Anyone can be a bone marrow donor, but when it comes to finding a match, race can be everything. There are certain genetic markers that doctors will look for when searching for a match -- and if a match is made, a transplant can then be scheduled. If someone is in need of a transplant, the process can be daunting, especially if there is only a small pool of donors that share a similar ethnicity.

There are many bone marrow donor services throughout the country, but the Asian American Donor Program (AADP) is a champion nonprofit dedicated to increasing the availability of potential stem cells donors for patients with life threatening diseases curable by a stem cell transplant. Based in Alameda, CA, AADP holds donor registration drives and outreach events to Asian, Pacific Islander, and mixed race communities in the Bay Area.

Stem cells are found inside bone marrow, and those cells can turn into red blood cells, white blood cells and platelets. AADP explains that red blood cells carry oxygen throughout the body; white blood cells help fight infections; and platelets help control bleeding. Diseases like leukemia, sickle cell anemia, blood cancers, and many other immune diseases can be treated with a bone marrow or stem cell transplant. This soft tissue is incredibly important to our health.

To learn more about why bone marrow donation is important, and why it is particularly important in Asian Pacific American and mixed race communities, I reached out to Ruby Law, AADP's Recruitment Director.

Hyphen: When does one need a bone marrow donation, and what does it do?

Ruby Law: Disease can affect the marrows ability to function. When this happens, a bone marrow or cord blood transplant could be the best treatment option. For some diseases, transplant offers the only potential cure. A bone marrow or cord blood transplant replaces unhealthy blood-forming cells with healthy ones. Blood-forming cells are also called blood stem cells. Blood stem cells are immature cells that can grow into red blood cells, white blood cells and platelets. Every year, 12,000 patients with blood diseases such as leukemia and lymphoma, sickle cell and other life-threatening diseases need a bone marrow or umbilical cord blood transplant.

Hyphen: Why is bone marrow donation important for Asian Pacific American and mixed-Asian Pacific Americans communities to address in discussions about health?

RL: A patient needs a matching donor for a successful transplant. The closer the match, the better for the patient. Patients are more likely to match someone from their own race or ethnicity. For example a Chinese patient will most likely need a Chinese donor, while a Japanese patient will most likely need a Japanese donor. Out of 10 million registrants in the United States, only 7% of the registrants are Asian and only 4% are of mixed race. Most Asian or Mixed Asian patients cannot find any matching donor in the registry because there are not enough Asian, mixed Asian and minority donors.

Ruby Law, Asian American Donor Program (AADP) Recruitment Director

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Down to the Bone: The Need for API Bone Marrow Donors

KINDHEARTED Andrew Gibson is giving somebody the gift of life, after being inspired by a workmates little boy.

Andrew, 29, from Southend, signed up to the bone marrow transplant register after hearing about 21-month-old Jack Kleinberg.

Jack, of St James Gardens, Westcliff, is facing the second bone marrow transplant in his short life to help him beat two life-threatening conditions.

His parents are hoping the op will fight the effects of Wiskott Aldrich syndrome and familial Mediterranean fever.

After hearing Jacks story, from Jacks mum, Vicki Parrott, a workmate at the Hood Groups Southend insurance office, Andrew donated stem cells for use by an un-named patient in need.

Andrew was disappointed to learn he wouldnt be a match for Jack, but decided to go ahead all the same and Ms Parrott is delighted her son's example is helping others in need.

She said: At the office Christmas party, I found out Andrew, who had joined the Anthony Nolan bone marrow register when Jack first got ill, was recently called up as a match. He donated his stem cells a month ago to a stranger.

I couldn't believe it. I was so emotional and hugged him loads. I dont know if well ever meet Jacks donor, so this is the closest thing weve had.

Its overwhelming to think theres someone out there whos had a second chance at life because of Jacks story. Itsmade my year.

Andrew said: There was an email going around at work, urging people to sign up to the Anthony Nolan register, as a way of showing our support for Vicki and her son Jack, who had just been diagnosed. Id never heard of Anthony Nolan before, but I didnt hesitate. Seeing Vicki at the Christmas party really made it sink in what Id done. It was an emotional moment and it was clear how much it meant to her.

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Andrew donates bone marrow after hearing about brave boy

NEW DELHI: India may soon have an official database on stem cell donors and recipients. The health ministry is evaluating a proposal along with All India Institute of Medical Sciences (AIIMS) to create a donor registry as part of the National Health Mission (NHM), a senior official told TOI.

The proposal suggests enrolling all district hospitals in the first phase to seek stem cell details from across the country. "Once a stem cell donor registry is in place, a willing donor can be contacted and one can coordinate easily. Also, this would enhance access to safe blood," the official said.

Stem cells, found in bone marrow, are like building blocks which can grow into any normal cell of the body such as red blood cells to carry oxygen, white blood cells to fight infection, or platelets to stop bleeding.

Apart from the donor registry, the ministry is also looking at creating facilities for human leucocyte antigen (HLA) typing. HLA-typing is a process conducted for matching donors and recipients of stem cell. HLA-typing is necessary to minimize rejection of stem cell transplant, experts say.

Once created, this would be the first government registry in the country. Till now, such registries have been run in the country by a few NGOs such as Bharat Stem Cells.

According to Bharat Stem Cells, there is usually 25% chance of a patient finding a matching donor within the family. The rest depend on unrelated voluntary stem cell donors.

Stem cell therapy has been shown to be effective in various blood disorders and in treatment of cancer. It is widely used in bone marrow transplantation. However, stem cell treatment remains expensive because of limited research as well as unavailability and lack of coordination between donors and recipients. Some private hospitals charge as much as Rs 1 lakh per session for stem cell therapy. On an average, stem cell treatment is estimated to cost around Rs 15-16 lakh.

According to the official, the idea behind including stem cell into NHM is to make it affordable by creating records and providing facilities.

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Stem cell registry will make cancer treatment cheaper

John and Leslie Malone pose with Maikel at Harmony Sporthorses, December 2, 2014.

The largest ever cash donation to Colorado State University stems from a novel treatment to get a dressage horse with a bum knee back into the show ring.

John and Leslie Malone's $42.5 million gift, announced Monday, will create the CSU Institute for Biologic Translational Therapies in the College of Veterinary Medicine and Biomedical Sciences, a 100,000-square-foot facility to develop stem-cell research into commercially viable treatments for animals and humans.

"This is the largest cash gift in the history of the university and it's absolutely staggering," said Brett Anderson, CSU's vice president for advancement. "It really allows us to be the best in the nation."

The Malone money will fund half of the $65 million cost to construct the facility. The school is looking for more donations to match the Malones' contribution. So far, an additional $10 million has been raised.

The Malones also provided $10 million to cover the Institute's operating expenses once the facility is built.

"The Malones have been so gracious. We asked them if they want to put their name on the building, but they said if it's helpful to you in order to get another major donor, we are happy to let you name it for someone else," Anderson said. "They are an incredible couple."

John Malone, who made his millions at the helm of Tele-Communications Inc. and now chairs the giant Liberty Media Corp., and his wife, Leslie, could not be reached for comment on Monday.

The Malones, who raise and train dressage and jumping horses on a ranch near Kiowa, last year donated $6 million to the school to establish the Leslie A. Malone Presidential Chair in Equine Sports Medicine.

They later brought Blixt, their dressage horse with a bad knee, to the vet school's Orthopaedic Research Center.

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Malones donate $42.5 million to CSU for new stem-cell research facility

CORDLIFE is now the largest network of private cord blood banks in Asia Pacific with state-of-the-art cord-blood and tissue processing and cryopreservation facilities in the country.

Once considered a medical waste, the blood left in the umbilical cordthe part of the placenta that delivers nutrients to a fetusafter a baby is delivery is now known to be a rich source of blood-forming stem cells.

These cells have been found to be potentially useful in treating diseases that require stem cell transplants (also called bone marrow transplants) such as certain kinds of leukemia or lymphoma, aplastic anemia (a blood disorder in which the bodys bone marrow doesnt make enough new blood cells), severe sickle cell disease and severe combined immunodeficiency.

Unlike with bone marrow, which is obtained through a painful medical procedure, there is only one chance to collect this seemingly precious stuff: immediately after the babys birth.

This is why a number of expectant parents in the country are being offered a chance to save stem cells from their babys umbilical cord blood via what is known as cord-blood banking.

Safeguard

Cordlife Philippines medical director Arvin Faundo said: Its a type of safeguard because the genetically unique stem cells have current and potential uses in medical treatment. No parent wishes his/her child to experience the heartbreaking effects of any illness. What we at Cordlife offer them is the chance to prepare for potential eventualitiesto secure the future well-being and happiness of their family.

Cordlife Philippines is a subsidiary of Cordlife Group Ltd., a company listed on the Singapore Exchange. Launched in February 2010 as the Philippines first and only cord-blood processing and cryopreservation facility, its facility was ISO-certified and built in accordance to global gold standards such as the American Association of Blood Banks.

The 365-day facility, located within UP-Ayala Land TechnoHub in Quezon City, is equipped with the worlds most advanced fully automated cord-blood processing system, the Swiss-made Sepax.

CordLife uses the US FDA-approved cryogenic storage pouch.

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Freezing newborns own stem cells for possible future use

Hard road: Cooper Densley gets a kiss from mother Olivia as brothers Jackson (left), and Fletcher play around him with father Andrew (right). Photo: Simon O'Dwyer

Santa Claus delivered some wonderful gifts to Cooper Densley this year, but none of them compare to one he received from his brother Jackson in October.

In a potentially life-saving exchange, Jackson Densley, 2, donated stem cells found in his bone marrow to his older brother Cooper, 4, three months ago.

Their parents,Oliviaand AndrewDensley, are hoping the transplant will help cure Cooper of a rare genetic condition he was diagnosed with last year: Wiskott-Aldrich Syndrome.

The disorder weakens the immune system, leaving sufferers vulnerable to infections, and it reduces the production of platelets - blood cells that keep bleeding under control.

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It means children suchasCooper can get extremely sick from common coughs and colds and a knock to the head while playing sport could trigger fatal bleeding in the brain.

The only known treatment is a stem cell transplant which can be derived from bone marrow or umbilical cord blood from a healthy donor whose tissue matches that of the recipient. When those cells are put in to the recipient's bloodstream, they can develop into normal immune cells and platelets.

Without a donation, the average life expectancy for people with the condition is 15 to 20 years.

Shortly after Mr and MrsDensleywere told about Cooper's diagnosis in 2013, MrsDensleyfell pregnant with their fifth baby, prompting hope blood from their newborn's umbilical cord could provide stem cells for Cooper.

Continue reading here:
Brother's transplant holds the gift of life for Densley family

Dr. Murray Howe and his hockey great father, Gordie Howe, on a fishing trip in Saskatchewan in 2013.

Hockey legend Gordie Howe is making a dramatic recovery from a serious stroke thanks to stem cell therapy developed by San Diego-based Stemedica, his family says. Some medical scientists aren't so sure, however.

Howe, 86, suffered the stroke in late October, leaving him unable to walk and disoriented. He began improving within hours after receiving the stem cells in early December, said Dr. Murray Howe, a radiologist and one of Howes sons. For example, Howe insisted on walking to the bathroom, which he previously could not do.

"If I did not witness my father's astonishing response, I would not have believed it myself," Murray Howe said by email Thursday. "Our father had one foot in the grave on December 1. He could not walk, and was barely able to talk or eat."

"Our father's progress continues," the email continued. "Today, Christmas, I spoke with him on FaceTime. I asked him what Santa brought him. He said 'A headache.' I told him I was flying down to see him in a week. He said, 'Thanks for the warning.'"

Howe is receiving speech and physical therapy at his home in Lubbock, Texas, and his therapists say he is much better than before receiving the stem cells.

Howe received the treatment from Novastem, a Mexican stem cell company that has licensed the use of Stemedica's cells for clinical trials approved by the Mexican government. Howe was given neural stem cells to help his brain repair damage, and stem cells derived from bone marrow to improve blood circulation in the brain. The procedure took place at Novastem's Clinica Santa Clarita in Tijuana.

Such use of unproven stem cell therapies outside the U.S. clinical trial system draws objections from some American health care professionals. They warn of the potential for abuse, say there's a lack of rigorous scientific standards, and call for tighter federal regulation of the proliferation of stem cell treatments.

Nevertheless, patients with ailments that don't response to approved treatments continue to seek such care. These patients and families say they have the right to make their own judgments. And they may not have time to wait for proof, so they're willing to take a chance.

Stemedica says it follows U.S. government law, and requires those licensing its stem cells in foreign countries to obey the laws of those countries.

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Did stem cells really help Gordie Howe?

Sacramento, Calif. (PRWEB) December 22, 2014

Using modified human stem cells, a team of UC Davis scientists has developed an improved gene therapy strategy that in animal models shows promise as a functional cure for the human immunodeficiency virus (HIV) that causes AIDS. The achievement, which involves an improved technique to purify populations of HIV-resistant stem cells, opens the door for human clinical trials that were recently approved by the U.S. Food and Drug Administration.

We have devised a gene therapy strategy to generate an HIV-resistant immune system in patients, said Joseph Anderson, principal investigator of the study and assistant professor of internal medicine. We are now poised to evaluate the effectiveness of this therapy in human clinical trials.

Anderson and his colleagues modified human stem cells with genes that resist HIV infection and then transplanted a near-purified population of these cells into immunodeficient mice. The mice subsequently resisted HIV infection, maintaining signs of a healthy immune system.

The findings are now online in a paper titled Safety and efficacy of a tCD25 pre-selective combination anti-HIV lentiviral vector in human hematopoietic stem and progenitor cells, and will be published in the journal Stem Cells.

Using a viral vector, the researchers inserted three different genes that confer HIV resistance into the genome of human hematopoietic stem cells cells destined to develop into immune cells in the body. The vector also contains a gene which tags the surface of the HIV-resistant stem cells. This allows the gene-modified stem cells to be purified so that only the ones resistant to HIV infection are transplanted. The stem cells were then delivered into the animal models, with the genetically engineered human stem cells generating an HIV-resistant immune system in the mice.

The three HIV-resistant genes act on different aspects of HIV infection one prevents HIV from exposing its genetic material when inside a human cell; another prevents HIV from attaching to target cells; and the third eliminates the function of a viral protein critical for HIV gene expression. In combination, the genes protect against different HIV strains and provide defense against HIV as it mutates.

After exposure to HIV infection, the mice given the bioengineered cells avoided two important hallmarks of HIV infection: a drop in human CD4+ cell levels and a rise in HIV virus in the blood. CD4+ is a glycoprotein found on the surface of white blood cells, which are an important part of the normal immune system. CD4+ cells in patients with HIV infection are carefully monitored by physicians so that therapies can be adjusted to keep them at normal level: If levels are too low, patients become susceptible to opportunistic infections characteristic of AIDS. In the experiments, mice that received the genetically engineered stem cells and infected with two different strains of HIV were still able to maintain normal CD4+ levels. The mice also showed no evidence of HIV virus in their blood.

Although other HIV investigators had previously bioengineered stem cells to be resistant to HIV and conducted clinical trials in human patients, efforts were stymied by technical problems in developing a pure population of the modified cells to be transplanted into patients. During the process of genetic engineering, a significant percentage of stem cells remain unmodified, leading to poor resistance when the entire population of modified cells is transplanted into humans or animal models. In the current investigation, the UC Davis team introduced a handle onto the surface of the bioengineered cells so that the cells could be recognized and selected. This development achieved a population of HIV-resistant stem cells that was greater than 94 percent pure.

Developing a technique to purify the population of HIV-resistant stem cells is the most important breakthrough of this research, said Anderson, whose laboratory is based at the UC Davis Institute for Regenerative Cures. We now have a strategy that shows great promise for offering a functional cure for the disease.

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New Technique for Bioengineering Stem Cells Shows Promise in HIV Resistance

A LITTLE boy faces his third bone marrow transplant before his second birthday.

Jack Kleinberg has battled against two life-threatening conditions as he suffers from familial mediterranean fever and WiskottAldrich syndrome, which affects one in 10 million children and means he has to live in virtual isolation.

His parents, Rob and Vicki, live with the knowledge that any part of his body can stop working at any time from a simple fall or infection.

The couple, of St James Gardens, Westcliff, spend much of their time travelling to Great Ormond Street Hospital for Jack to receive treatment to keep him alive.

The family includes Robs children from a previous relationship, Oliver, 14 and Sophia, ten.

Vicki, 28, said: Its a 24/7 job, but we wouldnt change it for the world. Oliver and Sophia didnt see Jack for the first year because he was in hospital. Its become normal for them to come home and wash and change into sterile clothes before they can see Jack, because of the danger of infection for him. Jack has had one full transplant and a top-up transplant and is waiting for a potential donor for a possible third transplant.

Vicki said: People think it is a painful process, but these days it is a stem cell transplant where if a donor is found to be suitable, they are given an injection the week before, which makes the body release bone marrowcells into the blood stream which are then taken like a normal blood donation. It takes just 20 minutes of someones time and saves so many lives. The transplants have given Jack 25 per cent of the cells he needs. Without them, he wouldnt have lived past his first birthday.

We are trying to get through Christmas and then we will decide on whether, if a donor is found, Jack has another full stem cell transplant or whether we let him live his life, with all its restrictions, for a while because he has spent so much time in hospital.

For more information about becoming a donor, visit http://www.anthonynolan.org

Rugby club is pitching in

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People urged to donate bone marrow as tot faces third transplant

SOUTH BEND, Ind.--- Mike and Katie have been a couple since college, but they've known each other much longer.

"We've been together forever," said Mike.

"I've actually known Mike since I was 5-years-old," said Katie.

A marriage and three kids later they've been through good times, and bad. The worst came nine-years-ago when Mike found out he had Huntington's disease.

Huntington's is a deadly, inherited disease that affects about 30,000 Americans; 150,000 more are at risk.

Until now there has been no hope for these patients, who typically die of the disease within 15 years of diagnosis.

"My father had it, said Mike. He died from it."

Huntington's causes uncontrollable movements and mental decline, there is no cure.

"Unfortunately, it ends in death, said Dr. Vicki Wheelock, a neurologist at UC Davis Health System. It's a fatal disease."

Now researchers are gearing up for a new trial in humans.

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Doctors think stem cell injections could provide hope for Huntington disease patients

Toddlers who undergo total body irradiation in preparation for bone marrow transplantation are at higher risk for a decline in IQ and may be candidates for stepped up interventions to preserve intellectual functioning, St. Jude Children's Research Hospital investigators reported. The findings appear in the current issue of the Journal of Clinical Oncology.

The results clarify the risk of intellectual decline faced by children, teenagers and young adults following bone marrow transplantation. The procedure is used for treatment of cancer and other diseases. It involves replacing the patient's own blood-producing stem cells with those from a healthy donor.

Researchers tracked IQ scores of 170 St. Jude patients before and for five years after transplantation, making this the most comprehensive effort yet to determine how the procedure affects intelligence. The patients ranged in age from 4 months to 23 years when their transplants occurred. The procedure had little lasting impact on the IQ scores of most patients.

"For the great majority of patients, these findings provide reassurance that transplantation will not have a significant negative impact on cognitive development," said corresponding author Sean Phipps, Ph.D., chair of the St. Jude Department of Psychology. "We have also identified a high-risk group of younger patients who may benefit from more intensive interventions, including developmental stimulation and other rehabilitative therapies designed to prevent a decline in intellectual functioning and aid in recovery."

The high-risk group includes patients whose transplants occurred when they were aged 3 years or younger and involved total body irradiation (TBI). TBI is used to prepare patients for transplantation by killing remaining cancer cells and protecting the transplanted cells from their immune systems. TBI is associated with a range of short-term and long-term side effects. At St. Jude, therapeutic advances have significantly reduced the use of TBI in bone marrow transplantations.

Previous studies of bone marrow transplantation survivors reported conflicting results about the long-term impact of age and TBI on cognitive abilities.

Before transplantation, the average IQ scores of all patients in this study were in the normal range. One year after transplantation, average IQ scores of patients aged 5 and younger had declined sharply. But scores of most patients rebounded in subsequent years. Five years after the procedure, IQ scores for most patients, even the youngest survivors, had largely recovered and fell within the range of normal intelligence.

Patients in the high-risk group were the lone exception. IQ scores of patients who were both aged 3 or younger when their transplants occurred and who received TBI failed to recover from the first-year decline. Five years after transplantation, these survivors had average IQ scores in the low-normal range of intelligence. Their scores were more than 16 points lower than the scores of patients who were just as young when their transplants occurred but did not receive TBI.

Of the 72 patients in this study whose transplants included TBI, researchers found there was a long-term impact on intellectual functioning only of patients who were aged 3 or younger at transplantation.

"The significant first-year decline reflects the intensity of transplantation, which our results suggest leads to greater disruption in development in the youngest children than was previously recognized," said the study's first author Victoria Willard, Ph.D., a St. Jude psychology department research associate.

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Youngest bone marrow transplant patients at higher risk of cognitive decline

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Newswise An innovative targeted payload immunotherapy that is being readied for a Phase 3 clinical trial (due to begin in the first half of 2015), received a favorable endorsement from Actinium Pharmaceuticals Scientific Advisory Board (SAB). The nod occurred after the members conducted its year-end meeting to review the progress of Iomab-B, a radiolabeled antibody being developed as a part of bone marrow transplant regimen initially in relapsed and refractory AML patients ages 55 and older.

The group met prior to the 56th American Society of Hematology (ASH) Annual Meeting and Exposition in San Francisco and was Chaired by John Pagel, MD PhD of the Fred Hutchinson Cancer Research Center and Swedish Cancer Institute in Seattle and included senior members from Memorial Sloan Kettering Cancer Center, MD Anderson Cancer Center and other leading institutions. The SABs goal is to further the development of Iomab-B as a myeloablative agent for older relapsed and refractory AML patients. If approved, Iomab-B should increase the number of patients eligible for curative bone marrow transplant (BMT, also known as HSCT) and improve clinical outcomes.

Richard Champlin MD, Chair of Stem Cell Transplantation and Cellular Therapy at MD Anderson Cancer Center, stated, We are impressed with progress in Iomab-B development and are looking forward to starting the trial. Iomab-B treatment would be an important new addition to our unfortunately very limited armamentarium for the most difficult-to-treat AML patients, and could potentially change the way refractory AML in older patients is treated.

As an international leader in the field of hematopoietic stem cell transplantation (HSCT), Dr. Champlin pioneered the use of donor transplants and lower doses of chemotherapy, reducing mortality rates along the way. Under his leadership, the MD Anderson HSCT program grew to become the largest in the world.

The Company updated the SAB on progress made in 2014, including refining and completing the Phase 3 protocol, progress in manufacturing centralization and scale-up, CRO engagement and the completion of other administrative items. Plans for 2015 were also reviewed, including assembly of the IND (Investigational New Drug) Application for submission to FDA early next year, clinical trial sites selection, preparation of ancillary materials and other items related to the upcoming pivotal trial. This study is planned as the final clinical trial prior to potential FDA clearance and approval.

Dr. Dragan Cicic, Chief Medical Officer of Actinium stated: "Actinium is committed to the ongoing development of Iomab-B with a multi-center Phase 3 pivotal trial due to begin in 2015. With the continued support and input from our world renowned scientific advisors, we are moving quickly to advance Iomab-B development. The SAB meeting further supported our belief that, if approved by FDA, Iomab-B could significantly change the treatment paradigm for elderly relapsed and refractory AML patients by providing a potentially curative pathway for majority of patients who today have a life expectancy of 5 or fewer months."

About AML Acute myeloid leukemia (AML) is an aggressive cancer of the blood and bone marrow. It is characterized by an uncontrolled proliferation of immature blast cells in the bone marrow. The American Cancer Society estimates there will be approximately 18,860 new cases of AML and approximately 10,460 deaths from AML in the U.S. in 2014, most of them in adults. Patients over age 60 comprise the majority of those diagnosed with AML, with a median age of a patient diagnosed with AML being 67 years. Treatment approaches in this population are limited because a majority of these individuals are judged too frail and unable to tolerate standard induction chemotherapy or having forms of disease generally unresponsive to currently available drugs. Elderly, high risk patients ordinarily have a life expectancy of 5 or fewer months if treated with standard chemotherapy, and only about a third of them receive this treatment because of toxicity of and limited responses to the available therapy. The other two-thirds receive best supportive care, with 2 months survival, according to Oran and Weisdorf (Haematologica 2012; 1916-24).

About Iomab-B Iomab-B will be used in preparing patients for hematopoietic stem cell transplantation (HSCT), the fastest growing hospital procedure in the U.S. The Company established an agreement with the FDA that the path to a Biologics License Application (BLA) submission could include a single, pivotal Phase 3 clinical study if it is successful. The trial population in this two arm, randomized, controlled, multicenter trial will be refractory and relapsed Acute Myeloid Leukemia (AML) patients over the age of 55. The trial size was set at 150 patients with 75 patients per arm. The primary endpoint in the pivotal Phase 3 trial is durable complete remission, defined as a complete remission lasting at least 6 months and the secondary endpoint will be overall survival at one year. There are currently no effective treatments approved by the FDA for AML in this patient population and there is no defined standard of care. Iomab-B has completed several physician sponsored clinical trials examining its potential as a conditioning regimen prior to HSCT in various blood cancers including the Phase 1/2 study in relapsed and/or refractory AML patients. The results of these studies in over 300 patients have demonstrated the potential of Iomab-B to create a new treatment paradigm for bone marrow transplants by: expanding the pool to ineligible patients who do not have any viable treatment options currently; enabling a shorter and safer preparatory interval for HSCT; reducing post-transplant complications; and showing a clear survival benefit including curative potential.

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Experts in Leukemia and Bone Marrow Transplant Prepare for Upcoming Pivotal Trial of Innovative Targeted Payload ...

High costs keep patients from using stem cells harvested from umbilical cords

Scientists are studying ways to treat HIV, cerebral palsy and other diseases using umbilical cord blood, although little of the collected blood will actually be used. Credit:Banc de Sang via flickr

Youd think doctors and patients would be clamoring for cells so versatile they could help reboot a body suffering from everything from leukemia to diabetes. But a new report shows that an important source of these stem cellsdiscarded umbilical cordsis rarely used because of high costs and the risk of failure.

Stem cells drawn from newborns umbilical cord blood are sometimes used to treat medical conditions, especially bone and blood cancers like multiple myeloma or lymphoma by replacing dysfunctional blood-producing cells in bone marrow. Generally the diseased cells are destroyed with chemotherapy and irradiation. Then new stem cells are transplanted into the patient to restore function. Cord blood stem cells are an alternative to bone marrow transplants and peripheral blood transplants, in which stem cells are gathered from the blood stream. Cord blood tends to integrate better with the body and it is easier to find a suitable donor than the alternatives.

Yet less than 3 percent of cord blood collected in the U.S. is ever used whereas the rest sits uselessly in blood banks, according to a recent report in Genetic Engineering & Biotechnology News. Immunologist Enal Razvi is author of the report and managing director of Select Biosciences, a biotechnology consulting agency. Razvi found that public cord blood banks, which store donated frozen units for transplants as needed, have only a 1 to 3 percent turnover annually. Most of their inventory sits unused year after year. For example, at Community Blood Services in New Jersey, patients have only used 278 of its 13,000 cords since it opened in 1996, according to business development director Misty Marchioni. Usage is even lower at private cord blood banks, which charge clients thousands of dollars to store a cord in the event a family member one day needs it.

Unlike bone marrow, the main alternative stem cell source, cells transplanted from cord blood carry little risk of graft-versus-host disease, a deadly condition in which the body rejects a transplant. Scientists believe this is because a babys immune system is closer to a blank slate, so their stem cells can integrate with the patients body more easily. But cord blood transplants also take longer to start working, requiring longer hospital stays and upping the bill. Due to storage and testing costs, the cords themselves also get pricy. The cost of the cord is prohibitively high, Razvi explains. Each unit of cord blood costs between $35,000 and $40,000 and most adults require two units for a successful transplant. Insurance companies will generally pay a set amount for a stem cell transplant regardless of where the cells come from. The price tag on a cord blood transplant can run up to $300,000, which may not be fully covered.

Cord blood stem cell transplants also have a higher failure rate than other transplant methods. If the transplant fails, it leaves patients with a compromised immune system in addition to their original disease and medical bills. Because the preparation for transplant includes wiping out the patients original bone marrow, the entire body has to be repopulated with stem cells able to replace it. There are not many stem cells in each cord. Compared with bone marrow or peripheral blood there is a greater chance that there will not be enough stem cells that actually implant and begin producing blood and bone marrow. Its like spreading a small amount of seeds in a big garden, says Mitchell Horwitz, who teaches cell therapy at Duke University Medical Center. Sometimes it just doesnt take.

Martin Smithmyer, chief executive of the private bank Americord, claims that more clients will eventually use their cords, especially as more applications are found for cord blood stem cells. But some scientists disagree. Steven Joffe, a professor of medical ethics at the University of Pennsylvania Perelman School of Medicine, says that many treatments cannot be done with a patients own stem cells because genetic diseases would already be present in the cord blood and that bone marrow might be a better option for relatives. The likelihood they are ever going to use that product is vanishingly small, he says.

Despite the low usage, advocates say cord blood programs have been crucial in improving transplant options for racial minorities, because it can be hard to find a bone marrow match for some groups. Cord blood does not need to match the patient as perfectly as bone marrow. This has transformed the treatment of minority patients, says Andromachi Scaradavou, medical director of the National Cord Blood Program, a public bank based in New York City. In the past we didnt have good donors to offer them. Community Blood Servicess Marchioni also maintains that cord blood is a good emergency option, because finding a compatible bone marrow or peripheral blood donor can take months or years. If you need a transplant quickly, she says, its easy to get cord blood off of a shelf.

Still, experts are working on more efficient ways of ensuring widespread availability of cord blood without having so much of it sit forever unused. Researchers are also continuing to look for ways to improve transplant success and to increase the number of stem cells obtained from each cord, potentially bringing down costs and making cord blood transplants feasible for more patients. If the cost could be lowered, Scaradavou says, it would help a lot of patients get the treatment they need.

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Vast Majority of Life-Saving Cord Blood Sits Unused

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4-Dec-2014

Contact: John Wallace wallacej@vcu.edu 804-628-1550 Virginia Commonwealth University @vcunews

Is the human immune system similar to the weather, a seemingly random yet dynamical system that can be modeled based on past conditions to predict future states? Scientists at VCU Massey Cancer Center's award-winning Bone Marrow Transplant (BMT) Program believe it is, and they recently published several studies that support the possibility of using next-generation DNA sequencing and mathematical modeling to not only understand the variability observed in clinical outcomes of stem cell transplantation, but also to provide a theoretical framework to make transplantation a possibility for more patients who do not have a related donor.

Despite efforts to match patients with genetically similar donors, it is still nearly impossible to predict whether a stem cell transplant recipient will develop potentially fatal graft-versus-host disease (GVHD), a condition where the donor's immune system attacks the recipient's body. Two studies recently published by the online journal Frontiers in Immunology explored data obtained from the whole exome sequencing of nine donor-recipient pairs (DRPs) and found that it could be possible to predict which patients are at greatest risk for developing GVHD and, therefore, in the future tailor immune suppression therapies to possibly improve clinical outcomes. The data provides evidence that the way a patient's immune system rebuilds itself following stem cell transplantation is representative of a dynamical system, a system in which the current state determines what future state will follow.

"The immune system seems chaotic, but that is because there are so many variables involved," says Amir Toor, M.D., member of the Developmental Therapeutics research program at Massey and associate professor in the Division of Hematology, Oncology and Palliative Care at the VCU School of Medicine. "We have found evidence of an underlying order. Using next-generation DNA sequencing technology, it may be possible to account for many of the molecular variables that eventually determine how well a donor's immune system will graft to a patient."

Toor's first study revealed a large and previously unmeasured potential for developing GVHD for which the conventional approach used for matching DRPs does not account. The conventional approach for donor-recipient compatibility determination uses human leucocyte antigen (HLA) testing. HLA refers to the genes that encode for proteins on the surface of cells that are responsible for regulating the immune system. HLA testing seeks to match DRPs who have similar HLA makeup.

Specifically, Toor and his colleagues used whole exome sequencing to examine variation in minor histocompatibility antigens (mHA) of transplant DRPs. These mHA are protein fragments presented on the HLA molecules, which are the receptors on cells' surface to which these fragments of degraded proteins from within a cell bind in order to promote an immune response. Using advanced computer-based analysis, the researchers examined potential interactions between the mHA and HLA and discovered a high level of mHA variation in HLA-matched DRPs that could potentially contribute to GVHD. These findings may help explain why many HLA-matched recipients experience GVHD, but why some HLA-mismatched recipients experience none remains a mystery. This seeming paradox is explained in a companion paper, also published in the journal Frontiers in Immunology. In this manuscript, the team suggests that by inhibiting peptide generation through immunosuppressive therapies in the earliest weeks following stem cell transplantation, antigen presentation to donor T cells could be diminished, which reduces the risk of GVHD as the recipients reconstitute their T-cell repertoire.

Following stem cell transplantation, a patient begins the process of rebuilding their T-cell repertoire. T cells are a family of immune system cells that keep the body healthy by identifying and launching attacks against pathogens such as bacteria, viruses or cancer. T cells have small receptors that recognize antigens. As they encounter foreign antigens, they create thousands of clones that can later be called upon to guard against the specific pathogen that presented the antigen. Over the course of a person's life, they will develop millions of these clonal families, which make up their T-cell repertoire and protect them against the many threats that exist in the environment.

This critical period where the patient rebuilds their immune system was the focus of the researchers' efforts. In previous research, Toor and his colleagues discovered a fractal pattern in the DNA of recipients' T-cell repertoires. Fractals are self-similar patterns that repeat themselves at every scale. Based on their data, the researchers believe that the presentation of minor histocompatability antigens following transplantation helps shape the development of T-cell clonal families. Thus, inhibiting this antigen presentation through immunosuppressive therapies in patients who have high mHA variation can potentially reduce the risk of GVHD by influencing the development of their T-cell repertoire. This is backed by data from clinical studies that show immune suppression soon after transplantation improves outcomes in unrelated DRPs.

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Predicting the storm: Can computer models improve stem cell transplantation?

Boston, MA (PRWEB) December 04, 2014

In a new report published in the online journal Cell Death and Disease, the Adult Stem Cell Technology Center, LLC (ASCTC) continues to demonstrate its special expertise in uncovering unknown properties that are unique to adult tissue stem cells. In particular, the new study continues to build the companys portfolio of technologies that make previously invisible adult stem cells not only identifiable, but also countable.

The studies were performed with mouse hair follicle stem cells. Because of the universal nature of adult tissue stem cell properties, the new findings are predicted to apply to stem cells in a wide range of human tissues as well.

For the past half century since the experimental demonstration of their existence, it has not been possible to identify adult tissue stem cells exclusive of other related cell types. Consequently, counting them has been impossible, too. Established stem cell therapies like bone marrow transplantation are suboptimal because of this limitation; and the current worldwide flood of thousands of clinical trials of tissue stem cell transplantation therapies has the same problem. Without being able to count potentially curative adult tissue stem cells, there is no way to optimize and standardize successful treatments.

The new report presents a discovery made during studies employing one of the ASCTCs recently defined biomarkers for detecting tissue stem cells. The new biomarker is a member of a family of cell factors called histones that package the cellular DNA into chromosomes. One of the less abundant members of this family is called H2A.Z. In 2011, the ASCTC discovered that H2A.Z is only accessible on the set of chromosomes that segregates to the stem cell sister when a stem divides to produce a non-stem sister cell. The non-stem sister differentiates to replenish lost mature tissue cells. Before a stem cell divides in this manner, the stem cell chromosomes and the non-stem cell chromosomes are distinct because of this difference in their H2A.Z access. This unique feature, called H2A.Z asymmetry, is a highly specific biomarker for identifying adult tissue stem cells.

Because detection of H2A.Z asymmetry does not disrupt other features of stem and non-stem chromosomes, it can be used as a specific landmark to discover other molecular differences between chromosomes destined for the stem cell sister and chromosomes destined for the non-stem sister. The new report describes how two well-known gene regulation modifications of an abundant histone family member, H3, also display asymmetry between stem cell chromosomes and differentiating cell chromosomes.

The newly discovered asymmetric chromosomal patterning of gene regulation modifications in adult tissue stem cells may reveal a long sought mechanism to explain how stem cell fate is maintained in mammalian tissues. This new insight into the function of tissue stem cells addresses a fundamental question in the field of stem cell biology research. ASCTC Director James L. Sherley anticipates that the new report will give stem cell scientists and bioengineers a new lead idea and new research tools for extending knowledge on the molecular workings of adult tissue stem cells. Such advances in knowledge are greatly needed currently to improve the scientific foundation for the increasing number of regenerative medicine clinical trials.

******************************************************************************************** The Adult Stem Cell Technology Center, LLC is a Massachusetts life sciences company. ASCTC Director and founder, James L. Sherley, M.D., Ph.D. is the foremost authority on the unique properties of adult tissue stem cells. The companys patent portfolio contains biotechnologies that solve the three main technical problems production, quantification, and monitoring that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing induced pluripotent stem cells. Currently, ASCTC is employing its technological advantages to pursue commercialization of facile methods for monitoring adult tissue stem cell number and function.

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The Adult Stem Cell Technology Center, LLCs New Report on Asymmetric Character of Stem Cell Chromosomes Advances ...

MIAMI (PRWEB) December 04, 2014

After a successful first run in Spain last month, Global Stem Cells Group, has announced the decision to take the biotech companys hands-on stem cell training course to additional European cities in 2015. GSCG subsidiary Stem Cell Training, Inc. and Dr. J. Victor Garcia conducted the Adipose Derived Harvesting, Isolation and Re-integration Training Course for medical professionals in Barcelona Nov. 22-23, 2014.

The two-day, hands-on intensive training course was developed for physicians and high-level practitioners to learn techniques in harvesting and reintegrating stem cells derived from adipose tissue and bone marrow. The objective of the training is to bridge the gap between bench science in the laboratory and the doctors office by teaching effective, in-office regenerative medicine techniques.

Global Stem Cells Group will release a schedule of cities and dates for future training classes in upcoming weeks.

For more information, visit the Stem Cell Training, Inc. website, email info(at)stemcelltraining(dot)net, or call 305-224-1858.

About Global Stem Cells Group: Global Stem Cells Group, Inc. is the parent company of six wholly owned operating companies dedicated entirely to stem cell research, training, products and solutions. Founded in 2012, the company combines dedicated researchers, physician and patient educators and solution providers with the shared goal of meeting the growing worldwide need for leading edge stem cell treatments and solutions.

With a singular focus on this exciting new area of medical research, Global Stem Cells Group and its subsidiaries are uniquely positioned to become global leaders in cellular medicine.

Global Stem Cells Groups corporate mission is to make the promise of stem cell medicine a reality for patients around the world. With each of GSCGs six operating companies focused on a separate research-based mission, the result is a global network of state-of-the-art stem cell treatments.

About Stem Cell Training, Inc.:

Stem Cell Training, Inc. is a multi-disciplinary company offering coursework and training in 35 cities worldwide. Coursework offered focuses on minimally invasive techniques for harvesting stem cells from adipose tissue, bone marrow and platelet-rich plasma. By equipping physicians with these techniques, the goal is to enable them to return to their practices, better able to apply these techniques in patient treatments.

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Global Stem Cells Group Hands-on Training Course in Barcelona Heading to Additional Euro Cities in 2015

PUBLIC RELEASE DATE:

3-Dec-2014

Contact: Glenna Picton picton@bcm.edu 713-798-4710 Baylor College of Medicine @bcmhouston

HOUSTON - (Dec. 3, 2014) - A novel mechanism - similar to how normal tissue stem cells respond to wounding - might explain why bladder cancer stem cells actively contribute to chemo-resistance after multiple cycles of chemotherapy drug treatment. Targeting this "wound response" of cancer stem cells can potentially provide a novel approach for therapeutic invention, said researchers from the National Cancer Institute-designated Dan L. Duncan Cancer Center at Baylor College of Medicine.

The results of their study appear online in the journal Nature today.

"Treatment for advanced bladder cancer is limited to surgery and chemotherapy. There are no targeted treatments available," said Dr. Keith Syson Chan, an assistant professor of molecular and cellular biology and of urology and the corresponding author on the report. "The chemotherapy response is far from ideal so the clinical goal is to advance research into this area and uncover a much more targeted approach."

Together with co-lead authors Antonina Kurtova, a graduate student in the Translational Biology and Molecular Medicine Program at Baylor, and Dr. Jing Xiao, research assistant in urology at Baylor, Chan and his team sought out to identify mechanisms underlying the development of resistance in bladder cancer that has invaded the muscles. They found that regrowth of cancer stem cells actively contributes to therapy resistance between drug treatment cycles.

"This is a paradoxical mechanism leading to resistance, one we didn't expect," said Chan. "The cancer stem cells actively regrow and respond to the induced damage or apoptosis (cell death) caused by chemotherapy in between the different cycles, similar to how normal tissue stem cells respond to wound-induced damages."

The proliferation is stimulated by the release of a metabolite (or factor) called prostaglandin E2 or PGE2 from the dying cells, which causes the cancer stem cells to repopulate tumors that were reduced in size by chemotherapy, they found.

In normal cells, this is a part of the wound repair process when PGE2 induces tissues stem cells to regrow; in cancer PGE2 ironically induces regrowth of more cancer stem cells in between chemotherapy cycles, Kurtova and Xiao said.

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'Wound response' of cancer stem cells may explain chemo-resistance in bladder cancer

Author: Ian Murnaghan BSc (hons), MSc - Updated: 11 September 2014 | Comment

A bone marrow stem cell transplant uses stem cells derived from bone marrow to provide a fresh and healthy source of new blood cells which in turn, allows for a patient to receive higher doses of chemotherapy to treat certain types of cancer such as leukaemia. This ultimately means that a person has a better chance of surviving cancer. The bone marrow stem cells may be allogeneic and therefore donated by a family member of stranger, or they may be autologous, which utilizes a patient's own stem cells.

Bone marrow stem cells are found in bone marrow and in a person's blood. After stem cells multiply, they form immature blood cells, which are then subject to a collection of changes that allow them to develop into mature blood cells. Once mature, the blood cells migrate from the marrow and are introduced into the bloodstream, where they provide important functions in keeping the body alive and healthy.

A patient will usually receive some chemotherapy to reduce cancer cells before stem cells are collected. The harvested stem cells are also treated to ensure that no cancer cells remain. Higher doses of chemotherapy are then given, sometimes alongside complete body radiation, to confirm that no cancer remains. Stem cells are then transplanted back into the body via a rapid injection. Stem cells will eventually migrate to the bone marrow, where they latch onto other cells there and develop into the different blood cells.

Stem cells are then infused into the patient via an intravenous line over several hours. Stem cells travel to the patient's bone marrow where they develop and produce the blood cells necessary for blood functioning. Patients may also still be given drug therapy for some time to reduce the chances of immune rejection.

Bone marrow stem cell harvests are clearly a life saving technique for those suffering from certain cancers such as leukaemia. They are one of the 'older' stem cell therapies and have been proven effective for decades now. There are, however, still issues of rejection that warrant further development and refinement of stem cell harvesting techniques. It is hoped that scientists will continue to focus on research to improve the odds of success for this important treatment.

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Sohaila Reda - 9-Aug-14 @ 5:06 PM

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NEW YORK (CBSNewYork) A New York woman battling leukemia was especially grateful this Thanksgiving, as she credited the kindness of a total stranger with helping save her life.

They found the donor, and it was just basically like a weight lifted off my shoulders, said Jeanine Walsh, 38.

As CBS2s Dr. Max Gomez reported Thursday, Walsh the mother of two young children has been battling leukemia for the second time in two years.

I was in total and complete shock, she said.

No members of Walshs family were a match for her, but a willing donor was found through the national registry. Peripheral stem cells were collected from the donor, located in the Western U.S., earlier this week.

The process took just a few hours.

We attach the patient, that is the donor, to a machine. The machine takes blood form the donor, filters out the stem cells if you will, and returns the rest of the blood to the donor, said Dr. Michael Schuster, director of stem cell transplantation at Stony Brook University Hospital.

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Stranger Donates Stem Cells In Hopes Of Curing New York Woman With Leukemia