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Research on treatment for ALS aided by stem cells

Paul Sherman/Daily
Neurology prof. Eva Feldman presents the results of phase I clinical trials of a new stem cell treatment for Lou Gerhig’s disease. Buy this photo

By Ian Dillingham, Daily Staff Reporter
Published October 2, 2013

Though the Food and Drug Administration remains closed due to the federal government shutdown, researchers at the University are pushing forward the development of stem-cell therapies, with the hope of improving the quality of life for individuals with life-threatening disabilities.

Researchers at University Hospital and the A. Alfred Taubman Medical Research Institute are exploring the use of stem cells in the treatment of amyotrophic lateral sclerosis — also known as Lou Gerhig’s disease, a neurodegenerative condition that causes cell death in spinal cord neurons that control movement. Patients with ALS suffer from loss of muscle control and often die of respiratory failure.

Neurology Prof. Eva Feldman presented recent results from her research at an event Wednesday evening at the Taubman Institute’s Kahn Auditorium for an audience of about 40 students and faculty. Feldman discussed the completion of Phase I trials of the new stem-cell therapy and her plans for Phase II.

While Phase I trials typically test the safety of a treatment in human patients, Phase II tests the treatment’s efficacy. Feldman’s research team received approval for Phase II of their research in May and has since begun tests.

Shortly before the event Wednesday afternoon, a third patient enrolled in the trial had the surgical procedure, in which a surgeon injects stem cells into specific regions of the spinal cord. Although it is too early to record changes in disease progression, Feldman said the three patients have experienced “no adverse consequences” from the procedure.

Stem cells have the unique ability to fulfill a wide variety of tasks by developing into specialized cells depending on their environment. When these cells are injected into the spinal cord of ALS patients, they surround diseased cells and slow the progression of cell death, Feldman said.

“Depending on how you grow them … they can become any cell in the body,” Feldman said.

Feldman’s treatment uses a relatively new strain of human embryonic stem cells developed at the University through partnerships with the National Institutes of Health. She referenced the work of Physiology Prof. Gary Smith at MStem Cell Laboratories — the University’s stem cell institution — as a crucial component to the development of the treatment.

“What we’ve done here at the University of Michigan … is make embryonic stem cell lines, which are now being used for understanding disease course as well as for treatment,” Feldman said.

Stem cells have the potential to aid in the treatment of not only ALS, but a wide range of debilitating and life-threatening diseases, including Parkinson’s disease, Alzheimer’s disease and multiple sclerosis, Feldman said.

“I know that the potential of human embryonic stem cells is really in its infancy,” Feldman said. “As we engage in understanding of what stem cells do, we will really then begin to more fully understand their complete potential. I really don’t think we’re even at the tip of the iceberg.”

Although the results from the trials are still preliminary, Feldman said four of 15 patients tested in Phase I experienced a “stable or improved disease course.” Prior to human studies, the research team demonstrated the capability of this treatment to slow the progression of symptoms in rats with the disease.

“Stem cells placed into the spinal cord of these rat models of ALS caused the large motor neurons to survive,” Feldman said. “And also actually allowed the rats to have continued function and not to die as quickly from their disease. We’re very excited about that.”

Prior to 2008, Feldman spent 10 years investigating ALS treatments at the University, while stem-cell research was still illegal in the state of Michigan.