of beta-catenin-Tf3, leading to upregulation of gene expressions of Oct4, Sox9, Klf4, c-Myc and Nanog skin-specific pluripotent factors that support hair follicular regeneration. These factors are also known as the Yamanaka Transcription Factors discovered by Dr. Shinya Yamanaka, joint-recipient of the 2012 Nobel Prize in Physiology or Medicine. Prof. Yamanaka discovered that mature cells can be reprogrammed to become pluripotent.

The researchers concluded that palm TRF suppression of epidermal E-cadherin induced beta-catenin and nuclear translocation is the novel pathway that leads to expressions of pluripotent factors and subsequently promotes anagen hair cycling in adult skin.

What we have shown is that Palm TRF can induce hair folliculogenesis, which means that it can enrich the skin stem cell reserves. This novel epidermal pathway of hair follicular regeneration can have widespread impact on skin function including skin aging and repair, says Prof. Chandan Sen, the lead researcher at Ohio State University Wexner Medical Center.

Prior to the above discovery, researchers from University Science Malaysia had reported and patented the unique benefits of tocotrienols (EVNol SupraBio) in supporting hair growth in subjects with on-going hair loss (2).

We are thrilled with this new discovery, especially this novel pathway that affirmed our previous clinical findings for EVNol SupraBio in hair growth, (US Patent No: 7,211,274; Trop. Life Sci. Res. 2010). Taken together this latest study and previous published papers explain the mechanism as to how EVNol SupraBio may help in promoting hair growth in subjects experiencing hair loss, says Bryan See, Business Development Manager, ExcelVite.

Source:

About ExcelVite

ExcelVite Sdn. Bhd., incorporated in Malaysia in 2013, is the leading and largest producer of natural full spectrum tocotrienol / tocopherol complex (EVNol, and EVNol SupraBio), natural mixed-carotene complex (EVTene), phytosterol complex (EVRol), and red palm oil concentrate (EVSpectra) in the world via a patented technology.

ExcelVite is the only tocotrienol producer that operates in accordance to GMP (PIC/S) Guide to Good Manufacturing Practice for Medicinal Products. Its laboratory is accredited with ISO/IEC 17025 accreditation.

EVNol SupraBio is a patented (US Patent No. 6,596,306) self-emulsifying palm tocotrienol complex that ensures optimal tocotrienols oral absorption.

ExcelVite manufactures and markets its products under the tradenames: EVNol, EVNol SupraBio, EVTene, EVRol, and EVSpectra. These branded ingredients are Non-GMO, Kosher and Halal certified. ExcelVite supports the production of certified sustainable palm oil (CSPO) through RSPO Credits.

Websites:www.excelvite.com andwww.tocotrienol.org

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Latest Research Unveiled Novel Pathway For T3 In Hair Follicle Regeneration - Natural Products INSIDER

MIAMI, Sept. 6, 2017 /PRNewswire/ --Longeveron LLC, a regenerative medicine company developing cellular therapies, announced today that it treated its first patient in the Company's Phase 2b clinical trial evaluating the safety and efficacy of Longeveron human Allogeneic Mesenchymal Stem Cells (LMSCs) in patients with Aging Frailty Syndrome. This trial is being conducted pursuant to an Investigational New Drug Application (IND) in conformance with U.S. Food & Drug Administration (FDA) regulations. Aging Frailty is a common geriatric medical condition that is serious and life-threatening, and for which there are currently no U.S. Food and Drug Administration-approved therapeutics available.

The clinical trial is designed to enroll 120 subjects from approximately 10 medical centers around the U.S. The primary objective of the study is to evaluate the effect that LMSCs have on functional mobility and exercise tolerance in elderly Aging Frailty subjects. Three different LMSC dose groups will be compared to placebo over 12 months in a randomized, double-blinded, parallel arm design.Specifically, the trial will evaluate changes to the following:

"Frailty Syndrome is a very common and difficult situation to manage from a clinician's and caregiver's standpoint," stated Marco Pahor, M.D., Director of the Institute on Aging at the University of Florida. "The goal of intervention is to stop or slow the progression towards dependence and adverse health outcomes common to the syndrome, and to restore the patient to a state of healthy aging and functional independence. Longeveron's regenerative medicine trial is an important step towards the development of an effective therapeutic."

Allogeneic mesenchymal stem cells (MSCs) were previously tested in a Phase I/2 proof-of-concept study conducted by investigators at the University of Miami'sMiller School of Medicine. In that study, MSCs were shown to be safe and well-tolerated in frail, elderly subjects in a Phase 1 open label single ascending dose trial (publication link here) with a similar safety profile observed in the randomized, placebo-controlled Phase 2 study (publication link here) Subjects treated with a dose of 100 million MSCs showed significant improvements in six minute walking distance, and significant decreases in systemic inflammation, both relative to baseline.

"As individuals age, stem cell production and proliferation decreases, systemic inflammation increases, and a person's ability to repair and regenerate worn out or damaged tissue diminishes," remarked Suzanne Liv Page, Longeveron Chief Operating Officer. "In frail individuals this is particularly problematic. Our hypothesis is that exogenously infused allogeneic mesenchymal stem cells that are derived from the bone marrow of a healthy young donor, and culture expanded in our lab, will have potent regenerative and restorative effects."

Participants in this study must be between the ages of 70 and 85, be diagnosed as mildly to moderately frail due primarily to aging, and be able to walk between 200 and 400 meters over six minutes. Detailed information about the trial, subject eligibility and participating centers can be found by clicking here or by visiting the website http://www.clinicaltrials.gov and entering trial ID: NCT03169231.

About LMSCs

LMSCs is an allogeneic product, which means it is produced from stem cells derived from human donor bone marrow, and not from the patient's own stem cells, (referred to as autologous). LMSCs are manufactured at Longeveron's Cell Processing Facility in Miami, Fl. using a proprietary ex vivo culture expansion process.

About Longeveron

Longeveron is a regenerative medicine therapy company founded in 2014. Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company's research focus areas include Alzheimer's disease, Aging Frailty and the Metabolic Syndrome. Longeveron produces LMSCs in its own state-of-the-art cGMP cell processing facility. http://www.longeveron.com

Contact:Suzanne Liv Pagerel="nofollow">spage@longeveron.com305.909.0850

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Longeveron Initiates Phase 2b Stem Cell Therapy Trial to Treat Aging Frailty - Markets Insider

by David M. Panchision*

Diseases of the nervous system, including congenital disorders, cancers, and degenerative diseases, affect millions of people of all ages. Congenital disorders occur when the brain or spinal cord does not form correctly during development. Cancers of the nervous system result from the uncontrolled spread of aberrant cells. Degenerative diseases occur when the nervous system loses functioning of nerve cells. Most of the advances in stem cell research have been directed at treating degenerative diseases. While many treatments aim to limit the damage of these diseases, in some cases scientists believe that damage can be reversed by replacing lost cells with new ones derived from cells that can mature into nerve cells, called neural stem cells. Research that uses stem cells to treat nervous system disorders remains an area of great promise and challenge to demonstrate that cell-replacement therapy can restore lost function.

The nervous system is a complex organ made up of nerve cells (also called neurons) and glial cells, which surround and support neurons (see Figure 3.1). Neurons send signals that affect numerous functions including thought processes and movement. One type of glial cell, the oligodendrocyte, acts to speed up the signals of neurons that extend over long distances, such as in the spinal cord. The loss of any of these cell types may have catastrophic results on brain function.

Although reports dating back as early as the 1960s pointed towards the possibility that new nerve cells are formed in adult mammalian brains, this knowledge was not applied in the context of curing devastating brain diseases until the 1990s. While earlier medical research focused on limiting damage once it had occurred, in recent years researchers have been working hard to find out if the cells that can give rise to new neurons can be coaxed to restore brain function. New neurons in the adult brain arise from slowly-dividing cells that appear to be the remnants of stem cells that existed during fetal brain development. Since some of these adult cells still retain the ability to generate both neurons and glia, they are referred to as adult neural stem cells.

These findings are exciting because they suggest that the brain may contain a built-in mechanism to repair itself. Unfortunately, these new neurons are only generated in a few sites in the brain and turn into only a few specialized types of nerve cells. Although there are many different neuronal cell types in the brain, we now know that these new neurons can quot;plug inquot; correctly to assist brain function.1 The discovery of these cells has spurred further research into the characteristics of neural stem cells from the fetus and the adult, mostly using rodents and primates as model species. The hope is that these cells may be able to replenish those that are functionally lost in human degenerative diseases such as Parkinson's Disease, Huntington's Disease, and amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease), as well as from brain and spinal cord injuries that result from stroke or trauma.

Scientists are applying these new stem cell discoveries in two ways in their experiments. First, they are using current knowledge of normal brain development to modulate stem cells that are harvested and grown in culture. Researchers can then transplant these cultured cells into the brain of an animal model and allow the brain's own signals to differentiate the stem cells into neurons or glia. Alternatively, the stem cells can be induced to differentiate into neurons and glia while in the culture dish, before being transplanted into the brain. Much progress has been made the last several years with human embryonic stem (ES) cells that can differentiate into all cell types in the body. While ES cells can be maintained in culture for relatively long periods of time without differentiating, they usually must be coaxed through many more steps of differentiation to produce the desired cell types. Recent studies, however, suggest that ES cells may differentiate into neurons in a more straightforward manner than may other cell types.

Figure 3.1. The NeuronWhen sufficient neurotransmitters cross synapses and bind receptors on the neuronal cell body and dendrites, the neuron sends an electrical signal down its axon to synaptic terminals, which in turn release neurotransmitters into the synapse that affects the following neuron. The brain neurons that die in Parkinson's Disease release the transmitter dopamine. Oligodendrocytes supply the axon with an insulating myelin sheath.

2001 Terese Winslow

Second, scientists are identifying growth (trophic) factors that are normally produced and used by the developing and adult brain. They are using these factors to minimize damage to the brain and to activate the patient's own stem cells to repair damage that has occurred. Each of these strategies is being aggressively pursued to identify the most effective treatments for degenerative diseases. Most of these studies have been carried out initially with animal stem cells and recipients to determine their likelihood of success. Still, much more research is necessary to develop stem cell therapies that will be useful for treating brain and spinal cord disease in the same way that hematopoietic stem cell therapies are routinely used for immune system replacement (see Chapter 2).

The majority of stem cell studies of neurological disease have used rats and mice, since these models are convenient to use and are well-characterized biologically. If preliminary studies with rodent stem cells are successful, scientists will attempt to transplant human stem cells into rodents. Studies may then be carried out in primates (e.g., monkeys) to offer insight into how humans might respond to neurological treatment. Human studies are rarely undertaken until these other experiments have shown promising results. While human transplant studies have been carried out for decades in the case of Parkinson's disease, animal research continues to provide improved strategies to generate an abundant supply of transplantable cells.

The intensive research aiming at curing Parkinson's disease with stem cells is a good example for the various strategies, successful results, and remaining challenges of stem cell-based brain repair. Parkinson's disease is a progressive disorder of motor control that affects roughly 2% of persons 65 years and older. Triggered by the death of neurons in a brain region called the substantia nigra, Parkinson's disease begins with minor tremors that progress to limb and bodily rigidity and difficulty initiating movement. These neurons connect via long axons to another region called the striatum, composed of subregions called the caudate nucleus and the putamen. These neurons that reach from the substantia nigra to the striatum release the chemical transmitter dopamine onto their target neurons in the striatum. One of dopamine's major roles is to regulate the nerves that control body movement. As these cells die, less dopamine is produced, leading to the movement difficulties characteristic of Parkinson's disease. Currently, the causes of death of these neurons are not well understood.

For many years, doctors have treated Parkinson's disease patients with the drug levodopa (L-dopa), which the brain converts into dopamine. Although the drug works well initially, levodopa eventually loses its effectiveness, and side-effects increase. Ultimately, many doctors and patients find themselves fighting a losing battle. For this reason, a huge effort is underway to develop new treatments, including growth factors that help the remaining dopamine neurons survive and transplantation procedures to replace those that have died.

The strategy to use new cells to replace lost ones is not new. Surgeons first attempted to transplant dopamine-releasing cells from a patient's own adrenal glands in the 1980s.2,3 Although one of these studies reported a dramatic improvement in the patients' conditions, U.S. surgeons were only able to achieve modest and temporary improvement, insufficient to outweigh the risks of such a procedure. As a result, these human studies were not pursued further.

Another strategy was attempted in the 1970s, in which cells derived from fetal tissue from the mouse substantia nigra was transplanted into the adult rat eye and found to develop into mature dopamine neurons.4 In the 1980s, several groups showed that transplantation of this type of tissue could reverse Parkinson's-like symptoms in rats and monkeys when placed in the damaged areas.The success of the animal studies led to several human trials beginning in the mid-1980s.5,6 In some cases, patients showed a lessening of their symptoms. Also, researchers could measure an increase in dopamine neuron function in the striatum of these patients by using a brain-imaging method called positron emission tomography (PET) (see Figure 3.2).7

The NIH has funded two large and well-controlled clinical trials in the past 15 years in which researchers transplanted tissue from aborted fetuses into the striatum of patients with Parkinson's disease.7,8 These studies, performed in Colorado and New York, included controls where patients received quot;shamquot; surgery (no tissue was implanted), and neither the patients nor the scientists who evaluated their progress knew which patients received the implants. The patients' progress was followed for up to eight years. Unfortunately, both studies showed that the transplants offered little benefit to the patients as a group. While some patients showed improvement, others began to suffer from dyskinesias, jerky involuntary movements that are often side effects of long-term L-dopa treatment. This effect occurred in 15% of the patients in the Colorado study.7 and more than half of the patients in the New York study.8 Additionally, the New York study showed evidence that some patients' immune systems were attacking the grafts.

However, promising findings emerged from these studies as well. Younger and milder Parkinson's patients responded relatively well to the grafts, and PET scans of patients showed that some of the transplanted dopamine neurons survived and matured. Additionally, autopsies on three patients who died of unrelated causes, years after the surgeries, indicated the presence of dopamine neurons from the graft. These cells appeared to have matured in the same way as normal dopamine neurons, which suggested that they were acting normally in the brain.

Figure 3.2. Positron Emission Tomography (PET) images from a Parkinson's patient before and after fetal tissue transplantation. The image taken before surgery (left) shows uptake of a radioactive form of dopamine (red) only in the caudate nucleus, indicating that dopamine neurons have degenerated. Twelve months after surgery, an image from the same patient (right) reveals increased dopamine function, especially in the putamen. (Reprinted with permission from N Eng J Med 2001;344(10) p. 710.)

Researchers in Sweden followed the severity of dyskinesia in patients for eleven years after neural transplantation and found that the severity was typically mild or moderate. These results suggested that dyskinesias were due to effects that were distinct from the beneficial effects of the grafts.9 Dyskinesias may therefore be related to the ways that transplantation disturbs other cells in the brain and so may be minimized by future improvements in therapy. Another study that involved the grafting of cells both into the striatum (the target of dopamine neurons) and the substantia nigra (where dopamine neurons normally reside) of three patients showed no adverse effects and some modest improvement in patient movement.10 To determine the full extent of therapeutic benefits from such a procedure and confirm the reliability of these results, this study will need to be repeated with a larger patient population that includes the appropriate controls.

The limited success of these studies may reflect variations in the fetal tissue used for transplantation, which is of limited quantity and can not be standardized or well-characterized. The full complement of cells in these fetal tissue samples is not known at present. As a result, the tissue remains the greatest source of uncertainty in patient outcome following transplantation.

The major goal for Parkinson's investigators is to generate a source of cells that can be grown in large supply, maintained indefinitely in the laboratory, and differentiated efficiently into dopamine neurons that work when transplanted into the brain of a Parkinson's patient. Scientists have investigated the behavior of stem cells in culture and the mechanisms that govern dopamine neuron production during development in their attempts to identify optimal culture conditions that allow stem cells to turn into dopamine-producing neurons.

Preliminary studies have been carried out using immature stem cell-like precursors from the rodent ventral midbrain, the region that normally gives rise to these dopamine neurons. In one study these precursors were turned into functional dopamine neurons, which were then grafted into rats previously treated with 6-hydroxy-dopamine (6-OHDA) to kill the dopamine neurons in their substantia nigra and induce Parkinson's-like symptoms. Even though the percentage of surviving dopamine neurons was low following transplantation, it was sufficient to relieve the Parkinson's-like symptoms.11 Unfortunately, these fetal cells cannot be maintained in culture for very long before they lose the ability to differentiate into dopamine neurons.

Cells with features of neural stem cells have been derived from ES-cells, fetal brain tissue, brain tissue from neurosurgery, and brain tissue that was obtained after a person's death. There is controversy about whether other organ stem cell populations, such as hematopoietic stem cells, either contain or give rise to neural stem cells

Many researchers believe that the more primitive ES cells may be an excellent source of dopamine neurons because ES-cells can be grown indefinitely in a laboratory dish and can differentiate into any cell type, even after long periods in culture. Mouse ES cells injected directly into 6-OHDA-treated rat brains led to relief of Parkinson-like symptoms. Further investigation showed that these ES cells had differentiated into both dopamine and serotonin neurons.12 This latter type of neuron is generated in an adjacent region of the brain and may complicate the response to transplantation. Since ES cells can generate all cell types in the body, unwanted cell types such as muscle or bone could theoretically also be introduced into the brain. As a result, a great deal of effort is being currently put into finding the right quot;recipequot; for turning ES cells into dopamine neuronsand only this cell typeto treat Parkinson's disease. Researchers strive to learn more about normal brain development to help emulate the natural progression of ES cells toward dopamine neurons in the culture dish.

The recent availability of human ES cells has led to further studies to examine their potential for differentiation into dopamine neurons. Recently, dopamine neurons from human embryonic stem cells have been generated.13 One research group used a special type of companion cell, along with specific growth factors, to promote the differentiation of the ES cells through several stages into dopamine neurons. These neurons showed many of the characteristic properties of normal dopamine neurons.13 Furthermore, recent evidence of more direct neuronal differentiation methods from mouse ES cells fuels hope that scientists can refine and streamline the production of transplantable human dopamine neurons.

One method with great therapeutic potential is nuclear transfer. This method fuses the genetic material from one individual donor with a recipient egg cell that has had its nucleus removed. The early embryo that develops from this fusion is a genetic match for the donor. This process is sometimes called quot;therapeutic cloningquot; and is regarded by some to be ethically questionable. However, mouse ES cells have been differentiated successfully in this way into dopamine neurons that corrected Parkinsonian symptoms when transplanted into 6-OHDA-treated rats.14 Similar results have been obtained using parthenogenetic primate stem cells, which are cells that are genetic matches from a female donor with no contribution from a male donor.15 These approaches may offer the possibility of treating patients with genetically-matched cells, thereby eliminating the possibility of graft rejection.

Scientists are also studying the possibility that the brain may be able to repair itself with therapeutic support. This avenue of study is in its early stages but may involve administering drugs that stimulate the birth of new neurons from the brain's own stem cells. The concept is based on research showing that new nerve cells are born in the adult brains of humans. The phenomenon occurs in a brain region called the dentate gyrus of the hippocampus. While it is not yet clear how these new neurons contribute to normal brain function, their presence suggests that stem cells in the adult brain may have the potential to re-wire dysfunctional neuronal circuitry.

The adult brain's capacity for self-repair has been studied by investigating how the adult rat brain responds to transforming growth factor alpha (TGF), a protein important for early brain development that is expressed in limited quantities in adults.16 Injection of TGF into a healthy rat brain causes stem cells to divide for several days before ceasing division. In 6-OHDAtreated (Parkinsonian) rats, however, the cells proliferated and migrated to the damaged areas. Surprisingly, the TGF-treated rats showed few of the behavioral problems associated with untreated Parkinsonian rats.16 Additionally, in 2002 and 2003, two research groups isolated small numbers of dividing cells in the substantia nigra of adult rodents.17,18

These findings suggest that the brain can repair itself, as long as the repair process is triggered sufficiently. It is not clear, though, whether stem cells are responsible for this repair or if the TGF activates a different repair mechanism.

Many other diseases that affect the nervous system hold the potential for being treated with stem cells. Experimental therapies for chronic diseases of the nervous system, such as Alzheimer's disease, Lou Gehrig's disease, or Huntington's disease, and for acute injuries, such as spinal cord and brain trauma or stoke, are being currently developed and tested. These diverse disorders must be investigated within the contexts of their unique disease processes and treated accordingly with highly adapted cell-based approaches.

Although severe spinal cord injury is an area of intense research, the therapeutic targets are not as clear-cut as in Parkinson's disease. Spinal cord trauma destroys numerous cell types, including the neurons that carry messages between the brain and the rest of the body. In many spinal injuries, the cord is not actually severed, and at least some of the signal-carrying neuronal axons remain intact. However, the surviving axons no longer carry messages because oligodendrocytes, which make the axons' insulating myelin sheath, are lost. Researchers have recently made progress to replenish these lost myelin-producing cells. In one study, scientists cultured human ES cells through several steps to make mixed cultures that contained oligodendrocytes. When they injected these cells into the spinal cords of chemically-demyelinated rats, the treated rats regained limited use of their hind limbs compared with un-grafted rats.19 Researchers are not certain, however, whether the limited increase in function observed in rats is actually due to the remyelination or to an unidentified trophic effect of the treatment.

Getting neurons to grow new axons through the injury site to reconnect with their targets is even more challenging. While myelin promotes normal neuronal function, it also inhibits the growth of new axons following spinal injury. In a recent study to attempt post-trauma axonal growth, Harper and colleagues treated ES cells with a combination of factors that are known to promote motor neuron differentiation.20 The researchers then transplanted these cells into adult rats that had received spinal cord injuries. While many of these cells survived and differentiated into neurons, they did not send out axons unless the researchers also added drugs that interfered with the inhibitory effects of myelin. The growth effect was modest, and the researchers have not yet seen evidence of functional neuron connections. However, their results raise the possibility that signals can be turned on and off in the correct order to allow neurons to reconnect and function properly. Spinal injury researchers emphasize that additional basic and preclinical research must be completed before attempting human trials using stem cell therapies to repair the trauma-damaged nervous system.

Since myelin loss is at the heart of many other degenerative diseases, oligodendrocytes made from ES cells may be useful to treat these conditions as well. For example, scientists recently cultured human ES cells with a combination of growth factors to generate a highly enriched population of myelinating oligodendrocyte precursors.21,22 The researchers then tested these cells in a genetically-mutated mouse that does not produce myelin properly. When the growth factor-cultured ES cells were transplanted into affected mice, the cells migrated and differentiated into mature oligodendrocytes that made myelin sheaths around neighboring axons. These researchers subsequently showed that these cells matured and improved movement when grafted in rats with spinal cord injury.23 Improved movement only occurred when grafting was completed soon after injury, suggesting that some post-injury responses may interfere with the grafted cells. However, these results are sufficiently encouraging to plan clinical trials to test whether replacement of myelinating glia can treat spinal cord injury.

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is characterized by a progressive destruction of motor neurons in the spinal cord. Patients with ALS develop increasing muscle weakness over time, which ultimately leads to paralysis and death. The cause of ALS is largely unknown, and there are no effective treatments. Researchers recently have used different sources of stem cells to test in rat models of ALS to test for possible nerve cell-restoring properties. In one study, researchers injected cell clusters made from embryonic germ (EG) cells into the spinal cord fluid of the partially-paralyzed rats.24 Three months after the injections, many of the treated rats were able to move their hind limbs and walk with difficulty, while the rats that did not receive cell injections remained paralyzed. Moreover, the transplanted cells had migrated throughout the spinal fluid and developed into cells that displayed molecular characteristics of mature motor neurons. However, too few cells matured in this way to account for the recovery, and there was no evidence that the transplanted cells formed functional connections with muscles. The researchers suggest that the transplanted cells may be promoting recovery in some other way, such as by producing trophic factors.

This possibility was addressed in a second study in which scientists grew human fetal CNS stem cells in culture and genetically modified them to produce a trophic factor that promotes the survival of cells that are lost in ALS. When grafted into the spinal cords of the ALS-like rats, these cells secreted the desired growth factor and promoted the survival of the neurons that are normally lost in the ALS-like rats.25 While promising, these results highlight the need for additional basic research into functional recovery in ALS disease models.

Stroke affects about 750,000 patients per year in the

U.S. and is the most common cause of disability in adults. A stroke occurs when blood flow to the brain is disrupted. As a consequence, cells in affected brain regions die from insufficient amounts of oxygen. The treatment of stroke with anti-clotting drugs has dramatically improved the odds of patient recovery. However, in many patients the damage cannot be prevented, and the patient may permanently lose the functions of affected areas of the brain. For these patients, researchers are now considering stem cells as a way to repair the damaged brain regions. This problem is made more challenging because the damage in stroke may be widespread and may affect many cell types and connections.

However, researchers from Sweden recently observed that strokes in rats cause the brain's own stem cells to divide and give rise to new neurons.26 However, these neurons, which survived only a couple of weeks, are few in number compared to the extent of damage caused. A group from the University of Tokyo added a growth factor, bFGF, into the brains of rats after stroke and showed that the hippocampus was able to generate large numbers of new neurons.27 The researchers found evidence that these new neurons were actually making connections with other neurons. These and other results suggest that future stroke treatments may be able to coax the brain's own stem cells to make replacement neurons.

Taking an alternative approach, another group attempted transplantation as a means to treat the loss of brain mass after a severe stroke. By adding stem cells onto a polymer scaffold that they implanted into the stroke-damaged brains of mice, the researchers demonstrated that the seeded stem cells differentiated into neurons and that the polymer scaffold reduced scarring.28 Two groups transplanted human fetal stem cells in independent studies into the brains of stroke-affected rodents; these stem cells not only survived but migrated to the damaged areas of the brain.29,30 These studies increase our knowledge of how stem cells are attracted to diseased areas of the brain.

There is also increasing evidence from numerous animal disease models that stem cells are actively drawn to brain damage. Once they reach these damaged areas, they have been shown to exert beneficial effects such as reducing brain inflammation or supporting nerve cells. It is hoped that, once these mechanisms are better understood, this stem cell recruitment can potentially be exploited to mobilize a patient's own stem cells.

Similar lines of research are being considered with other disorders such as Huntington's Disease and certain congenital defects. While much attention has been called to the treatment of Alzheimer's Disease, it is still not clear if stem cells hold the key to its treatment. But despite the fact that much basic work remains and many fundamental questions are yet to be answered, researchers are hopeful that repair for once-incurable nervous system disorders may be amenable to stem cell based therapies.

Considerable progress has been made the last few years in our understanding of stem cell biology and devising sources of cells for transplantation. New methods are also being developed for cell delivery and targeting to affected areas of the body. These advances have fueled optimism that new treatments will come for millions of persons who suffer from neurological disorders. But it is the current task of scientists to bring these methods from the laboratory bench to the clinic in a scientifically sound and ethically acceptable fashion.

Notes:

* Chief, Developmental Neurobiology Program, Molecular, Cellular & Genomic Neuroscience Research Branch, Division of Neuroscience and Basic Behavioral Science, National Institute of Mental Health, National Institutes of Health, Email: panchisiond@mail.nih.gov

Chapter 2|Table of Contents|Chapter 4

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Repairing the Nervous System with Stem Cells | stemcells ...

NEW YORK--(BUSINESS WIRE)--Americord, the fastest growing cord blood bank in the country, and a leader in the advancement of umbilical cord blood, cord tissue, and placental tissue banking, has expanded customer options to now offer placental tissue banking as a stand-alone service.

As one of the only companies to offer placental tissue banking, Americord believes in the importance of offering new mothers an opportunity to preserve their stem cells for potential future use. We are therefore launching placental tissue banking as a stand-alone service, without the need to bank umbilical cord blood.

While many families have embraced Americords cord blood and tissue bundles, some have expressed interest in storing only placental tissue, commented Erin Willigan, Vice-President of Marketing at Americord. We wanted to respond to their desire to select individual services that best fit their budget and future plans.

Placental tissue contains mesenchymal stem cells (MSCs) that are a genetic match to the mother. These stem cells are multipotent, meaning that they can differentiate into many different types of cells, including organ and muscle tissue, skin, bone, cartilage, and fat cells. The placenta uses these stem cells to grow and function during pregnancy. After baby is delivered, stem cells from the placenta can be collected and stored for potential future use.

Due to their ability to multiply and become many different types of tissue, MSCs hold great promise for regenerative treatments. Over 50 clinical trials are currently researching therapeutic uses for MSCs, including treatments for Type 1 Diabetes, Alzheimers, and spinal cord injuries.

About Americord Registry LLC (Americord)

Americord Registry LLC is a leader in the advancement of umbilical cord blood, cord tissue and placenta tissue banking. Americord collects, processes, and stores newborn stem cells from umbilical cord blood for future medical or therapeutic use, including the treatment of more than 80 blood diseases such as sickle cell anemia and leukemia. Founded in 2008, Americord is registered with the FDA and operates in all 50 states. The companys laboratory is CLIA Certified, accredited by the AABB and complies with all federal and state guidelines and applicable licenses. Americord is headquartered in New York, NY. For more information, visit http://www.americordblood.com.

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Americord Offers New Option for Banking Placental Tissue - Business Wire (press release)

This article is about imaging techniques and modalities for the human body. For imaging of animals in research, see Preclinical imaging.

Medical imaging is the technique and process of creating visual representations of the interior of a body for clinical analysis and medical intervention, as well as visual representation of the function of some organs or tissues (physiology). Medical imaging seeks to reveal internal structures hidden by the skin and bones, as well as to diagnose and treat disease. Medical imaging also establishes a database of normal anatomy and physiology to make it possible to identify abnormalities. Although imaging of removed organs and tissues can be performed for medical reasons, such procedures are usually considered part of pathology instead of medical imaging.

As a discipline and in its widest sense, it is part of biological imaging and incorporates radiology which uses the imaging technologies of X-ray radiography, magnetic resonance imaging, medical ultrasonography or ultrasound, endoscopy, elastography, tactile imaging, thermography, medical photography and nuclear medicine functional imaging techniques as positron emission tomography (PET) and Single-photon emission computed tomography (SPECT).

Measurement and recording techniques which are not primarily designed to produce images, such as electroencephalography (EEG), magnetoencephalography (MEG), electrocardiography (ECG), and others represent other technologies which produce data susceptible to representation as a parameter graph vs. time or maps which contain data about the measurement locations. In a limited comparison these technologies can be considered as forms of medical imaging in another discipline.

Up until 2010, 5billion medical imaging studies had been conducted worldwide.[1] Radiation exposure from medical imaging in 2006 made up about 50% of total ionizing radiation exposure in the United States.[2]

Medical imaging is often perceived to designate the set of techniques that noninvasively produce images of the internal aspect of the body. In this restricted sense, medical imaging can be seen as the solution of mathematical inverse problems. This means that cause (the properties of living tissue) is inferred from effect (the observed signal). In the case of medical ultrasonography, the probe consists of ultrasonic pressure waves and echoes that go inside the tissue to show the internal structure. In the case of projectional radiography, the probe uses X-ray radiation, which is absorbed at different rates by different tissue types such as bone, muscle and fat.

The term noninvasive is used to denote a procedure where no instrument is introduced into a patients body which is the case for most imaging techniques used.

In the clinical context, invisible light medical imaging is generally equated to radiology or clinical imaging and the medical practitioner responsible for interpreting (and sometimes acquiring) the images is a radiologist. Visible light medical imaging involves digital video or still pictures that can be seen without special equipment. Dermatology and wound care are two modalities that use visible light imagery. Diagnostic radiography designates the technical aspects of medical imaging and in particular the acquisition of medical images. The radiographer or radiologic technologist is usually responsible for acquiring medical images of diagnostic quality, although some radiological interventions are performed by radiologists.

As a field of scientific investigation, medical imaging constitutes a sub-discipline of biomedical engineering, medical physics or medicine depending on the context: Research and development in the area of instrumentation, image acquisition (e.g., radiography), modeling and quantification are usually the preserve of biomedical engineering, medical physics, and computer science; Research into the application and interpretation of medical images is usually the preserve of radiology and the medical sub-discipline relevant to medical condition or area of medical science (neuroscience, cardiology, psychiatry, psychology, etc.) under investigation. Many of the techniques developed for medical imaging also have scientific and industrial applications.[3]

Two forms of radiographic images are in use in medical imaging. Projection radiography and fluoroscopy, with the latter being useful for catheter guidance. These 2D techniques are still in wide use despite the advance of 3D tomography due to the low cost, high resolution, and depending on application, lower radiation dosages. This imaging modality utilizes a wide beam of x rays for image acquisition and is the first imaging technique available in modern medicine.

A magnetic resonance imaging instrument (MRI scanner), or nuclear magnetic resonance (NMR) imaging scanner as it was originally known, uses powerful magnets to polarize and excite hydrogen nuclei (i.e., single protons) of water molecules in human tissue, producing a detectable signal which is spatially encoded, resulting in images of the body.[4] The MRI machine emits a radio frequency (RF) pulse at the resonant frequency of the hydrogen atoms on water molecules. Radio frequency antennas (RF coils) send the pulse to the area of the body to be examined. The RF pulse is absorbed by protons, causing their direction with respect to the primary magnetic field to change. When the RF pulse is turned off, the protons relax back to alignment with the primary magnet and emit radio-waves in the process. This radio-frequency emission from the hydrogen-atoms on water is what is detected and reconstructed into an image. The resonant frequency of a spinning magnetic dipole (of which protons are one example) is called the Larmor frequency and is determined by the strength of the main magnetic field and the chemical environment of the nuclei of interest. MRI uses three electromagnetic fields: a very strong (typically 1.5 to 3 teslas) static magnetic field to polarize the hydrogen nuclei, called the primary field; gradient fields that can be modified to vary in space and time (on the order of 1kHz) for spatial encoding, often simply called gradients; and a spatially homogeneous radio-frequency (RF) field for manipulation of the hydrogen nuclei to produce measurable signals, collected through an RF antenna.

Like CT, MRI traditionally creates a two dimensional image of a thin slice of the body and is therefore considered a tomographic imaging technique. Modern MRI instruments are capable of producing images in the form of 3D blocks, which may be considered a generalization of the single-slice, tomographic, concept. Unlike CT, MRI does not involve the use of ionizing radiation and is therefore not associated with the same health hazards. For example, because MRI has only been in use since the early 1980s, there are no known long-term effects of exposure to strong static fields (this is the subject of some debate; see Safety in MRI) and therefore there is no limit to the number of scans to which an individual can be subjected, in contrast with X-ray and CT. However, there are well-identified health risks associated with tissue heating from exposure to the RF field and the presence of implanted devices in the body, such as pace makers. These risks are strictly controlled as part of the design of the instrument and the scanning protocols used.

Because CT and MRI are sensitive to different tissue properties, the appearance of the images obtained with the two techniques differ markedly. In CT, X-rays must be blocked by some form of dense tissue to create an image, so the image quality when looking at soft tissues will be poor. In MRI, while any nucleus with a net nuclear spin can be used, the proton of the hydrogen atom remains the most widely used, especially in the clinical setting, because it is so ubiquitous and returns a large signal. This nucleus, present in water molecules, allows the excellent soft-tissue contrast achievable with MRI.

A number of different pulse sequences can be used for specific MRI diagnostic imaging (multiparametric MRI or mpMRI). It is possible to differentiate tissue characteristics by combining two or more of the following imaging sequences, depending on the information being sought: T1-weighted (T1-MRI), T2-weighted (T2-MRI), diffusion weighted imaging (DWI-MRI), dynamic contrast enhancement (DCE-MRI), and spectroscopy (MRI-S). For example, imaging of prostate tumors is better accomplished using T2-MRI and DWI-MRI than T2-weighted imaging alone.[5] The number of applications of mpMRI for detecting disease in various organs continues to expand, including liver studies, breast tumors, pancreatic tumors, and assessing the effects of vascular disruption agents on cancer tumors.[6][7][8]

Nuclear medicine encompasses both diagnostic imaging and treatment of disease, and may also be referred to as molecular medicine or molecular imaging & therapeutics.[9] Nuclear medicine uses certain properties of isotopes and the energetic particles emitted from radioactive material to diagnose or treat various pathology. Different from the typical concept of anatomic radiology, nuclear medicine enables assessment of physiology. This function-based approach to medical evaluation has useful applications in most subspecialties, notably oncology, neurology, and cardiology. Gamma cameras and PET scanners are used in e.g. scintigraphy, SPECT and PET to detect regions of biologic activity that may be associated with disease. Relatively short lived isotope, such as 99mTc is administered to the patient. Isotopes are often preferentially absorbed by biologically active tissue in the body, and can be used to identify tumors or fracture points in bone. Images are acquired after collimated photons are detected by a crystal that gives off a light signal, which is in turn amplified and converted into count data.

Fiduciary markers are used in a wide range of medical imaging applications. Images of the same subject produced with two different imaging systems may be correlated (called image registration) by placing a fiduciary marker in the area imaged by both systems. In this case, a marker which is visible in the images produced by both imaging modalities must be used. By this method, functional information from SPECT or positron emission tomography can be related to anatomical information provided by magnetic resonance imaging (MRI).[12] Similarly, fiducial points established during MRI can be correlated with brain images generated by magnetoencephalography to localize the source of brain activity.

Medical ultrasonography uses high frequency broadband sound waves in the megahertz range that are reflected by tissue to varying degrees to produce (up to 3D) images. This is commonly associated with imaging the fetus in pregnant women. Uses of ultrasound are much broader, however. Other important uses include imaging the abdominal organs, heart, breast, muscles, tendons, arteries and veins. While it may provide less anatomical detail than techniques such as CT or MRI, it has several advantages which make it ideal in numerous situations, in particular that it studies the function of moving structures in real-time, emits no ionizing radiation, and contains speckle that can be used in elastography. Ultrasound is also used as a popular research tool for capturing raw data, that can be made available through an ultrasound research interface, for the purpose of tissue characterization and implementation of new image processing techniques. The concepts of ultrasound differ from other medical imaging modalities in the fact that it is operated by the transmission and receipt of sound waves. The high frequency sound waves are sent into the tissue and depending on the composition of the different tissues; the signal will be attenuated and returned at separate intervals. A path of reflected sound waves in a multilayered structure can be defined by an input acoustic impedance (ultrasound sound wave) and the Reflection and transmission coefficients of the relative structures.[11] It is very safe to use and does not appear to cause any adverse effects. It is also relatively inexpensive and quick to perform. Ultrasound scanners can be taken to critically ill patients in intensive care units, avoiding the danger caused while moving the patient to the radiology department. The real time moving image obtained can be used to guide drainage and biopsy procedures. Doppler capabilities on modern scanners allow the blood flow in arteries and veins to be assessed.

Elastography is a relatively new imaging modality that maps the elastic properties of soft tissue. This modality emerged in the last two decades. Elastography is useful in medical diagnoses, as elasticity can discern healthy from unhealthy tissue for specific organs/growths. For example, cancerous tumours will often be harder than the surrounding tissue, and diseased livers are stiffer than healthy ones.[13][14][15][16] There are a several elastographic techniques based on the use of ultrasound, magnetic resonance imaging and tactile imaging. The wide clinical use of ultrasound elastography is a result of the implementation of technology in clinical ultrasound machines. Main branches of ultrasound elastography include Quasistatic Elastography/Strain Imaging, Shear Wave Elasticity Imaging (SWEI), Acoustic Radiation Force Impulse imaging (ARFI), Supersonic Shear Imaging (SSI), and Transient Elastography.[14] In the last decade a steady increase of activities in the field of elastography is observed demonstrating successful application of the technology in various areas of medical diagnostics and treatment monitoring.

Tactile imaging is a medical imaging modality that translates the sense of touch into a digital image. The tactile image is a function of P(x,y,z), where P is the pressure on soft tissue surface under applied deformation and x,y,z are coordinates where pressure P was measured. Tactile imaging closely mimics manual palpation, since the probe of the device with a pressure sensor array mounted on its face acts similar to human fingers during clinical examination, slightly deforming soft tissue by the probe and detecting resulting changes in the pressure pattern. Figure on the right presents an experiment on a composite tissue phantom examined by a tactile imaging probe illustrating the ability of tactile imaging to visualize in 3D the structure of the object.

This modality is used for imaging of the prostate,[17] breast,[18]vagina and pelvic floor support structures,[19] and myofascial trigger points in muscle.[20]

Photoacoustic imaging is a recently developed hybrid biomedical imaging modality based on the photoacoustic effect. It combines the advantages of optical absorption contrast with ultrasonic spatial resolution for deep imaging in (optical) diffusive or quasi-diffusive regime. Recent studies have shown that photoacoustic imaging can be used in vivo for tumor angiogenesis monitoring, blood oxygenation mapping, functional brain imaging, and skin melanoma detection, etc.

Tomography is the imaging by sections or sectioning. The main such methods in medical imaging are:

When ultrasound is used to image the heart it is referred to as an echocardiogram. Echocardiography allows detailed structures of the heart, including chamber size, heart function, the valves of the heart, as well as the pericardium (the sac around the heart) to be seen. Echocardiography uses 2D, 3D, and Doppler imaging to create pictures of the heart and visualize the blood flowing through each of the four heart valves. Echocardiography is widely used in an array of patients ranging from those experiencing symptoms, such as shortness of breath or chest pain, to those undergoing cancer treatments. Transthoracic ultrasound has been proven to be safe for patients of all ages, from infants to the elderly, without risk of harmful side effects or radiation, differentiating it from other imaging modalities. Echocardiography is one of the most commonly used imaging modalities in the world due to its portability and use in a variety of applications. In emergency situations, echocardiography is quick, easily accessible, and able to be performed at the bedside, making it the modality of choice for many physicians.

FNIR Is a relatively new non-invasive imaging technique. NIRS (near infrared spectroscopy) is used for the purpose of functional neuroimaging and has been widely accepted as a brain imaging technique.[21]

Using superparamagnetic iron oxide nanoparticles, magnetic particle imaging (MPI) is a developing diagnostic imaging technique used for tracking superparamagnetic iron oxide nanoparticles. The primary advantage is the high sensitivity and specificity, along with the lack of signal decrease with tissue depth. MPI has been used in medical research to image cardiovascular performance, neuroperfusion, and cell tracking.

In response to increased concern by the public over radiation doses and the ongoing progress of best practices, The Alliance for Radiation Safety in Pediatric Imaging was formed within the Society for Pediatric Radiology. In concert with The American Society of Radiologic Technologists, The American College of Radiology and The American Association of Physicists in Medicine, the Society for Pediatric Radiology developed and launched the Image Gently Campaign which is designed to maintain high quality imaging studies while using the lowest doses and best radiation safety practices available on pediatric patients.[22] This initiative has been endorsed and applied by a growing list of various Professional Medical organizations around the world and has received support and assistance from companies that manufacture equipment used in Radiology.

Following upon the success of the Image Gently campaign, the American College of Radiology, the Radiological Society of North America, the American Association of Physicists in Medicine and the American Society of Radiologic Technologists have launched a similar campaign to address this issue in the adult population called Image Wisely.[23] The World Health Organization and International Atomic Energy Agency (IAEA) of the United Nations have also been working in this area and have ongoing projects designed to broaden best practices and lower patient radiation dose.[24][25][26]

Medical imaging may be indicated in pregnancy because of pregnancy complications, intercurrent diseases or routine prenatal care. Magnetic resonance imaging (MRI) without MRI contrast agents as well as obstetric ultrasonography are not associated with any risk for the mother or the fetus, and are the imaging techniques of choice for pregnant women.[27]Projectional radiography, X-ray computed tomography and nuclear medicine imaging result some degree of ionizing radiation exposure, but have with a few exceptions much lower absorbed doses than what are associated with fetal harm.[27] At higher dosages, effects can include miscarriage, birth defects and intellectual disability.[27]

The amount of data obtained in a single MR or CT scan is very extensive. Some of the data that radiologists discard could save patients time and money, while reducing their exposure to radiation and risk of complications from invasive procedures.[28] Another approach for making the procedures more efficient is based on utilizing additional constraints, e.g., in some medical imaging modalities one can improve the efficiency of the data acquisition by taking into account the fact the reconstructed density is positive.[29]

Volume rendering techniques have been developed to enable CT, MRI and ultrasound scanning software to produce 3D images for the physician.[30] Traditionally CT and MRI scans produced 2D static output on film. To produce 3D images, many scans are made, then combined by computers to produce a 3D model, which can then be manipulated by the physician. 3D ultrasounds are produced using a somewhat similar technique. In diagnosing disease of the viscera of abdomen, ultrasound is particularly sensitive on imaging of biliary tract, urinary tract and female reproductive organs (ovary, fallopian tubes). As for example, diagnosis of gall stone by dilatation of common bile duct and stone in common bile duct. With the ability to visualize important structures in great detail, 3D visualization methods are a valuable resource for the diagnosis and surgical treatment of many pathologies. It was a key resource for the famous, but ultimately unsuccessful attempt by Singaporean surgeons to separate Iranian twins Ladan and Laleh Bijani in 2003. The 3D equipment was used previously for similar operations with great success.

Other proposed or developed techniques include:

Some of these techniques[examples needed] are still at a research stage and not yet used in clinical routines.

Neuroimaging has also been used in experimental circumstances to allow people (especially disabled persons) to control outside devices, acting as a brain computer interface.

Many medical imaging software applications (3DSlicer, ImageJ, MIPAV, ImageVis3D, etc.) are used for non-diagnostic imaging, specifically because they dont have an FDA approval[31] and not allowed to use in clinical research for patient diagnosis.[32] Note that many clinical research studies are not designed for patient diagnosis anyway.[33]

Used primarily in ultrasound imaging, capturing the image produced by a medical imaging device is required for archiving and telemedicine applications. In most scenarios, a frame grabber is used in order to capture the video signal from the medical device and relay it to a computer for further processing and operations.[34]

The Digital Imaging and Communication in Medicine (DICOM) Standard is used globally to store, exchange, and transmit medical images. The DICOM Standard incorporates protocols for imaging techniques such as radiography, computed tomography (CT), magnetic resonance imaging (MRI), ultrasonography, and radiation therapy.[35] DICOM includes standards for image exchange (e.g., via portable media such as DVDs), image compression, 3-D visualization, image presentation, and results reporting.[36]

Medical imaging techniques produce very large amounts of data, especially from CT, MRI and PET modalities. As a result, storage and communications of electronic image data are prohibitive without the use of compression. JPEG 2000 is the state-of-the-art image compression DICOM standard for storage and transmission of medical images. The cost and feasibility of accessing large image data sets over low or various bandwidths are further addressed by use of another DICOM standard, called JPIP, to enable efficient streaming of the JPEG 2000 compressed image data.

There has been growing trend to migrate from PACS to a Cloud Based RIS. A recent article by Applied Radiology said, As the digital-imaging realm is embraced across the healthcare enterprise, the swift transition from terabytes to petabytes of data has put radiology on the brink of information overload. Cloud computing offers the imaging department of the future the tools to manage data much more intelligently.[37]

Medical imaging has become a major tool in clinical trials since it enables rapid diagnosis with visualization and quantitative assessment.

A typical clinical trial goes through multiple phases and can take up to eight years. Clinical endpoints or outcomes are used to determine whether the therapy is safe and effective. Once a patient reaches the endpoint, he or she is generally excluded from further experimental interaction. Trials that rely solely on clinical endpoints are very costly as they have long durations and tend to need large numbers of patients.

In contrast to clinical endpoints, surrogate endpoints have been shown to cut down the time required to confirm whether a drug has clinical benefits. Imaging biomarkers (a characteristic that is objectively measured by an imaging technique, which is used as an indicator of pharmacological response to a therapy) and surrogate endpoints have shown to facilitate the use of small group sizes, obtaining quick results with good statistical power.[38]

Imaging is able to reveal subtle change that is indicative of the progression of therapy that may be missed out by more subjective, traditional approaches. Statistical bias is reduced as the findings are evaluated without any direct patient contact.

Imaging techniques such as positron emission tomography (PET) and magnetic resonance imaging (MRI) are routinely used in oncology and neuroscience areas,.[39][40][41][42] For example, measurement of tumour shrinkage is a commonly used surrogate endpoint in solid tumour response evaluation. This allows for faster and more objective assessment of the effects of anticancer drugs. In Alzheimers disease, MRI scans of the entire brain can accurately assess the rate of hippocampal atrophy, while PET scans can measure the brains metabolic activity by measuring regional glucose metabolism,[38] and beta-amyloid plaques using tracers such as Pittsburgh compound B (PiB). Historically less use has been made of quantitative medical imaging in other areas of drug development although interest is growing.[43]

An imaging-based trial will usually be made up of three components:

Lead is the main material used for radiographic shielding against scattered X-rays.

In magnetic resonance imaging, there is MRI RF shielding as well as magnetic shielding to prevent external disturbance of image quality.

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Microneedling has quickly become one of the most popular skin rejuvenation treatments. If youre considering trying it, here is what you need to know.

Microneedling, also called collagen-induction therapy, uses small needles that pierce the outermost layer of skin to create tiny microchannels. These microchannels help stimulate the production of collagen and elastin within the skin. They also promote new capillaries.

This can lead to an improved skin texture, reduction of acne or other scarring and help with discoloration, such as brown spots caused by sun damage. Microneedling may be combined with platelet-rich plasma, stem cells, or pure hyaluronic acid to enhance results further.

Microneedling can also be used on the scalp to help stimulate hair rejuvenation.

Prior to your first microneedling session, you will be asked to avoid sun exposure for at least 24 hours. Some doctors will tell you to avoid blood-thinning medications and herbal supplements like aspirin, ibuprofen and St. Johns wort to reduce bruising.

Each microneedling session takes about 20 to 30 minutes. First, your face will be cleansed and a numbing cream will be applied. Multiple treatment sessions, spaced a few weeks apart, are recommended. Most doctors recommend three to six treatments but many will notice an improvement in the tone and texture of their skin after just one treatment.

Immediately after your microneedling session, you will likely notice some redness that can last for several days. In my practice, we recommend that patients do not touch their face for at least four hours after treatment and do not apply anything to the face for 24 hours. It is crucial to avoid sun exposure for three days after the procedure.

You should avoid strenuous activity and exercise for the first 12 hours after treatment to prevent redness and bruising. For the first three days after treatment, you should use a gentle non-foaming cleanser, a barrier repair moisturizer, and a physical SPF. If swelling or bruising are a concern, you can take arnica supplements both before and after treatment to help minimize these side effects.

Once any redness or swelling diminishes, you should notice an immediate improvement in the way your skin looks and feels. Over the next several weeks, your skins appearance should continue to improve.

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What is microneedling and why is the skin treatment so popular? - Miami Herald

A new therapy changes the balance of osteoblasts (pictured here) and fat cells in the bone marrow, leading to stronger bones.

Science Picture Co/Science Source

By Emma YasinskiSep. 5, 2017 , 2:59 PM

A drug that can reverse diabetes and obesity in mice may have an unexpected benefit: strengthening bones. Experiments with a compound called TNP (2,4,6-trinitrophenol, which is also known as picric acid), which researchers often use to study obesity and diabetes, show that in mice the therapy can promote the formation of new bone. Thats in contrast to many diabetes drugs currently in wide use that leave patients bones weaker. If TNP has similar effects in humans, it may even be able to stimulate bone growth after fractures or prevent bone loss due to aging or disuse.

As more and more patients successfully manage diabetes with drugs that increase their insulin sensitivity, doctors and researchers have observed a serious problem: Thedrugs seem to decrease the activity of cells that produce bone, leaving patients prone to fractures and osteoporosis.

There are millions and millions of people that have osteoporosis [with or without diabetes], and it's not something we can cure, says Sean Morrison, a stem cell researcher at University of Texas Southwestern in Dallas. We need new agents that promote bone formation.

Morrison and his colleagues have shown that a high-fat diet causes mice to develop bones that contain more fat and less bone. The diet increased the levels of leptina hormone produced by fat cells that usually signals satiety in the brainin the bone marrow, which promoted the development of fat cells instead of bone cells. That suggests that nutrition has a direct effect on the balance of bone and fat in the bone marrow.

After reading Morrisons work, Siddaraju Boregowda, a stem cell researcher at the Scripps Research Institute in Jupiter, Florida, was reminded of genetically altered mice that dont gain body fat or develop diabetes, even when fed high-fat diets. He and his boss, stem cell researcher Donald Phinney, wondered whetherthose mice were also protected from the fattening of the bone marrow that accompanies a high-fat diet.

They contacted Anutosh Chakraborty, a molecular biologist who was studying such mice down the hall at Scripps at the time. The animals lack the gene for an enzyme called inositol hexakisphosphate kinase 1 (IP6K1), which is known to play a role in fat accumulation and insulin sensitivity. The scientists suspected that the lost enzyme might affect the animals' mesenchymal stem cells (MSCs)stem cells found in the bone marrow that are capable of developing into both thebone cells and fat cells that make up our skeletons. If too many fat cells develop, they take the place of bone cells, weakening the bone.

The researchers fed genetically altered and normal mice a high-fat diet for 8weeks. Not only did the genetically altered mice develop fewer fat cells than their normal counterparts, but their production of bone cells was higher than that of the normal mice, the team reported last month in Stem Cells.

The scientists then set out to see whetherthey could use a drug to achieve the same effect in normal mice. For 8weeks, they fed normal mice a high-fat diet and gave them daily injections of either TNP, a well-known IP6K1 inhibitor, or a placebo. When they analyzed the animals bones and marrow, they found that mice that had received TNP had significantly more bone cells, fewer fat cells, and greater overall bone area. The IP6K1 inhibitor apparently protected the mice from the detrimental effects of the high-fat diet.

The study provided thesurprising result that one new therapy currently being explored to lower insulin resistance promotes, rather than decreases, the formation of bone in mice, says DarwinProckop,a stem cell researcher at Texas A&M College of Medicine in Temple, who was not involved in the work.

The researchers still need to figure out how to deliver TNPs effects only to MSCs, instead of the entire body, given that it sometimes blocks other enzymes along with IP6K1. Inhibition of IP6K1 is a promising target for patients with both diabetes and obesity, Boregowda says. He says he and his colleagues are now enthusiastic about testing their findings in a wide range of bone-related diseases and disorders. It might even help heal broken bones, he speculates.

Phinney, on the other hand, is aiming even higher. He wonders whetherthe therapy could also be useful for space travel, because bones are especially vulnerable to deterioration in zero gravity. Its a whole new field of science and drug discovery.

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New therapy could protect diabetic bones - Science Magazine

Kathmandu, September 4

Civil Hospital is launching haploidentical stem cell transplant within a few months.

Its a treatment process for patients with blood-related cancers and certain blood disorders.

Patients who need a stem cell transplant and cant find a donor who matches their tissue type will benefit from the transplant. Haploidentical transplant is a modified form of stem cell transplant in which a healthy first degree relative a parent, or sibling can often serve as a donor.

When no matched donor is available, half-matched related (haploidentical) donors are safely used in stem cell transplantation, informed Dr Bishesh Poudyal, associate professor and chief of Clinical Hematology and Bone Marrow Transplant Unit at the hospital.

The cost of the transplant will be around 12 to 15 lakh rupees. People suffering from blood cancer, aplastic anaemia, sickle cell anaemia and thalassemia will benefit from the transplant.

The hospital has been performing allogeneic and autotransplant stem cell transplant where only siblings can be donors.

Nine patients had undergone autotransplant and one had undergone allogeneic stem cell transplant in the hospital after it started bone marrow transplant in the hospital in 2016.

A version of this article appears in print on September 05, 2017 of The Himalayan Times.

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Stem cell transplant to be launched - Himalayan Times

TEL AVIV, Israel, Sept. 5, 2017 /PRNewswire/ -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of stem cells selection technology, announced today that theU.S. Food and Drug Administration(FDA) has granted orphan drug designation for Cellect's ApoGraft for the prevention of acute and chronic graft versus host disease(GvHD) in transplant patients.

GvHD is a transplant associated disease representing an outcome of two immune systems crashing into each other. In many transplantations from donors, and especially in Bone Marrow Transplantations (BMT), the transplanted immune mature cells (as opposed to stem cells) attack the host (patient receiving the transplant) and create severe morbidity and in many cases even death.

This disease happens as a result of current practices being unable to separate the GvHD causing cells from the much needed stem cells.Cellect's ApoGraft was designed to eliminate immune responses in any transplantation of foreign cells and tissues.

Cellect's AppoGraft technology can be utilized already today to help thousands of development and research centers globally engaged in adult stem cells based therapeutics by providing them with a simplified and cost efficient enriched stem cells for use as a raw material for a wide range of stem cells based therapeutics R&D. Before Cellect's ApoGraft, such procedures were extremely complex, inefficient and required substantial resources in both cost, time and infrastructure requirements. ApoGraft can now be used to significantly advance the use of stem cells across multiple therapeutics indications as well as research and biobanking purposes.

The FDA Orphan Drug Act provides incentives for companies to develop products for rare diseases affecting fewer than 200,000 people inthe United States. Incentives may include tax credits related to clinical trial expenses, an exemption from theFDAuser fee, FDAassistance in clinical trial design and potential market exclusivity for seven years following approval.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (NASDAQ: "APOP", "APOPW") has developed a breakthrough technology for the selection of stem cells from any given tissue that aims to improve a variety of stem cell applications.

The Company's technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. Cellect's technology is applicable to a wide variety of stem cell related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss the Company's pathway for commercialization of its technology. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov. and in the Company's period filings with the SEC and the Tel-Aviv Stock Exchange.

ContactCellect Biotechnology Ltd. Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

View original content:http://www.prnewswire.com/news-releases/fda-grants-orphan-drug-status-to-cellects-apograft-for-acute-gvhd-and-chronic-gvhd-300513675.html

SOURCE Cellect Biotechnology Ltd.

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FDA Grants Orphan Drug Status to Cellect's ApoGraft for Acute GvHD and Chronic GvHD - Markets Insider