Archive for the ‘Bone Marrow Stem Cells’ Category

Patients with rare blood cancer brought back to normal The Hindu All the three patients who underwent bone marrow transplantation of matching stem cells from donors, including one from Germany, crossed 100 days which is considered an important milestone in the transplant process, said Padmaja Lokireddy, consultant, ... and more »

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Patients with rare blood cancer brought back to normal - The Hindu

Updated June 09, 2017 10:59:15

Leukaemia patient Jake Cooper, 14, is desperately searching for the cure to his cancer, but his hopes of a life-saving fix now rest with strangers, not scientists.

Jake has chronic myeloid leukaemia and as his condition progresses will need a bone marrow transplant.

So why, when there are 29 million accessible donors on worldwide bone marrow registries, do so many patients, including Jake, struggle to find a match?

The answer is ethnicity, where your cultural background can turn your chance of survival into a desperate needle-in-a-haystack search.

Bone marrow transplants, sometimes called stem cell transplants, can be used to treat patients with cancers such as leukaemia, lymphoma and multiple myeloma.

But first a suitable donor needs to be found and that in itself can be a months or years-long process, one that usually starts with a patient's siblings, Red Cross bone marrow donor centre operations manager Paul Berghofer said.

"There's a one-in-four chance that [any one] sibling will be a match," he said.

While those odds aren't bad, and obviously improve if you're from a big family, they don't always deliver a match.

Then, the search broadens to the Australian Bone Marrow Donor Registry and beyond that, to a global registry, but for many patients these offer little hope.

While donors of north-west European backgrounds are over-represented on the registries, other ethnic groups are desperately under-represented.

"The chance of finding you a matched donor who is not related to you is best with people of a similar ethnic background," Mr Berghofer said.

The process wasn't drawn out but there were a bunch of general health check-ups, the initial typing and there was a discussion about the process involved.

I was told I could stop anytime up until a point of no return, I think a week or two before the transplant. You can't pull out in the last week because the [recipient] will almost certainly die.

Part of the workup is self-administering a course of an artificial hormone for five days into the flesh of my stomach. By day four or five, everything ached - like growing pains or shin splints.

The procedure itself was sitting in a chair, sticking my arms out by my side and local anaesthetic [being injected] in the crook of both elbows. To prevent damaging blood and increase the flows, they use big needles, they were ridiculous. That's why you have the anaesthetic!

Then I just lay in a chair and listened to music for four hours.

The process wasn't painful or bad, just uncomfortable and a little bit cold.

I don't regret it at all, I'd definitely do it again - it helped someone and it might be their only chance.

For Jake, whose dad is Samoan and German, and his mum Australian and British, his "incredibly rare" DNA means, despite monthly checks of the global registry, there is no bone marrow match available to tackle the "monster" in his body.

"The condition is bad enough but if it came to him [urgently] needing the transplant, I'd have to say to him, 'there's nothing I can do, we don't have a match'," his mum Renee Cooper said.

Complicating Jake's search is the fact Samoa does not have its own bone marrow registry, meaning thousands of potential matches are missed.

"It drives me crazy, there's not a day I don't think about it that someone is out there [possibly] with the cure," she said.

"The most frustrating part is I could be walking past them in a shopping centre and not even know."

Ms Cooper started Jake's Quest for a Cure on Facebook, a page she hoped would be shared to spread Jake's search around the world.

She said a lack of awareness of the registry and misinformation about the donation process were hurting patients' chances of a cure.

"There are no advertisements on bone marrow, there's no education around it in schools the way there is with organ donation," she said.

"People hear bone marrow and they think, 'oh my God, they're cutting my bones out'. People just have no idea at all."

Mr Berghofer said in 80 per cent of cases, the donation process was done as a peripheral blood stem cell collection which was not dissimilar to donating blood.

"The donor gets a needle in one arm, the blood goes through the apheresis machine and filters off blood stem cells and returns the rest of the blood back into their other arm," he said.

Pamela Bousejean, founder of Ur the Cure, an organisation striving to boost ethnic diversity on bone marrow registries, said potential donors were "slipping through our fingers everyday".

"People don't even know the bone marrow registry exists and how easy it is to donate stem cells if you're called up. You're saving a life doing something so simple," she said.

Ms Bousejean launched her campaign for a more representative registry after her own search for a donor, when she was diagnosed with Hodgkin lymphoma in 2010.

After chemotherapy and radiation treatments failed, she was told her "last chance" was to have a stem cell transplant.

"But they also told me it was going to be difficult to find me a match because of my Lebanese background," she said.

"That was really hard to hear."

For the next six months while his sister waited for a life-saving match, Ms Bousejean's brother took matters into his own hands, launching a social media campaign to find a donor.

"You're stuck in this limbo state," Ms Bousejean said.

"You know the cure to my cancer is out there in someone else's body."

In many ways, the campaign was successful raising awareness of the need for ethnic diversity on the Australian and international bone marrow registries but it didn't deliver the adult match she had been hoping for.

Instead, a "plan b" treatment in the form of a cord-blood donation gave the marketing professional her cure and she went into remission in 2012.

Now, she is on a mission to improve education programs targeted at ethnic and Indigenous communities and boost opportunities to recruit ethnically diverse bone marrow donors.

"We can make some small changes that would make a big difference," she said.

Topics: blood, diseases-and-disorders, leukaemia, perth-6000, australia

First posted June 09, 2017 06:00:36

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Bone marrow transplants: When your heritage leads to a needle-in ... - ABC Online

The BMT program at MedStar Georgetown is a joint effort with specialists from John Theurer Cancer Center and a key component of the Lombardi Comprehensive Cancer Center, the only cancer program in the Washington, D.C. region designated by the National Cancer Institute (NCI) as a comprehensive cancer center.

"Once considered experimental, BMT is today's established gold standard for treating patients with a number of malignant and other non-malignant diseases of the immune system, blood, and bone marrow, including multiple myeloma, lymphoma, and acute and chronic leukemia. For some conditions, blood stem cell transplant can provide a cure in patients who have failed conventional therapies," says Scott Rowley, M.D., chief of the BMT program at MedStar Georgetown as well as a member of the John Theurer Cancer Center's Blood and Marrow Stem Cell Transplantation. "For some conditions, it can actually be a cure; for others, it prolongs survival and improves quality of life. Having performed 100 BMTs at MedStar Georgetown including allogenic transplantation illustrates the strength and maturity of our program achieved in rather short time."

MedStar Georgetown's program is also the only comprehensive BMT center within Washington, D.C. and southern Maryland with accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT) for adult autologous procedures, where the patient donates his or her own cells.

The BMT program at John Theurer Cancer Center is one of the top 10 transplant programs in the United States, with more than 400 transplants performed annually.

A BMT involves a two-step process: first, collecting bone marrow stem cells from the patient and storing them for future use. Then, a week or so later, patients receive high dose chemotherapy to eliminate their disease. The previously stored cells are reinfused back into the bloodstream, where after reaching the bone marrow, they begin repopulating and allow the patient to recover their blood counts over the following 2 weeks.

"Even though BMT is considered standard therapy for myeloma worldwide, in the United States fewer than 50 percent of the patients who could benefit from BMT are referred for evaluation," says David H. Vesole, M.D., Ph.D., co- chief and director of Research of John Theurer Cancer Center's Multiple Myeloma division and director of MedStar Georgetown's Multiple Myeloma Program.

"That's mostly due to physicians' concerns that a patient is too old or compromised from other health conditions like diabetes, cardiac disease or renal failure. But new techniques and better supportive care have improved both patient outcomes and the entire transplant process, extending BMT to more patients than ever before."

The MedStar Georgetown/Georgetown Lombardi Blood and Marrow Stem Cell Transplant Program is part of a collaborative cancer research agenda and multi-year plan to form an NCI-recognized cancer consortium. This recognition would support the scientific excellence of the two centers and highlight their capability to integrate multidisciplinary, collaborative research approaches to focus on all the aspects of cancer.

The research areas include expansion of clinical bone marrow transplant research; clinical study of "haplo" transplants use of half-matched stem cell donor cells; re-engineering the function and focus of key immune cells; and the investigation of "immune checkpoint" blocking antibodies that unleash a sustained immune response against cancer cells.

"In this partnership, we've combined John Theurer's strength in clinical care with Georgetown Lombardi's strong research base that significantly contributes to clinical excellence at MedStar Georgetown. By working together, we have broadened our cancer research to offer more effective treatment options for tomorrow's patients," says Andrew Pecora, M.D., FACP, CPE, president of the Physician Enterprise and chief innovations officer, Hackensack Meridian Health. "This is one of many clinical and research areas that have been enhanced by this affiliation."

"Our teams are pursuing specific joint research projects we feel are of the utmost importance and significance in oncology particularly around immuno-oncology as well as precision medicine," says Andr Goy, M.D., MS, chairman of the John Theurer Cancer Center and director of the division chief of Lymphoma; chief science officer and director of Research and Innovation, RCCA; professor of medicine, Georgetown University. "Together our institutions have a tremendous opportunity to transform the delivery of cancer care for our patient populations and beyond."

ABOUT THE JOHN THEURER CANCER CENTER AT HACKENSACK UNIVERSITY MEDICAL CENTER John Theurer Cancer Center at Hackensack University Medical Center is New Jersey's largest and most comprehensive center dedicated to the diagnosis, treatment, management, research, screenings, and preventive care as well as survivorship of patients with all types of cancers. The 14 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in the New Jersey/New York metropolitan area turn to the John Theurer Cancer Center for cancer care than to any other facility in New Jersey. Housed within a 775-bed not-for-profit teaching, tertiary care, and research hospital, the John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguish the John Theurer Cancer Center from other facilities.www.jtcancercenter.org.

ABOUT MEDSTAR GEORGETOWN UNIVERSITY HOSPITAL MedStar Georgetown University Hospital is a not-for-profit, acute-care teaching and research hospital with 609 beds located in Northwest Washington, D.C. Founded in the Jesuit principle of cura personaliscaring for the whole personMedStar Georgetown is committed to offering a variety of innovative diagnostic and treatment options within a trusting and compassionate environment. MedStar Georgetown's centers of excellence include neurosciences, transplant, cancer and gastroenterology. Along with Magnet nurses, internationally recognized physicians, advanced research and cutting-edge technologies, MedStar Georgetown's healthcare professionals have a reputation for medical excellence and leadership. For more information please visit: medstargeorgetown.org/bmsct

ABOUT HACKENSACK MERIDIAN HEALTH HACKENSACK UNIVERSITY MEDICAL CENTER Hackensack Meridian Health Hackensack University Medical Center, a 775-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the county's first hospital, it is now part of one of the largest networks in the state comprised of 28,000 team members and more than 6,000 physicians. Hackensack University Medical Center was listed as the number one hospital in New Jersey in U.S. News & World Report's 2016-17 Best Hospital rankings - maintaining its place atop the NJ rankings since the rating system was introduced. It was also named one of the top four New York Metro Area hospitals. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades America's 50 Best Hospitals Award for five or more years in a row. Becker's Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2017. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 25 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its fifth consecutive designation in 2014. Hackensack University Medical Center has created an entire campus of award-winning care, including: the John Theurer Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Women's and Children's Pavilion, which houses the Joseph M. Sanzari Children's Hospital and Donna A. Sanzari Women's Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guide's list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to The Northern Trust PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Children's Cancer Institute housed at the Joseph M. Sanzari Children's Hospital. To learn more, visit http://www.HackensackUMC.org.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/john-theurer-cancer-center-and-medstar-georgetown-university-hospital-announce-100th-blood-stem-cell-transplant-300471445.html

SOURCE Hackensack Meridian Health

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John Theurer Cancer Center and MedStar Georgetown University Hospital Announce 100th Blood Stem Cell Transplant - PR Newswire (press release)

June 8, 2017 One example of STAT1 GOF Mutation phenotype. Credit: Hiroshima University

Researchers report the first-ever study assessing how patients with "gain of function" mutation of the STAT1 gene respond to stem cell transplantation. It involved 15 young patients from nine different countries, each suffering a range of complications caused by the gene's mutation.

Of these, only six survived a regime of stem cell transplantationwith five completely cured and disease free by the study's conclusion.

The study was carried out by Dr. Satoshi Okada (Hiroshima University), Professor Jennifer Leiding (University of Florida), Professor Tomohiro Morio (Tokyo Medical and Dental University), and Professor Troy Torgerson (University of Washington).

Dr. Okada, who first discovered the STAT1 gain of function mutation in 2011, says, "Overall, this result is disappointing but the fact that five patients were cured proves that treatment with stem cells can work, and we now need to learn from these 15 individual cases."

The STAT1 gene plays a vital role in the body's immune system. Rare mutations can lead to STAT1's over-activation (GOF) and autoimmunity.

While the majority of patients afflicted typically show mild to moderate symptoms involving fungal (mostly Candida), bacterial, and viral infectionsabout 10 percent of cases are severe and life threatening.

Until now, developing suitable treatments has been challenging; e.g. anti-fungal drugs temporarily treat the symptoms but not the source mutation, and immunosuppressive therapies often do more harm than good by knocking out already overburdened immune systems.

With only one confirmed case prior to this study of a sufferer being successfully cured using stem cell transplantation, researchers are keen to build an understanding of best practices in order to offer real hope for the typically young sufferers of this condition.

The 15 selected patients were sourced via an international appeal to transplant centers and consortiums. Their ages ranged from 13 months to 33 years at the time of treatment. Screening by HU researchers confirmed that each had the STAT1-GOF mutation, and that the mutation was the source of their ailments.

Treatment was carried out independently by centers around the world. It used chemotherapy to eradicate the host's bone marrowthe source of the damaging STAT1 mutation in these patients. Healthy stem cell cultures sourced from donors were then transplanted into the subjects with the aim of reconstituting their bone marrow to a mutation-free, disease-fighting state.

The researchers suspect three reasons for the low 40 percent success rate:

In response, the researchers have made several proposals for improving this treatment. Due to most of the patients having mild to moderate ailments, only those suffering from severe symptoms should undergo this treatment. In addition, the chemotherapy dosage should be reduced. Those who received low-dose chemotherapy reacted better.

However, a balance must be struck. Low-dose chemotherapy may not eradicate host bone marrow to the extent required for its reconditioning the chance of transplant rejection is thus increased. With this in mind, support treatment may be required to neutralize host antibodies and prevent attacks of introduced stem cells.

Finally, due to the relative success seen in younger patients, stem cell transplantation should occur at as early an age as possible. Due to recent advancements in STAT1-GOF diagnosis, early detection is now a very real possibility hopefully leading to greater success rates, and less suffering for those carrying this potentially devastating mutation.

Explore further: 'Smart' genetic library makes disease diagnosis easier

More information: Jennifer W. Leiding et al. Hematopoietic stem cell transplantation in patients with Gain of Function STAT1 Mutation, Journal of Allergy and Clinical Immunology (2017). DOI: 10.1016/j.jaci.2017.03.049

Researchers at Hiroshima University have developed a smart genetic reference library for locating and weeding out disease-causing mutations in populations.

A single blood test and basic information about a patient's medical status can indicate which patients with myelodysplastic syndrome (MDS) are likely to benefit from a stem cell transplant, and the intensity of pre-transplant ...

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year ...

Physicians at the University of Illinois Hospital & Health Sciences System have cured 12 adult patients of sickle cell disease using a unique procedure for stem cell transplantation from healthy, tissue-matched siblings.

Using a technique that avoids the use of high-dose chemotherapy and radiation in preparation for a stem cell transplant, physicians at the University of Illinois Hospital & Health Sciences System have documented the first ...

A large, nationwide study published in the journal JAMA Oncology found that people who received transplants of cells collected from a donor's bone marrow the original source for blood stem cell transplants, developed decades ...

Researchers at UC Berkeley have found unexpected effects of viral infections, a discovery that may explain why viruses can make people feel so lousy.

In news that may bring hope to asthma sufferers, scientists discover a mechanism that provides a possible new target for allergy treatments.

(HealthDay)Administration of allergen immunotherapy (AIT) in patients with allergic asthma leads to lower short-term symptom and medication scores, according to a review published online May 19 in Allergy.

A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland.

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Virtually the entire population of sub-Saharan Africa, and some 70% of African Americans, carry a gene variant (allele) which results in a trait referred to as Duffy-negative. It has long been known that carriers of this ...

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Stem cell treatment for lethal STAT1 gene mutation produces mixed results - Medical Xpress

The Lipogems technology has great promise, but experts say itll take time to assess how successful the new procedure is.

What if you could put that little bit of body fat around your midsection to good use?

A procedure called Lipogems utilizes a persons body fat as a source of stem cells to help treat arthritis and joint conditions.

At least thats the promise.

Lipogems was approved for widespread use by the Food and Drug Administration (FDA) in November 2016, and its already garnering a lot of attention.

Rush University Medical Center recently became the first sports medicine specialists in the Midwest to offer treatment with the device.

The technology is ideal for patients with certain orthopedic conditions, such as painful joints including the knee, ankle, or shoulder with limited range of motion. Additionally, it can be used in soft tissue defects located in tendons, ligaments, and/or muscles to improve the biologic environment, said Dr. Brian Cole, professor of orthopedic surgery, and section head of the Rush Cartilage Restoration Center, in a press release.

Read more: Stem cell therapies offering hope for MS patients

Stem cells work by growing and differentiating themselves into different cells in the body based on the site of injection.

They are believed to help the natural regenerative processes in the body.

Hence they have earned the nickname as mini drug stores based on their ability to secrete a spectrum of bioactive molecules and support the natural regeneration of focal injuries.

Stem cells can be harvested from certain parts of the human body, most notably bone marrow and adipose tissue (fat).

Harvesting bone marrow stem cells is a significantly more invasive and time-consuming procedure that is performed using general anesthesia.

Lipogems offers a novel approach to orthopedic stem cell treatments by using a persons own fat.

The procedure uses a small incision into an area of subcutaneous fat, from which a quantity of fat tissue is harvested and processed by the Lipogems apparatus.

The technology itself, which really is the device that processes the fat, creates a concentration of fat that has been cleansed of all the extraneous things like red blood cells and fibrous tissues, Cole told Healthline.

The concentrated stem cells within that fat tissue are then applied to the problematic joint or bone area.

The procedure can be completed in under 30 minutes.

Read more: Stem cell therapy a possible treatment for rheumatoid arthritis

Lipogems offers a streamlined procedure for stem cell treatment, but there is nothing new about the science itself.

The use of stem cells to treat a variety of conditions has been ongoing for some time now.

As Healthline reported earlier this year, stem cells have been touted as a breakthrough treatment for some time, but real proof of efficacy is still being researched.

The same is true for Lipogems.

What were lacking is really good data at this point in the clinical setting, Cole said. There is substantial data in the laboratory suggesting that these cells may function in the way Ive described: reducing inflammation and so forth. But, we really dont have yet much in the way of good solid clinical data saying that definitively this is making a difference.

He further cautions individuals thinking that the new procedure, or that stem cells in general, are a panacea.

Read more: Unproven stem cell treatments offer hope but also risks

Instead, he would like those seeking orthopedic treatment to understand that Lipogems is just one part of a much larger and more complex suite of tools used by physicians.

It has to be taken into context of all the other possible treatment options, from simply icing down a swollen ankle, to changing your daily activity, to surgery.

The unfortunate thing is that people think, well this is the solution that can be used instead of, say, a joint replacement and no longer do we need to do surgery, said Cole.

Nothing could be further from the truth.

Nonetheless, Cole and his team are still excited about the possibilities of the Lipogems procedure.

Using a readily available and easily accessible substance like fat as a source of stem cells could have far-reaching implications for procedures in the future.

Were optimistic and intuitively there is a good argument to be made that this is as good or better than any other source of stem cells, said Cole.

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New Technology Uses Body Fat to Help Relieve Joint Pain - Healthline

This week alone in the news weve seen air strikes, suicide bombings and murders caused by hate. Violence over the hue of someones skin, the way they speak or how they dress. Hate-filled speech by neighbors at meetings and on Facebook. At dinner yesterday, my 8-year-old step-daughter asked her dad, Whats a bomb? My heart is heavy.

Its easy to forget that we are more alike than we are unalike. I offer to you a different perspective.

Six years ago my brother got the call he had Hodgkin Lymphoma, a cancer that starts in cells that are part of the bodys immune system. He was 28 years old. It started as a visible lump under his collar bone, and sometimes you wonder how can so much suffering be caused by such a little lump? And so my story begins.

About one year into his treatment, he reached remission, and from there he was required to undergo an autologous stem cell transplant (a transplant using his own stem cells) to replace his bone marrow and stem cells that were destroyed by chemotherapy and radiation. Fast-forward 10 or so months and my brothers cancer returned. This time the treatment plan had to change his body needed help actually fighting the cancer cells, rather than just a replenishment of normal blood cells. This time around, he required an allogeneic stem cell transplant (a transplant using the stem cells from a healthy donor) and as his sister, I needed to be tested to see if I was a tissue match.

This was all new to me and our family. You hear a lot about cancer. We all know someone who has it, if you dont have cancer yourself. But I knew nothing about stem cell transplants or what it meant to be a donor. First we had to find out if my brother and I were a match.

I received a kit in the mail and all I had to do was swab the inside of my cheek, place the swab inside a sealed bag, and mail it back to the hospital. A week or so later, my brother got the news from his doctor that changed our lives. I was, in fact, a match a near perfect match and we could move forward with his second stem cell transplant.

At this point in my story, youre probably thinking, Of course, youd be a match, youre his sister. I assumed so, as well. Read on.

On Aug. 12, 2016, my brother and I underwent our stem cell transplant at Dana Farber/Brigham and Womens Hospital in Boston. There are two different ways to donate stem cells peripheral blood stem cells (stem cells extracted from your blood after receiving five days of injections of a drug called filgrastim, used to increase the number of blood-forming cells in your bloodstream) and bone marrow (a surgical procedure where doctors use needles to withdraw liquid marrow from both sides of the back of your pelvic bone). Due to my brothers specific treatment plan, he required pure bone marrow, and my bone marrow was taken from my pelvis. Two liters worth of my bone marrow was processed at Dana Farber and then brought to my brother immediately, who received it via an IV drip.

So how does my story end? Why am I telling you all this?

My brother is thriving. My pelvis has healed. And we were absolutely blessed to find a match right within our family.

The reality is that fewer than 30 percent of patients with a blood cancer or blood disease will find a related-donor; the other 70 percent, thousands of patients with blood cancers like leukemia and lymphoma, sickle cell anemia or other life-threatening diseases, depend on the national bone marrow registry to find a match to save their life. Some day you or someone you love might depend on a complete stranger who might be a Muslim, a Republican, gay or straight. But it wont matter because from the inside, they will be the same.

I plead with you to remember that we are more alike than we are unalike, and to do something positive for humanity.

You can visit http://www.bethematch.org and join the Be The Match national bone marrow registry.

Or you can attend one of my in-person donor drives in Greenfield over the next few months. The first will be this Saturday, June 10, from 2 to 4 p.m. at the Pints in the Park event at the Greenfield Energy Park.

If you are between the ages of 18 and 44, patients especially need you. You could be someones cure.

I note the obvious differences

between each sort and type,

but we are more alike, my friends,

than we are unalike.

We are more alike, my friends,

than we are unalike.

From Human Family, a poem by Maya Angelou

Ashli Stempel is a Greenfield resident and a member of the Greenfield Town Council.

Excerpt from:
My Turn: Do something within your power to save another life - The Recorder

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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Mice with ALS improve with stem cell therapy - The Ledger

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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Nepal's sole bone marrow transplant doctor - Republica

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 toChrisvanDoomwith 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.

Link:
Vancouver woman's family pleading for help finding a bone marrow donor - CBC.ca

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."

Go here to see the original:
Pitting avocados against leukemia stem cells - CBC.ca

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.

Visit link:
Donor appeal for poorly toddler 'may have saved other lives' - Bicester Advertiser

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.

The rest is here:
Chippewa Valley Schools hosts bone marrow registration drive - The Macomb Daily

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

More here:
Bone marrow transplant patient credits positive recovery to staying close to home - KSFY

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.

Visit link:
Oxford University staff join bone marrow stem cell donor drive for Oxford toddler Ally Kim - Witney Gazette

(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.

Click here to read full story

Continue reading here:
Stem Cells Fast Facts - KABC

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 ...

Link:
Want to save a life? Cuban American searches for bone marrow donor - Miami Herald

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.

Original post:
Oxford University staff join bone marrow stem cell donor drive for ... - Oxford Mail

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.

Read more:
Donor appeal for poorly toddler 'may have saved other lives' - Witney Gazette

Blood stem cells are things of wonder: hidden inside each single cell is the power to reconstitute an entire blood system, like a sort of biological big bang.

Yet with great power comes greater vulnerability. Once these master cells are compromised, as in the case of leukemia and other blood disorders, treatment options are severely limited.

A bone marrow transplant is often the only chance for survival. The surgery takes a healthy donors marrowrich with blood stem cellsand reboots the patients blood system. Unfortunately, like organ transplants, finding a matching donor places a chokehold on the entire process.

According to Dr. George Daley at Harvard Medical School, a healthy sibling gives you a one in four chance. A stranger? One in a million.

For 20 years, scientists have been trying to find a way to beat the odds. Now, two studies published in Nature suggest they may be tantalizingly close to being able to make a limitless supply of blood stem cells, using the patients own healthy tissues.

"This step opens up an opportunity to take cells from patients with genetic blood disorders, use gene editing to correct their genetic defect and make functional blood cells," without depending on donors, says Dr. Ryohichi Sugimura at Boston Childrens Hospital, who authored one of the studies with Daley.

Using a magical mix of seven proteins called transcription factors, the team coaxed lab-made human stem cells into primordial blood cells that replenished themselves and all components of blood.

A second study led by Dr. Shahin Rafii, a stem cell scientist at Weill Cornell Medical College took a more direct route, turning mature cells from mice straight into genuine blood stem cells indiscernible from their natural counterparts.

This is the first time researchers have checked all the boxes and made blood stem cells, says Dr. Mick Bhatia at McMaster University, who was not involved in either study, That is the holy grail.

The life of a blood stem cell starts as a special cell nestled on the walls of a large blood vesselthe dorsal aorta.

Under the guidance of chemical signals, these cells metamorphose into immature baby blood stem cells, like caterpillars transforming into butterflies. The exact conditions that prompt this birthing process are still unclear and is one of the reasons why lab-grown blood stem cells have been so hard to make.

These baby blood stem cells dont yet have the full capacity to reboot blood systems. To fully mature, they have to learn to respond to all sorts of commands in their environment, like toddlers making sense of the world.

Some scientists liken this learning process to going to school, where different external cues act as textbooks to train baby blood stem cells to correctly respond to the body.

For example, when should they divide and multiply? When should they give up their stem-ness, instead transforming into oxygen-carrying red blood cells or white blood cells, the immune defenders?

Both new studies took aim at cracking the elusive curriculum.

In the first study, Daley and team started with human skin and other cells that have been transformed back into stem cells (dubbed iPSCs, or induced pluripotent stem cells). Although iPSCs theoretically have the ability to turn into any cell type, no one has previously managed to transform them into blood stem cells.

A lot of people have become jaded, saying that these cells dont exist in nature and you cant just push them into becoming anything else, says Bhatia.

All cells in an organism share the same genes. However, for any given cell only a subset of genes are turned into proteins. This process is what gives cells their identitiesmay it be a heart cell, liver cell, or blood stem cell.

Daley and team focused on a family of transcription factors. Similar to light switches, these proteins can flip genes on or off. By studying how blood vessels normally give birth to blood stem cells, they found seven factors that encouraged iPSCs to grow into immature blood stem cells.

Using a virus, the team inserted these factors into their iPSCs and injected the transformed cells into the bone marrow of mice. These mice had been irradiated to kill off their own blood stem cells to make room for the lab-grown human replacements.

In this way, Daley exposed the immature cells to signals in a blood stem cells normal environment. The bone marrow acts like a school, explains Drs. Carolina Guibentif and Berthold Gttgens at the University of Cambridge, who are not involved in the study.

It worked. In just twelve weeks, the lab-made blood stem cells had fully matured into master cells capable of making the entire range of cells normally found in human blood. Whats more, when scientists took these cells out and transplanted them into a second recipient, they retained their power.

This a major step forward compared with previous methods, says Guibentif.

In contrast, the second study took a more direct route. Rafii and team took cells lining a mouses vessels, based on the finding that these cells normally turn into blood stem cells during development.

With a set of four transcription factors, the team directly reprogrammed them into baby blood stem cells, bypassing the iPSC stage.

These factors act like a maternity ward, allowing the blood stem cells to be born, says Guibentif.

To grow them to adulthood, Rafii and team laid the cells onto a blanket of supporting cells that mimics the blood vessel nursery. Under the guidance of molecular cues secreted by these supporting cells, the blood stem cells multiplied and matured.

When transplanted into short-lived mice without a functional immune system, the cells sprung to action. In 20 weeks, the mice generated an active immune response when given a vaccine. Whats more, they went on to live a healthy 1.5 yearsroughly equivalent to 60 years old for a human.

Rafii is especially excited about using his system to finally crack the stem cell learning curriculum.

If we can figure out the factors that coax stem cells to divide and mature, we may be able to unravel the secrets of their longevity and make full-fledged blood stem cells in a dish, he says.

Calling both experiments a breakthrough, Guibentif says, this is something people have been trying to achieve for a long time.

However, she points out that both studies have caveats. A big one is cancer. The transcription factors that turn mature cells into stem cells endow them with the ability to multiply efficientlya hallmark of cancerous cells. Whats more, the virus used to insert the factors into cells may also inadvertently turn on cancer-causing genes.

That said, neither team found evidence of increased risk of blood cancers. Guibentif also acknowledges that future studies could use CRISPR in place of transcription factors to transform cells into blood stem cells on demand, further lowering the risk.

The techniques will also have to be made more efficient to make lab-grown blood stem cells cost efficient. Itll be years until human use, says Guibentif.

Even so, the studies deter even the most cynical of critics.

After 20 years, were finally tantalizingly close to generating bona fide human blood stem cells in a dish,"says Daley.

Image Credit: Pond5

Read more from the original source:
Limitless Lab-Grown Blood Is 'Tantalizingly Close' After 20 Years - Singularity Hub

Malaria parasites (green) are killing this red blood cell, but a new study suggests they also damage bone.

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By Mitch LeslieJun. 2, 2017 , 2:00 PM

Malaria parasites leave a trail of destruction in an infected persons body. The microscopic invaders massacre red blood cells, produce harmful chemicals, and sometimes damage the brain. A new mouse study suggests that the parasites can also weaken bones. If they do the same in people, they could stunt the growth of children infected with the disease. But the study also provides some good news, identifying a potential way to prevent the skeletal decline with a compound similar to vitamin D.

Its important work, says parasitologist Regina Joice Cordy of Emory University in Atlanta, who wasnt connected to the study. Its taken us a step further, she adds, in understanding the long-term effects of malaria infections.

Malaria parasites, which are transmitted through the bite of an infected mosquito, cause the most destruction during the part of their life cycle when they dwell in red blood cells circulating through the body. There, they reproduce and feast on oxygen-carrying hemoglobin proteins, releasing noxious byproducts. The parasites eventually explode from the blood cells, killing them in droves. Although researchers have also detected the parasites in bone marrow, where blood-forming stem cells reside, no one has known until now whether they damage the skeleton.

To find out, a team led by graduate student Michelle Lee and immunologist Cevayir Coban of Osaka University in Japan infected mice with either of two species of malaria parasites. The rodents immune systems fought off the parasites, but the animals skeletons showed the effects of the infection. We found bone loss for both types of infections, Coban says. In adult mice, the spongy material inside the bones began to break down. It contained more gaps, and support structures were thinner and less numerous. Similar changes occur in the bones of people with osteoporosis, Coban says.

In young mice, the bones also grew slower than normal. As a result, the animals thigh bones were about 10% shorter than those of their uninfected counterparts, the researchers report online today in Science Immunology.

The parasites might trigger these problems, the scientists hypothesized, by upsetting the normal balance between cells known as osteoclasts, which dissolve bone, and cells called osteoblasts, which build it back up. The researchers discovered that both types of cells shut down when the mice were infected with malaria. Once the animals had eliminated the parasites, both cell types started working again. But bone breakdown outpaced bone restoration, suggesting that osteoclasts were working harder.

Why do the mices bones deteriorate even after their immune system ousted the parasites? Lee, Coban, and colleagues suspected that the culprit was chemical waste released by the parasites, including the residue of digested hemoglobin, a molecule called hemozoin. In malaria-infected mice, the researchers found, hemozoin seeped into the bones, turning them black. It was still there 2 months after the parasites had been eliminated. To gauge the impact of hemozoin and other parasite wastes, the team cultured bone marrow cells in a cocktail of these substances. The mixture spurred the cells to release inflammation-promoting molecules known to spur osteoclast production.

That mechanism suggested a way to block the parasites bone-destroying effects. Coban and colleagues gave infected mice alfacalcidol, a derivative of vitamin D that treats osteoporosis by suppressing osteoclasts and stimulating osteoblasts. The drug prevented bone loss in the mice.

Cordy says the proposed bone-destroying mechanism is plausible. The key question, she says, is whether it occurs in humans. So far, Coban says, the researchers dont have direct evidence that malaria triggers bone loss in people. Children in malaria-prone areas often grow abnormally slowly, but researchers arent sure whether malaria or other diseases that are prevalent in these areas are to blame. If further studies confirm the new findings, treating kids with alfacalcidol or related molecules, along with antimalarials, might lead to a growth spurt.

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Malaria may weaken the skeleton - Science Magazine

Two separate research teams have succeeded in generating blood stem cells using completely different procedures. One team was led by stem cell biologist Dr. George Q. Daley of Harvard Medical School and Boston Childrens Hospital. The other team was spearheaded by Dr. Shahin Rafii of the Weill Cornell Medicines Ansary Stem Cell Institute in New York.

In both cases, reprogrammed blood stem cells were able to successfully produce blood cells when implanted into mice. And if either or both procedures turn out to be viable for humans, a future where blood donors will no longer be needed may soon be in the horizon because science has provided us with a way to produce unlimited blood supply.

Stem cells are specially programmed cells that are responsible for creating all of the bodys other cells. There are two types of stem cells embryonic and adult. Embryonic stem cells are located you guessed it in the embryo where they stay before they start to specialise. Adult stem cells are the ones used to repair and replace worn out or old cells.

Those are the natural types. Theres another type, though. Theyre called induced pluripotent stem cells (iPS cells for short). Unlike the first two types, iPS cells arent naturally present. Theyre actually adult stem cells that were converted back to their primitive state, which means they can be coaxed to turn into any type of cell.

Dr. Daley and his team chose to use both embryonic stem cells and iPS cells for their research. Using a combination of proteins, they coaxed the cells to turn into hemogenic endothelium a kind of embryonic tissue that eventually turns into blood stem cells. Next, they tested several transcription factors genes that tell other genes what to do until they came up with the combination (specifically: ERG, HOXA5, HOXA9, HOXA10, LCOR, RUNX1, and SPI1) that pushed the hemogenic endothelium into a blood-forming or blood stem cell state. They then injected those modified cells into the bone marrow of their mice subjects. After several weeks, portions of the mices blood and bone marrow developed different types of blood cells, including red blood cells, white blood cells, and even immune cells.

As Daley described the feat: Were tantalizingly close to generating bona fide human blood stem cells in a dish.

On the other hand, Rafii and his team chose a different route. They didnt make use of iPS cells. Instead, they created true blood stem cells, starting off by extracting stem cells from the blood vessel lining of mature mice. Next, they inserted transcription factors (Fosb, Gfi1, Runx1, and Spi1) into the genomes of the extracted cells, then kept these cells in Petri dishes designed to replicate the environment within human blood vessels.

Over time, the cells turned into blood stem cells and multiplied. They then injected those stem cells into mice treated with radiation (which meant most of their blood and immune cells were gone). The stem cells regenerated not just the blood, but the immune cells too. Consequently, the mice recovered and went on to live for over 1.5 years in the lab.

As described by Rafii, the procedure they used is similar to a direct aeroplane flight, while Daleys is like a flight that took a detour prior to reaching its ultimate destination. Doing away with the iPS part kind of makes Rafiis method slightly better than Daleys because it minimizes the threat of tumors forming or the body rejecting the stem cells, which is a typical reaction that iPS cells might cause. But if Daleys team is able to refine their process to eliminate this risk, then that will level the playing field, so to speak.

Whatever happens from here on, both procedures are nonetheless considered significant breakthroughs. And even though its not yet certain which method will turn out to be the better one for humans, whats clear is that both methods have the potential to be game-changers when it comes to any kind of treatment involving blood infusion and transfusion.

Both studies have been published in the journal Nature, with Daleys under the title Haematopoietic stem and progenitor cells from human pluripotent stem cells and Rafiis under the title Conversion of adult endothelium to immunocompetent haematopoietic stem cells.

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Scientists Close to Generating Unlimited Blood Supply from Stem Cells - Wall Street Pit

The Hindu

Unrelated donor transplants to aid thalassemics The Hindu Transplantation of a special kind of stem cells found in the bone marrow has been the only curative option for patients with thalassemia major (genetic inability to produce normal, adult haemoglobin leading to severe anaemia). Since only 30-35% of such ...

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Unrelated donor transplants to aid thalassemics - The Hindu

Kansas City Star

Sickle cell cure is real, as this Kansas patient proves Kansas City Star Intense pain. Fatigue. Repeated infections, emergency room visits and hospitalizations. Desiree Ramirez endured them often until she became the first adult cured at a Kansas hospital of sickle cell disease. Bone marrow stem cells donated by a ...

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Sickle cell cure is real, as this Kansas patient proves - Kansas City Star

By By Elianna Novitch, The Gazette

May 29, 2017 at 5:00 am | Print View

IOWA CITY More than 20 million people are registered as bone marrow donors in the Be the Match registry, the largest and most diverse donor registry in the world.

But none can help Calder Wills, a 12-year-old Iowa City boy battling stage 4 T-cell lymphoma, or cancer of the blood.

Only one person has been identified as a 100-percent match for Calder, but that person was deemed medically unable to donate bone marrow.

This has left the Wills family with few options.

And so, friends of the family are hosting a donor registry drive on Tuesday to raise awareness about the need for more marrow donors and to perhaps find a match for Calder and others like him.

The event takes place from 3 to 8 p.m. inside the gym at Hoover Elementary School, 2200 E. Court St., Iowa City. Those who attend can join the Be The Match registry. Those who are unable to attend can register online at bethematch.org.

Calder was diagnosed with lymphoma in February 2016. He went into remission within the first 30 days but found out on April 11 the day after his 12th birthday that he had relapsed and would need a bone-marrow transplant. He is one of thousands searching for a match.

He is among the 70 percent of patients who surprisingly dont have a match in their own family, explained Colleen Reardon, manager of the Iowa Marrow Donor Program at the University of Iowa Hospitals & Clinics. We are looking for a tissue type match and each sibling has about a 25 percent chance of being a match.

Calder has three siblings, a twin brother Grayson and sisters Charlotte, 7, and Arden, 5, all of whom were not matches. The next best chance a patient has, statistically, is to find an unrelated donor that is a 100-percent match.

Calders mother Brianna Wills described it as devastating when the family found out that the 58-year-old woman who matched with Calder was deemed medically unable to donate.

That left us with no match, no options, she said. Weve decided to pursue cord blood for his transplant, Wills said. He is going to have a cord blood transplant at the University of Minnesota because a bone marrow match wasnt available and he couldnt wait until one became available.

According to the Be The Match website, cord blood is one of three sources of blood-forming cells used in transplant. The others are bone marrow and peripheral blood stem cells. Cord blood can be used to treat more than 80 diseases, including blood cancers like leukemia and lymphoma. Cord blood comes from a babys umbilical cord.

Wills said that even though Calder is receiving a different type of transplant, she does not want people to not register as a marrow donor.

I dont want that to dissuade people from continuing to do it because he has about a two out of three chance that this transplant will fail because he has T-cell lymphoma that is very aggressive and very hard to treat, Wills said. Realistically, statistically, we are looking at him needing a second transplant down the road and thats when we hope that well find a donor and we can use a bone marrow match then.

Please still do it and not just for Calder, do it for the thousands of people who also dont have a match.

According to Reardon, of every 540 people who register as a donor, only one will be identified as that perfect match for someone and be asked to donate.

Were not realistically hoping to find Calders donor, I mean that would be amazing, but really were hoping to expand the database. Were just hoping that some family in Texas or somewhere else in the world is also doing this and maybe theyll find Calders donor, Wills said. If were all doing it, were going to expand the database for everyones benefit.

Wills recognizes that even though the drive is in Calders honor, it is truly to the benefit of thousands of other people who dont have donors.

There are other ethnic groups that have very little participation and to be a match you need to be matched with donors that have similar ethnic background as you do, Wills said. So African Americans, Hispanics, people that have mixed races, or Asian background wed love to have them come because there are people waiting for donors of all kinds of backgrounds.

What: Bone Marrow Donor Drive

When: 3 to 8 p.m. Tuesday

Where: Hoover Elementary School, 2200 E. Court St., Iowa City

Details: Join the Iowa Marrow Donor Program and Be The Match Registry using a simple cheek swab.

Info: join.bethematch.org/CalderStrong or call the Iowa Marrow Donor Program at (319) 356-3337.

l Comments: (319) 368-8538; elianna.novitch@thegazette.com

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A Devon family are swimming to Great Ormond Street Hospital to help a mum whose 15-month-old son has been diagnosed with very rare syndrome called Diskeratosis Congenita. Little Max Hilton's treatment is reliant on a steady supply of stem cell donors and after being around children with similar conditions Max's mum, Becca, is determined to encourage donors to come forward.

Through a touching Facebook group called Be There For Buzz Man Becca, has charted her son's journey and the difficulty they both face.

Becca's North Devon family have sprung into action to help spread the message that the UK needs more Stem Cell donors and to raise funds for the Antony Nolan Register, an organisation dedicated to researching stem cells and matching donors to those in need of help.

"We're delighted with the support we have received by so many people in aid of raising money for Anthony Nolan, including Reef, Tace and Aimee who are based in North Devon, have organised a charity swim called Swimming to Max; swimming 250 miles from Barnstaple to Romford, the distance between them and Max, over 20 weeks to raise as much as they can for the charity," said Becca.

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"Nobody ever expects their newborn child to be diagnosed with such a rare condition, to see him fighting every day is extremely painful, and to see so many brave children in the same ward really does showcase the need of more stem cell and bone marrow donors. Great Ormond Street Hospital are doing all they can, and we'd like to thank the staff for providing invaluable support to both Max and our family.

"For us, converting the negativity we have experienced with Max into making a positive impact for other patients in the same position will make our day.

"If just one person who reads our story decides to see if they're eligible, that could then continue to save a life. Please don't let it affect someone you love to then decide to register. There are so many patients waiting for suitable donors."

For Becca, telling Max's story is not just important for friends and family, but primarily to raise awareness of the desperate need for donors.

To see if you're eligible to donate stem cells, you must be 16 or over, and it is as easy as spitting in a cup to provide a saliva sample for Anthony Nolan to then assess eligibility to then donate - all done through a free sample kit sent via post, from their website.

Donating bone marrow and stem cells is not invasive at all; 9 out of 10 people donate stem cells via the bloodstream, in a procedure called peripheral blood stem cell collection. One in 10 people will have stem cells taken from the bone marrow itself, whilst under general anaesthetic.

Neither procedure hurts, and it's time more is done to increase the people on the register so patients, similar to Max, have a chance in recovering from their rare conditions with the help of those that are genetically matched to their blood type.

The Be There For Buzz Man Facebook page can be found at http://www.facebook.com/buzzman11, and to find out how to donate stem cells visit http://www.anthonynolan.org.

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Baby Max can only survive with a constant supply of stem cells - Devon Live