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Category Archives: Stem Cell Treatments
Stem cell treatment for heart failure takes small step forward
Posted: May 24, 2012 at 5:10 pm
Researchers at the Technion-Israel Institute of Technology in Haifa, Israel, reported Tuesday that they had removed skin cells from two patents with heart failure, returned those cells to an embryonic state, and then transformed them into beating heart cells that could communicate with the patients existing heart tissue.
We have shown that it is 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 healthy and young the equivalent to this stage of his heart cells when he was just born, study leader Dr. Lior Gepstein said in a statement.
The discovery marks a small step toward a long-sought goal: using stem cells to regrow the cardiac tissue that is damaged in heart attacks. (The Times reported on the quest in February, 2011.) But it doesnt mean that patients with heart failure are likely to get shiny new hearts through stem cell treatments anytime soon.
Several hurdles stand in the way of using induced pluripotent stem cells, as the skin-derived cells are called, to reverse heart attack damage. The Israeli researchers acknowledged several. Such cells are known to spin out of control and cause cancer. Stem cell-derived cardiac cells have also had problems coordinating with normal heart rhythms. The team will need to be able to generate larger numbers of the cells before it can test the treatment, and will need to perfect transplant methods.
And if all those hurdles are crossed, scientists still wont know if the technology will work in people. What we produce in an animal model or in a petri dish is hardly what happens in a human. This is a first step. It will take five, 10, 15, maybe 20 years to reach fruition at the soonest, said Dr. Shephal Doshi, director of electrophysiology and pacing at Saint Johns Health Center in Santa Monica.
Most patients will have to wait to take advantage of other types of stem cell cures for heart failure as well including treatments that use cells derived from bone marrow to stimulate heart regeneration, treatments that use cardiac stem cells removed from the heart to build heart tissue and insert it back into the diseased organ and treatments that attempt to stimulate cardiac stem cells in place in the heart into action to rebuild tissues. These technologies are in varying stages of testing and development.
The Centers for Disease Control and Prevention has more information about heart failure in the U.S.
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Medical success or boondoggle?
Posted: May 20, 2012 at 7:12 am
Indian clinic's stem cell therapy real?
STORY HIGHLIGHTS
For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.
New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.
Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.
His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.
The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.
Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.
After five weeks of treatment, Cash and his parents returned home to the U.S.
That's when Cash began walking with the aid of braces for the first time.
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Canada approves stem cell therapy
Posted: May 19, 2012 at 5:10 am
Osiris Therapeutics Inc says Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.
Osiris shares rose 14 percent to $6.00 in extended trading after the news was announced.
Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient's body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.
The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.
Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company's chief executive, said in an interview that could take three to four years.
Some investment analysts have been skeptical about Prochymal's future. In 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.
Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD -- those who have more or less failed all other therapies -- Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 percent of patients.
In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.
The Canadian authorities approved the drug on the basis of that data, the company said.
FDA SUBMISSION THIS YEAR
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Canada approves stem cell therapy
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Osiris stem cell therapy approved in Canada
Posted: May 19, 2012 at 5:10 am
NEW YORK (AP) -- Osiris Therapeutics Inc. said Thursday that Canadian regulators approved its stem cell therapy Prochymal, which is intended to treat a deadly side effect of bone marrow transplants.
The company said Health Canada approved Prochymal for use against graft vs. host disease, a condition in which transplanted bone marrow cells attack the body of the patient. Prochymal is approved for use in children who are not responding to steroid therapy. Osiris said about 80 percent of child who contract graft vs. host disease die.
Osiris is still running clinical trials of Prochymal to support full marketing approval, but the drug was granted a type of conditional approval because of the lack of effective treatments for graft vs. host disease. Osiris is required to run additional studies to confirm the treatment works.
Marketing of Prochymal has not been approved in the U.S., but patients can get the treatment under certain conditions. The Food and Drug Administration allows the use of Prochymal in treatment of both adults and children with graft vs. host disease after steroids have failed.
Osiris, based in Columbia, Md., was developing Prochymal and a second stem cell treatment, Chondrogen, as part of a partnership with Genzyme, a unit of French drugmaker Sanofi. But in February Sanofi said it discontinued late-stage testing of Prochymal. Osiris said it believes Sanofi has terminated the collaboration and that it should regain all rights to Prochymal. Osiris said Sanofi disagrees and the companies are discussing their status.
Prochymal is also being studied as a treatment for Crohn's disease and other conditions.
Shares of Osiris Therapeutics dipped 5 cents to $5.26 on Thursday and climbed 74 cents, or 14.1 percent, to $6 in after-hours trading.
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Osiris stem cell therapy approved in Canada
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First stem cell drug approved for systemic disease treatment
Posted: May 19, 2012 at 5:10 am
Osiris Therapeutics Inc said on Thursday that Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.
Osiris shares rose 14 percent to $6.00 in extended trading after the news was announced.
Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient's body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting.
The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.
Prochymal was approved with the condition that Osiris carry out further testing after it reaches the market. C. Randal Mills, the company's chief executive, said in an interview that could take three to four years.
Some investment analysts have been skeptical about Prochymal's future. In 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease, though it showed promise in certain subgroups of patients.
Since then, the company has mined data from all its clinical trials to show that in patients with severe refractory acute GvHD -- those who have more or less failed all other therapies -- Prochymal demonstrated a clinically meaningful response at 28 days after therapy began in 61-64 percent of patients.
In addition, treatment with Prochymal resulted in a statistically significant improvement in survival when compared with a historical control population of pediatric patients with refractory GvHD.
The Canadian authorities approved the drug on the basis of that data, the company said.
FDA submission this year
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Vet undertakes stem cell surgery
Posted: May 16, 2012 at 7:11 am
Animal stem cell regenerative therapy is the newest service at the Animal Hospital of Tiffin.
"We are the official first site for the therapy in Ohio," said veterinarian Bob McClung.
The technology uses an adult animal's stem cells to heal itself.
Veterinarian Mike Brothers performed the surgery Monday on his dog, Tucker, a 2-year-old labrador retriever. It was the second surgery performed at the clinic.
Brothers said his dog's joint problems are hereditary and he's had problems since he was a puppy.
"What we've been able to do is slow down the arthritis," Brothers said. The cause of the degeneration will continue, but the fatty tissue removed from the dog can be used for future treatments.
From a piece of fatty tissue of the size removed from Tucker, McClung estimated $3.2 billion stem cells were harvested.
Each injection uses about 90 million cells, so there will be enough of the material for future treatments.
"We have basically 2 billion cells to bank," he said. "We use cryo-preservation."
In the freezing process, the cells are gradually cooled to prevent damage and stored in liquid nitrogen at temperatures of minus 80 to minus 90 degrees Fahrenheit.
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Vet undertakes stem cell surgery
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Boston scientists grow lung tissue from cystic fibrosis patients’ skin cells
Posted: May 10, 2012 at 11:11 pm
By Carolyn Y. Johnson, Globe Staff
Two teams of Boston scientists have developed new ways to turn stem cells into different types of lung tissue, surmounting a major hurdle for scientists trying to harness the power of stem cell biology to study and develop treatments for major lung diseases.
One team then used skin cells from cystic fibrosis patients to create embryonic-like stem cells, then working in lab dishes used those cells to grow tissue that lines the airways and contains a defect responsible for the rare, fatal disease. The technique -- essentially a recipe for growing such lung tissue -- could provide a powerful platform to screen drugs and study the biology of the disease.
Growing lung tissue in the laboratory has long been a goal of stem cell scientists, but has been more technically difficult than growing other types of tissues, such as brain cells or heart cells. Such lung tissue is valuable because it could be used to screen potential drugs and more closely probe the problems that underlie diseases such as asthma, emphysema, and rare genetic diseases. Such techniques may also one day help researchers grow replacement tissues and devise ways to restore or repair injured lung tissue.
A team led by Massachusetts General Hospital researchers created lung tissue from a patient with the genetic mutation that most commonly underlies cystic fibrosis and researchers hope the technique will also be a powerful tool to study other diseases that affect the airway tissue, such as asthma and lung cancer. The other team, led by Boston University School of Medicine scientists, was able to derive cells that form the delicate air sacs of the lung from mouse embryonic stem cells. The team is hoping to refine the recipe for making the cells so that they can be used to derive lung tissue from a bank of 100 stem cell lines of patients with lung disease. Both papers were published Thursday in the journal Cell Stem Cell.
Vertex Pharmaceuticals, a Cambridge biotechnology company, earlier this year received approval for Kalydeco -- the first drug to directly target the underlying cause of cystic fibrosis. That compound was discovered by screening massive numbers of potential drugs against cells engineered to carry the same defect that underlies cystic fibrosis.
We had to use engineered cells, and certainly using more native human cells ... would be potentially beneficial, said Dr. Frederick Van Goor, head of biology for Vertexs cystic fibrosis research program. We had to rely on donor tissue obtained from patients with cystic fibrosis, and its a bit more challenging, because the number of donor lungs you can get and the number of cells you can derive from there are more limited.
Van Goor said it was too soon to say whether the company would use the new technology in screening, but noted that the tests the company had used to determine whether a drug was likely to work against the disease had, in some cases, given scientists false leads. Some molecules that worked on the engineered cells did not work in the complicated biology of the lung.
Its a significant event for the lung field, said Dr. Thiennu Vu, associate professor of medicine at the University of California San Francisco, who was not involved in the research. She added that much work remains before such cells could be used to repair or replace damaged tissue, and even before such cells would necessarily be useful for drug screening. It will be important, she said, to refine the recipe to ensure that the technique yields pure populations of the specific types of functional lung cells.
In the competitive world of science, where credit for being the first to do something is crucially important, the two research teams accomplishments are an unusual example of competitors turning into collaborators -- forging a relationship that both teams felt helped speed up progress.
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Stem cells boost brain tumor treatments for some patients, study finds
Posted: May 10, 2012 at 11:11 pm
(CBS News) Patients with brain cancer may face devastating side effects from chemotherapy, but a new study offers a possible solution: stem cells.
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The stem cells form a shield of sorts against the toxic side effects from chemo, according to the researchers behind the study. It was a small trial that involved only three patients with glioblastoma, the most aggressive and common form of a malignant brain tumor that's usually fatal.
Two of the patients survived longer than predicted with help from the stem cell treatment - an average of 22 months - and a third man from Alaska remains alive today with no disease progression almost three years following treatment.
How does it work?
Many patients with the deadly form of brain cancer possess a gene called MGMT. The MGMT gene is typically turned on and counters the effects from some chemotherapy agents, such as temozolomide, rendering them less effective. As such, people with such a gene often have a particularly poor prognosis.
A drug called benzylguanine can block the MGMT gene, thus making tumors more receptive to chemotherapy, but the combination of the drug and chemo are often too toxic for healthy bone marrow cells.
That's where the new stem cell treatment comes in. By combining bone marrow stem cells with a modified version of MGMT in the form of the new treatment, patients' cells were protected from the toxic effects of the cancer drugs and chemotherapy while keeping the tumor cells targeted.
"This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded," study author Dr. Jennifer Adair, a researcher at the Fred Hutchinson Cancer Research Center in Seattle, said in a news release.
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Stem cell that may aid healing and repair found in brain
Posted: April 25, 2012 at 10:13 pm
Washington, April 24 : Scientists have discovered a new stem cell in the adult brain and they hope to put the discovery to use to develop methods that can repair diseases and injury to the brain.
These cells can proliferate and form several different cell types - most importantly, they can form new brain cells.
Analysing brain tissue from biopsies, the researchers at the Lund University for the first time found stem cells located around small blood vessels in the brain.
The cell's specific function is still unclear, but its plastic properties suggest great potential. A similar cell type has been identified in several other organs where it can promote regeneration of muscle, bone, cartilage and adipose tissue.
In other organs, researchers have shown clear evidence that these types of cells contribute to repair and wound healing.
Scientists suggested that the curative properties might also apply to the brain. The next step is to try to control and enhance stem cell self-healing properties with the aim of carrying out therapies targeted to a specific area of the brain.
"Our findings show that the cell capacity is much larger than we originally thought, and that these cells are very versatile," said Gesine Paul-Visse, Ph.D., Associate Professor of Neuroscience at Lund University.
"Most interesting is their ability to form neuronal cells, but they can also be developed for other cell types. The results contribute to better understanding of how brain cell plasticity works and opens up new opportunities to exploit these very features," Paul-Visse added.
The study is of interest to a broad spectrum of brain research. Future possible therapeutic targets range from neurodegenerative diseases to stroke.
"We hope that our findings may lead to a new and better understanding of the brain's own repair mechanisms. Ultimately the goal is to strengthen these mechanisms and develop new treatments that can repair the diseased brain," said Dr. Paul-Visse.
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Second U-M Stem Cell Line Now Publicly Available to Help Researchers Find Treatments for Nerve Condition
Posted: April 25, 2012 at 10:13 pm
Newswise ANN ARBOR, Mich. The University of Michigans second human embryonic stem cell line has just been placed on the U.S. National Institutes of Healths registry, making the cells available for federally-funded research. It is the second of the stem cell lines derived at U-M to be placed on the registry.
The line, known as UM11-1PGD, was derived from a cluster of about 30 cells removed from a donated five-day-old embryo roughly the size of the period at the end of this sentence. That embryo was created for reproductive purposes, tested and found to be affected with a genetic disorder, deemed not suitable for implantation, and would therefore have otherwise been discarded when it was donated in 2011.
It carries the gene defect responsible for Charcot-Marie-Tooth disease, a hereditary neurological disorder characterized by a slowly progressive degeneration of the muscles in the foot, lower leg and hand. CMT, as it is known, is one of the most common inherited neurological disorders, affecting one in 2,500 people in the United States. People with CMT usually begin to experience symptoms in adolescence or early adulthood.
The embryo used to create the cell line was never frozen, but rather was transported from another IVF laboratory in the state of Michigan to the U-M in a special container. This may mean that these stem cells will have unique characteristics and utilities in understanding CMT disease progression or screening therapies in comparison to other human embryonic stem cells.
We are proud to provide this cell line to the scientific community, in hopes that it may aid the search for new treatments and even a cure for CMT, says Gary Smith, Ph.D., who derived the line and also is co-director of the U-M Consortium for Stem Cell Therapies, part of the A. Alfred Taubman Medical Research Institute. Once again, the acceptance of these cells to the registry demonstrates our attention to details of proper oversight, consenting, and following of NIH guidelines.
U-M is one of only four institutions including two other universities and one private company to have disease-specific stem cell lines listed in the national registry. U-M has several other disease-specific hESC lines submitted to NIH and awaiting approval, says Smith, who is a professor in the Department of Obstetrics and Gynecology at the University of Michigan Medical School. The first line, a genetically normal one, was accepted to the registry in February.
Stem cell lines that carry genetic traits linked to specific diseases are a model system to investigate what causes these diseases and come up with treatments, says Sue OShea, Ph.D., professor of Cell and Developmental Biology at the U-M Medical School, and co-director of the Consortium for Stem Cell Therapies.
Each line is the culmination of years of preparation and cooperation between U-M and Genesis Genetics, a Michigan-based genetic diagnostic company. This work was made possible by Michigan voters' November 2008 approval of a state constitutional amendment permitting scientists to derive embryonic stem cell lines using surplus embryos from fertility clinics or embryos with genetic abnormalities and not suitable for implantation.
The amendment also made possible an unusual collaboration that has blossomed between the University of Michigan and molecular research scientists at Genesis Genetics, a company that has grown in only eight years to become the leading global provider of pre-implantation genetic diagnosis (PGD) testing. PGD is a testing method used to identify days-old embryos carrying the genetic mutations responsible for serious inherited diseases. During a PGD test, a single cell is removed from an eight-celled embryo. The other seven cells continue to multiply and on the fifth day form a cluster of roughly 100 cells known as a blastocyst.
Genesis Genetics performs nearly 7,500 PGD tests annually. Under the arrangement between the company and U-M, patients with embryos that test positive for a genetic disease now have the option of donating those embryos to U-M if they have decided not to use them for reproductive purposes and the embryos would otherwise be discarded.
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Second U-M Stem Cell Line Now Publicly Available to Help Researchers Find Treatments for Nerve Condition
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