Stem cells are getting serious. Two decades after they were discovered, human embryonic stem cells (hESCs) are being tested as a treatment for two major diseases: heart failure and type 1 diabetes.

Treatments based on hESCs have been slow coming because of controversy over their source and fears that they could turn into tumours once implanted. They have enormous potential because hESCs can be grown into any of the body's 200 tissue types, unlike the stems cells isolated from adult tissues that have mostly been used in treatments until now.

In the most rigorous test of embryonic stems cells' potential yet, six people with heart failure will be treated in France with a patch of immature heart cells made from hESCs, and 40 people with diabetes in the US will receive pouches containing immature pancreatic cells made from hESCs.

The hope is that the heart patch will help to regenerate heart muscle destroyed by heart attacks. Trials in monkeys showed that the patch could regenerate up to 20 per cent of the lost muscle within two months.

The pancreatic cells are supposed to mature into beta cells, which produce the hormone insulin. These would act as a substitute for the cells that are destroyed by the immune systems of people with type 1 diabetes.

Although treatments based on hESCs have already been given to people with a type of age-related blindness and with spinal paralysis, the latest trials are the therapy's first foray into major fatal diseases. Heart disease is the biggest killer in the world, and cases of type 1 diabetes are growing.

"Both are landmark studies, and are different from what we've had up to now," says Chris Mason, head of regenerative medicine at University College London. "The blindness already being treated is serious, but diabetes and heart failure are killers, and things we don't have solutions for, so this brings hESCs into the mainstream."

Some people with heart disease and diabetes have received experimental treatments based on stem cells isolated from adult tissue, often from bone marrow, with varying degrees of success. These mesenchymal stem cells, or MSCs, can mature into several tissues including muscle, bone, cartilage and fat but there is no guarantee that they will grow into cardiac muscle.

A recent review of 23 trials involving 1255 people with heart disease found that there is some evidence that recipients of stem cell therapy are less likely to die or be readmitted to hospital a year or more after treatment than people who received standard treatment.

The hope is that using hESCs in place of MSCs will improve these outcomes further because they can be grown from scratch into cells exactly suited to their medical purpose. "We think our cells are more committed to the heart lineage," says Philippe Menasch, head of the French trial at the Georges Pompidou European Hospital in Paris.

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Embryonic stem cells to tackle major killer diseases

PUBLIC RELEASE DATE:

30-Sep-2014

Contact: Laura Bailey baileylm@umich.edu 734-647-1848 University of Michigan @umich

ANN ARBORThe discovery of a gene mutation that causes a rare premature aging disease could lead to the development of drugs that block the rapid, unstoppable cell division that makes cancer so deadly.

Scientists at the University of Michigan and the U-M Health System recently discovered a protein mutation that causes the devastating disease dyskeratosis congenita, in which precious hematopoietic stem cells can't regenerate and make new blood. People with DC age prematurely and are prone to cancer and bone marrow failure.

But the study findings reach far beyond the roughly one in 1 million known DC patients, and could ultimately lead to developing new drugs that prevent cancer from spreading, said Jayakrishnan Nandakumar, assistant professor in the U-M Department of Molecular, Cellular, and Developmental Biology.

The DC-causing mutation occurs in a protein called TPP1. The mutation inhibits TPP1's ability to bind the enzyme telomerase to the ends of chromosomes, which ultimately results in reduced hematopoietic stem cell division. While telomerase is underproduced in DC patients, the opposite is true for cells in cancer patients.

"Telomerase overproduction in cancer cells helps them divide uncontrollably, which is a hallmark of all cancers," Nandakumar said. "Inhibiting telomerase will be an effective way to kill cancer cells."

The findings could lead to the development of gene therapies to repair the mutation and start cell division in DC patients, or drugs to inhibit telomerase and cell division in cancer patients. Both would amount to huge treatment breakthroughs for DC and cancer patients, Nandakumar said.

Nandakumar said that a major step moving forward is to culture DC patient-derived cells and try to repair the TPP1 mutation to see if telomerase function can be restored. Ultimately, the U-M scientist hopes that fixing the TPP1 mutation repairs telomerase function and fuels cell division in the stem cells of DC patients.

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Disease decoded: Gene mutation may lead to development of new cancer drugs

PUBLIC RELEASE DATE:

29-Sep-2014

Contact: Sasha Walek newsmedia@mssm.edu 212-241-6738 The Mount Sinai Hospital / Mount Sinai School of Medicine @mountsinainyc

Patients with Multiple Sclerosis (MS) were able to safely tolerate treatment with cells cultured from human placental tissue, according to a study published today in the journal Multiple Sclerosis and Related Disorders. The study, which is the first of its kind, was conducted by researchers at Mount Sinai, Celgene Cellular Therapeutics subsidiary of Celgene Corporation and collaborators at several other institutions.

While designed to determine safety of the treatment, early signals in the data also suggested that a preparation of cultured cells called PDA-001 may repair damaged nerve tissues in patients with MS. PDA-001 cells resemble mesenchymal, stromal stem cells found in many tissues of the body. Since the cells are expanded in cell cultures, one donor is able to supply enough cells for many patients.

"This is the first time placenta-derived cells have been tested as a possible therapy for multiple sclerosis," said Fred Lublin, MD, Director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Professor of Neurology at Icahn School of Medicine at Mount Sinai and the lead investigator of the study. "The next step will be to study larger numbers of MS patients to assess efficacy of the cells, but we could be looking at a new frontier in treatment for the disease."

MS is a chronic autoimmune disease in which the body's immune system mounts recurring assaults on the myelin--the fatty, protective coating around nerve fibers in the central nervous system. This causes nerves to malfunction and can lead to paralysis and blindness. The disease usually begins as an episodic disorder called relapsing-remitting MS (RRMS), and for many sufferers, evolves into a chronic condition with worsening disability called secondary progressive MS (SPMS).

The new safety study was conducted on 16 MS patients (10 with RRMS and six with SPMS) between the ages of 18 and 65. Six patients were given a high dose of PDA-001, another six were given a lower dose, and four patients were given placebo. Any time the immune system is altered, say by an experimental treatment, there is always a risk for MS to worsen, noted Dr. Lublin. All subjects were given monthly brain scans over a six-month period to ensure they did not acquire any new or enlarging brain lesions, which would indicate a worsening of MS activity. No subjects showed any paradoxical worsening on MRI and after one year, the majority had stable or improved levels of disability.

"We're hoping to learn more about how placental stromal cells contribute to myelin repair," said Dr. Lublin. "We suspect they either convert to a myelin making cell, or they enhance the environment of the area where the damage is to allow for natural repair. Our long-term goal is to develop strategies to facilitate repair of the damaged nervous system."

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Cells from placentas safe for patients with multiple sclerosis

Patients with Multiple Sclerosis (MS) were able to safely tolerate treatment with cells cultured from human placental tissue, according to a study published today in the journal Multiple Sclerosis and Related Disorders. The study, which is the first of its kind, was conducted by researchers at Mount Sinai, Celgene Cellular Therapeutics subsidiary of Celgene Corporation and collaborators at several other institutions.

While designed to determine safety of the treatment, early signals in the data also suggested that a preparation of cultured cells called PDA-001 may repair damaged nerve tissues in patients with MS. PDA-001 cells resemble "mesenchymal," stromal stem cells found in connective tissue in bone marrow, but unlike their bone-marrow derived counterparts, stromal cells from the placenta are more numerous, with one donor able to supply enough cells for many patients.

"This is the first time placenta-derived cells have been tested as a possible therapy for multiple sclerosis," said Fred Lublin, MD, Director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Professor of Neurology at Icahn School of Medicine at Mount Sinai and the lead investigator of the study. "The next step will be to study larger numbers of MS patients to assess efficacy of the cells, but we could be looking at a new frontier in treatment for the disease."

MS is a chronic autoimmune disease in which the body's immune system mounts recurring assaults on the myelin--the fatty, protective coating around nerve fibers in the central nervous system. This causes nerves to malfunction and can lead to paralysis and blindness. The disease usually begins as an episodic disorder called relapsing-remitting MS (RRMS), and for many sufferers, evolves into a chronic condition with worsening disability called secondary progressive MS (SPMS).

The new safety study was conducted on 16 MS patients (10 with RRMS and six with SPMS) between the ages of 18 and 65. Six patients were given a high dose of PDA-001, another six were given a lower dose, and four patients were given placebo. Any time the immune system is altered, say by an experimental treatment, there is always a risk for MS to worsen, noted Dr. Lublin. All subjects were given monthly brain scans over a six-month period to ensure they did not acquire any new or enlarging brain lesions, which would indicate a worsening of MS activity. No subjects showed any paradoxical worsening on MRI and after one year, the majority had stable or improved levels of disability.

"We're hoping to learn more about how placental stromal cells contribute to myelin repair," said Dr. Lublin. "We suspect they either convert to a myelin making cell, or they enhance the environment of the area where the damage is to allow for natural repair. Our long-term goal is to develop strategies to facilitate repair of the damaged nervous system."

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The above story is based on materials provided by Mount Sinai Medical Center. Note: Materials may be edited for content and length.

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Cells from placentas safe for patients with multiple sclerosis, study shows

Edgar Irastorza was just 31 when his heart stopped beating in October 2008.

A Miami property manager, break-dancer and former high school wrestler, Irastorza had recently gained weight as his wifes third pregnancy progressed. I kind of got pregnant, too, he said.

During a workout one day, he felt short of breath and insisted that friends rush him to the hospital. Minutes later, his pulse flat-lined.

He survived the heart attack, but the scar tissue that resulted cut his hearts pumping ability by a third. He couldnt pick up his children. He couldnt dance. He fell asleep every night wondering if he would wake up in the morning.

Desperation motivated Irastorza to volunteer for a highly unusual medical research trial: getting stem cells injected directly into his heart.

I just trusted my doctors and the science behind it, and said, This is my only chance, he said recently.

Over the last five years, by studying stem cells in lab dishes, test animals and intrepid patients like Irastorza, researchers have brought the vague, grandiose promises of stem cell therapies closer to reality.

Stem cells broke into the public consciousness in the early 1990s, alluring for their potential to help the body beat back diseases of degeneration like Alzheimers, and to grow new parts to treat conditions like spinal cord injuries.

Progress has been slow. The Michael J. Fox Foundation for Parkinsons Research, an early supporter of stem cell research, pulled its financial backing two years ago, saying that it preferred to invest in research that was closer to providing immediate help for Parkinsons disease patients.

But researchers have been slowly learning how to best use stem cells, what types to use and how to deliver them to the body findings that arent singularly transformational, but progressive and pragmatic.

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Stem cell therapies making slow but promising progress

Scientists at a Birmingham university have carried out research which could help find a cure for leukaemia.

The study by researchers at the University of Birmingham has been described as a key step in the process to understanding how the disease develops.

The research specifically investigated acute myeloid leukaemia and looked at the way blood cells behave in patients suffering from the illness.

University Professor Constanze Bonifer explained: Stem cells in the bone marrow generate billions of different blood cells each day. The process resembles a production line with genes acting as regulators to control each step of the blood formation.

Leukaemia arises when the DNA encoding regulators in the stem cells is changed by a mutation.

When a mutation occurs in the relevant regulator genes, the finely balanced order of the production line is disrupted with drastic consequences.

A chain reaction occurs, with the function of other regulators in the process being altered. The new cells no longer develop into normal blood cells, but leukemic cells that multiply and begin to take over the body.

The team, which carried out the research alongside experts from Newcastle University, used state-of-the-art technology to see how the cells could be manipulated to stop them from causing the disease.

Professor Olaf Heidenreich, of Newcastle University, said: One aberrant regulator reprograms thousands of genes. If targeting it can reverse the changes it is making to the cellular production line then it would ultimately point towards new avenues for a more precise treatment of leukaemia.

Knowing that the production line can be restored to normal function gives us real hope. Of course, that is much easier to do in the lab that it is in the human body.

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Leukaemia: Birmingham scientists hope research could pave way for cure

PUBLIC RELEASE DATE:

25-Sep-2014

Contact: Guido Silvestri gsilves@emory.edu 404-727-9139 PLOS

A study published on September 25th in PLOS Pathogens reports a new primate model to test treatments that might cure HIV/AIDS and suggests answers to questions raised by the "Berlin patient", the only human thought to have been cured so far.

Being HIV-positive and having developed leukemia, the Berlin patient underwent irradiation followed by a bone-marrow transplant from a donor with a mutation that abolishes the function of the CCR5 gene. The gene codes for a protein that facilitates HIV entry into human cells, and the mutationin homozygous carriers who, like the donor, have two defective copiesprotects against HIV infection.

Several factors could have contributed to the cure of HIV/AIDS in the patient: (1) the ablation of blood and immune cells following irradiation killed all or many of the viral reservoir cells that are not eliminated by antiretroviral treatment (ART); (2) the CCR5 deletion mutation in the donor cells protected them and their progeny from HIV infection; (3) a "graft versus host" reaction occurred, where the transplanted cells and their progeny recognize the host cells as foreign and attacked and eliminated HIV-positive reservoir cells that survived the irradiation.

Guido Silvestri, from Emory University in Atlanta, USA, and colleagues investigated the relative contribution of the irradiation to eliminate the reservoir of HIV-infected cells. The scientists worked with the animal model of Simian Immunodeficiency Virus (SIV, a close relative of HIV that infects primates and causes a disease similar to AIDS) infection in rhesus macaques. Using a total of six monkeys (three of which served as controls and did not receive transplants) they performed, for the first time, hematopoietic stem cell transplantation in rhesus macaques infected with a chimeric simian/human immunodeficiency virus (SHIV) and treated with ART.

The researchers harvested hematopoetic stem cells from three macaques prior to infection (of all six animals) with SHIV. They also treated the macaques with ART to reduce viral load and mimic the situation in human HIV-infected patients on ART. They then exposed the three monkeys from which they had collected hematopietic stem cells to a high dose of radiation. This killed most of their existing blood and immune cells, including between 94 and 99% of their CD4-T cellsthe main target of HIV infectionin the blood. The irradiation was followed by transplantation of each monkey's own virus-free hematopoietic stem cells. The latter can regenerate the blood and immune cells, and did so in all three monkeys within 3 to 6 weeks. Because the transplanted cells are not from a different donor, no graft versus host disease would be expected, and none was observed.

After that time, the scientists stopped ART in all six monkeys. As expected, the virus rebounded rapidly in the control animals. Of the three transplanted animals, two also showed a rapid rebound. The third monkey developed kidney failure two weeks after ART was stopped and was euthanized. It still had undetectable levels of virus in the blood at that time, but post-mortem analysis showed low levels of viral DNA in a number of tissues, arguing that none of the three transplanted monkeys was cured.

The researchers acknowledge a number of limitations of the study, including the small number of monkeys, and the relatively short period of ART prior to irradiation and transplantation. Nonetheless, they say their study "supports the hypothesis that myeloablative total body irradiation can cause a significant decrease in the viral reservoir in blood cells, even though it was not sufficient to eliminate all reservoirs". Their results, they say, suggest that in the cure of the Berlin patient, "the use of the CCR5 mutant donor and/or the presence of graft versus host disease played a significant role".

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Stem cell transplant does not cure SHIV/AIDS after irradiation of infected rhesus macaques

PUBLIC RELEASE DATE:

24-Sep-2014

Contact: Michael Bernstein m_bernstein@acs.org 202-872-6042 American Chemical Society @ACSpressroom

The liver provides critical functions, such as ridding the body of toxins. Its failure can be deadly, and there are few options for fixing it. But scientists now report in the journal ACS Applied Materials & Interfaces a way to potentially inject stem cells from tonsils, a body part we don't need, to repair damaged livers all without surgery.

Byeongmoon Jeong and colleagues point out that currently, the only established method for treating liver failure or severe cases of liver disease is complete or partial transplantation. But the need is much greater than the number of available organs. Plus, surgery has inherent risks and a hefty price tag. A promising alternative in development is transplanting liver cells. One such approach involves using adult stem cells to make liver cells. Stem cells from bone marrow could be used, but they have limitations. Recently, scientists identified another source of adult stem cells that could be used for this purpose tonsils. Every year, thousands of surgeries are performed to remove tonsils, and the tissue is discarded. Now it could have a new purpose, but scientists needed a way to grow them on a 3-D scaffold that mimics real liver tissue. Jeong's team set out to do just that.

The researchers encapsulated tonsil-derived stem cells in a heat-sensitive liquid that turns into a gel at body temperature. They added substances called growth factors to encourage the stem cells to become liver cells. Then, they heated the combination up to a normal body temperature. The result was a 3-D, biodegradable gel that contained functioning liver cells. The researchers conclude that the same process has promise with some further tweaking for ideal conditions as an injectable tissue engineering technique to treat liver disease without surgery.

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The authors acknowledge funding from the National Research Foundation of Korea.

The American Chemical Society is a nonprofit organization chartered by the U.S. Congress. With more than 161,000 members, ACS is the world's largest scientific society and a global leader in providing access to chemistry-related research through its multiple databases, peer-reviewed journals and scientific conferences. Its main offices are in Washington, D.C., and Columbus, Ohio.

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Newswise LOUISVILLE, Ky. William Tse, M.D., associate professor of medicine and eminent scholar in hematologic malignancies research at the Mary Babb Randolph Cancer Center at West Virginia University, has been named the new director of Bone Marrow Transplantation at the University of Louisville James Graham Brown Cancer Center, a part of KentuckyOne Health. Tse will join UofL Nov. 1.

Tse will hold the Marion F. Beard Endowed Chair in Hematology Research at UofL and become a member of the cancer centers Developmental Biology Program.

Dr. Tse is emerging as one of the thought leaders in bone marrow transplantation, said Donald Miller, M.D., Ph.D., director of the JGBCC. He has trained and worked at several of the leading blood cancer programs in the nation. We look forward to his leading our program at UofL.

Tse has been at West Virginia since 2009, where he also is the co-leader the Osborn Hematologic Malignancies Program. Prior to joining West Virginia, Tse was on the faculty at the University of Colorado Denver, where he was the director of translational research program for bone marrow transplantation and hematologic malignancies. He also previously was with Case Western Reserve University and the Fred Hutchinson Cancer Research Center/University of Washington Medical Center.

Tse is active in national organizations, serving in several capacities with the American Society of Hematology, including section chair for the annual meetings Oncogene Section and bone marrow transplantation outcome section, as well as the American Society of Clinical Oncology as an annual meeting abstract reviewer and the section chair on geriatric oncology. Tse also serves leadership roles on several editorial boards including as the senior editor of the American Journal of Blood Research, stem cell biomarkers section editor for Biomarker Research, senior editor of the American Journal of Stem Cells and the academic editor of PLoS One.

A graduate of the Sun Yat-Sen University School of Medicine in Guangzhou, Guangdong, in China, he did a thoracic surgical oncology residency at Sun Yat-Sen University Cancer Center in Guangzhou before completing postdoctoral research fellowships in medical biophysics, immunology and cancer at the Princess Margaret Hospital/Ontario Cancer Institute and the Hospital for Sick Children in Ontario, Canada. He completed clinical pathology and internal medicine residencies at North Shore-Long Island Jewish Hospital before undertaking a senior medical fellowship in clinical research and medical oncology divisions at the Fred Hutchinson Cancer Research Center at the University of Washington Medical Center.

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Tse Named Director of Bone Marrow Transplantation Division at University of Louisville

A teenager who gave stem cells to save the life of a stranger is backing a national campaign to find more donors.

In June, Celyn Evans, 17, became one of the youngest people in the UK to donate stem cells.

The Colchester Royal Grammar School sixth-form student is supporting Anthony Nolans Save a Life at 16 campaign.

The charity wants HMRC to include details about stem cell donation when it writes to teens with their National Insurance numbers ahead of their 16th birthday.

Celyn, of West Mersea, said: You often hear that young people are self absorbed and not interested in helping others, but I think thats wrong.

People just need to be made aware of how they can help. That is why I am supporting this campaign.

Celyn joined the bone marrow donor register last September when his brothers friend developed leukaemia.

He was not able to help the family friend, but in February, Anthony Nolan contacted him to say he was a possible match for another patient in need of a potentially life-saving transplant.

Celyn agreed to donate and, after a series of check-ups, made the donation in London in June.

Like 90 per cent of donors, he gave his stem cells through a simple, outpatient process similar to giving blood.

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Colchester teen becomes one of the UK's youngest stem cell donors

When a child became ill, Jim Pattison was one of many who stepped up as a potential bone marrow donor.

Herald photo by Jodi Schellenberg

Jim Pattison was given two paperweights for his stem cell donation. He decided to become a donor in 1996, but was not a match until after 2010.

In 1996, Pattison was one of many who went on the bone marrow transplant list to help a one and a half year old child who was diagnosed with acute myeloid leukemia. The organizers of the donor drive expected maybe 50 people to show up and were shocked by the close to 400 who attended.

Sadly, the family didnt find a match and the girl died, but Pattison decided to stay on the registry.

They asked if I wanted to stay on and my answer was that if I would do it for Abigail I would do it for anybody, he said.

Throughout the years, Pattison was asked to test for more markers to see if he would be a match for someone else. He did his last test in 2010 and heard back a short time later with the news he was a match.

Pattison was chosen for a peripheral stem cell donation, which is different from a bone marrow transplant because it is less invasive.

I first went to where the stem cells are collected and had a physical, he explained. They sent me back with some drugs that I had to have injected here, that stimulate the stem cells to grow. I had four injections before I went.

They were looking to make sure I had a high enough level of stem cells to make the donation, he added.

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Local man shares his story of stem cell donation

Celyn Evans, 17, from Colchester, has donated stem cells to save the life of a complete stranger. Pictured with the stem cells.

Monday, September 22, 2014 10:49 AM

A selfless teenager from Colchester who donated stem cells to a stranger is backing a campaign to help find more young heroes.

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In June Celyn Evans, 17, became one of the youngest such donors in the UK.

He was contacted by the Anthony Nolan Trust as a possible match after joining the bone marrow register last September when his brothers friend developed leukaemia.

Now he is supporting Anthony Nolans Save A Life At 16 campaign, calling on HMRC to include details about stem cell donation when it writes to people with their National Insurance number ahead of their 16th birthday.

Celyn said: You often hear that young people are self absorbed and not interested in helping others, but I think thats wrong. People just need to be made aware of how they can help.

Its a very simple process, and I am surprised more people dont do it. But I think its just down to people knowing about it, which is where Anthony Nolans idea comes in.

For more information or to join the register visit the Anthony Nolan Trust website.

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Colchester: Selfless teen stem cell donor Celyn Evans backs campaign to find more young heroes

About three million Briton currently suffer osteoporosis which is affected by a number factors such as genes, a lack of exercise and poor diet and results in about 60,000 hip, 50,000 wrist and 120,000 spinal fractures every year, according to the National Osteoporosis Society, costing about 1.7 billion in health and social care.

Dr Ifty Ahmed, a researcher at Nottingham University, said his team wanted to provide a preventative treatment, strengthening the bones of those at risk before they suffered a fracture.

Speaking at the Regener8 conference on regenerative medicine, in Leeds last week, he said: Our aim would be to use screening to spot people who are at risk, then strengthen their bones before they get fractures.

It means that rather than waiting until people have a fall and break something, we would try to stop that ever happening, along with the consequences, loss of independence, surgery and secondary illnesses.

Previous attempts have been made to find ways of strengthening thinning bones but the difficulties of protecting the fragile stem cells has meant no such treatments have yet been developed.

Dr Ahmeds team hope to overcome this problem by puncturing the tiny hollow spheres of calcium phosphate allowing the stem cells to migrate inside them where they are protected.

The experimental treatment has not yet been trialled on humans.

It would involve extracting stem cells from a patients bone marrow and mixing them with the microspheres before injecting the paste into the vulnerable bones.

Dr Ahmed said: "If it works, this kind of treatment could be done in a day.

Until now the team have been funded by the Engineering and Physical Sciences Research Council but they are now looking for a commercial partner.

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Bone paste could provide treatment for ostoeporosis

Investigators from the Institute for Research in Immunology and Cancer (IRIC) at the Universit de Montral have just published, in the journal Science, the announcement of the discovery of a new molecule, the first of its kind, which allows for the multiplication of stem cells in a unit of cord blood. Umbilical cord stem cells are used for transplants aimed at curing a number of blood-related diseases, including leukemia, myeloma and lymphoma. For many patients this therapy comprises a treatment of last resort.

Directed by Dr. Guy Sauvageau, principal investigator at IRIC and hematologist at the Maisonneuve-Rosemont Hospital, the research has the potential to multiply by 10 the number of cord blood units available for a transplant in humans. In addition, it will considerably reduce the complications associated with stem cell transplantation. And it will be particularly useful for non-Caucasian patients for whom compatible donors are difficult to identify.

A clinical study using this molecule, named UM171 in honor of the Universit de Montral, and a new type of bioreactor developed for stem culture in collaboration with the University of Toronto will be initiated in December 2014 at the Maisonneuve-Rosemont Hospital.

According to Dr. Guy Sauvageau, "This new molecule, combined with the new bioreactor technology, will allow thousands of patients around the world access to a safer stem cell transplant. Considering that many patients currently cannot benefit from a stem cell transplant for lack of matching donors, this discovery looks to be highly promising for the treatment of various types of cancer."

The Centre of Excellence for Cellular Therapy at the Maisonneuve-Rosemont Hospital will serve as production unit for these stem cells, and grafts will then be distributed to patients in Montreal, Quebec City and Vancouver for this first Canadian clinical study. Tangible results should be available one year later, that is, in December 2015. The significance of this new discovery is such that over time, conclusive clinical results could revolutionize the treatment of leukemia and other blood-related illnesses.

"These extraordinary advances result from the efforts of a remarkable team that includes extremely gifted students and postdoctoral investigators working in the IRIC laboratories," adds Dr. Guy Sauvageau. "Among them, the first authors of this publication: Iman Fars, doctoral student, and Jalila Chagraoui, research officer, along with the professionals in IRIC's medical chemistry core facility under the direction of Anne Marinier, who optimized the therapeutic properties of this new molecule."

Context

Umbilical cord blood from newborn children is an excellent source of hematopoietic stem cells for stem cell transplants, since their immune system is still immature and the stem cells have a lower probability of inducing an adverse immune reaction in the recipient.

Furthermore, it is not necessary for the immunological compatibility between donor and recipient to be perfect, unlike in a bone marrow transplant. However, in most cases the number of stem cells obtained from an umbilical cord is much too low for treating an adult, and its use is confined above all to the treatment of children. With the new molecule UM171 it will be possible to multiply stem cells in culture and to produce enough of them to treat adults, especially those who are not Caucasian, and who because of the lack of donors have limited access to transplants.

Collaborators from the Maisonneuve-Rosemont Hospital, the British Columbia Cancer Agency, the Ontario Cancer Institute and the Fred Hutchison Cancer Research Center also played an important role in evaluating the biological properties of this new molecule, and those from the University of Toronto in developing the bioreactor.

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New molecule allows for up to 10-fold increase in stem cell transplants

Two men who changed each others lives forever by being on the giving and receiving ends of a bone marrow transplant met for the first time today and had their first chance to say, Thank you, face-to-face.

Thank you so much, Joe Yannantuono, 33, said to his bone marrow donor, Justin Jenkins, 35, as he embraced him in a hug in a live, emotional meeting on Good Morning America.

Yannantuono, not very long ago, was waging a two-year long battle for his life against stage 4 lymphoma.

WATCH: Robin Roberts Celebrates 1-Year Anniversary of Bone Marrow Transplant

Toddler Meets Life-Saving Bone Marrow Donor

As his wife, Christine Buono, and his 4-year-old son, JJ Yannantuono, stood by his side, the family, from Staten Island, N.Y., got the unbelievable news that a man in Texas, a stranger, was a rare 10 for 10 genetic bone marrow match.

That stranger in Texas, Jenkins, of Dallas, had registered to be a bone marrow donor by chance 15 years ago when he was 21-years-old and donated blood because they were offering free snacks.

Soon after Jenkins was found to be a match, his stem cells were transported by airplane to New York and transplanted into Yannatuonos body in December 2012 at Memorial Sloan Kettering Cancer Center.

For more than one year after the successful transplant, Yannantuono had no idea whose cells he was now carrying in his body.

As Yannantuono was rebuilding his life, Jenkins life was thrown a tragic curveball. His mother, who raised him on her own and had been a big part of his donation journey, was killed in a car crash.

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Bone Marrow Recipient Meets Donor Who Saved His Life

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Newswise Investigators from the Institute for Research in Immunology and Cancer (IRIC) at the Universit de Montral have just published, in the prestigious magazine Science, the announcement of the discovery of a new molecule, the first of its kind, which allows for the multiplication of stem cells in a unit of cord blood. Umbilical cord stem cells are used for transplants aimed at curing a number of blood-related diseases, including leukemia, myeloma and lymphoma. For many patients this therapy comprises a treatment of last resort.

Directed by Dr. Guy Sauvageau, principal investigator at IRIC and hematologist at the Maisonneuve-Rosemont Hospital, this world breakthrough has the potential to multiply by 10 the number of cord blood units available for a transplant in humans. In addition, it will considerably reduce the complications associated with stem cell transplantation. And it will be particularly useful for non-Caucasian patients for whom compatible donors are difficult to identify.

A clinical study using this molecule, named UM171 in honor of the Universit de Montral, and a new type of bioreactor developed for stem culture in collaboration with the University of Toronto will be initiated in December 2014 at the Maisonneuve-Rosemont Hospital.

According to Dr. Guy Sauvageau, This new molecule, combined with the new bioreactor technology, will allow thousands of patients around the world access to a safer stem cell transplant. Considering that many patients currently cannot benefit from a stem cell transplant for lack of matching donors, this discovery looks to be highly promising for the treatment of various types of cancer.

The Centre of Excellence for Cellular Therapy at the Maisonneuve-Rosemont Hospital will serve as production unit for these stem cells, and grafts will then be distributed to patients in Montreal, Quebec City and Vancouver for this first Canadian clinical study. Tangible results should be available one year later, that is, in December 2015. The significance of this new discovery is such that over time, conclusive clinical results could revolutionize the treatment of leukemia and other blood-related illnesses.

These extraordinary advances result from the efforts of a remarkable team that includes extremely gifted students and postdoctoral investigators working in the IRIC laboratories, adds Dr. Guy Sauvageau. Among them, the first authors of this publication: Iman Fars, doctoral student, and Jalila Chagraoui, research officer, along with the professionals in IRICs medical chemistry core facility under the direction of Anne Marinier, who optimized the therapeutic properties of this new molecule.

Context

Umbilical cord blood from newborn children is an excellent source of hematopoietic stem cells for stem cell transplants, since their immune system is still immature and the stem cells have a lower probability of inducing an adverse immune reaction in the recipient.

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World Breakthrough: A New Molecule Allows for an Increase in Stem Cell Transplants

PUBLIC RELEASE DATE:

18-Sep-2014

Contact: Luke Harrison l.harrison.1@bham.ac.uk University of Birmingham @unibirmingham

A key step in understanding the nature of the fight for superiority between mutated genes and normal genes could lead to new therapies to combat leukaemia, say researchers from the University of Birmingham and Newcastle University.

The study, published in Cell Reports, investigated Acute Myeloid Leukaemia to understand why leukemic cells are not able to develop normally into mature blood cells.

Stem cells in the bone marrow generate billions of different blood cells each day. The process resembles a production line with genes acting as regulators to control each step of the blood formation.

Leukaemia arises when the DNA encoding regulators in the stem cells is changed by a mutation. When a mutation occurs in the relevant regulator genes, the finely balanced order of the production line is disrupted with drastic consequences.

A chain reaction occurs, with the function of other regulators in the process being altered. The new cells no longer develop into normal blood cells, but leukemic cells that multiply and begin to take over the body.

Professor Constanze Bonifer, of the University of Birmingham, explained, "This particular leukaemia is characterised by a mutation in a gene that produces a rogue regulator. That is, one that is not normally made and behaves in a different way. The knock-on effect of that one mutation is huge."

The team showed that this aberrant regulator switches off hundreds of other genes, many of them regulators themselves, by using state of the art technology that looks at the activity of all genes within a cell. As a consequence of the drastically altered production line, normal blood formation cannot happen, and leukemic cells are formed.

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The war on leukemia: How the battle for cell production could be decisive

MISSISSAUGA Dorothy Vernon-Brown is on the search of her life.

When she was diagnosed with acute myaloid leukemia in August, 2013, the Mississauga mother said it was like a huge kick in the gut.

She underwent chemotherapy shortly after the diagnosis, which helped her into remission, but to survive, she has been told her only chance is a bone marrow transplant.

Bone marrow cells rescue patients from the lethal effect of chemotherapy, says Vernon-Browns oncologist, Dr. Mark Minden.

Since starting a relationship with Canadas stem cell and marrow network, One Match, Vernon-Browns search has come up empty.

Vernon-Brown says she is desperate: Recent tests show lower white blood cell counts in her blood an indication the cancer may be coming back.

But shes also worried about how few donors are from the black community. Vernon-Brown has been told her chance of finding a compatible donor are one in 10,000.

Genetics are key in finding potential stem cell and bone marrow donors says, One Match patient and transplant liaison, MaryLynn Pride said.

There is only a 25 per cent chance that even a sibling will be a match, says Pride. We have patients from all ethnic communities that are currently in need of a stem cell transplant.

Black Canadians makes up only 1 percent of all Canadians registered as potential donors, according to Pride.

Link:
Desperate search for bone marrow donor goes private for Mississauga woman

Written by Lidia Dinkova on September 17, 2014

A partnership between University of Miami researchers and a Georgia-based biomedical company this month made a new type of stem cell commercially available for bone regeneration and healing.

The marrow-isolated adult multi-lineage inducible cell, known as the MIAMI cell, is the result of 15 years of research by the University of Miami.

Since the stem cell was made commercially available, about 21 patients have been treated with a MIAMI cell infusion.

We are controlling the release to make sure it goes very smoothly, said Tracy S. Anderson, president and CEO of Vivex Biomedical Inc.

Vivex invested in the research and development of the cell and licensed the technology from UM for orthopedic use. The company has contracted with the universitys tissue bank to develop the cell for commercial use and pays an undisclosed royalty back to UM from sales.

Before the MIAMI cell goes to full national release in January 2015, Vivex is controlling the use of the cell.

I am going very slowly and selectively with the surgeons we are working with, Mr. Anderson said. Anytime you have a new product like this, you have to make sure that it goes smoothly.

Mr. Anderson didnt want to disclose revenue generated from the sale of the MIAMI cell, only saying that it has been significant.

So far, the MIAMI cell has been used in bone regeneration and healing in Utah, Florida, Georgia, Michigan, Illinois and Ohio.

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21 infused with new UM stem cell

A Quebec womans desperate, months-long search for a compatible stem cell or umbilical cord match is over.

Mai Duong, a 34-year-old Vietnamese-Canadian battling acute leukemia, announced Tuesday that she has finally found a match.

"I'm going to have the transplant and hopefully everything will go well and hopefully I'll have a new marrow," Duong tearfully told reporters Tuesday. "I just hope I'm going to beat cancer once and for all."

"A woman gave birth to her child and has donated her baby's umbilical cord to save another life," reads a post on the Save Mai Duong Facebook page. "Thank you dear mommy, we cannot fathom the importance of your gesture. I am very moved."

Duong beat cancer last year, after chemotherapy that she had to terminate a 15-week pregnancy to undergo.

She was in remission until May, when blood tests revealed the leukemia had returned.

"Seventy per cent of people who had that type of leukemia were just cured with chemotherapy and unfortunately I'm in the 30 per cent," she said at the time.

Doctors said Duong would need a bone marrow transplant or cord blood stem cells and she needed it fast. Despite being on the international list, doctors struggled to find a match.

Duong said, for people who aren't Caucasian, finding the right donor can be like searching for a needle-in-a-haystack.

"Less than one per cent of the 25 million donors worldwide are Vietnamese," she wrote on her website. "All ethnic communities are severely under-represented in the world donor bank, making finding a compatible donor very difficult for me and countless others who are currently waiting for a transplant."

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Quebec leukemia patient Mai Duong finds stem cell donor