The study used stem cells originally designed in Svendsen’s laboratory to produce a protein called glial cell line-derived neurotrophic factor (GDNF). And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS,” Svendsen says.Īimed at preserving leg function in patients with ALS, the engineered cells could pave the way to a therapeutic option for this disease that causes progressive muscle paralysis, robbing people of their ability to move, speak and breathe. “We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. In a news release, senior author Clive Svendsen, PhD, executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, says using stem cells shows lots of promise in treating patients with ALS. There is no effective therapy for the disease. People with the disease lose the ability to move their muscles and, over time, the muscles atrophy and people become paralyzed and eventually die. The disease results when the cells in the brain or spinal cord that instruct muscles to move-called motor neurons-die off. About 6,000 people are diagnosed with ALS each year in the U.S., and the average survival time is two to five years. What causes ALS?ĪLS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. The findings were reported in the peer-reviewed journal Nature Medicine. In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Lou Gehrig’s disease. With funding support from the California Institute for Regenerative Medicine (CIRM), Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. Senior author of the study Clive Svendsen, PhD (center)
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