Why banking on cord blood isn't necessarily a good idea

CARLY WEEKS From Saturday's Globe and Mail Published Saturday, May. 26, 2012 6:00AM EDT

Its a straightforward pitch to expecting parents: Pay a private clinic to store your babys stem-cell-rich umbilical-cord blood, and rest assured that he or she has protection for life. Multiple sclerosis, cerebral palsy, diabetes, traumatic brain injury, stroke, brain tumours and even Alzheimers disease are just a few of the ailments stem cells may be able to treat or cure in the future.

The optimism is contagious. Tens of thousands of Canadian families have made the decision to pay thousands of dollars to bank cord blood. But beyond the websites and brochures featuring photos of smiling babies and testimonials from families, a different picture is emerging of an industry that uses inflated arguments, aggressive marketing and misleading information to convince parents to buy in.

I dont know if the families are walking away with an entirely honest picture of what theyre buying, says John Doyle, former head of blood and marrow transplants at Torontos Hospital for Sick Children. I dont think that parents truthfully understand the limits.

Theres a long-standing history of overinflated promises by the cord-blood banks, agrees Donna Wall, director of the blood and marrow transplant program at CancerCare Manitoba. I could have retired many times over if I had gotten into the business. Its just not the right thing to do.

Full of promise

The stem cells found in umbilical-cord blood have the ability to turn into red or white blood cells or blood-clotting cells. For that reason, they offer promising treatments for leukemia, lymphoma, sickle cell disease and other blood, bone, immune and metabolic disorders.

Adults also carry these stem cells, which is why Canadian Blood Services has a campaign to recruit people to join OneMatch, its network to connect stem-cell and bone-marrow donors to patients. But finding a suitable donor is much more difficult than simply matching blood types. Patient and donor cells must match 10 out of 10 human leukocyte antigens or proteins found on the surface of cells. Donor registries are limited and seldom diverse enough to serve patients of all ethnicities.

Hence the excitement over umbilical-cord-blood stem cells: Not only are they young and less likely to lead to complications, they need not match as precisely as adult cells.

This has just opened up so many more possibilities to patients in need, says Sue Smith, executive director for stem cells at Canadian Blood Services.

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Why banking on cord blood isn't necessarily a good idea

UC San Diego Scientists Net $12 Million For Stem Cell Research

Five UC San Diego scientists have received almost $12 million combined from the California Institute for Regenerative Medicine to pay for stem cell-based research, the university announced today.

A team led by Lawrence Goldstein, of the Department of Cellular and Molecular Medicine and director of the UC San Diego Stem Cell Program, was given $1.8 million to continue looking for new methods to find and test possible medications for Alzheimer's disease, according to UCSD. They use reprogrammed stem cells in their work.

Dr. Mark Tuszynski, professor of neurosciences and director of the Center for Neural Repair, received $4.6 million to develop more potent stem cell-based treatments for spinal cord injuries.

Gene Yeo, assistant professor in the Department of Cellular and Molecular Medicine, was awarded $1.6 million to continue research into treatments for amyotrophic lateral sclerosis. His research hopes to take advantage of recent discoveries about ALS, or Lou Gehrig's disease, which center on mutations in RNA-binding proteins that cause dysfunction and death in neurons.

Dr. Eric David Adler, an associate clinical professor of medicine and cardiologist, was granted $1.7 million to screen potential drugs for Danon disease, a type of inherited heart failure that frequently kills patients by their 20s.

Yang Xu, a professor in the Division of Biological Sciences, was given $1.8 million to research the use of human embryonic stem cells to produce a renewable source of heart muscle cells that replace cells damaged or destroyed by disease, while overcoming biological resistance to new cells.

"With these new awards, the (institute) now has 52 projects in 33 diseases at varying stages of working toward clinical trials,'' said Jonathan Thomas, chairman of the CIRM governing board. "Californians should take pride in being at the center of this worldwide research leading toward new cures.''

CIRM was established in November 2004 with voter passage of the California Stem Cell Research and Cures Act. UC San Diego has received $112 million since CIRM began providing grants six years ago.

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UC San Diego Scientists Net $12 Million For Stem Cell Research

Fat stem cells may help treat kidney ailments

Fat stem cells may help treat kidney ailments BS Reporter / Mumbai/ AhmedabadMarch 06, 2007 In a breakthrough in the stem cell research, scientist from Ahmedabad have developed a technique to encourage a new kind of stem cells called Mesenchymal stem cells generated from fat (adipose tissue) of donors, which can be used in treating kidney diseases. Mesenchymal stem cells generated from fat of donors hold great promise for the treatment of kidney diseases, claims H L Trivedi, director, Institute of Kidney Diseases and Research Center (IKDRC), Ahmedabad. We will soon patent the research, he added. The institute will soon convene a meeting of scientists working on the project and take a decision on securing the patent for the research. A team of scientists from the IKDRC, led by Trivedi, has clinically proved that when presented in the right physical context, certain growth factors encourage the survival and proliferation of fat mesenchymal stem cells grown outside the body. Trivedi says the research offers hope of cent per cent recovery for patients suffering from severe kidney diseases as the mesenchymal stem cells will nullify the rejection rate of the body, thus inducing the body to accept the newly transplanted kidney as part of its own body. Emphasising on the success of mesenchymal stem cells for kidney treatment, Trivedi further said mesenchymal stem cells were the best repair stem cells compared to other stem cells. The worlds first recipient of these kinds of stem cells is a kidney patient - Hetalben Mewada, a 30-year-old housewife from Palanpur in Gujarat, claims the scientist from IKDRC. Speaking about the financial aspect of the kidney treatment, Trivedi said, Mesenchymal stem cells using fat is simple and cheap. The latest invention would cut the cost of surgery drastically and make it affordable for the needy people. It will also reduce the chances of recurrence and complexity in the post surgery situation, he said. Mesenchymal stem cells are available in bone marrow and peripheral blood cells in smaller quantity, but the cells are not economically feasible. Mesenchymal stem cells can be easily derived from fat and is economically viable. Mesenchymal stem cells that are already in the fats are separated and grown through culturing technique in laboratory. IKDRCs scientists carried out a kidney transplant operation using Mesenchymal stem cells derived from fats along with adult hematopoietic stem cells infused into the transplanted kidney to create tolerance or acceptance by the patients. This eliminates the chance of rejection, and the patients would not need medication. Under this procedure, the mesenchymal stem cells act as a big brother to adult hematopoietic stem cells. Mesenchymal stem cells protect these hematopoietic stem cells and help their grafting into different organs by themselves getting grafted and making space for their younger siblings (adult hematopoietic stem cells) to live along with them in their vicinity. Fat stem cells may help treat kidney ailments BS Reporter / Mumbai/ Ahmedabad Mar 06, 2007, 22:52 IST

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Fat stem cells may help treat kidney ailments

UC San Diego researchers receive new CIRM funding

Public release date: 25-May-2012 [ | E-mail | Share ]

Contact: Scott LaFee slafee@ucsd.edu 619-543-6163 University of California - San Diego

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

With a $1.8 million award, Lawrence Goldstein, PhD, professor in the Department of Cellular and Molecular Medicine, Howard Hughes Medical Institute Investigator and director of the UC San Diego Stem Cell Program, and colleagues will continue their work developing new methods to find and test drug candidates for Alzheimer's disease (AD). Currently, there is no effective treatment for AD. The researchers screen novel candidates using purified human brain cells made from human reprogrammed stem cells. Already, they have discovered that these human brain cells exhibit a unique biochemical behavior that indicates early development of AD in a dish.

Mark H. Tuszynski, MD, PhD, professor of neurosciences and director of the Center for Neural Repair at UC San Diego, and colleagues seek to develop more potent stem cell-based treatments for spinal cord injuries. By combining grafts of neural stem cells with scaffolds placed at injury sites, the researchers have reported substantial progress in restoring functional improvement in impaired animal models. The new $4.6 million grant will fund work to identify the optimal human neural stem cells for preclinical development and, in an unprecedented step, test this treatment in appropriate preclinical models of spinal cord injury, providing the strongest validation for human translation.

Amyotrophic lateral sclerosis or ALS (Lou Gehrig's disease) is a progressive neurological condition that is currently incurable. Gene Yeo, PhD, assistant professor in the Department of Cellular and Molecular Medicine, and colleagues will use a $1.6 million grant to exploit recent discoveries that specific mutations in RNA-binding proteins cause neuronal dysfunction and death. They will use neurons generated from patient cells containing the mutations to identify the unique RNA "signature" of these doomed neurons and screen for drug-like compounds that bypass the mutations to correct the RNA signature to obtain healthy neurons.

Eric David Adler, MD, an associate clinical professor of medicine and cardiologist, studies heart failure, including the use of stem cells to treat it. His $1.7 million award will fund research into Danon disease, a type of inherited heart failure that frequently kills patients by their 20s. Adler and colleagues will turn stem cells created from skin cells of patients with Danon disease into heart cells, then screen hundreds of thousands of drug candidates for beneficial effects. The most promising drugs will subsequently be tested on mice with a genetic defect similar to Danon disease, with the ultimate goal of identifying a suitable candidate for human clinical trials. The research may have broader applications for other conditions with similar pathogenesis, such as cancer and Parkinson's disease.

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UC San Diego researchers receive new CIRM funding

State awards stem cell grants to medical researchers

SACRAMENTO California's stem cell agency today approved two grants to UC Davis Health System researchers for their innovative work in regenerative medicine.

Kyriacos A. Athanasiou, distinguished professor of orthopaedic surgery and professor and chair of biomedical engineering, and the Child Family Professor of Engineering at UC Davis, is investigating the use of skin-derived stem cells to heal cartilage injuries and debilitating conditions of the knee such as osteoarthritis.

W. Douglas Boyd, professor of surgery, plans to further refine a novel approach to treating cardiovascular injuries suffered during a heart attack by using stem cells and a tissue-like scaffold to repair cardiac damage.

The pair received individual grants totaling approximately $6.6 million from the California Institute for Regenerative Medicine's (CIRM) governing board.

Athanasiou's and Boyd's multi-year grants were among the proposals submitted to CIRM for its third round of Early Translational Awards, which are intended to enable clinical therapies to be developed more rapidly.

"Both of these scientists are conducting exciting research that could have far-reaching implications in health care," said Jan Nolta, director of the UC Davis Institute for Regenerative Cures and the university's stem cell program director. "Dr. Athanasiou is bioengineering new cartilage that could have the same physiological integrity as the cartilage a person is born with. Dr. Boyd is developing a treatment that uses a paper-thin patch embedded with stem cells to harness their regenerative powers to repair damaged heart muscle."

Boyd, who's a pioneering cardiothoracic surgeon, pointed out in his CIRM proposal that heart disease is the nation's number-one cause of death and disability. An estimated 16.3 million Americans over the age of 20 suffer from coronary heart disease, which in 2007 accounted for an estimated 1 in 6 deaths in the U.S. Boyd plans to use bone-marrow derived stem cells -- known as mesenchymal stem cells -- in combination with a bioengineered framework known as an extracellular matrix, to regenerate damaged heart tissue, block heart disease and restore cardiac function, something currently not possible except in cases of a complete and very invasive heart transplant.

An expert in biomedical engineering, Athanasiou is focusing on developing a cellular therapy using stem cells created from an individual's own skin -- known as autologous skin-derived stem cells -- which have shown great promise in animal models. He plans to use the new funding to conduct extensive toxicology and durability tests to determine the technique's long-term safety and efficacy. Such tests are among the many steps needed to advance toward human clinical trials.

Cartilage is the slippery tissue that covers the ends of bones in joints, allowing bones to glide over each other and absorbing the shock of movement. Cartilage defects from injuries and lifelong wear and tear can eventually degenerate into osteoarthritis. According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases, osteoarthritis is the most common form of arthritis and affects an estimated 27 million Americans over the age of 25.

"For anyone suffering from osteoarthritis or other debilitating cartilage conditions, Dr. Athanasiou's goal of using stem cells to regenerate new tissue could have enormous quality-of-life and economic benefits," said Nolta, who is the recipient of a prior translational grant from CIRM to develop potential therapies for Huntington's disease . "Dr. Boyd's work is equally promising because he's using a bioengineered structure to encourage cardiac tissue repair, which could have important benefits in the treatment of heart disease."

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State awards stem cell grants to medical researchers

5 scientists receive stem-cell research grants

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities.

The May 24 grants bring UC San Diego's total to more than $112 million in CIRM funding since the first awards in 2006.

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From stem cell to brain cell: New technique mimics the brain

ScienceDaily (May 24, 2012) A new technique that converts stem cells into brain cells has been developed by researchers at Lund University. The method is simpler, quicker and safer than previous research has shown and opens the doors to a shorter route to clinical cell transplants.

By adding two different molecules, the researchers have discovered a surprisingly simple way of starting the stem cells' journey to become finished brain cells. The process mimics the brain's natural development by releasing signals that are part of the normal development process. Experiments in animal models have shown that the cells quickly adapt in the brain and behave like normal brain cells.

"This technique allows us to fine-tune our steering of stem cells to different types of brain cells. Previous studies have not always used the signals that are activated during the brain's normal development. This has caused the transplanted cells to develop tumours or function poorly in the brain," says Agnete Kirkeby, one of the authors of the study.

Since the method effectively imitates the brain's own processes, it reduces the risk of tumour formation, one of the most common obstacles in stem cell research. The quick, simple technique makes the cells mature faster, which both makes the transplant safer and helps the cells integrate better into the brain. The results of the study bring stem cell research closer to transplant trials in the human brain.

"We have used the new protocol to make dopamine neurons, the type of neuron that is affected by Parkinson's disease, and for the first time, we are seriously talking about these cells as being good enough to move forward for transplantation in patients. The next step is to test the process on a larger scale and to carry out more pre-clinical safety tests," explains Malin Parmar, research team leader.

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From stem cell to brain cell: New technique mimics the brain

City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells

DUARTE, Calif.--(BUSINESS WIRE)--

City of Hope was granted a $5,217,004 early translational research award by the California Institute for Regenerative Medicine (CIRM) to support the development of a T cell-based immunotherapy that re-directs a patients own immune response against glioma stem cells. City of Hope has been awarded more than $49.7 million in grant support from CIRM since awards were first announced in 2006.

City of Hope is a pioneer in T cell immunotherapy research, helping to develop genetically modified T cells as a treatment for cancer. This strategy, termed adoptive T cell therapy, focuses on redirecting a patients immune system to specifically target tumor cells, and has the potential to become a promising new approach for treatment of cancer.

In this research, we are genetically engineering a central memory T cell that targets proteins expressed by glioma stem cells, said Stephen J. Forman, M.D., Francis and Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation and director of the T Cell Immunotherapy Research Laboratory. Central memory T cells have the potential to establish a persistent, lifelong immunity to help prevent brain tumors from recurring.

The American Cancer Society estimates that more than 22,000 people in the U.S. will be diagnosed with a brain tumor this year, and 13,700 will die from the disease. Glioma is a type of brain tumor that is often difficult to treat and is prone to recurrence. Currently, less than 20 percent of patients with malignant gliomas are living five years after their diagnosis. This poor prognosis is largely due to the persistence of tumor-initiating cancer stem cells, a population of malignant cells similar to normal stem cells in that they are able to reproduce themselves indefinitely. These glioma stem cells are highly resistant to chemotherapy and radiation treatments, making them capable of re-establishing new tumors.

Researchers at City of Hope previously have identified several proteins as potential prime targets for the development of cancer immunotherapies, such as interleukin 13 receptor alpha 2, a receptor found on the surface of glioma cells, and CD19, a protein that is active in lymphoma and leukemia cells. Both investigational therapies are currently in phase I clinical trials. Forman is the principal investigator for the newly granted study which will develop a T cell that targets different proteins expressed by glioma stem cells. Christine Brown, Ph.D., associate research professor, serves as co-principal investigator, and Michael Barish, Ph.D., chair of the Department of Neurosciences, and Behnam Badie, M.D., director of the Brain Tumor Program, serve as co-investigators on the project.

Because cancer stem cells are heterogeneous, our proposed therapy will target multiple antigens to cast as wide a net as possible over this malignant stem cell population, said Brown.

While in this effort, we are targeting a neurological cancer, our approach will lead to future studies targeting other cancers, including those that metastasize to the brain, added Barish.

The CIRM grant will help us to build a targeted T cell therapy against glioma that can offer lasting protection, determine the best way to deliver the treatment, establish an efficient process to manufacture these T cells for treatment, and get approval for a human clinical trial, said Badie.

City of Hope is also a collaborative partner providing process development, stem cell-derived cell products and regulatory affairs support in two other CIRM-funded projects that received early translational research grants. Larry Couture, Ph.D., senior vice president of City of Hopes Sylvia R. & Isador A. Deutch Center for Applied Technology Development and director of the Center for Biomedicine & Genetics, is working with Stanford University and Childrens Hospital of Orange County Research Institute on their respective projects.

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City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells

Nobelist Speaks Out on Genetic Modification, Synthetic Biology, Stem Cell Research

ASTANA, Kazakhstan, May 24, 2012 /PRNewswire/ --Sir Richard Roberts, the eminent British biologist and Nobel Prize laureate, said today European opposition to genetically modified organisms is political rather than scientific in nature.

He also said "personal medicine" based on human genome research holds large-scale promise to improve the health of the world's people on an individualized basis.

Roberts, who won the Nobel in 1993 for his shared discovery of split genes, made his remarks at the Astana Economic Forum, a global conference of scientists, academics, multinational executives and government leaders.

"On a political level, governments must embrace genetically modified organisms (GMOs) and not give way to European prophets of doom, who oppose the use of GMOs for purely political reasons," said Roberts. "It is important to note there is a complete absence of evidence that GMOs can cause any harm. Indeed to any well-informed scientist, traditionally bred plants seem much more likely to be harmful than GMOs."

Roberts predicted growing knowledge of the human genome will yield better medical treatments and diagnostics. "It is just as important that we learn more about the bacteria that colonize our bodies since they are an essential part of what it means to be human," he said.

He also predicated synthetic biology will enable scientists to build novel microorganisms from "scratch."

"Most exciting is the promise of stem cells where the challenge is to understand how they drive their differentiation into all of the other cell types in our bodies," Roberts said. "While I do not advocate prolonging life indefinitely, I am very much in favor of ensuring that as we age, the quality of our life does not diminish."

The annual Astana Economic Forum this year has drawn thousands of participants from more than 80 nations to this rapidly growing Central Asian nation. There has been much focus at the current sessions on the Greek financial crisis and turbulence in the Euro currency, in addition to the broader economic, scientific and international trade issues that are a traditional mainstay at Astana.

Deal making is a big part of both the official and the unofficial agenda at Astana. Multinationals represented include Chevron, Toyota, Nestle, Microsoft, BASF, Total, General Electric.

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Nobelist Speaks Out on Genetic Modification, Synthetic Biology, Stem Cell Research

Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells

Editor's Choice Main Category: Cardiovascular / Cardiology Article Date: 25 May 2012 - 0:00 PDT

Current ratings for: 'Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells'

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This achievement is significant, as it opens up the prospect of treating heart failure patients with their own, human-induced pluripotent stem cells (hiPSCs) to fix their damaged hearts.

Furthermore, the cells would avoid being rejected as foreign as they would be derived from the patients themselves. The study is published in the European Heart Journal. However, the researchers state that it could take a minimum of 5 to 10 years before clinical trials could start due to the many obstacles that must be overcome before using hiPSCs in humans is possible.

Although there has been advances in stem cell biology and tissue engineering, one of the major problems scientists have faced has been lack of good sources of human heart muscle cells and rejection by the immune system. Furthermore, until now, scientific have been unable to demonstrate that heart cells created from hiPSCs could integrate with existing cardiovascular tissue.

"What is new and exciting about our research is that we have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are health and young - the equivalent to the stage of his heart cells when he was just born," said Professor Lior Gepstein, Professor of Medicine (Cardiology) and Physiology at the Sohnis Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine, Technion-Israel Institute of Technology and Rambam Medical Center in Haifa, Israel, who led the study.

In the study, Professor Gepstein, Ms Limor Zwi-Dantsis, and their colleagues retrieved skin cells from two male heart failure patients, aged 51 and 61 years, and reprogrammed the cells by delivering 3 transcription factors (Sox2, Oct4, and Klf4) in addition to a small molecule called valproic acid, to the cell nucleus. The team did not include a transcription factor called c-Myc as it is a known cancer-causing gene.

Professor Gepstein said:

In addition, the team used an alternative strategy involving a virus transferred reprogramming data to the cell nucleus. However, the team removed the virus after the information had been transferred in order to avoid insertional oncogenesis.

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Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells