“Latest Stem Cells News”

39-year-old Ted Harada was diagnosed with ALS, also known as Lou Gehrig‘s disease. It’s one of the worst diagnoses anyone could get.
He and his doctors expected his health to have severely declined by now. But thanks to an experimental stem cell treatment, he has tossed his cane and is once again playing in the pool with his three kids (…)

Then his neurologist told him about an experiment at Emory University that was recruiting ALS patients to test a stem cell treatment.
The surgeons told Harada that injecting the stem cells into his spine likely would not help him personally, and might even cause harm. But the study would hopefully help scientists find an effective treatment in the future. Harada had nothing to lose and expected nothing – he became study subject number 11 and underwent surgery on March 9 (…)

The Emory surgeons injected 1 million neural stem cells into 10 locations in Harada’s spine (earlier patients received fewer cells; the dosage was gradually increased as the trial progressed). All of the cells came from a single voluntarily aborted and donated two-month-old fetus. Using technology developed by Neuralstem, scientists multiplied the cells and created enough of them to treat all of the patients in this trial and beyond.

“We took one small part of the spinal cord and isolated one million stem cells which are now going to, we hope, treat millions of people around the world,” Dr. Karl Johe, chief scientific officer at Neuralstem told me.

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Professor Jeanne Loring

Stem cells show great potential to enable treatments for conditions such as spinal injuries or Lou Gehrig’s disease, and also as research tools. One of the greatest problems slowing such work is that researchers have found major complications in purifying cell mixtures, for instance to remove stem cells that can cause tumors from cells developed for use in medical treatments. But a group of Scripps Research scientists, working with colleagues in Japan, have developed a clever solution to this purification problem that should prove more reliable than other methods, safer, and perhaps 100 times cheaper.

The work appears in the current edition of the journal Cell Research.

Effective tricks for separating stem cells from other types are essential for many emerging medical treatments. These techniques begin with researchers inducing stem cells to take specific forms, or differentiate, for instance into nerve cells. These differentiated cells might then be used to repair a spinal cord injury. Other cells might enable a diabetic’s body to produce adequate insulin.

A key problem is that in the differentiation process, at least some stem cells inevitably remain in their undifferentiated, or pluripotent, state. These cells can grow to form tumors in patients if injected along with differentiated cells, a concern that has already led the US Food and Drug Administration (FDA) to delay clinical trials for promising stem cell-based therapies.

A New Approach

To date, almost all attempts at purification have focused on developing antibodies—immune system attack cells—that can remove or destroy stem cells in mixtures. But this approach has had shortcomings. Effective antibodies are difficult and expensive to develop, and their use in medical therapies raises safety issues because they are produced in animals.

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The Governing Board of the California Institute for Regenerative Medicine, the State Stem Cell Agency, approved a $25 million award to support the first FDA-approved clinical trial based on cells derived from human embryonic stem cells.

The award to Menlo Park-based Geron, Corp, will support the company’s on-going early phase trial for people with spinal cord injury. This is the first time the agency, which was created by the passage of proposition 71 in 2004, has funded a human clinical trial testing a stem cell-derived therapy.

“Supporting the Geron trial is a landmark step for CIRM,” said Robert Klein, CIRM chairman. “However, we must remember that there will be successes and interim failures as human trials proceed through the refinements necessary to achieve a successful human therapy. We need to be prepared to stand by the heroic patients and the companies as they face these challenges and solve the problems that stand in the way of the recovery of patients from paralysis. When the people of California voted for proposition 71 they did so with the hope of seeing new therapies for disabling diseases like Alzheimer’s disease, Parkinson’s disease, diabetes and other chronic diseases and injuries. By funding this trial, CIRM is taking a major step toward making that hope a reality. ”

The initial phase of the trial will include just a small number of people with recent spinal cord injuries who will receive injections of oligodendrocyte progenitor cells derived from embryonic stem cells into the site of the injury. In animal models, those cells mature into oligodendrocytes, which produce the insulating layer surrounding neurons. The initial phase of the three-year project is designed to test whether the cells are safe. Later phases will include different levels of spinal cord injury and will test increasing doses of the cells. One person has already received injections of the cells at a clinical trial site in Georgia. Stanford University Medical Center is another of the trial locations.

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An innovative experimental treatment for boosting the effectiveness of blood stem-cell transplants with umbilical cord blood has a favorable safety profile in long-term animal studies, according to Harvard Stem Cell Institute (HSCI) scientists at Dana-Farber Cancer Institute (DFCI), Beth Israel Deaconess Medical Center (BIDMC), and Children’s Hospital Boston (CHB).

Analysis of long-term safety testing in nonhuman primates, published online by the journal Cell Stem Cell in a new section called “Clinical Progress,” revealed that a year following transplant umbilical cord blood units treated with a signaling molecule called 16,16-dimethyl PGE2 reconstituted all the normal types of blood cells, and none of the animals receiving treated cord blood units developed cancer. Wolfram Goessling is the first author of the paper; his HSCI colleague Trista North is the senior author.

The results of long-term safety studies in mice were previously submitted to the Food and Drug Administration to gain permission for a Phase I clinical trial under an investigational new drug (IND) application. Principal investigator Corey Cutler, a Dana-Farber transplant specialist, initiated the trial in 2009 at Dana-Farber and Massachusetts General Hospital. The IND is sponsored by Fate Therapeutics Inc. of San Diego.

Goessling and North were postdoctoral fellows in the laboratory of co-author Leonard Zon, a stem cell researcher at CHB and a scientific founder of Fate Therapeutics, when they hit upon 16,16-dimethyl PGE2 while looking for compounds that could regulate the production of hematopoietic stem cells (blood stem cells). The initial testing made use of zebrafish models.

“This is the first time a compound discovered in zebrafish has received a nod from the FDA for a clinical trial,” said Goessling.

One of the limitations of cord blood as a transplant source is that the cells engraft, or “take,” in the recipient’s bone marrow more slowly than matched donor cells form bone marrow. In addition, there is a higher failure rate for cord blood transplants. Thus there is a need for ways to improve the speed and quality of cord blood transplantation.

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Discovery sheds new light on the process of stem cell generation, and will help promote safer stem-cell based studies and future clinical trials.

Dr. Andras Nagy’s laboratory at the Samuel Lunenfeld Research Institute of Mount Sinai Hospital and Dr. Timo Otonkoski’s laboratory at Biomedicum Stem Cell Center (University of Helsinki), as well as collaborators in Europe and Canada have identified genetic abnormalities associated with reprogramming adult cells to induced pluripotent stem (iPS) cells. The findings give researchers new insights into the reprogramming process, and will help make future applications of stem cell creation and subsequent use safer. The study was published online today in Nature.

The team showed that the reprogramming process for generating iPS cells (i.e., cells that can then be ‘coaxed’ to become a variety of cell types for use in regenerative medicine) is associated with inherent DNA damage. This damage is detected in the form of genetic rearrangements and ‘copy number variations,’ which are alterations of DNA in which a region of the genome is either deleted or amplified on certain chromosomes. The variability may either be inherited, or caused by de novo mutation.

“Our analysis shows that these genetic changes are a result of the reprogramming process itself, which raises the concern that the resultant cell lines are mutant or defective,” said Dr. Nagy, a Senior Investigator at the Lunenfeld. “These mutations could alter the properties of the stem cells, affecting their applications in studying degenerative conditions and screening for drugs to treat diseases. In the longer term, this discovery has important implications in the use of these cells for replacement therapies in regenerative medicine.”

“Our study also highlights the need for rigorous characterization of generated iPS lines, especially since several groups are currently trying to enhance reprogramming efficiency,” said Dr. Samer Hussein, a McEwen post-doctoral scientist who initiated these studies with Dr. Otonkoski, before completing them with Dr. Nagy. “For example, increasing the efficiency of reprogramming may actually reduce the quality of the cells in the long run, if genomic integrity is not accurately assessed.”

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From wheel chair to walk again

William Orr hasn’t always been in that wheelchair, 24 years ago he survived a tragic accident while riding his bike, leaving him paralyzed from the waist down. Today, he speaks with high hopes, optimism, and an emotional strength he will one day walk again, thanks to a Bonita Springs doctor.

Bill Orr is not quite walking. But he’s getting close. And his progress may be one of the best stories of 2010 for a whole lot of reasons.

The 50-year-old Aurora man has been a quadriplegic for half his life — since a car hit him while he was riding his bike home on Route 34 in Oswego back in 1986.

In August, Orr underwent what many believe is a first of its kind stem cell procedure in Naples, Fla. — using bone marrow from his hip that doctors hope will rejuvenate the dead spine cells.

The procedure is showing “amazing” promises (…)

The enthusiasm is bigger than Bill Orr, however. Grekos believes stem cell therapy has the potential to revolutionize medicine, but it’s not going to happen through the FDA or pharmaceutical companies. “It’s going to take a grass-roots movement,” he said (…)

Whatch video…

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