Ally Little described the past month of her life as a nightmare from which she just cant wake up.

On Dec. 22, the University of Southern Maine soccer player learned she had a rare and life-threatening disease in which her bone marrow stops producing healthy blood cells. However, the words severe aplastic anemia meant nothing to Little at the time.

Its really hard because I didnt know what this was before I had it, said Little, a 20-year-old sophomore from Stoneham, Massachusetts. No one has really heard of aplastic anemia or what the treatment is.

A bone marrow transplant is the cure for this disease, and Little has yet to find a matching donor. Littles teammates have organized a bone marrow donor registry drive from 9 a.m. to 1 p.m. Wednesday at Abromson Mezzanine at the USM Portland campus and from 2:30 to 5:30 p.m. at Costello Complex at the Gorham campus.

Diagnosed during winter break, Little broke the news to her teammates on social media.

It hit home, said USM womens soccer coach Lisa Petruccelli. This is really the first time someone their age at this juncture is struggling with something like this.

Littles initial symptoms didnt seem serious. She started getting pounding headaches around Thanksgiving, but she had gotten headaches before. Physical activities such as skiing or working out for soccer became unusually exhausting, which Little attributed to dehydration. She didnt go to her doctor until she noticed blood in her stool.

(Aplastic anemia) is believed to be an autoimmune system gone wrong, said Paul Scribner, Senior Director of Patient Advocacy Programs with the Aplastic Anemia and MDS International Foundation (AAMDS). The disease usually results from the destruction of bone marrow stem cells by the immune system. Other symptoms include infections and the tendency to bruise and bleed easily. With such innocuous warning signs, Scribner said a lot of people find out when they go to their doctor because theyre feeling run down.

After bloodwork, Little was told that her results were very abnormal. She spent the next few days in the hospital undergoing tests while doctors prepared her for the worst case scenario leukemia.

That was obviously horrifying, Little said. We didnt find out until about three days later that it was severe aplastic anemia.

Aplastic anemia is rare and can occur at any age. In the United States, about 600 to 900 people are diagnosed each year, according to AAMDS. The disease is considered severe when all three types of blood cells red blood cells (carry oxygen), white blood cells (fight infections) and platelets (help blood to clot) are very low in number.

I was kind of relieved it wasnt cancer, Little said. Then, doctors explained to me that its really not that good. It was devastating.

Little couldnt go back to school. With her compromised immune system, crowds are off limits. She cant play contact sports or do anything that could put her at risk of internal bleeding. She gets blood transfusions every week, and she can tell when shes due for another by the dizziness and headaches she gets. The long-term risk of too many transfusions, Scribner said, is iron overload.

Most days, I feel OK, Little said. I dont really feel sick, which is good. But its hard to remember I cant do certain things.

Little is buying time until she can get a bone marrow transplant. Bone marrow is the spongy tissue inside of the bones that produces the bodys blood cells. She didnt find a match among her family or with Be The Match a national bone marrow registry that contains 22.5 million adult donors.

Registering at the drive is simple. Potential donors must be between 18-44 years old and fill out basic paperwork and get their cheek swabbed to have their tissue type added to the registry a process that takes just a few minutes. After that, they will remain registered until age 61, unless they withdraw.

However for those in need of bone marrow finding a perfect match is not so easy.

Think about Megabucks and how hard it is to match that, said Jackie McLoon, Assistant Account Executive with Rhode Island Blood Center as well as a bone marrow donor. McLoon, a representative with Be the Match, has helped the USM soccer team organize its drive. Everyday, there are donors getting added to the database. Hopefully, her match shows up one of these days.

Only 30 percent of patients in need of a marrow transplant have a matching donor in their family. Be The Match helps the 14,000 patients a year who suffer from leukemia, lymphoma or a variety of bone marrow functioning diseases. McLoon said a protein called human leukocyte antigen (HLA) is used to match patients with donors, and potential matches will then undergo bloodwork to determine if they would be a good fit. Only about 1 in 500 registrants go on to actually donate marrow.

There are 10 things that they are supposed to match, Little said. They think one of my 10 is very rare.

But her teammates are optimistic. On Saturday, they attended a home basketball game clad in T-shirts adorned with the phrase: All for Ally. They gushed about Littles kind personality and reminisced about all the times they crashed in her room.

Shes the best teammate ever. Shes so sweet oh my god, I love her, said Jessica Preble, a sophomore on the team. If you ask anything of her, shell drop everything and do it.

This team is kind of used to bad things happening to our girls, said Dayna Staffiere, noting that one of their teammates lost her dad at sea last season when the cargo ship El Faro sank after encountering Hurricane Joaquin. It just brings us all closer.

When Little isnt at the hospital, shes usually working on her online classes or walking her dog. She said the support from family, friends and her soccer team is what keeps her going.

Were the ones who are supposed to be strong for her, but shes so strong for us, Preble said. Just a cheek swab and some paperwork could help save her life.

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USM women's soccer players organize bone marrow drive for teammate with rare disease - Press Herald

February 08, 2017 09:45 ET | Source: BioRestorative Therapies, Inc.

MELVILLE, N.Y., Feb. 08, 2017 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. ("BRT" or the Company") (OTCBB:BRTX), a life sciences company focused on stem cell-based therapies, today announced that it has received clearance by the U.S. Food and Drug Administration (FDA) to commence a Phase 2 clinical trial using its lead cell therapy candidate, BRTX-100, to treat chronic lower back pain due to degenerative disc disease related to protruding/bulging discs.

The Phase 2 clinical trial is a 72 patient, randomized, double-blind, controlled, multi-center study designed to evaluate safety and efficacy of a single dose of BRTX-100 in treating chronic lower lumbar disc disease. BRTX-100 will be administered via intradiscal injection into one disc of a subject with chronic lumbar disc disease and whose pain is not responsive to conservative treatment measures (e.g., oral medication, epidural injections and physical therapy). The primary goal of the treatment is to both reduce pain and increase function in these patients.

In January 2017, the Company had submitted an Investigational New Drug Application (IND) to the FDA to obtain clearance to commence this clinical trial using BRTX-100. BRTX-100 is a product formulated using a patients own cell population (autologous), which consists of hypoxic (low oxygen) cultured mesenchymal stem cells (MSCs) that are optimized for specific use for a non-surgical, conservative intradiscal procedure that can be performed in a physicians office.

"We are excited to be able to begin our clinical development of BRTX-100 with this Phase 2 clinical trial," saidMark Weinreb, President and Chief Executive Officer of BioRestorative Therapies. This treatment has the potential to positively impact millions of Americans suffering from chronic lumbar disc disease as an alternative to surgery. We believe that this technology can be truly transformative and addresses a large market underserved by current therapies.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. The BRTX-100 production process involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery.

Metabolic Program (ThermoStem): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including those set forth in the Company's Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

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BioRestorative Therapies Receives FDA Clearance to Initiate Phase 2 Clinical Trial for the Treatment of Patients ... - GlobeNewswire (press release)

An act of kindness can change your life in a positive way. But how much better would it be if that same act ends up changing someone elses life in an amazing way too? Jillian Altenburg can answer that.

It will change me for the better, but I am even happier I have the ability to change someone elses life for the better, Jillian said.

This month, Jillian will go through a bone marrow donor procedure for a little child struggling with leukemia. Not only will this change Jillian forever, but it gives a small child a new chance at life.

Jillian, the daughter of Gary Altenburg and Lori Altenburg, is a Cut Bank High School 2013 graduate. She will be part of another graduation this May when she receives her nursing degree from the MSU-BSN college of nursing program in Great Falls.

Jillian first learned about being a bone marrow donor while in her freshman year of college at MSU-Bozeman. It was during one of my classes we heard about a child who was looking for a bone marrow match and after class the organization called Be the Match was introduced to us.

Be the Match is an organization that matches bone marrow donors to patients in need of a bone marrow transplant. Jillian became a potential donor that day after class when she had her cheek swabbed and entered into the program.

It was a few years before she heard anything from Be the Match, but this past December Jillian received a call saying she could be a match for a patient needing bone marrow.

I didnt know if I was the only match or if there were other potential matches too, Jillian stated. They asked me to do some blood work and they said it usually takes 60 days to determine if I would be a perfect match. But it only took a week for them to get back to me and let me know I was a perfect match for this little child with leukemia.

It was then that things started happening fast for Jillian. They told me that the child was in remission and that there is a window of time to do the procedure, so they gave me the date it would be done and where I needed to be for the procedure and explained what would be happening. And Be the Match would be picking up the tab for everything.

According to Jillian, there are two ways to collect bone marrow. One of those is by putting needles in my arm to gather stem cells. The other is by cutting a slit on both sides of my pelvis and inserting a hollow needle into the bone to pull out what they need. Normally they can take up to six cups of bone marrow, but with this being a child needing the bone marrow, they probably will not need that much.

After the procedure, Jillian will be discharged but will need to stay close to the hospital for another night in a hotel to make sure all is okay. Once that is determined, she can return home.

They say the procedure is painful and I will be sore. But ever since I learned I was a match, there was no choice in this for me. I actually feel like the lucky one, having this opportunity to help this child, Jillian said.

Within seven days of harvesting Jillians bone marrow, the child will receive the bone marrow that is so desperately needed for survival. During that time, Jillian should feel better and better each day. And within two weeks of having made the donation, Jillians body will have replaced the bone marrow taken from her.

Even though Be the Match tells all their potential donors that they can opt out of the program at any time, that was not an option for Jillian. Once they start prepping the patient to receive a bone marrow transplant, they really dont want people to say they have changed their minds. I have no intention of doing that anyway. When they called me, it was not a decision I had to think about. I knew I was going to do it. It feels good to be able to help someone and change their life for the better, she said.

The day Jillian shared the news with her mom Lori, that she was a potential bone marrow match for someone, Lori said, I got that warm, fuzzy feeling, but as we spoke longer it turned to worry, mostly for this child who has had to deal with these awful circumstances. I knew Jillian would be fine. She is strong, very physically fit and young. She has everything going for her in being a good donor.

Lori will be accompanying Jillian when she has the procedure done and will be there for her all the way through to recovery. We have always teased Jillian that she is still attached to her moms umbilical cord, so we both know I have to go, even though I know it doesnt stretch quite that far, laughed Lori.

Lori admitted she did not know Jillian had even put her name into the Be the Match program. We have lost many loved ones due to cancer, with number one being Jillians Grama Nancy (Loris mom). Even so, when I got the call from Jillian that she was contacted by the Be the Match program because she was a possible bone marrow match, it was very, very surprising. But I feel super proud and very blessed that she was chosen. To say I am not a little nervous would be a fib, but she will be in good hands and we know friends and family are praying for her and the child who will receive the donation, Lori shared.

For a year following the transplant, Jillian will not know the name of the child she made the donation to. I can find out through Be the Match how this little one is doing, but for up to a year they want me to remain anonymous. After a year, we can have contact.

As Jillian said, she knew without a doubt, she would be going through this procedure the minute she was called and told she was a perfect match. And while she admitted she is a little scared, she countered that with, It will be worth it.

There are two lives being changed with one procedure. Jillians life will be forever changed by this. And the young child? With Jillians donation, there is hope that many years can be added to that young little life.

Excerpt from:
Jillian Altenburg: Sharing her gift of lifeand bone marrowwith young leukemia patient - Cut Bank Pioneer Press

Stem cells are often in the news. These days its usually about some advance in research. Sometimes the controversy about using embryonic stem cells resurfaces. Despite all the coverage (pro or con) stem cells are not well understood. What are they and why are they important?

In more ways than one, its the potential of stem cells that makes them important. At the moment most of the work with stem cells is still in the laboratory; but thats changing. Within the next few years stem cells, in one form or another, will be at work in medical applications such as repairing a damaged pancreas or a heart. In fact, stem cells will be used to repair or even re-grow tissues all over the body skin, liver, lungs, bone marrow. The production of stem cells, their delivery, and procedures for using them will become the basis of an industry. In the not too distant future stem cells, or the knowledge we gain from working with them, will be used in sophisticated repair of the brain and as part of the development of replacement organs. The potential is enormous.

What are stem cells?

Stem cells are found in most multicellular creatures and come in different varieties; all have an important ability: They can fully reproduce themselves almost indefinitely. For example, in mammals like human beings, blood stem cells (hematopoietic stem cells) are active all our lives in the marrow of bones, where they continually produce the many different kinds of blood cells. Therein is another key property for most stem cells; they can become other kinds of cells. The word for this process is differentiate; blood stem cells can differentiate into red blood cells, white blood cells, blood platelets and so forth. The ability to produce different kinds of cells is why stem cells may be used, for example, to repair or replace damaged heart cells something mature heart cells cannot do on their own.

Stem cell jargon

When you read about stem cells, there are a number of words that jump out jargon, yes, but still descriptive. Stem cells are classified by their potency, that is, what other kinds of cells they can become, or put another way, their ability to differentiate into other cells. There is a rank order from more to less potent:

Totipotent sometimes also called omnipotent stem cells can construct a complete and viable organism. In short, they are the same as a cell created by the fusion of the egg and a sperm (an embryonic cell). Totipotent cells can become any type of cell.

Pluripotent stem cells are derived from totipotent cells and are nearly as versatile. They can become any type of cell, except embryonic.

Multipotent stem cells can become a wide variety of cells, but only those of a close family, for example blood stem cells (hematopoietic cells) can become any of the blood cells, but not other kinds of cells.

Oligopotent stem cells are limited to becoming specific types of cells, such as endoderm, ectoderm, and mesoderm.

Unipotent stem cells can only produce cells of their own type, for example skin cells. They can renew themselves (replicate indefinitely), which distinguishes them from non-stem cells.

To a certain extent the potency of a stem cell relates to its usefulness. In one view of an ideal (lab) world, only totipotent stem cells would be used because they can become any other kind of cell. The real world (lab or otherwise) doesnt work that way. For one thing, stem cells of lesser versatility than totipotent cells are valuable for use in specific applications. Even unipotent stem cells, lowest on the potency poll, are arguably better suited for some targeted uses than more generic stem cells. Most importantly, for many uses, especially for medical purposes, pluripotent stem cells are extremely versatile and less controversial.

Avoiding embryonic stem cells

The true totipotent stem cell is a fertilized egg one embryonic cell. To obtain it means detecting and collecting the cell shortly after fertilization and before it begins to divide. Collecting embryonic stem cells one at a time is very difficult and very expensive. Also, in some parts of the world, using embryonic stem cells is highly controversial, usually on religious grounds. Collecting embryonic stem cells can be considered abortion, since the procedure means the cell(s) will not become an embryo. The label abortion is also applied to collecting embryonic stem cells (by gastrulation) shortly after the first fertilized cell begins to divide. These cells, obviously more numerous, are pluripotent and have been the mainstay of stem cell research.

The history of opposition to the use of embryonic stem cells goes back to the 1990s, when stem cell research was in its own infancy. At that time the only source of viable laboratory stem cells was from in vitro living donors. Most of these were harvested from fertilization clinics. They were so difficult to acquire that only a few stem cell lines (painstakingly cultivated generations of embryonic stem cells) were available. Even those were controversial. The United States banned the taking of embryonic stem cells except for 23 grandfathered lines. (This ban was lifted in 2009.)

The controversy over embryonic stem cells can be avoided primarily in two ways. One way is to use adult stem cells. The word adult is a bit misleading since the cells may be derived from fetuses, newborns, and children, which is why theyre sometimes called somatic stem cells. It means that these stem cells come from relatively mature tissue, cells that are already differentiated to a certain degree. Thats why adult stem cells are almost always classified as multipotent, oligopotent, or unipotent. The other way is to transform adult stem cells into pluripotent stem cells. Many approaches to this transformation are being explored in labs all over the world. Some approaches are derived from fetal/newborn substances such as amniotic fluid and placental or umbilical tissue. Other approaches use mature (differentiated) stem cells, such as those from skin, and genetically modify them until they become pluripotent. Such cells are called induced pluripotent stem cells, often abbreviated as iPSC.

At the moment, it is not possible to say which approaches to stem cell production and application will be the most effective. Even some that seem unlikely (stem cells from skin cells?) may turn out to be the most economical and useful. Still, this is where the payoff for stem cell research lies both in terms of scientific knowledge and in profits for medical applications. Consequently the amount of research work in progress is substantial, and often competitive.

Stem Cell Tourism

Because experimental medical techniques and human desperation can add up to big money, there is a developing market for stem cell applications for a variety of medical disorders. Unfortunately, at least for now, with the exception of blood cell transplants and skin cell treatments, most of these applications are either fraudulent or based on shaky experimental results. In general, most stem cell treatments are at best unethical and often illegal; however, their status around the world is a patchwork quilt of laws and regulations (or their absence). It is a near ideal situation for scam artists to lure desperate people into traveling long distances for stem cell treatment that is illegal in their own country. Hence the name: stem cell tourism.

Tracking the Impact of Stem Cell Research

In relative terms, stem cell research is just getting started. Researchers have been at it since the 1950s; but one of the most important discoveries so far induced pluripotent stem cells dates back to only 2006. This means that stem cells are: a. Not yet well understood and b. Their use in medicine is largely experimental and tentative. Heres a useful listing of what the National Institute of Health (U.S. NIH) considers some of the major open questions about adult stem cells:

How many kinds of adult stem cells exist, and in which tissues do they exist? How do adult stem cells evolve during development and how are they maintained in the adult? Are they leftover embryonic stem cells, or do they arise in some other way? Why do stem cells remain in an undifferentiated state when all the cells around them have differentiated? What are the characteristics of their niche that controls their behavior? Do adult stem cells have the capacity to transdifferentiate, and is it possible to control this process to improve its reliability and efficiency? If the beneficial effect of adult stem cell transplantation is a trophic effect, what are the mechanisms? Is donor cell-recipient cell contact required, secretion of factors by the donor cell, or both? What are the factors that control adult stem cell proliferation and differentiation? What are the factors that stimulate stem cells to relocate to sites of injury or damage, and how can this process be enhanced for better healing? [Source: U.S. National Institute of Health]

SciTechStory Impact Area: Stem Cells

Theres not much debate on the importance of stem cell research. It has already had major impact on our understanding of cell biology, and it will provide more. It is just beginning to have an impact on medicine, with much more to come. In fact, news about stem cell research already occurs once or twice a week (on average) that pace is likely to increase. As a matter of keeping up, its necessary to attempt sorting lab work from practical application, which is to say sorting promise from delivery. Even at that it will be difficult to select which stem cell stories are significant.

Continued here:
Stem Cells - SciTechStory

Louisville, Ky. man saves stranger's life

Julia Rose, WHAS 1:17 PM. EST February 07, 2017

Eric Gurevich and Ron Dreben

LOUISVILLE (WHAS11) -- It's hard to believe that up until a few months ago, Eric Gurevich and Ron Dreben were total strangers.

I got to see him for the first time and I just gave him this big bear hug and he was crying and his wife was there and his 81-year-old mother was there, Gurevich said.

That bear hug was years in the making but the two were bonded long before as blood brothers.

Because he received those, he was kind of given a new lease on life, Gurevich said.

Gurevich lives in Louisville. He donated his stem cells to Dreben who lives in Washington D.C. in 2014, one, quick decision that changed the lives of two people. Gurevich remembers the call from the organization Gift of Life like it was yesterday.

They said that there was a 54-year-old man with MDS and his life was dire and I am the only potential match, Gurevich said.

Without hesitation, he hopped on a plane from Louisville to D.C. and a week later started the donation process. Despite some concerns from family members who weren't totally sold on the idea of him undergoing the major medical procedure for a man he didn't even know, Gurevich says his decision was a no-brainer.

You're a little kid and you dream of being a superhero or helping someone or saving someone's life and you get this call. You get an opportunity. How could you not? Gurevich said.

He says donating 1.5 billion stem cells is painless, just like the cheek swab he did back in 2008, the reason he was even a possible match for Dreben.

Didn't think anything of it, got my cheek swabbed and then forgot about it pretty much the next day, Gurevich said.

That cheek swab was taken on his Birthright trip to Israel, a once in a lifetime opportunity that he never expected to lead to another once in a life time journey.

Gurevichs stem cell donation saved Dreben's life and for a full year after the transplant, they wondered about one another. By law, they weren't allowed to know each other's identities but that didn't stop them from exchanging cards and small gifts.

Eric Gurevich donated his stem cells to Dreben who lives in Washington D.C. in 2014, one, quick decision that changed the lives of two people.

He sent me a magnet that says 'life is a journey not a destination' and I have it right on my fridge and I think about him just about every day, Gurevich said.

Finally, after more than a year, the pair met face to face in Miami in November, a moment captured in a picture and forever captured in their hearts.

When you donate to a stranger you always wonder, you know who is on the other side and I was just so grateful that it was him, Gurevich said.

Gurevich says he and Dreben text each other often, sending pictures of their families back and forth and they plan to meet up again in the future.

If you're interested in becoming a potential stem cell or bone marrow donor with Gift of Life, you can find more information here: http://www.giftoflife.org.

There are also two local organizations dedicated to stem cell research and transplants: sharingamericasmarrow.com & nationalstemcellfoundation.org.

( 2017 WHAS)

Here is the original post:
Ky. man saves stranger's life with stem cell transplant - WHAS11.com

Physicians at the Johns Hopkins Kimmel Cancer Center report they have successfully treated 16 patients with a rare and lethal form of bone marrow failure called severe aplastic anemia using partially matched bone marrow transplants followed by two high doses of a common chemotherapy drug. In a report on the new transplant-chemo regimen, published online Dec. 22, 2016, in Biology of Blood and Marrow Transplantation, the Johns Hopkins team says that more than a year after their transplants, all of the patients have stopped taking immunosuppressive drugs commonly used to treat the disorder and have no evidence of the disease.

"Our findings have the potential to greatly widen treatment options for the vast majority of severe aplastic anemia patients," according to Robert Brodsky, M.D., professor of medicine and oncology at the Johns Hopkins Kimmel Cancer Center and an author of the report.

Results of the small clinical trial have already prompted the organization of a larger national trial being led by Amy DeZern, M.D., an assistant professor of oncology and medicine at the Johns Hopkins Kimmel Cancer Center, with plans to involve patients at 25 medical centers across the country.

Diagnosed in about one in 250,000 people each year, aplastic anemia occurs when one's own immune system damages blood-making bone marrow cells, which gradually stop producing red and white blood cells and platelets.

Patients must receive frequent blood transfusions, take multiple medicines to suppress the autoimmune response that damages the marrow, take other drugs to prevent infections, and limit contact with the outside world to avoid infection and even minor injury. Over the long term, most patients eventually die of infections.

When immunosuppressive therapy fails to keep the disease in check -- in as many as 30 to 40 percent of patients -- doctors usually prescribe a drug called eltrombopag, which is used in a variety of blood disorders to increase platelets. The drug, according to the Johns Hopkins experts, works only in about 30 percent of patients and usually leads to a partial, not complete, response.

Brodsky and DeZern say that the only curative treatment is a bone marrow transplant, but few patients have donors who are "fully matched" -- sharing the same collection of immune-stimulating proteins that decorate every cell in the body.

In an effort to overcome the donor shortage and offer transplant to more patients, DeZern, Brodsky and their colleagues enrolled 16 patients between 11 and 69 years of age in this study from July 2011 through August 2016.

Each of the patients had failed to respond to immunosuppressive therapy or other drug treatments. None had access to a related fully matched bone marrow donor but did have an available and willing donor who was a half match. Three patients used unrelated donors.

After administering a cocktail of drugs designed to suppress their immune system and prevent rejection of the donor marrow, the patients received half-matched bone marrow transplants, some from siblings or parents, and others from unrelated donors.

Three and four days after their transplants, the patients received high doses of the chemotherapy drug cyclophosphamide. For the next year, or slightly longer, they remained on immunosuppressive medications, including tacrolimus, then stopped taking them.

Within weeks of their transplants, tests showed that each of the patients' red and white blood cell and platelet counts had returned to normal levels without the need for blood transfusions. Once immunosuppressive therapy was stopped, none of the patients required further treatment related to their disease, the Johns Hopkins team reported.

Although 13 patients were able to discontinue immunosuppressive drugs a year after their transplant, three developed mild graft-versus-host disease (GVHD), a common complication of bone marrow transplants that occurs when immune cells in the transplant attack the newly transplanted cells. Two patients had mild GVHD that appeared on their skin, and one patient's GVHD occurred in the mouth and skin. After a few extra months of immunosuppressive therapy, their GVHD subsided, and they also were able to stop taking these medications.

Ending all therapy related to their disease has been life-changing for the patients, says DeZern. "It's like night and day," she says. "They go from not knowing if they have a future to hoping for what they'd hoped for before they got sick. It's that transformative."

Successful transplants using partial match donors, Brodsky says, open up the transplant option to nearly all patients with this condition, especially minority patients. Seven of the 16 patients treated at Johns Hopkins self-identified as nonwhite.

A full sibling only has a 25 percent chance of being a full match. However, 100 percent of parents and 50 percent of siblings or half-siblings are half matches, regardless of ethnicity. The average person in the United States has about four half matches or better. "Now, a therapy that used to be available to 25 to 30 percent of patients with severe aplastic anemia is potentially available to more than 95 percent," says Brodsky.

The idea of using cyclophosphamide after a partial-match transplant was first pioneered decades ago by Johns Hopkins Kimmel Cancer Center experts. Brodsky says the drug destroys patient's diseased immune system cells but does not harm the donor's blood stem cells, which create new disease-free blood cells in the patient.

Bone marrow transplants are costly -- sometimes exceeding more than $300,000. However, Brodsky and DeZern say that full and half-matched transplants are life-saving for many, and there is cost-saving potential when aplastic anemia patients can avoid a lifetime of immunosuppressive therapy, hospitalizations, medications and blood transfusions.

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16 aplastic anemia patients free of disease after bone marrow transplant and chemo - Science Daily

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Small sheets of healthy skin are being grown from scratch at a Stanford University lab, proof that gene therapy can help heal a rare disease that causes great human suffering.

The precious skin represents growing hope for patients who suffer from the incurable blistering disease epidermolysis bullosa and acceleration of the once-beleaguered field of gene therapy, which strives to cure disease by inserting missing genes into sick cells.

It is pink and healthy. Its tougher. It doesnt blister, said patient and research volunteer Monique Roeder, 33, of Cedar City, Utah, who has received grafts of corrected skin cells, each about the size of an iPhone 5, to cover wounds on her arms.

More than 10,000 human diseases are caused by a single gene defect, and epidermolysis bullosa is among the most devastating. Patients lack a critical protein that binds the layers of skin together. Without this protein, the skin tears apart, causing severe pain, infection, disfigurement and in many cases, early death from an aggressive form of skin cancer.

The corrected skin is part of a pipeline of potential gene therapies at Stanfords new Center for Definitive and Curative Medicine, announced last week.

The center, a new joint initiative of Stanford Healthcare, Stanford Childrens Health, and the Stanford School of Medicine, is designed to accelerate cellular therapies at the universitys state-of-the-art manufacturing facility on Palo Altos California Avenue. Simultaneously, itisaiming to bring cures to patients faster than before and boost the financial value of Stanfords discoveries before theyre licensed out to biotech companies.

With trials such as these, we are entering a new era in medicine, said Dr. Lloyd B. Minor, dean of the Stanford University School of Medicine.

Gene therapy was dealt a major setback in 1999 when Jesse Gelsinger, an Arizona teenager with a genetic liver disease, had a fatal reaction to the virus that scientists had used to insert a corrective gene.

But current trials are safer, more precise and build on better basic understanding. Stanford is also using gene therapy to target other diseases, such as sickle cell anemia and beta thalassemia,a blood disorder that reduces the production of hemoglobin.

There are several diseases that are miserable and worthy of gene therapy approaches, said associate professor of dermatology Dr. Jean Tang, who co-led the trial with Dr. Peter Marinkovich. But epidermolysis bullosa, she said, is one of the worst of the worst.

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It took nearly 20 years for Stanford researchers to bring this gene therapy to Roeder and her fellow patients.

It is very satisfying to be able to finally give patients something that can help them, said Marinkovich.In some cases, wounds that had not healed for five years were successfully healed with the gene therapy.

Before, he noted, there was only limited amounts of what you can do for them. We can treat their wounds and give them sophisticated Band-Aids. But after you give them all that stuff, you still see the skin falling apart, Marinkovich said. This makes you feel like youre making a difference in the world.

Roeder seemed healthy at birth. But when her family celebrated her arrival by imprinting her tiny feet on a keepsake birth certificate, she blistered. They encouraged her to lead a normal childhood, riding bicycles and gentle horses. Shes happily married. But shes grown cautious, focusing on photography, writing a blog and enjoying her pets.

Scarring has caused her hands and feet digits to become mittened or webbed. Due to pain and risk of injury, she uses a wheelchair rather than walking long distances.

Every movement has to be planned out in my head so I dont upset my skin somehow, she said. Wound care can take three to six hours a day.

She heard about the Stanford research shortly after losing her best friend, who also had epidermolysis bullosa, to skin cancer, a common consequence of the disease. Roeder thought: Why dont you try? She didnt get the chance.

The team of Stanford experts harvested a small sample of skin cells, about the size of a pencil eraser, from her back. They put her cells in warm broth in a petri dish, where they thrived.

To this broth they added a special virus, carrying the missing gene. Once infected, the cells began producing normal collagen.

They coaxed these genetically corrected cells to form sheets of skin. The sheets were then surgically grafted onto a patients chronic or new wounds in six locations. The team reported their initial results in Novembers Journal of the American Medical Association.

Historically, medical treatment has had limited options: excising a sick organ or giving medicine, said Dr. Anthony E. Oro of Stanfords Institute for Stem Cell Biology and Regenerative Medicine. When those two arent possible, theres only symptom relief.

But the deciphering of the human genome, and new tools in gene repair, have changed the therapeutic landscape.

Now that we know the genetic basis of disease, we can use the confluence of stem cell biology, genome editing and tissue engineering to develop therapies, Oro said.

Its not practical to wrap the entire body of a patient with epidermolysis bullosa in vast sheets of new skin, like a mummy, Oro said.

But now that the team has proved that gene therapy works, they can try related approaches, such as using gene-editing tools directly on the patients skin, or applying corrected cells like a spray-on tan.

A cure doesnt take one step, said Tang. It takes many steps towards disease modification, and this is the first big one. Were always looking for something better.

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Stanford team is growing healthy skin for diseased patients - The Mercury News

For many people suffering from disabling conditions, such as Parkinsons disease, spinal injury and paralysis, heart disease, and even cancer, announcements in the press around breakthroughs in stem cell research undoubtedly bring hope.

Keeping the balance between hope and hype is a difficult one, particularly when there are vulnerable and suffering people relying on the hope medical research offers. As Australian of the Year, Emeritus Professor Alan Mackay-Sim, stated in his acceptance speech, there are now many clinical trials being performed in Australia and around the globe, to determine whether the delivery of certain types of cells, including some grown from stem cells, into the spinal column can allow patients with spinal cord injury to regain function.

For these individuals, even a small gain of functionis a major advance. However, as yet there is no stem cell silver bullet.

And stem cells that have shown promise can also cause complications. It was also reported a paraplegic woman developed a growth in her spine many years after an unsuccessful spinal stem cell treatmentHence, more research to test these and other types of cells in well-run clinical trials is required to move from anecdote to safe and effective therapies.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:The future of stem cells: tackling hype versus hope

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Hype versus hope: Deciphering news about stem cell breakthroughs - Genetic Literacy Project

A New Delhi clinic that has claimed to help paralyzed Canadians walk again by injecting them with stem cells now says it can use the same treatment to make children with Down syndrome almost near normal.

Nutech Mediworld says it has treated up to 16 newborns, toddlers and older children with Down syndrome. According to its medical director, Geeta Shroff, we have seen that patients actually start improving clinically they become almost at par for their age.

Canadian experts say the bold claim risks raising false expectations and public confusion, much like the now-discredited Liberation therapy for multiple sclerosis, and that its playing off the over-hyped belief stem cells have the potential to cure almost anything.

Its also the latest controversy over stem cell tourism, and the growing number of clinics worldwide marketing pricey, unregulated and unproven treatments.

Nutech Mediworld charges US$5,000 to $6,000 per week for its stem cell-based therapies. The clinic says it has treated such incurable conditions as spinal cord injury and cerebral palsy. Around 20 Canadians have sought treatment at the clinic for paralyzing spinal cord injuries, spending upwards of $US48,000 each. Shroff says some of her patients have regained the ability to walk with walkers.

More recently, she began working with Down syndrome, one of the most common chromosomal disorders worldwide.

Most cases are caused by a random error in cell division. The child ends up with three copies of chromosome 21, instead of the usual two.

That extra copy causes abnormal neuronal development and changes in the central nervous system, Shroff says, leading to persistent developmental delays.

Human embryonic stem cells injected into a childs muscles and bloodstreamcan regenerate and repair that damaged brain, she says. They also work at the genetic level, she claims.

In a single case published last year, Shroff reported treating a two-month-old baby boy in September 2014 diagnosed with Down syndrome at birth. The infant had delayed milestones, lack of speech, subnormal understanding and subnormal motor skills, she wrote.

After two stem cell therapy sessions, the baby started babbling and crawling, she reported. He had improved muscle tone. He was social and was able to recognize near ones.

The child became almost as near normal as possible cognitively

The child became almost as near normal as possible cognitively, Shroff told the Post in an interview. Today, hes talking; hes walking. He was at par with normal children on analysis.

The former infertility specialist uses embryonic stem cells developed from a single fertilized egg donated by an IVF patient 17 years ago. According to Shroff, We have witnessed no adverse events at all.

The Down syndrome treatments, reported by New Scientist, have raised skepticism and alarm. Its not at all clear what cells shes actually putting in patients, says renowned developmental biologist Janet Rossant, senior scientist at the Hospital for Sick Children Research Institute in Toronto.

By just putting them into the bloodstream theres no way to imagine they could contribute to the right tissues.

Embryonic stem cells can also form teratomas benign tumours and masses composed of lung cells, tufts of hair, teeth, bone and other tissues.

The gold standard for any therapy would be a clinical trial comparing treated with untreated children and vetted through proper regulatory systems that clearly she is not going through, Rossant says.

The Ottawa Hospitals Dr. Duncan Stewart, who is leading the first trial in the world of a genetically enhanced stem cell therapy for heart attack, says theres a remote chance embryonic stem cells could help with Down syndrome. But its a stretch. The injected cells would also likely be rejected and die off with days, he believes. If the cells are disappearing within days, how are they working?

This is a very vulnerable population Theyre very vulnerable to people who are selling hope and have no basis for it

This is a very vulnerable population, Stewart adds. Theyre very vulnerable to people who are selling hope and have no basis for it.

But stem cells have taken on almost mystical appeal.

Theyve become a pop culture phenomenon, says healthy policy expert Timothy Caulfield, of the University of Alberta. The field itself is guilty of making breathless announcements about breakthroughs and cutting edge, he says. And people can market that kind of language.

This kind of nonsense doesnt help.

Email: jskirkey@postmedia.com | Twitter:

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From Down syndrome to 'near normal'? New Delhi clinic makes stem cell claims that worry experts - National Post

A new revolutionary stem cell technique is being used to treat those suffering from damaged muscles without the cancer risk that was previously present. This was the first time that researchers had successfully implanted synthetic stem cardiac cells that managed to repair the muscle that a previous heart attack has weakened. Cancer was previously a risk with traditional stem cell therapy as scientists were unable to stop formertumors as the cells continued to replicate.

This procedure is mostly performed on those suffering from blood or bone marrow cancers such as leukemia. But, researchers are also working on developing effective stem cell treatments for those diagnosed with neurodegenerative diseases such as Parkinsons and heart disease too.

Synthetic stem cells are very handy because unlike natural stem cells, theyre easy to preserve and can be adapted to be used in various parts of the body. Ke Cheng, associate professor of molecular biomedical sciences at North Carolina State University, said, We are hoping that this may be the first step towards a truly off-the-shelf cell product that would enable people to receive beneficial stem cell therapies when theyre needed, without costly delays.

[The study can be found here.]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Pioneering Stem Cell Technique Promise Muscle Regeneration Without Cancer Risk

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Regrowing heart muscles without cancer risk, using synthetic stem cells - Genetic Literacy Project

Globalnews.ca

Quebec family hopes to raise awareness for patients in need with stem cell registry drive Globalnews.ca Natasha Camacho-Gomes (middle right, standing) organized a bone marrow registry drive Saturday, Feb. 4, 2017 to raise awareness for patients in need. Her Fianc Kevin Butterfill is one of those patients. He was diagnosed with leukemia in January.

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Quebec family hopes to raise awareness for patients in need with stem cell registry drive - Globalnews.ca

By filling out a form, and swabbing his mouth, Harlee O'Watch could save a life.

"To find a match, because the list of donors is so low, is really unlikely," said the 22-year-old.

O'Watch is one of four young adults from Carry the Kettle First Nation who registered with the OneMatch program, which connects donors with people in need of bone marrow or stem cell transplants.

A problem for the 14 indigenous people currently waiting for a match is that, out of the 17,000 people on the Canadian registry, fewer than one per cent are indigenous.

"It doesn't give me much hope if I ever get sick and need a blood transfusion or bone marrow transplant, said OWatch.

It doesn't give me much hope because, if there's no potential matches, I'm going to die, bottom line, and I don't want to die."

Robyn Henwood works for Canadian Blood Services, which runs OneMatch. She covers Alberta to Northern Ontario and the Northwest Territories, including the Prairies, and visited Carry the Kettle to recruit. A match requires a genetic twin and indigenous people are only in Canada.

"It does get more complicated [with] these different ethnic backgrounds. . . even within First Nations that get brought into it, said Henwood.

The chances of finding a match becomes that much more difficult."

This means someone who is Cree cannot donate to someone who is Mohawk, she said.

In the past year, Canadian Blood Services has visited less than 12 reserves to help find matches for indigenous people. Carry the Kettle is Henwoods third community.

"We have been leaving messages and voicemails, not getting a lot of response back, she said.

I'm hoping a new technique will work. Things like this, this is so important to spread our message."

According to Indigenous and Northern Affairs Canada, more than 50 per cent of indigenous people live in urban centres. And yet, Henwood says finding indigenous donors in cities is also a struggle.

"Trying to get someone to sign up and commit for the next 30 to 40 years, to potentially save a stranger's life is not an easy thing to do," she said.

Henwood says informing indigenous people about one match will empower more to donate. Until then, the chance of survival for those waiting on the registry is low.

Excerpt from:
Program seeks to boost bone marrow, stem cell donations from indigenous people - CTV News

BUCKEYE, AZ - As the saying goes: "blood is thicker than water." But when it comes to bone marrow, it is truer than ever especially because family is usually the only people to turn to for a match.

But, one Buckeye family is finding that the phrase could not be more perfectfor them because a brother has been serving as the lifeline for his sibling over the last few years.

Gloria Mesquias calls her 11-year-old son, Shaun, "the warrior."

"He takes every jab he gets and just rolls through," said Mesquias.

Shaun's 13-year-old brother Malik is called "the hero."

"They are actually like night and day, "Mesquias said. "They're brothers."

The three of them, and other supportive family members, have spent months at Phoenix Children's Hospital. Shaun has a condition where his body isn't producing new blood cells.

"He was diagnosed with severe aplastic anemia," Mesquias said.

That happened when he was just about 1 year old. Ever since then, he's been in and out of the hospital.

But, Malikhas served as a bone marrow and stem cell donor to his b brother not once, not twice but, three different times.

And the fight for this little warrior, is not over yet. If his treatment goes well over the next few days, he will have a fourth surgery on Wednesday; another stem cell transplant.

Mesquias doing this all as a single mother. She also has a 5-year-old daughter, who has not seen Shaun since before Christmas.

"She understand;she gets it," Mesquias explained. "She knows brother is sick and she knows mom is here with Shaun."

But, while she tries to keep it together, all of the stress and days away from home are weighing on Mesquias. But, it's something she will never let her family see.

"At night time, I can go in the restroom and cry my eyes out or ball my face out in the pillow," Mesquias said. "But, I just don't do it in front of him."

So, the boys' Buckeye teacher, Carrie Brown, has also taken action to try and do something special for the family.

"She would never ask for help," Brown said. "She's not that kind of person. So, I just thought that this was one thing that I could do to relieve some of the worry that she has and to give her a little bit of comfort."

Brown started a GoFundMe page to try and help the family who has given so much to each other.

And Mesquias said she is making sure all of them get out of that hospital together.

"He has his moments too where he says he wants to go home," Mesquias explained. "And... I'm like, 'I'm not going home until you're going home. So, we're good."

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Buckeye boy donates bone marrow to sick brother - ABC15 Arizona - ABC15 Arizona

The 2017 Australian of the Year award went to Professor Alan Mackay-Sim for his significant career in stem cell science.

The prize was linked to barbeque-stopping headlines equating his achievements to the scientific equivalent of the moon landing and paving the road to recovery for people with spinal cord injuries.

Such claims in the media imply that there is now a scientifically proven stem cell treatment for spinal cord injury. This is not the case.

For now, any clinic or headline claiming miracle cures should be viewed with caution, as they are likely to be trading on peoples hope.

Put simply, injury to the spinal cord causes damage to the nerve cells that transmit information between the brain and the rest of the body.

Depending on which part of the spine is involved, the injury can affect the nerves that control the muscles in our legs and arms; those that control bowel and bladder function and how we regulate body temperature and blood pressure; and those that carry the sensation of being touched. This occurs in part because injury and subsequent scarring affect not just the nerves but also the insulation that surrounds and protects them. The insulation the myelin sheath is damaged and the body cannot usually completely replace or regenerate this covering.

Stem cells can self-reproduce and grow into hundreds of different cell types, including nerves and the cells that make myelin. So the blue-sky vision is that stem cells could restore some nerve function by replacing missing or faulty cells, or prevent further damage caused by scarring.

Studies in animals have applied stem cells derived from sources including brain tissue, the lining of the nasal cavity, tooth pulp, and embryos (known as embryonic stem cells).

Dramatic improvements have been shown on some occasions, such as rats and mice regaining bladder control or the ability to walk after injury. While striking, such improvement often represents only a partial recovery. It holds significant promise, but is not direct evidence that such an approach will work in people, particularly those with more complex injuries.

The translation of findings from basic laboratory stem cell research to effective and safe treatments in the clinic involves many steps and challenges. It needs a firm scientific basis from animal studies and then careful evaluation in humans.

Many clinical studies examining stem cells for spinal repair are currently underway. The approaches fit broadly into two categories:

using stem cells as a source of cells to replace those damaged as a result of injury

applying cells to act on the bodys own cells to accelerate repair or prevent further damage.

One study that has attracted significant interest involves the injection of myelin-producing cells made from human embryonic stem cells. Researchers hoped that these cells, once injected into the spinal cord, would mature and form a new coating on the nerve cells, restoring the ability of signals to cross the spinal cord injury site. Preliminary results seem to show that the cells are safe; studies are ongoing.

Other clinical trials use cells from patients own bone marrow or adipose tissue (fat), or from donated cord blood or nerves from fetal tissue. The scientific rationale is based on the possibility that when transplanted into the injured spinal cord, these cells may provide surrounding tissue with protective factors which help to re-establish some of the connections important for the network of nerves that carry information around the body.

The field as it stands combines years of research, and tens of millions of dollars of investment. However, the development of stem cell therapies for spinal cord injury remains a long way from translating laboratory promise into proven and effective bedside treatments.

Each case is unique in people with spinal cord injury: the level of paralysis, and loss of sensation and function relate to the type of injury and its location. Injuries as a result of stab wounds or infection may result in different outcomes from those incurred as a result of trauma from a car accident or serious fall. The previous health of those injured, the care received at the time of injury, and the type of rehabilitation they access can all impact on subsequent health and mobility.

Such variability means caution needs to accompany claims of man walking again particularly when reports relate to a single individual.

In the case that was linked to the Australian of the Year award, the actual 2013 study focused on whether it was safe to take the patients own nerves and other cells from the nose and place these into the damaged region of the spine. While the researchers themselves recommended caution in interpreting the results, accompanying media reports focused on the outcome from just one of the six participants.

While the outcome was significant for the gentleman involved, we simply do not know whether recovery may have occurred for this individual even without stem cells, given the type of injury (stab wounds), the level of injury, the accompanying rehabilitation that he received or a combination of these factors. It cannot be assumed a similar outcome would be the case for all people with spinal injury.

Finding a way to alleviate the suffering of those with spinal cord injury, and many other conditions, drives the work of thousands of researchers and doctors around the globe. But stem cells are not a silver bullet and should not be immune from careful evaluation in clinical trials.

Failure to proceed with caution could actually cause harm. For example, a paraplegic woman who was also treated with nasal stem cells showed no clinical improvement, and developed a large mucus-secreting tumour in her spine. This case highlights the need for further refinement and assessment in properly conducted clinical trials before nasal stem cells can become part of mainstream medicine.

Its also worth noting that for spinal cord injury, trials for recovery of function are not limited to the use of stem cells but include approaches focused on promoting health of surviving nerves (neuroprotection), surgery following injury, nerve transfers, electrical stimulation, external physical supports known as exoskeletons, nanotechnology and brain-machine interfaces.

Ultimately, determining which of these approaches will improve the lives of people with spinal injury can only be done through rigorous, ethical research.

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Yes there's hope, but treating spinal injuries with stem cells is not a reality yet - The Conversation AU

ANDREW and Judy Kim are still searching the globe for a donor for their two-year-old son after he was diagnosed with a rare genetic condition.

The couple's son Alastair was diagnosed with chronic granulomatous disorder (CGD) in February last year.

Mr and Mrs Kim launched an appeal for help in September but the search is still on for a matching donor and their son still needs hospital treatment.

The life-threatening condition wipes out his immune system, meaning even the most minor infections leave him seriously ill.

A course of genetic therapy treatment to help him fight infections has been launched and Alastair has been treated at Oxford Children's Hospital and Great Ormond Street Hospital in London.

The only hope of a permanent cure lies in a bone marrow stem cell donor but it needs to be a 90 per cent genetic match and the family is calling for more East Asians to sign up as donors.

Mr Kim, 37, a medical research engineer, said: "It is not easy to find a match and we pray every day that it will work out.

"We have to make sure that Alastair does not get a cut because it could get infected and he does not have the ability to fight off bacteria.

"That could cascade down the line to something very dangerous for him.

"If we get ill then we have to stay away from him he loves our dog Choco Pie but he is not allowed to stroke her.

"We are doing our best to stay positive and raise awareness about his condition."

Mr and Mrs Kim, who live near Longworth with their other son Micah, five, have already searched the international register of more than four million donors but without success.

They are both of Korean descent so a matching donor will most likely be of Korean, Japanese or Chinese heritage.

The number of East Asians on international donor registers is very limited of the 617,000 registered donors in the UK just 0.5 per cent are east Asian.

The couple, who moved to Oxfordshire from Chicago nine years ago, are now appealing for people around the world, particularly East Asians, to order a free kit through a website they have set up, and take a two-minute home test to see if they could help.

Alastair has had numerous infections since he was born in September 2014.

He spent the first year-and-a-half of his life in and out of hospital but CGD is so rare, doctors never thought to test him for it but eventually a doctor at the John Radcliffe Hospital in Oxford decided to test Alastair for the condition.

The couple desperately want to find a matching donor, but also want to increase the number of East Asians on the donor register.

The couple have run several blood drives at Mrs Kim's office at Oxford University and at Harwell Oxford.

More than 90 people came forward and of those, five were able to donate blood that helped Alastair to fight infections.

Mr Kim added: "At a couple of blood drives we have found matches for other people and hopefully one day a match will found for Alastair."

To join the register go to allysfight.com

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Search goes on for bone marrow match for little Longworth lad ... - Oxford Mail

Human skin can be morphed into genetically modified, cancer-killing brain stem cells, according to a new study. This latest advance has only been tested in mice but eventually, its possible that it could be translated into a personalized treatment for people with a deadly form of brain cancer.

The study builds on an earlier discovery that brain stem cells have a weird affinity for cancers. So researchers, led by Shawn Hingtgen, a professor at University of North Carolina at Chapel Hill, created genetically engineered brain stem cells out of human skin. Then they armed the stem cells with drugs to squirt directly onto the tumors of mice that had been given a human form of brain cancer. The treatment shrank the tumors and extended survival of the mice, according to results recently published in the journal Science Translational Medicine.

The treatment shrank the tumors and extended survival

Usually we think about stem cell therapy in the context of rebuilding or regrowing a broken body part like a spinal cord. But if they could be modified to become cancer-fighting homing missiles, it would give patients with a deadly and incurable brain cancer called glioblastoma a better chance at survival. Glioblastomas typically affect adults, and are highly fatal because they send out a web of cancerous threads. Even when the main mass is removed, those threads remain despite chemotherapy and radiation treatment. This cancer has caused a number of high-profile deaths including Senator Edward (Ted) Kennedy in 2009, and possibly Beau Biden more recently. Approximately 12,000 new cases of glioblastoma are estimated to be diagnosed in 2017.

We really have no drugs, no new treatment options in years to even decades, Hingtgen says. [We] just really want to create new therapy that can stand a chance against this disease.

But theres a problem: brain stem cells arent exactly easy to get. Brain stem cells, more properly known as neural stem cells, hang out in the walls of the brains irrigation canals areas filled with cerebrospinal fluid, called ventricles. They generate the cells of the nervous system, like neurons and glial cells, throughout our lives.

They could be modified to become cancer-fighting homing missiles

A research group at the City of Hope in California conducted a clinical trial to make sure it was safe to treat glioblastoma patients with genetically engineered neural stem cells. But they used a neural stem cell line that theyd obtained from fetal tissue. Since the stem cells werent the patients own, people who were genetically more likely to reject the cells couldnt receive the treatment at all. For the people who could, treatment with the neural stem cells turned out to be relatively safe although at this phase of clinical trials, it hasnt been particularly effective.

More personalized treatments have been held up by the challenge of getting enough stem cells out of the patients own brains, which is virtually impossible, says stem cell scientist Frank Marini at the Wake Forest School of Medicine, who was not involved in this study. You cant really generate a bank of neural stem cells from anybody because you have to go in and resect the brain.

So instead, Hingtgen and his colleagues figured out a way to generate neural stem cells from skin which in the future, could let them make neural stem cells personalized to each patient. For this study, though, Hingtgen and his colleagues extracted the skin cells from chunks of human flesh leftover as surgical waste. That really is the magic piece here, Marini says. Now, all of a sudden we have a neural stem cell that can be used as a tumor-homing vehicle.

That really is the magic piece here.

Using a disarmed virus to infect the cells with a cocktail of new genes, the researchers morphed the skin cells into something in between a skin cell and a neural stem cell. People have turned skin cells back into a more generalized type of stem cell before. But then turning those basic stem cells into stem cells for a certain organ like the brain takes another couple of steps, which takes more time. Thats something that people with glioblastoma dont have.

The breakthrough here is that Hingtgens team figured out how to go straight from skin cells to something resembling a neural stem cell in just four days. The researchers then genetically engineered these induced neural stem cells to arm them with one of two different weapons: One group was equipped with an enzyme that could convert an anti-fungal drug into chemotherapy, right at the cancers location. The other was armed with a protein that binds to the cancer cells and makes them commit suicide in an orderly process called apoptosis.

The researchers tested these engineered neural stem cells in mice that had been injected with human glioblastoma cells, which multiplied out of control to create a human cancer in a mouse body. Both of the weaponized stem cell groups were able to significantly shrink the tumors and keep the mice alive by about an extra 30 days (for scale, mice usually live an average of two years).

Were working as fast as we can.

But injecting the cells directly into the tumor doesnt really reflect how the therapy would be used in humans. Its more likely that a person with glioblastoma would get the bulk of the tumor surgically removed. Then, the idea is that these neural stem cells, generated from the patients own skin, will be inserted into the hole left in the brain. So, the researchers tried this out in mice, and the tumors that regrew after surgery were more than three times smaller in the mice treated with the neural stem cells.

Its a promising start, but it could take a few years still before its in the clinic, Hingtgen says. He and his colleagues started a company called Falcon Therapeutics to drive this new therapy forward. Were working as fast as we can, Hingtgen says. We probably cant help the patients today. Hopefully in a year or two, well be able to help those patients.

One of the things theyll have to figure out first is whether the neural stem cells can travel the much bigger distances in human brains, and whether theyll be able to eliminate every remaining cancer cell. The caveats on this are that, of course, its a mouse study, and whether or not that directly converts to humans is unclear, Marini says. Still, he adds, Theres a very high probability in this case.

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The next weapon against brain cancer may be human skin - The Verge

Mouse and human skin cells can be reprogrammed to hunt down tumors and deliver anticancer therapies.

Imagine cells that can move through your brain, hunting down cancer and destroying it before they themselves disappear without a trace. Scientists have just achieved that in mice, creating personalized tumor-homing cells from adult skin cells that can shrink brain tumors to 2% to 5% of their original size. Althoughthe strategy has yet to be fully tested in people, the new method could one day give doctors a quick way to develop a custom treatment for aggressive cancers like glioblastoma, which kills most human patients in 1215 months. It only took 4 days to create the tumor-homing cells for the mice.

Glioblastomas are nasty: They spread roots and tendrils of cancerous cells through the brain, making them impossible to remove surgically. They, and other cancers, also exude a chemical signal that attracts stem cellsspecialized cells that can produce multiple cell types in the body. Scientists think stem cells might detect tumors as a wound that needs healing and migrate to help fix the damage. But that gives scientists a secret weaponif they can harness stem cells natural ability to home toward tumor cells, the stem cells could be manipulated to deliver cancer-killing drugs precisely where they are needed.

Other research has already exploited this methodusing neural stem cellswhich give rise to neurons and other brain cellsto hunt down brain cancer in mice and deliver tumor-eradicating drugs. But few have tried this in people, in part because getting those neural stem cells is hard, says Shawn Hingtgen, a stem cell biologist at the University of North Carolina inChapel Hill. Right now, there are three main ways. Scientists can either harvest the cells directly from the patient, harvest them from another patient, or they can genetically reprogram adult cells. But harvesting requires invasive surgery, and bestowing stem cell properties on adult cells takes a two-step process that can increase the risk of the final cells becoming cancerous. And using cells from someone other than the cancer patient being treated might trigger an immune response against the foreign cells.

To solve these problems, Hingtgens group wanted to see whetherthey could skip a step in the genetic reprogramming process, which first transforms adult skin cells into standard stem cells and then turns those into neural stem cells. Treating the skin cells with a biochemical cocktail to promote neural stem cell characteristics seemed to do the trick, turning it into a one-step process, he and his colleague report today in Science Translational Medicine.

But the next big question was whether these cells could home in on tumors in lab dishes, and in animals, like neural stem cells. We were really holding our breath, Hingtgen says. The day we saw the cells crawling across the [Petri] dish toward the tumors, we knew we had something special. The tumor-homing cells moved 500 micronsthe same width as five human hairsin 22 hours, and they could burrow into lab-grown glioblastomas. This is a great start, says Frank Marini, a cancer biologist at the Wake Forest Institute forRegenerative Medicine in Winston-Salem, North Carolina,who was not involved with the study. Incredibly quick and relatively efficient.

The team also engineered the cells to deliver common cancer treatments to glioblastomas in mice. Mouse tumors injected directly with the reprogrammed stem cells shrank 20- to 50-fold in 2428 days compared withnontreated mice. In addition, the survival times of treated rodents nearly doubled. In some mice, the scientists removed tumors after they were established, and injected treatment cells into the cavity. Residual tumors, spawned from the remaining cancer cells, were 3.5 times smaller in the treated mice than in untreated mice.

Marini notes that more rigorous testing is needed to demonstrate just how far the tumor-targeting cells can migrate. In a human brain, the cells would need to travel a matter of millimeters or centimeters, up to 20 times farther than the 500 microns tested here, he says. And other researchers question the need to use cells from the patients own skin. An immune response, triggered by foreign neural stem cells, could actually help attack tumors, says Evan Snyder, a stem cell biologist at Sanford Burnham Prebys Medical Discovery Institute in San Diego, California, and one of the early pioneers of the idea of using stem cells to attack tumors.

Hingtgens group is already testing how far their tumor-homing cells can migrate using larger animal models. They are also getting skin cells from glioblastoma patients to make sure the new method works for the people they hope to help, he says. Everything were doing is to get this to the patient as quickly as we can.

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Reprogrammed skin cells shrink brain tumors in mice | Science | AAAS - Science Magazine

Glioblastoma is an aggressive form of brain cancer that kills most patients within two years of diagnosis. In tests on mice last year, a team at the University of North Carolina at Chapel Hill showed that adult skin cells could be transformed into stem cells and used to hunt down the tumors. Building on that, they've now found that the process works with human cells, and can be administered quickly enough to beat the ticking time-bombs.

Treatments for glioblastoma include the usual options of surgery, radiation therapy and chemotherapy, but none of them are particularly effective. The tumors are capable of spreading tendrils out into the brain and it can grow back in a matter of months after being removed. As a result, the median survival rate of sufferers is under 18 months, and there's only a 30 percent chance of living more than two years.

"We desperately need something better," says Shawn Hingtgen, the lead researcher on the study.

To find that something better, last year the scientists took fibroblasts a type of skin cell that generates collagen and connective tissue from mice and reprogrammed them into neural stem cells. These stem cells seek out and latch onto cancer cells in the brain, but alone are powerless to fight the tumor. To give them that ability, the scientists engineered them to express a particular cancer-killing protein. The result was mice that lived between 160 and 220 percent longer.

The next step was to test the process with human cells, and in the year since, the team has found that the results are just as promising. The technique differs slightly when scaled up to humans. The patient would be administered with a substance called a prodrug, which by itself does nothing, until it's triggered. The stem cells are engineered to carry a protein that acts as that trigger, activating the prodrug only in a small halo around itself instead of affecting the entire body. That allows the drug to target only a small desired area, ideally reducing the ill side effects that treatments like chemotherapy can induce.

Importantly, the technique can be administered quickly, to give the patients the best chance at survival.

"Speed is essential," says Hingtgen. "It used to take weeks to convert human skin cells to stem cells. But brain cancer patients don't have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient."

The treatment is an important step, but there's still a long way to go.

"We're one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers," says Hingtgen. "This is a big step toward a real treatment and making a real difference."

The research was published in the journal Science Translational Medicine.

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Stem cells beat the clock for brain cancer - New Atlas

Brain cancers can be really tricky to treat. Some, such as glioblastomas, spread roots through the brain tissue, meaning they are often impossible to remove surgically, leading to tragically low survival rates. But researchers are working on a way touse stem cells to track down the cancer, kill it, and then melt it away. By doing this, theyve managed to shrink brain tumors in mice to2 to 5 percent of their original size.

The trick has already been tried before using neural stem cells to hunt down and deliver cancer-killing drugs to tumors in mice. But there is a problem: It's tricky to getneural stem cells from humans. The safest way of doing this would be to take adult cells and then induce them in a two-step process to become neural stem cells. This, however, takes time.

Speed is essential, saysShawn Hingtgen, who led the research published in Science Translational Medicine. It used to take weeks to convert human skin cells to stem cells. But brain cancer patients dont have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient.

The researchers found a way to speed the process up byremoving one of the steps entirely, allowing them to produce the neural stem cells from adult skin cells in just four days. Usually, researchers would need to take the skin cell, induce it to become a generic stem cell, and then push it towards becoming a neural stem cell.

But by treating the skin cells with a cocktail of biochemicals, they were able to get the cells to turn straight into neural stem cells. They then tested these to see if they still had the same properties as original neutral stem cells and home in on tumors both in a petri dish and in animals models. They found they behaved exactly the same.

The final step was to see if they could somehow engineer these newly created cells to deliver drugs that are targeted at the cancer. They therefore got the stem cells to carry a particular protein that activates what is called a prodrug, which the researchers describe as forming a halo of drugs around the stem cell.

Were one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers, says Hingtgen. This is a big step toward a real treatment and making a real difference.

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Scientists Reprogram Skin Cells To Hunt Down And Shrink Brain Tumors - IFLScience

The Scientist

Regulators OK Clinical Trials Using Donor Stem Cells The Scientist WIKIPEDIA, TMHLEEResearchers in Japan who have been developing a cell therapy for macular degeneration received support from health authorities this week (February 1) to begin a clinical trial using donor-derived induced pluripotent stem (IPS) cells ...

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Regulators OK Clinical Trials Using Donor Stem Cells - The Scientist