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SpaceX launches CU-built heart, bone health experiments to space station – CU Boulder Today

By Dr. Matthew Watson

Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch occured on Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

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3D-Printed Patch Mends Hearts – Photonics.com

By raymumme

Photonics.com Jun 2017 MINNEAPOLIS, June 6, 2017 A new 3D-laser-printed patch has been developed that can help heal scarred heart tissue after a heart attack.

Researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

"This is a significant step forward in treating the No. 1 cause of death in the U.S.," said Brenda Ogle, an associate professor of biomedical engineering at the University of Minnesota. "We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years."

The patch is modeled after a digital 3D scan of the structural proteins of native heart tissue. It is then made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells.

"We were quite surprised by how well it worked, given the complexity of the heart," Ogle said. "We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch."

The researchers will soon begin working on a larger patch and testing it on a pig heart, which is similar to a human heart.

The research study is published in the American Heart Association journal Circulation Research (doi: 10.1161/CIRCRESAHA.116.310277).

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Mice with ALS improve with stem cell therapy – The Ledger

By Dr. Matthew Watson

TAMPA Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease amyotrophic lateral sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USFs Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS.

USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

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Vancouver woman’s family pleading for help finding a bone marrow donor – CBC.ca

By raymumme

Vancouver surgeon and UBC professorRonald Lett is appealing tothe public forhelp in finding a bone marrow transplant for his wife Elizabeth Nega, who has an aggressive form of leukemia.

Nega, better known as Elsa, discovered that she had acute lymphoblasticleukemia in February and urgently needs a bone marrow transplant.However, the Ethiopian Canadian wife and mother of two has been unable to find a match because of the low number of African donors.

Ronald and Elsa are now reaching out to people of African descent to register as bone marrowdonors. They've started a website, match4elsa.com, as well as Facebook and Twitter accounts, to find Elsa and other African-Canadians life saving transplants.

"I love to live. I want to be with my kids. I want to smile again. I want to play with them again. If you save my life, you will save my whole family," said Elsa Nega in her video appeal for a donor.

Lett is the founder and international director of the charity, Canadian Network for International Surgery(CNIS). He met Elsa in Ethiopia while he was there training local doctors to perform essential surgeries.

After dedicating his life to helping others, Lett says being unable to help his wife in her time of need has been difficult.

"I helplessly watch as the love of my life suffers terribly, has devastating complications from her treatmentbut has no promise of a cure," said Lett.

"Transplant, which only works half the time, is our only hopeand all the news concerning a match for Elsahas been bad too."

Elizabeth Nega, Ronald Lett and their two children are running out of time to find Elsa a bone marrow donor. (Helen Goddard)

Since discovering that she had leukemia, Elsahas beenput through several rounds of chemotherapy, but after failing to go into remission, obtaining stem cells from a bone marrow transplant has become her only hope of recovery.

Her brother and sister in Ethiopia were her best chance, but neither were a match.

The larger issue in finding a donor for Elsa is the lack of diversity in the donor registry.

Of the 405,000 Canadians on the stem cell registry, only 800 have an African background, and none are a match for Elsa, according toChrisvan Doornwith the One Match Program.

Even among the 29 million people on the international registry, no match has been found.

Lett and Elsa's children, Lana, 8, and Lawrence, 6, have contributed to the effort.

They're in a video reading a letter appealing to Ethiopians around the world, including Canadian-Ethiopian R & B singerThe Weeknd, asking for help to save their mom.

In the meantime, Elsa's health is declining, and she's hoping for a miracle, even if it's not for her.

"If they save somebody, that's like a lotteryor a big blessing, you know.It's a big chance to get somebody to match to you and save your life.You know many people can't do this." saidNega.

People interested in registering to be a bone marrow donor can register at blood.ca,must be between 17 and 35 years old and in good health.

The test involves a cheek swab at the nearest clinicor a kit can be mailed out.

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Nepal’s sole bone marrow transplant doctor – Republica

By raymumme

Dr Bishesh Poudyal of the Civil Service Hospital in Kathmandu is the doctor who carried out all 18 transplants. At Civil the cost per transplant is between Rs 400,000 to Rs 500,000. KATHMANDU, June 7:A total of 18 bone marrow transplants have been successfully carried out in Nepal by a single doctor in Kathmandu since 2012.

A bone marrow transplant is a medical procedure performed to replace bone marrow that has been damaged or destroyed by disease, viral infection, or chemotherapy. This procedure involves transplanting blood stem cells, which travel to the bone marrow where they produce new blood cells and promote growth of new marrow.

Bone marrow is the spongy, fatty tissue inside the bones. It creates the red blood cells that carry oxygen and nutrients throughout the body, white blood cells that fight infection, and platelets that are responsible for the formation of clots.

Dr Bishesh Poudyal of the Civil Service Hospital in Kathmandu is the doctor who carried out all 18 transplants. "I am going to carry out bone marrow transplants on another six patients in near future," said Poudyal, who was born at Jawalakhel of Lalitpur.

Dr Poudyal, who passed SLC 24 years ago from Adarsha Vidya Mandir, was inspired by his father to pursue studies in hematology and bone marrow transplant. After completing his MBBS from China and MD from India under government scholarships, he started working at the Bir Hospital. "I served there for two years at Bir Hospital as per the government rule for scholarship students," he said.

Then, Dr Poudyal left the Bir Hospital as he came to know that bone marrow transplant was not possible at Bir and joined Civil Service Hospital. He also practised at the Nobel Medical College Hospital at Sinamangal where he started bone marrow transplant in 2012. "As I came to know Nobel was charging patients between Rs 800,000 to Rs 1 million per transplant, I quit the hospital," he said.

At his initiation, the Civil Hospital started bone marrow transplant about a year ago. At Civil the cost per transplant is between Rs 400,000 to Rs 500,000. The transplant recepients ranged from 22 years old to 64 years. Two patients died after about nine months of transplant. "One died of tuberculosis infection and another died of disease complications," according to Dr Poudyal.

"Bone marrow is transplanted in cancer and other blood diseases. Bone marrow is transplanted in different ways-- by treating patients' bone marrow, using siblings' and parents' bone marrow and matched unrelated donor (MUD). "We have not transplanted bone marrow under MUD category," said Dr Poudyal. "MUD is a condition of matching gene with other persons. A person's genes match those of only one percent of the population of the entire world," he added.

There is no actual data of patients with bone marrow problems in the country. However, 400 to 600 patients visit Civil Service Hospital for treatment of acute lukemia and other blood cancer cases per year. "Forty to 50 percent patients of blood cancer recover fully while the recovery rate among bone marrow recepients is 70-80 percent," said Dr Poudyal.

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Pitting avocados against leukemia stem cells – CBC.ca

By raymumme

Paul Spagnuolo is working on creating a drug with an avocado compound that targets cancer cells. (Paul Spagnuolo)

A Guelph food science researcher is getting $100,000 from the Ontario Institute for Cancer Research to fund investigations into using an avocado compound as a possible treatment for leukemia.

Paul Spagnuolo discovered that Avocatin B, a compound mainly found in avocado pits can kill leukemia stem cells in 2015.

"Getting funds to do any type of research is a reason to celebrate," said Spagnuolo told CBC News.

The funding will further his research by allowing his lab to use better equipment and collaborate with cancer researchers from the University of Toronto, Princess Margaret Cancer Centre, Ottawa University and McMaster University.

Spagnuolo's lab tested more than 800 natural compounds for their ability to kill leukemia stem cells and discovered Avocatin B was the most potent and only targetedcancer cells.

Avocatin B kills leukemia stem cells by stopping fatty acid oxidation in the cells, a process necessary for the cancer cell to digest fat as a fuel source in order to live and grow.

"Our cells can utilize glucose primarily and some other parts, but leukemia cells are rewired so that if you inhibit the oxidation process, they will die," he said.

Spagnulo and his lab are now looking to develop a way to detect whether or not Avocatin B is circulating in the blood and bone marrow.

Leukemia cells live in the bloodstream or bone marrow, so it's important for the drug to make it to those parts to kill the cancer cells.

"We want to be able to detect our drug inside the blood so that we can understand how we can formulate products better to get our product into the blood," said Spagnuolo.

Moving forward, Spagnuolo's lab will have to report to OICR quarterly, it's a condition of the funding which is spread over two years and has the possibility of renewal for another two years.

"(It's) a lot more intense than I anticipated, but I think the key here is it's very results oriented," said Spagnuolo, "There's no complacency here."

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Stem cells delivered via Skin gun can reduce second degree …

By NEVAGiles23

RenovaCare is developing breakthrough technologies to address Americas $45 billion wound and burn treatment market. Our flagship CellMist System makes use of a patients own stem cells, which are sprayed onto wounds using our novel SkinGun device.

For patients suffering severe burns and other wounds, the prospect of a quick-healing, gentle spray containing their own stem cells will be a promising alternative to conventional skin graft surgery, which can be painful, prone to complications, and slow-to-heal. Based on preliminary case studies, CellMist System patients can be treated within 90 minutes of arriving in an emergency room; a patients stem cells are isolated, processed, and sprayed on to wound sites for rapid healing.

Preliminary investigational use in Europe and the United States indicate the potential efficacy and safety of RenovaCares technologies. Clinical observations point to the potential for regeneration of new skin in as little as four days, rather than the many weeks of painful and risky recovery required by traditional skin graft techniques. These technologies are the result of nearly a decade of ongoing research and development dedicated to finding the most effective way to access the regenerative properties of a patients own skin stem cells, and the most efficient way to deliver these potent cells to heal moderate to severe skin wounds. We believe that RenovaCares CellMist System and SkinGun spray device are the worlds most advanced technologies of their kind.

This device system requires further clinical evaluation and data collection prior to submission of a premarketing application to the US FDA. At this time it is an investigational system and is not available for general use or sales in the United States.

The CellMist System RenovaCares CellMist System is comprised of two components:

Wikipedia indicates that so far the skin gun treatment has been used exclusively with second degree burns, though there is strong evidence that the treatment will be successful in treating a variety of skin wounds and skin disorders. Patients with infected wounds or with delay in wound healing are suitable for cell grafting treatment. Third-degree burns, however, completely deprive victims of both their epidermis and dermis skin levels, which exposes the tissue surrounding the muscles. The skin gun has not progressed to the point where it can be used for such advanced wounds, and these patients must seek more traditional treatment methods. The skin gun is generally not used for burn victims with anything less than a second-degree burn either. First degree-burns still maintain portions of the epidermis and can readily heal on their own, thus they do not need this expensive technology.

Currently, the skin guns applications have not been extended to include the regeneration of skin lost due to other injuries or skin diseases. It is also limited in that it is only effective immediately following the burn incident.

The average healing time for patients with second degree burns is three to four weeks. This is reduced to a matter of days with skin gun treatment

Traditional skin grafting can be risky, in that chances for infection are relatively high. The skin gun alleviates this concern because the increased speed in which the wound heals directly correlates to the decreased time the wound can be vulnerable to infection. Because of the rapid re-epithelialization associated with skin gun treatment, harmful side effects that can result from an open wound are significantly reduced. Applying the skin cells is quick and doesnt harm the patient because only a thin layer of the patients healthy skin is extracted from the body into the aqueous spray. The electronic spray distributes the skin cells uniformly without damaging the skin cells, and patients feel as if they are sprayed with salt water.

Because the skin cells are actually the patients own cells, the skin that is regenerated looks more natural than skin grown from traditional methods. During recovery, the skin cells grow into fully functional layers of the skin, including the dermis, epidermis, and blood vessels.[17] The regenerated skin leaves little scarring. The basic idea of optimizing regenerative healing techniques to damaged biological structures demonstrated by the skin gun in the future may also be applied to engineering reconstruction of vital organs, such as the heart and kidneys.

There are major limitations: the method will not work on deep burns that go through bone and muscle, specifically below the dermis. As of 2011, only several dozen patients have been treated; it remains an experimental, not a proven, method. As of 2011, the skin gun was still in its prototyping stage, since it has only treated a dozen patients in Germany and the US, compared to over 50,000 treated with Dermagraft bioengineered skin substitute. There is thus a lack of published peer reviewed clinical evidence, and no knowledge of long-term stability of the newly generated skin

Skingun Procedure

There is a seven page review of the skingun at the International Journal of Pharmacometrics and Integrated Biosciences (IJPIB)

Skingun Procedure Initially stamp-sized healthy skin of the injured patient is taken and stem cells were collected from it. Then they are harvested by using suitable enzymes. The prepared cell suspension is injected into sterile syringe and inserted into the gun. This gun helps in uniform spreading of the cells on wound. These cells will migrate, multiple, and differentiate forming a new tissue. The complete process occurs with in 2 hr. Full regeneration of skin occurs in 2 weeks and complete formation of texture tools 2-3 months

Stage 1

The CellMist Solution is a liquid suspension containing a patients own regenerative skin stem cells. A small sample (as little as a square inch) of the patients skin is quickly processed to liberate the stem cells from surrounding tissue. The resulting product is referred to as the CellMist Solution. The CellMist Solution is placed in the SkinGun for spray application onto the patients wound.

The CellMist Solution, containing the patients stem cells, is transferred to the SkinGun. The SkinGun sprays the cells onto wound sites to begin healing. Unlike conventional aerosol and pump systems, our next-generation fluid sprayer does not expose fragile cells to strong forces that can tear them apart. Instead our SkinGun gently delivers the CellMist Solution directly to the wound site using a positive-pressure air stream.

SOURCES RenovaCare, Wikipedia, International Journal of Pharmacometrics and Integrated Biosciences (IJPIB)

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This Stem Cell Gun Helps Burn Victims Grow New Skin Faster – GineersNow (press release) (registration) (blog)

By Dr. Matthew Watson

New skin begins to regenerate as soon as 4 days after this has been sprayed on patients, compared to skin grafting surgery which may take weeks of pain and possible infections.

Patients who suffer from burn wounds and scars that cant heal on their own only have 1 option: skin graft surgery. This can be very painful, and usually leads to several other complications, and it takes forever to actually heal.

However, RenovaCare has developed a new breakthrough piece of tech: CellMist, a gun that sprays stem cells into a patients burn wound, effectively allowing healthy skin to grow out of it.

It works literally like we described it as. Within 90 minutes of a patient being brought to the emergency room, they stem cells are isolated, processed, put in a liquid suspension, and then loaded into the CellMist gun. CellMist then gently sprays the stem cells onto the patients burn wound.

Tests conducted in Europe and the US have shown that new skin begins to regenerate as soon as 4 days after its been sprayed on patients, compared to skin grafting surgery which may take weeks of pain and possible infections.

Source: Next Big Future

So far, CellMist has only been used to treat second degree burns. However, evidence has shown that it can be used for other skin wounds and skin disorders. They dont think itll work for third degree burns though, as this kind of burn wound has damaged the entire epidermis and dermis levels. CellMist isnt advanced enough to heal such a deep burn wound, and victims would unfortunately have to stick to more traditional methods of treatment. First degree burn wounds on the other hand only barely touch the epidermis, meaning it can still heal on its own, thus not needing such an expensive piece of technology.

It is good to note that even though there is evidence that it might be able to heal things other than burns, CellMist wasnt built to regenerate skin lost from other kinds of injuries or diseases. Its also pretty limited, because as we stated earlier, it should be used immediately after the burn incident has occurred, or else it wont work.

Its still a pretty handy invention. The reason why skin grafting is so risky is because it involves cutting the skin open and leaving it open for 3-4 weeks. This means that nasty bacteria and fungi can easily get into the open wound within that time, causing several infections and complications.

Source: Next Big Future

With CellMist, however, simply involves extracting a thin layer of the patients healthy skin and stem cells and turning it into a spray, and then distributing the stem cells into the burn wound evenly, without damaging other healthy skin cells. The healing time only takes a few days, so there is little chance for an infection to occur if treated properly. And since the patients own skin cells are used in the process, the regenerated skin looks much more natural, with only little scarring. The stem cells grow into fully functioning layers of skin, from the dermis, to the epidermis, to even blood vessels.

Hopefully, this cool new invention will make way for other forms of stem cell treatments for the reconstruction of other organs, like ones heart and kidneys.

Article Sources:

Deccan Chronicle

Next Big Future

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Chippewa Valley Schools hosts bone marrow registration drive – The Macomb Daily

By NEVAGiles23

As a third grade teacher at Ottawa Elementary School, Kelly Gianotti teaches students many important life lessons along with reading and math.

The most important lesson she has instructed was taught by example: how to save the life of a blood cancer patient.

Gianotti donated her stem cells in 2013 to help save the life of a blood cancer patient. The patient was in need of a bone marrow stem cell transplant and had no donor match in her family.

I had seen a flier at a local gym for a high school student who was looking for a match. That intrigued me. I went online to register, Gianotti said.

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A year later Gianotti learned she was a potential match, but not for the high school patient. She went through more testing and did the outpatient donation procedure.

Gianotti later found out her donation assisted MaryAnn Hastings, who lived near Boston, Mass. The two chatted via e-mail and were able to meet in 2016, when Gianotti traveled to Boston.

The lady I donated for died last February of a different type of cancer. I wanted to honor her and spread the word, Gianotti said, adding that Hastings family indicated she was able to give Hastings three extra years of life with her donation.

The donation experience motivated Gianotti to host the first DKMS bone marrow registration drive through Chippewa Valley Schools district. DKMS is an international nonprofit organization dedicated to the fight against blood cancer and blood disorders, according to its website.

The goal of the drive is to help register potential donors. It will be held Tuesday from 4 p.m. to 9 p.m. at Cheyenne Elementary School in Macomb Township. Gianotti said she hopes to register between 100 and 200 potential donors.

Requirements to join the bone marrow registry are that the donor be in good health and between the ages of 18 and 55. The process involves filling out a form, understanding the donation methods and swabbing the inside of each cheek for 30 seconds with a cotton swab. Donors swab their cheeks in a circular motion.

There is no cost to register, although donations are accepted. The donations assist DKMS in covering the $65 registration processing fee.

According to DKMS, 70 percent of people suffering from blood-related illnesses rely on donors other than their families.

If selected as a match for a patient, there are two different methods of donation, according to the DKMS website.

According to the DKMS website, a donation method used in about 25 percent of cases is a one or two hour surgical procedure performed under anesthesia to collect marrow cells from the back of the pelvic bone using a syringe.

To obtain more information about the drive or to make a monetary donation, visit fb.com/cvsgetsswabbed. Those who want to join the bone marrow registry but are unable to attend the June 6 drive can register at dkms.org.

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Bone marrow transplant patient credits positive recovery to staying close to home – KSFY

By Sykes24Tracey

Multiple myeloma is cancer that involves our bone marrow with a specific cell called a plasma cell that patients can develop. Most patients will need a bone marrow transplant.

Patients needing bone marrow transplants dont have to travel far to receive this potentially life-saving transplant.

The actual day of the diagnosis was November 18th of 2015 and it was a diagnosis for multiple myeloma, said Steven Simpson.

Simpson was ready to fight from that day on. He learned from Dr. Kelly McCaul, the director of Avera Hematology Transplant Program, that he would need a bone marrow transplant.

There are many different types of transplant that we do. Theres basically an autologous transplant where patients would be their own donors for their stem cells and then theres allogenic transplant which are some sort of donor process. And so Steve has multiple myeloma. We would normally look at autologous transplant as the preferred pathway for patients with that disease, said Dr. McCaul.

Weve never had to leave anywhere other than here. This is it, said Simpson.

Simpson and his immediate family live no further than 20 minutes away from Avera McKenna so getting the transplant elsewhere was out of the question. But that didnt come without resistance from his insurance company.

Youre asking somebody to go three or four hours out of the way minimum for a period of time that could last anywhere from a week to whatever the process is. You lose your doctors. You lose the ability to have any local family support there as you need them and you dont really know what youre getting into. You just know what youre told, said Simpson.

Simpson and his insurance company worked together and was able to stay at Avera for his transplant.

I came in the day before scheduled for the transplant but left three hours after the transplant because I didnt have any reactions. Plus, we all knew that I had somebody available to watch me 24/7 for the period of time that we would have. The fact that you have your doctors here, your oncologist, your lab people, your nursing staff, everybodys here. They know who you are, said Simpson.

17 years ago when I first looked at this program one of the big things I looked at was the need in the community and it was felt from my perspective, and obviously Avera, that our need in the community was high. And it allows patients to stay within the community, close to family members, without having to drive four, five hours away, said Dr. McCaul.

Today, Simpson is well on his way to feeling like his old self, something he credits to staying close to home for his transplant.

For more information just call 877-AT-AVERA

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Meet the Cambridge scientist on verge of curing Multiple Sclerosis – Cambridge News

By LizaAVILA

Dr Su Metcalfe is sitting quietly reading through some documents in the lobby of the Judge Business School when I arrive for our interview. It would be easy to walk right past her and not know you were in the presence of a woman who could be on the verge of curing multiple sclerosis.

MS, an auto-immune condition which affects 2.3 million people around the world, attacks cells in the brain and the spinal cord, causing an array of physical and mental side effects including blindness and muscle weakness. At the moment theres no cure, but Su and her company, LIFNano, hope to change that.

Some people get progressive MS, so go straight to the severe form of the disease, but the majority have a relapsing or remitting version, she says.

It can start from the age of 30, and theres no cure, so all you can do is suppress the immune response, but the drugs that do that have side effects, and you cant repair the brain. The cost of those drugs is very high, and in the UK there are a lot of people who dont get treated at all.

But now a solution could be in sight thanks to Su, who has married one of the bodys cleverest functions with some cutting-edge technology. The natural side of the equation is provided by a stem cell particle called a LIF.

Su was working at the universitys department of surgery when she made her big breakthrough: I was looking to see what controls the immune response and stops it auto-attacking us, she explains.

I discovered a small binary switch, controlled by a LIF, which regulates inside the immune cell itself. LIF is able to control the cell to ensure it doesnt attack your own body but then releases the attack when needed.

That LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy. In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged.

So I thought, this is fantastic. We can treat auto-immune disease, and weve got something to treat MS, which attacks both the brain and the spinal cord. So you have a double whammy that can stop and reverse the auto-immunity, and also repair the damage caused in the brain.

Presumably Su, who has been in Cambridge since she was an undergraduate but retains a soft accent from her native Yorkshire, was dancing a jig of delight around her lab at this point, but she soon hit a snag; the LIF could only survive outside the cell for 20 minutes before being broken down by the body, meaning there was not enough time to deploy it in a therapy. And this is where the technology, in the form of nano-particles, comes in.

They are made from the same material as soluble stitches, so theyre compatible with the body and they slowly dissolve, says Su.

We load the cargo of the LIF into those particles, which become the delivery device that slowly dissolve and deliver the LIF over five days. The nano-particle itself is a protective environment, and the enzymes that break it down cant access it. You can also decorate the surface of the particles with antibodies, so it becomes a homing device that can target specific parts of the brain, for example. So you get the right dose, in the right place, and at the right time.

The particles themselves were developed at Yale University, which is listed as co-inventor with Su on the IP. But LIFNano has the worldwide licence to deploy them, and Su believes we are on the verge of a step-change in medicine.

She says: Nano-medicine is a new era, and big pharma has already entered this space to deliver drugs while trying to avoid the side effects. The quantum leap is to actually go into biologics and tap into the natural pathways of the body.

Were not using any drugs, were simply switching on the bodys own systems of self-tolerance and repair. There arent any side effects because all were doing is tipping the balance. Auto-immunity happens when that balance has gone awry slightly, and we simply reset that. Once youve done that, it becomes self-sustaining and you dont have to keep giving therapy, because the body has its balance back.

LIFNano has already attracted two major funding awards, from drug firm Merck and the Governments Innovate UK agency. Su herself is something of a novice when it comes to business, but has recruited cannily in the form of chairman Florian Kemmerich and ceo Oliver Jarry, both experienced operators in the pharma sector. With the support of the Judge, the company hopes to attract more investment, with the aim of starting clinical trials in 2020.

The 2020 date is ambitious, but with the funding weve got and the funding were hoping to raise, it should be possible, says Su.

Weve got everything we need in place to make the nano-particles in a clinically compliant manner, its just a case of flicking the switch when we have the money. Were looking at VCs and big pharma, because they have a strong interest in this area. Were doing all our pre-clinical work concurrently while bringing in the major funds the company needs to go forward in its own right.

Immune cells have been a big part of Sus career, and as we talk, her passion for her subject is obvious. I wanted to understand something that was so simple on one level but also so complex, she says.

The immune cell is the only single cell in the body that is its own unity, so it functions alone. Its probably one the most powerful cells in the body because it can kill you, and if you havent got it you die because you havent got it.

And MS may just be the start for LIFNano.

MS is our key driver at the moment, but its going to be leading through to other major auto-immune disease areas, Su adds.

Psoriasis is high up on our list, and diabetes is another. Downstream there are all the dementias, because a LIF is a major health factor for the brain. So if we can get it into the brain we can start protecting against dementia.

Now that would be something.

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Meet the Cambridge scientist on verge of curing Multiple Sclerosis - Cambridge News

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What Are Stem Cells – Checkbiotech.org (press release)

By NEVAGiles23

Tissue-specific stem cells

Tissue-specific stem cells, which are sometimes referred to as adult or somatic stem cells, are already somewhat specialized and can produce some or all of the mature cell types found within the particular tissue or organ in which they reside. Because of their ability to generate multiple, organ-specific, cell types, they are described as multipotent. For example, stem cells found

Stem cells are the foundation cells for every organ and tissue in our bodies. The highly specialized cells that make up these tissues originally came from an initial pool of stem cells formed shortly after fertilization. Throughout our lives, we continue to rely on stem cells to replace injured tissues and cells that are lost every day, such as those in our skin, hair, blood and the lining of our gut. Stem cells have two key properties: 1) the ability to self-renew, dividing in a way that makes copies of themselves, and 2) the ability to differentiate, giving rise to the mature types of cells that make up our organs and tissues.

Tissue-specific stem cells Tissue-specific stem cells, which are sometimes referred to as adult or somatic stem cells, are already somewhat specialized and can produce some or all of the mature cell types found within the particular tissue or organ in which they reside. Because of their ability to generate multiple, organ-specific, cell types, they are described as multipotent. For example, stem cells found within the adult brain are capable of making neurons and two types of glial cells, astrocytes and oligodendrocytes. Tissue-specific stem cells have been found in several organs that need to continuously replenish themselves, such as the blood, skin and gut and have even been found in other, less regenerative, organs such as the brain. These types of stem cells represent a very small population and are often buried deep within a given tissue, making them difficult to identify, isolate and grow in a laboratory setting. Neuron Dr. Gerry Shaw, EnCor Biotechnology Inc. Astrocyte Abcam Inc. Oligodendrocyte Dhaunchak and Nave (2007). Proc Natl Acad Sci USA 104:17813-8 http://www.isscr.org Embryonic stem cells Embryonic stem cells have been derived from a variety of species, including humans, and are described as pluripotent, meaning that they can generate all the different types of cells in the body. Embryonic stem cells can be obtained from the blastocyst, a very early stage of development that consists of a mostly hollow ball of approximately 150-200 cells and is barely visible to the naked eye. At this stage, there are no organs, not even blood, just an inner cell mass from which embryonic stem cells can be obtained. Human embryonic stem cells are derived primarily from blastocysts that were created by in vitro fertilization (IVF) for assisted reproduction but were no longer needed. The fertilized egg and the cells that immediately arise in the first few divisions are totipotent. This means that, under the right conditions, they can generate a viable embryo (including support tissues such as the placenta). Within a matter of days, however, these cells transition to become pluripotent. None of the currently studied embryonic stem cell lines are alone capable of generating a viable embryo (i.e., they are pluripotent, not totipotent). Why are embryonic stem cells so valuable? Unlike tissue-specific (adult) stem cells, embryonic stem cells have the potential to generate every cell type found in the body. Just as importantly, these cells can, under the right conditions, be grown and expanded indefinitely in this unspecialized or undifferentiated state. These cells help researchers learn about early human developmental processes that are otherwise inaccessible, study diseases and establish strategies that could ultimately lead to therapies designed to replace or restore damaged tissues. Induced pluripotent stem cells One of the hottest topics in stem cell research today is the study of induced pluripotent stem cells (iPS cells). These are adult cells (e.g., skin cells) that are engineered, or reprogrammed, to become pluripotent, i.e., behave like an embryonic stem cell. While these iPS cells share many of the same characteristics of embryonic stem cells, including the ability to give rise to all the cell types in the body, it is important to understand that they are not identical. The original iPS cells were produced by using viruses to insert extra copies of three to four genes known to be important in embryonic stem cells into the specialized cell. It is not yet completely understood how these three to four reprogramming genes are able to induce pluripotency; this question is the focus of ongoing research. In addition, recent studies have focused on alternative ways of reprogramming cells using methods that are safer for use in clinical settings. Disease- or patient-specific pluripotent stem cells One of the major advantages of iPS cells, and one of the reasons that researchers are very interested in studying them, is that they are a very good way to make pluripotent stem cell lines that are specific to a disease or even to an individual patient. Disease-specific stem cells are powerful tools for studying the cause of a particular disease and then for testing drugs or discovering other approaches to treat or cure that disease. The development of patientspecific stem cells is also very attractive for cell therapy, as these cell lines are from the patient themselves and may minimize some of the serious complications of rejection and immunosuppression that can occur following transplants from unrelated donors. Moving stem cells into the clinic Clinical translation is the process used to turn scientific knowledge into real world medical treatments. Researchers take what they have learned about how a tissue usually works and what goes wrong in a particular disease or injury and use this information to develop new ways to diagnose, stop or fix what goes wrong. Before being marketed or adopted as standard of care, most treatments are tested through clinical trials. Sometimes, in attempting new surgical techniques or where the disease or condition is rare and does not have a large enough group of people to form a clinical trial, certain treatments might be tried on one or two people, a form of testing sometimes referred to as innovative medicine. For more information on how science becomes medicine, please visit http://www.closerlookatstemcells.org. Current therapies Blood stem cells are currently the most frequently used stem cells for therapy. For more than 50 years, doctors have been using bone marrow transplants to transfer blood stem cells to patients, and more advanced techniques for collecting blood stem cells are now being used to treat leukemia, lymphoma and several inherited blood disorders. Umbilical cord blood, like bone marrow, is often collected as a source of blood stem cells and in certain cases is being used as an alternative to bone marrow transplantation. Additionally, some bone, skin and corneal diseases or injuries can be treated by grafting tissues that are derived from or maintained by stem cells. These therapies have also been shown to be safe and effective. Potential therapies Other stem cell treatments, while promising, are still at very early experimental stages. For example, the mesenchymal stem cell, found throughout the body including in the bone marrow, can be directed to become bone, cartilage, fat and possibly even muscle. In certain experimental models, these cells also have some ability to modify immune functions. These abilities have created considerable interest in developing ways of using mesenchymal stem cells to treat a range of musculoskeletal abnormalities, cardiac disease and some immune abnormalities such as graft-versus-host disease following bone marrow transplant. Remaining challenges Despite the successes we have seen so far, there are several major challenges that must be addressed before stem cells can be used as cell therapies to treat a wider range of diseases. First, we need to identify an abundant source of stem cells. Identifying, isolating and growing the right kind of stem cell, particularly in the case of rare adult stem cells, are painstaking and difficult processes. Pluripotent stem cells, such as embryonic stem cells, can be grown indefinitely in the lab and have the advantage of having the potential to become any cell in the body, but these processes are again very complex and must be tightly controlled. iPS cells, while promising, are also limited by these concerns. In both cases, considerable work remains to be done to ensure that these cells can be isolated and used safely and routinely. Second, as with organ transplants, it is very important to have a close match between the donor tissue and the recipient; the more closely the tissue matches the recipient, the lower the risk of rejection. Being able to avoid the lifelong use of immunosuppressants would also be preferable. The discovery of iPS cells has opened the door to developing patient-specific pluripotent stem cell lines that can later be developed into a needed cell type without the problems of rejection and immunosuppression that occur from transplants from unrelated donors. Third, a system for delivering the cells to the right part of the body must be developed. Once in the right location, the new cells must then be encouraged to integrate and function together with the bodys other cells. http://www.isscr.org Glossary Blastocyst A very early embryo that has the shape of a ball and consists of approximately 150-200 cells. It contains the inner cell mass, from which embryonic stem cells are derived, and an outer layer of cells called the trophoblast that forms the placenta. Cell line Cells that can be maintained and grown in a dish outside of the body. Clinical translation The process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury. Differentiation The process of development with an increase in the level of organization or complexity of a cell or tissue, accompanied by a more specialized function. Embryo The early developing organism; this term denotes the period of development between the fertilized egg and the fetal stage. Embryonic stem cell Cells derived from very early in development, usually the inner cell mass of a developing blastocyst. These cells are self-renewing (can replicate themselves) and pluripotent (can form all cell types found in the body). Induced pluripotent stem (iPS) cell Induced pluripotent cells (iPS cells) are stem cells that were engineered (induced) from non-pluripotent cells to become pluripotent. In other words, a cell with a specialized function (for example, a skin cell) that has been reprogrammed to an unspecialized state similar to that of an embryonic stem cell. Innovative medicine Treatments that are performed on a small number of people and are designed to test a novel technique or treat a rare disease. These are done outside of a typical clinical trial framework. In vitro fertilization A procedure in which an egg cell and sperm cells are brought together in a dish to fertilize the egg. The fertilized egg will start dividing and, after several divisions, forms the embryo that can be implanted into the womb of a woman and give rise to pregnancy. Mesenchymal stem cells Mesenchymal stem cells were originally discovered in the bone marrow, but have since been found throughout the body and can give rise to a large number of connective tissue types such as bone, cartilage and fat. Multipotent stem cells Stem cells that can give rise to several different types of specialized cells, but in contrast to a pluripotent stem cell, are restricted to a certain organ or tissue types. For example, blood stem cells are multipotent cells that can produce all the different cell types that make up the blood but not the cells of other organs such as the liver or brain. Pluripotent stem cells Stem cells that can become all the cell types that are found in an implanted embryo, fetus or developed organism. Embryonic stem cells are pluripotent stem cells. Self-renewal The process by which a cell divides to generate another cell that has the same potential. Stem cells Cells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells. Tissue-specific stem cells (also known as adult or somatic stem cells) Stem cells found in different tissues of the body that can give rise to some or all of the mature cell types found within the particular tissue or organ from which they came, i.e., blood stem cells can give rise to all the cells that make up the blood, but not the cells of organs such as the liver or brain. Totipotent stem cells Stem cells that, under the right conditions, are wholly capable of generating a viable embryo (including the placenta) and, for humans, exist until about four days after fertilization, prior to the blastocyst stage from which embryonic stem cells are derived.

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Stem Cell Research Facts – Adult Stem Cell Success

By Dr. Matthew Watson

"I Will Walk Again" The Laura Dominguez Story If there was ever a woman on a mission, its Laura Dominguez. Doctors once told her shed never walk again. And while shes not ready to run a marathon, shes already proving them wrong, with the best yet to come.

An oil spill on a San Antonio freeway is blamed for the car crash that sent Laura and her brother directly into a retaining wall one summer afternoon in 2001. Laura was just 16 years old at the time and the crash left her completely paralyzed from the neck down. Surgeons say she suffered whats known as a C6 vertebrae fracture that severely damaged her spinal cord.

I refused to accept their prognosis that I never would walk again and began searching for other options, says Laura. After stays in several hospitals for nearly a year, Laura and her mother relocated to San Diego, CA so that she could undergo extensive physical therapy. While in California, they met a family whose daughter was suffering from a similar spinal cord injury. They were also looking for other alternatives to deal with spinal cord injuries.

After extensive research and consultations with medical experts in the field of spinal cord injuries, they decided to explore a groundbreaking new surgical procedure using adult stem cells pioneered by Dr. Carlos Lima of Portugal.

The surgery involved the removal of tissue from the olfactory sinus area at the back of the nose--and transplanting it into the spinal cord at the injury site. Both procedures, the harvesting of the tissue and the transplant, were done at the same time. Laura was the tenth person in the world and the second American to have this procedure done and was featured in a special report by PBS called Miracle Cell. (Link to Miracle Cell (PBS) Episode)

Following the surgery she returned to California where she continued with the physical therapy regimen, then eventually returned home to San Antonio. Upon her return home, an MRI revealed her spinal cord was beginning to heal. Approximately 70% of the lesion now looked like normal spinal cord tissue. More importantly to Laura, she began to regain feeling in parts of her upper body and within six months of the surgery regained feeling down to her abdomen.

Improvements in sensory feelings have continued until the present time. She can feel down to her hips, and has regained feeling and some movement in her legs. Lauras upper body has gained more strength and balance and one of the most evident improvements has been her ability to stand and remain standing, using a walker, and with minimal assistance. When she stands she can contract her quadriceps and hamstring muscles.

Every week it seems Im able to do something new, something different that I hadnt done the week before, says Laura.

Now Lauras story is poised to take a new, potentially groundbreaking turn. In the Fall of 2009, she traveled again to Portugal where adult stem cells were extracted from her nose for culturing. As this story is written, she is preparing to fly back to Portugal where scar tissue at her injury site will be removed and her own adult stem cells injected in the area of her original wound.

The Laura Dominguez story is not complete. The next chapter may or may not yield the results she seeksbut no one can deny the determination and courage of Laura. For her part, she has one goal in mind: I will walk again.

We shall update this site and keep you informed on her progress.

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Stem Cell Research Facts - Adult Stem Cell Success

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[ June 3, 2017 ] SpaceX rocket again set for station delivery after scientists swap mice, fruit flies Mission Reports – Spaceflight Now

By Dr. Matthew Watson

The Falcon 9 rocket is raised at launch pad 39A early Saturday for a second launch attempt. Credit: Spaceflight Now

A Falcon 9 rocket is again standing upright on launch pad 39A at NASAs Kennedy Space Center in Florida after ground teams lowered the booster Friday to swap out mice heading to the International Space Station for medical experiments.

Liftoff is set for 5:07 p.m. EDT (2107 GMT) to begin a nearly two-day journey to the space station, where the Dragon supply ship fixed to the top of the Falcon 9 rocket will arrive Monday.

The Dragon capsule, the first cargo craft SpaceX has refurbished and reused after a previous flight, is carrying nearly 6,000 pounds of experiments and equipment, including 40 mice inside specially-designed transporters for an investigation into a treatment that could combat bone loss in astronauts on long-duration space missions and osteoporosis in patients on the ground.

Once the mice arrive at the space station, astronauts will treat the rodents with NELL-1, a therapeutic treatment designed to promote bone growth, according to Chia Soo, the chief scientist for the experiment and a professor of plastic, reconstructive and orthopaedic surgery at UCLA.

Men and women past the age of 50, on the average, lose about a half-percent of bone mass per year, Soo said. But in microgravity conditions, the astronaut, on average, loses anywhere from 1 to 2 percent of bone mass per month.

She added that bone loss in astronauts has tremendous implications for humans with respect to long-term space travel or space habitation in microgravity because we end up progressively losing bone mass.

Twenty of the mice will return to Earth alive with the SpaceX Dragon supply ship in early July, the first time the commercial spacecraft has landed with live animals on-board. The 20 mice that come back alive will go to UCLAs laboratories for additional research and treatment.

The other 20 mice will remain on the space station for more observation and comparative studies with the mice on Earth. All of the animals will eventually be euthanized.

If successful, this will have tremendous implications for patients on Earth because if you look at statistics approximately one in every two to three females over the age of 50, or one in every four to five males over the age of 50, will have an osteoporosis-related fracture, Soo said.

We are hoping this study will give us some insights on how NELL-1 can work under these extreme conditions and if it can work for treating microgravity-related bone loss, which is a very accelerated, severe form of bone loss, then perhaps it can (be used) for patients one day on Earth who have bone loss due to trauma or due to aging or disease, Soo said.

After the Falcon 9 launch attempts scrub Thursday, teams lowered the launcher at pad 39A and installed a temporary white room on the Dragon capsules hatch to change out the rodent habitats and several other experiments.

The logistics are complicated, as you might imagine,Louis Stodieck, director of BioServe Space Technologies at the University of Colorado Boulder, wrote in an email to Spaceflight Now. We would normally be okay for two back-to-back launch attempts, but because orbital mechanics would not permit a launch attempt (Friday), the first scrub was automatically done for 48 hours rather than 24.

This forced us to reload with new animals and new Transporters (spaceflight habitats for the ride to space for the mice), Stodieck wrote. We plan for additional groups of mice just for such contingencies.

NASA spokesperson Dan Huot said other experiments that required a changeout for the two-day launch delay included a swarm of fruit flies launching to the space station to examine how prolonged spaceflight affects their heart function.

The hearts of the insects beat at about same rate as the human heart, making it a useful analog, scientists said.

We were back in the lab the night of the scrub setting up new egg collections and adult fly vials, said Karen Ocorr, a co-investigator on the fruit fly experiment from theSanford Burnham Research Institute. These replaced the original set of vials and have now been loaded onto the Dragon for todays attempt.

Researchers are sending between 4,000 and 6,000 fruit fly eggs to the space station, where they will hatch before coming back to Earth aboard the Dragon spacecraft.

We would like to understand the role of microgravity on astronaut heart function in order to try to prevent long-term effects when they are in space for long periods and after they come back, Ocorr said.

But there are real-world implications as well for people who are spending long periods of time in bedrest or immobilized, Ocorr said. We expect that what we find in our studies on the ISS will have implications for maintaining cardiac function in those sorts of situations.

Huot said two crystal growth expeiments and a payload to study how microgravity affects cardiac stem cells also needed to be replaced with the two-day launch delay.

Email the author.

Follow Stephen Clark on Twitter: @StephenClark1.

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Oxford University staff join bone marrow stem cell donor drive for Oxford toddler Ally Kim – Witney Gazette

By raymumme

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy.

Two-year-old Alastair Ally Kim has Chronic Granulomatous Disorder (CGD), a life-threatening condition.

He has now become the fourth person in the world to start an experimental gene therapy course at Great Ormond Street Hospital.

In the meantime, his parents have spearheaded 200 international donor drives to find their son a match, signing up 7,000 would-be donors in the process - some of whom have since been matched with other patients.

Father Andrew Kim, 37, of Hinton Waldrist near Longworth, said: We want to use whatever momentum Allys story has to help someone else. We know that matches have come through our drives for other people. Its awesome that someone will benefit from all this.

On Thursday, May 25 family friend Cathy Oliveira organised a drive at the Oxford Universitys Old Road research building, signing up 80 staff members in a day.

Ms Oliveira said: When everything happened with Ally I wanted to show support in any way we could; this is directly beneficial not just for Ally but for others.

Allys CGD means his immune system is compromised and the tiniest infection could leave him seriously ill.

His only chance of a permanent cure is a bone marrow stem cell donation, with a match likely to be of Korean or East Asian origin.

In April the youngster and mum Judy Kim, 36, an Oxford University researcher, travelled to London for him to begin a pioneering new gene therapy treatment.

After a week of chemotherapy to wipe out Allys immune system, cells taken from him are modified in a lab and re-introduced to correct the disorder.

Mr Kim said: Bone marrow would give him back 100 per cent functionality and gene therapy is 10 to 15 per cent; its enough to live in the real world, and not be scared he will die every time he gets an infection.

It has been a roller-coaster of a year, but theres nothing to do but move forward. We are really excited at the thought of him being able to come home this summer.

Blood cancer charity DKMS supported last weeks donor drive in Oxford.

Senior donor recruitment manager Joe Hallet said: Around 30 per cent of patients in need of a blood stem cell donor will find a matching donor within their own family.

The remaining 70 per cent, like Ally, will need to find an unrelated donor to have a second chance of life, so events like these are crucial.

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Want to save a life? Cuban American searches for bone marrow donor – Miami Herald

By daniellenierenberg


Miami Herald
Want to save a life? Cuban American searches for bone marrow donor
Miami Herald
According to Gift of Life, a nonprofit, Boca Raton-based bone marrow and blood stem cell registry, 55 percent of Hispanic cancer patients and 75 percent of multiracial patients are never matched, some dying while waiting to get a transplant. The data ...

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Stem Cells Fast Facts | KABC-AM – KABC

By Sykes24Tracey

(CNN) Here is some background information about stem cells.

Scientists believe that stem cell research can be used to treat medical conditions including Parkinsons disease, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis and rheumatoid arthritis.

About Stem Cells:Stem cell research focuses on embryonic stem cells and adult stem cells.

Stem cells have two characteristics that differentiate them from other types of cells:- Stem cells are unspecialized cells that replicate themselves for long periods through cell division.- Under certain physiologic or experimental conditions, stem cells can be induced to become mature cells with special functions such as the beating cells of the heart muscle or insulin-producing cells of the pancreas.

There are four classes of stem cells: totipotent, multipotent, pluripotent, and unipotent.- Totipotent stem cells that develop into cells that make up all the cells in an embryo and fetus. (Ex: The zygote/fertilized egg and the cells at the very early stages following fertilization are considered totipotent)- Multipotent stem cells can give rise to multiple types of cells, but all within a particular tissue, organ, or physiological system. (Ex: blood-forming stem cells/bone marrow cells, most often referred to as adult stem cells)- Pluripotent stem cells (ex: embryonic stem cells) can give rise to any type of cell in the body. These cells are like blank slates, and they have the potential to turn into any type of cell.- Unipotent stem cells can self-renew as well as give rise to a single mature cell type. (Ex: sperm producing cells)

Embryonic stem cells are harvested from four to six-day-old embryos. These embryos are either leftover embryos in fertility clinics or embryos created specifically for harvesting stem cells by therapeutic cloning. Only South Korean scientists claim to have successfully created human embryos via therapeutic cloning and have harvested stem cells from them.

Adult stem cells are already designated for a certain organ or tissue. Some adult stem cells can be coaxed into or be reprogrammed into turning into a different type of specialized cell within the tissue type for example, a heart stem cell can give rise to a functional heart muscle cell, but it is still unclear whether they can give rise to all different cell types of the body.

The primary role of adult stem cells is to maintain and repair the tissue in which they are found.

Uses of Stem Cell Research:Regenerative (reparative) medicine uses cell-based therapies to treat disease.

Scientists who research stem cells are trying to identify how undifferentiated stem cells become differentiated as serious medical conditions, such as cancer and birth defects, are due to abnormal cell division and differentiation.

Scientists believe stem cells can be used to generate cells and tissues that could be used for cell-based therapies as the need for donated organs and tissues outweighs the supply.

Stem cells, directed to differentiate into specific cell types, offer the possibility of a renewable source of replacement cells and tissues to treat diseases, including Parkinsons and Alzheimers diseases, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis, and rheumatoid arthritis.

Policy Debate:Cloning human embryos for stem cells is very controversial.

The goal of therapeutic cloning research is not to make babies, but to make embryonic stem cells, which can be harvested and used for cell-based therapies.

Using fertilized eggs left over at fertility clinics is also controversial because removing the stem cells destroys them.

Questions of ethics arise because embryos are destroyed as the cells are extracted, such as: When does human life begin? What is the moral status of the human embryo?

Timeline:1998 President Bill Clinton requests a National Bioethics Advisory Commission to study the question of stem cell research.

1999 The National Bioethics Advisory Commission recommends that the government allow federal funds to be used to support research on human embryonic stem cells.

2000 During his campaign, George W. Bush says he opposes any research that involves the destruction of embryos.

2000 The National Institutes of Health (NIH) issues guidelines for the use of embryonic stem cells in research, specifying that scientists receiving federal funds can use only extra embryos that would otherwise be discarded. President Clinton approves federal funding for stem cell research but Congress does not fund it.

August 9, 2001 President Bush announces he will allow federal funding for about 60 existing stem cell lines created before this date.

January 18, 2002 A panel of experts at the National Academy of Sciences (NAS) recommends a complete ban on human reproductive cloning, but supports so-called therapeutic cloning for medical purposes.

February 27, 2002 For the second time in two years, the House passes a ban on all cloning of human embryos.

July 11, 2002 The Presidents Council on Bioethics recommends a four-year ban on cloning for medical research to allow time for debate.

February 2005 South Korean scientist Hwang Woo Suk publishes a study in Science announcing he has successfully created stem cell lines using therapeutic cloning.

December 2005 Experts from Seoul National University Hwang of faking some of his research. Hwang asks to have his paper withdrawn while his work is being investigated and resigns his post.

January 10, 2006 An investigative panel from Seoul National University accuses Hwang of faking his research.

July 18, 2006 The Senate votes 63-37 to loosen President Bushs limits on federal funding for embryonic stem-cell research.

July 19, 2006 President Bush vetoes the embryonic stem-cell research bill passed by the Senate (the Stem Cell Research Enhancement Act of 2005), his first veto since taking office.

June 20, 2007 President Bush vetoes the Stem Cell Research Enhancement Act of 2007, his third veto of his presidency.

January 23, 2009 The FDA approves a request from Geron Corp. to test embryonic stem cells on eight to 10 patients with severe spinal cord injuries. This will be the worlds first test in humans of a therapy derived from human embryonic stem cells. The tests will use stem cells cultured from embryos left over in fertility clinics.

March 9, 2009 President Barack Obama signs an executive order overturning an order signed by President Bush in August 2001 that barred the NIH from funding research on embryonic stem cells beyond using 60 cell lines that existed at that time.

August 23, 2010 US District Judge Royce C. Lamberth issues a preliminary injunction that prohibits the federal funding of embryonic stem cell research.

September 9, 2010 A three-judge panel of the US Court of Appeals for the D.C. Circuit grants a request from the Justice Department to lift a temporary injunction that blocked federal funding of stem cell research.

September 28, 2010 The US Court of Appeals for the District of Columbia Circuit lifts an injunction imposed by a federal judge, thereby allowing federally funded embryonic stem-cell research to continue while the Obama Administration appeals the judges original ruling against use of public funds in such research.

October 8, 2010 The first human is injected with cells from human embryonic stem cells in a clinical trial sponsored by Geron Corp.

November 22, 2010 William Caldwell, CEO of Advanced Cell Technology, tells CNN that the FDA has granted approval for his company to start a clinical trial using cells grown from human embryonic stem cells. The treatment will be for an inherited degenerative eye disease.

April 29, 2011 The US Court of Appeals for the District of Columbia lifts an injunction, imposed last year by a federal judge, banning the Obama administration from funding embryonic stem-cell research.

May 11, 2011 Stem cell therapy in sports medicine is spotlighted after New York Yankee pitcher Bartolo Colon is revealed to have had fat and bone marrow stem cells injected into his injured elbow and shoulder while in the Dominican Republic.

July 27, 2011 Judge Lamberth dismisses a lawsuit that tried to block funding of stem cell research on human embryos.

February 13, 2012 Early research published by scientists at Cedars-Sinai Medical Center and Johns Hopkins University show that a patients own stem cells can be used to regenerate heart tissue and help undo damage caused by a heart attack. It is the first instance of therapeutic regeneration.

May 2013 Scientists make the first embryonic stem cell from human skin cells by reprogramming human skin cells back to their embryonic state, according to a study published in the journal, Cell.

April 2014 For the first time scientists are able to use cloning technologies to generate stem cells that are genetically matched to adult patients,according to a study published in the journal, Cell Stem Cell.

October 2014 Researchers say that human embryonic stem cells have restored the sight of several nearly blind patients and that their latest study shows the cells are safe to use long-term. According to a report published in The Lancet, the researchers transplanted stem cells into 18 patients with severe vision loss as a result of two types of macular degeneration.

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Scientists Hope to Use Stem Cells to Reverse Death in … – Futurism – Futurism

By raymumme

In BriefBioquark is about to begin a trial that will attempt to bringbrain-dead patients back to life using stem cells. However, thetrial is raising numerous scientific and ethical questions forother experts in the field. Back From The Dead

Researchers seem to be setting their sights on increasinglylofty goals when it comes to the human body from the worlds first human head transplant, to fighting aging, and now reversing death altogether. Yes, you read that right. A company called Bioquarkhopes to bring people who have been declared clinically brain-dead back to life. The Philadelphia-based biotech company is expected to start on the project later this year.

This trial was originally intended to go forward in 2016 in India, but regulators shut it down. Assuming this plan will be substantially similar, it will enroll 20 patients who will undergo various treatments. The stem cell injection will come first, with the stem cells isolated from that patients own blood or fat. Next, the protein blend gets injected directly into the spinal cord, which is intended to foster growth of new neurons. The laser therapy and nerve stimulation follow for 15 days, with the aim of prompting the neurons to make connections. Meanwhile, the researchers will monitor both behavior and EEGs for any signs of the treatment causing any changes.

While there is some basis in science for each step in the process, the entire regimen is under major scrutiny. The electrical stimulation of the median nerve has been tested, but most evidence exists in the form of case studies. Dr. Ed Cooper has described dozens of these cases, and indicates that the technique can have some limited success in some patients in comas. However, comas and brain death are very different, and Bioquarks process raises more questions for most researchers than it answers.

One issue researchers are raising about this study is informed consent. How can participants in the trial consent, and how should researchers complete their trial paperwork given that the participants are legally dead and how can brain death be conclusively confirmed, anyway? What would happen if any brain activity did return, and what would the patients mental state be? Could anything beyond extreme brain damage even be possible?

As reported by Stat News, In 2016, neurologist Dr. Ariane Lewis and bioethicist Arthur Caplan wrote in Critical Care that the trial is dubious, has no scientific foundation, and suffers from an at best, ethically questionable, and at worst, outright unethical nature. According to Stat News, despite his earlier work with electrical stimulation of the median nerve, Dr. Cooper also doubts Bioquarks method, and feels there is no way this technique could work on someone who is brain-dead. The technique, he said, relies on there being a functional brain stem one of the structures that most motor neurons go through before connecting with the cortex proper. If theres no functional brain stem, then it cant work.

Pediatric surgeon Charles Cox, who is not involved in Bioquarks work, agrees with Cooper, commenting to Stat News on Bioquarks full protocol, its not the absolute craziest thing Ive ever heard, but I think the probability of that working is next to zero. I think [someone reviving] would technically be a miracle.

Pastor remains optimistic about Bioquarks protocol. I give us a pretty good chance, he said. I just think its a matter of putting it all together and getting the right people and the right minds on it.

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SpaceX to launch CU-built heart, bone health experiments to space station – CU Boulder Today

By Dr. Matthew Watson

Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch has been rescheduled for Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

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Trends in Drug Discovery Outsourcing: A Perspective – BSA bureau (press release)

By raymumme

Seven drugs of the current top-10 best selling drugs are biologics, the penetration of biologic drugs is expected to reach 30% by 2020 of the global pharmaceutical market and some of the key modalities include monoclonal antibodies, recombinant proteins, peptides, cell and gene therapy products.

Dr Subir Basak Chief Business Officer, GVK Biosciences

Over the last two decades, the Pharmaceutical industry has seen a radical change. The unprecedented downsizing of the internal discovery of big pharmaceuticals, patent expiration, shift towards biologics have seen a surge in the externalization and outsourcing activities. As the industry is looking for new sources of discovery and innovation with limited resources, there is a growing preference to move towards externalization and willingness to embrace the concept of outsourcing.

Seven drugs of the current top- 10 best selling drugs are biologics, the penetration of biologic drugs is expected to reach 30% by 2020 of the global pharmaceutical market and some of the key modalities include monoclonal antibodies, recombinant proteins, peptides, cell and gene therapy products. Global R&D spend in the biopharmaceutical industry is estimated to be $194 billion in 2016 and according to industry experts, 75-80% of the expenses can be outsourced. However, current penetration rate is around 58% which presents a huge opportunity for the CROs to tap the Trends in Drug Discovery Outsourcing: A Perspective market. The global pharmaceutical outsourcing market was estimated to be $113.7 billion in 2016 and out of which 49% is accounted for CROs. Among the $55.7 billion CRO market, 31.2% accounts for discovery-based service i.e. $17.4 billion in 2016 and the remaining 68.8% accounts for Preclinical and clinical services.

Biology related services segment is a high growth area with huge potential and expected to grow faster at a CAGR of 17.2% compared to the small molecules segment due to increase in budget allocations for R&D by biopharmaceutical companies.

The drug discovery CRO industry is witnessing increased consolidation. Many Asia-based companies are increasing their foothold in Europe and North America. GVK BIO, a Contract Research & Development Organization (CRDO) from India has taken over Aragen Bioscience. Aragen has early stage discovery biologics capabilities and played a leading role in oncology and fibrosis based animal models for preclinical biotechs in bay area. Similarly, ChemPartner established research facility in South San Francisco. Also, WuXi AppTec acquired HD Biosciences (HDB), a biology focused preclinical drug discovery CRO.

Advancement in drug discovery technologies such as iPS cells, automated high content screening, patch clamp, gene editing and DNAencoded libraries have expedited the drug discovery process with increased efficiency. There is an increased interest in the use of DNAencoded libraries (small molecules tagged with DNAs) by major pharma companies.Majority of the companies offering DNA-encoded library services are from US (DiCE, X-Chem, Ensemble therapeutics) and Europe (Nuevolution, Vipergen, Cominnex, Philochem). Thereseems to be very little competition in APAC. This trend should push some of the CROs from APAC region to acquire companies with proprietary technology in DNA-encoded libraries or to build capabilities and this seems likely to be a focus point for majority of the CROs especially from APAC.

Evolving business models including risk-based and insourcing are facilitating better collaboration between pharmaceutical companies and CROs. Some of the companies established a new business model known as insourcing which is a new sourcing for pharma where CROs work on-site at customer location in an integrated fashion. This new model provides outstanding performance with efficient cost and time.

CROs should build capabilities to differentiate in the area of Target Identification/Target Validation on how to use human disease pathology knowledge/primary tissues from humans clubbed with Omics knowledge to further validate the concepts. As most of the CROs propose targets from literature and sponsor companies consider it as risky option to invest in such projects without substantial evidences. As a de-risking strategy some CROs are investing internally and validating the concept by siRNA, knockdown approaches and take the concept to a level-up and then approach the sponsors companies who are working in similar area. This approach would increase the sponsor confidence in the CRO program.

There is a huge demand for the novel therapeutics addressing the unmet needs, for example, there are no FDA approved drugs or any therapies for NASH treatment and there is a tremendous opportunity for CROs to work on novel targets, preclinical models and biomarker to come-up with some early stage assets for partnering. Owing to market attractiveness, there is funding provided by venture capitalists to promising players, while some investors are even launching new companies to specifically work on NASH projects. For instance in February 2017, Versant Ventures formed Jecure Therapeutics through $20 million investment for NASH program development. Similarly, Third Rock Ventures formed Pliant Therapeutics with $45 million investment for TGF- signaling based NASH treatment. These companies could potentially outsource majority of the work to CROs in APAC region.

Asia is emerging as a preferred destination for outsourcing drug discovery activities due to the vast availability of skilled manpower, lower costs, favorable regulatory environment and quality data. In addition the local governments are focusing on development of healthcare and pharmaceutical industryby ensuring focus on high quality & compliance in terms of higher regulatory surveillance and training programs. Japan being the second largest pharmaceutical market in the world provides huge opportunity for CROs from APAC. Chinese and Indian pharmaceutical markets are one of the fastest growing in the world and are considered to be the preferred locations for drug discovery outsourcing primarily because of the end-to-end technological capabilities developed over several years. Asian CROs have strong capabilities in biologics research services and built new technology platforms for high-throughput screening, genomics and proteomics research panel screening, enzymatic, and binding assays. They are also well equipped with transgenic and disease animal models that have been developed for target validation, efficacy, and safety studies, thereby providing clients with end-to-end services. Indian CROs typically focus more on new chemical entities and offer integrated discovery services at much lower cost.

Therapeutic area gap analysis research indicates that the key contract research organizations in Asia pacific region are majorly focusing on Oncology, metabolic diseases, Inflammation and CNS. However, majority of Pharma companies in addition to the above therapeutic areas are also focusing on other areas like cardiovascular, immunology, infectious diseases. Since there is a high gap in these therapeutic areas, the CROs should increase their focus in order to tap the opportunity.

Growth in biologics research and orphan drugs, innovative technological platforms and evolving business models encourage pharma and biotech companies to outsource. Even though, big pharma is moving towards research institutions and academia to accelerate knowledge and leverage innovation and technology platforms, they lack the infrastructure to move the drugs from early stages of drug discovery. These factors are expected to enhance drug discovery outsourcing market in APAC region for the coming years.

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