ALS + REGENERATIVE MEDICINE
Frank Diaz, MD, PhD
Department of Neurology
Assistant Professor of Neurology
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Adam Mamelak, MD
Director, Functional Neurosurgery Program
Professor of Neurosurgery
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Clive Svendsen, PhD
Executive Director, Board of Governors Regenerative Medicine Institute
Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine
Professor of Biomedical Sciences and Medicine
After promising findings in a safety trial for patients with ALS focusing on stem cell injections in lower motor neurons, researchers are now studying the investigative therapy in the motor cortex. The stem cells, programmed into astrocytes that secrete glial cell line-derived neurotrophic factor (GDNF), may keep existing nerve cells healthier and preserve muscle control longer.
If motor cortex injections—done in collaboration with neurosurgeons and functional MRI experts—are safe, dose-finding efforts will begin. The early trials aim to provide support for the concept’s safety and potential to impact ALS symptoms.
"Our ALS patients are selfless: It's important for them to participate in this trial so that we can learn more about the disease and, hopefully, help others along the line."
— Frank Diaz, MD, PhD
"It is too early to say how this trial will impact outcomes for ALS patients. If proven safe, it will certainly open up an entire new field for both techniques of cellular delivery—which we can do safely already—and therapy options for ALS patients."
— Adam Mamelak, MD
Future Stem Cell Prospects
The lower neuron trial, documented in Nature Medicine, revealed valuable information about cell migration, which should improve future trials’ ability to target the transplant more accurately. Eventually, the ALS experts at Cedars-Sinai hope a combined approach targeting both the lower and upper motor neurons will offer patients effective delay of disease progression or even improvement in motor symptoms with a one-time treatment. A technique could also be developed to get stem cells to regions controlling breathing and swallowing.
If even minor success is demonstrated in ALS, GDNF-enhanced stem cell therapy may garner tremendous interest for exploration in numerous neurodegenerative and neuropsychiatric conditions.
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In the first trial of its kind, a Cedars-Sinai team showed that a combined stem cell and gene therapy treatment can be delivered past the blood-brain barrier and could protect diseased motor neurons in the spinal cord of patients with ALS.
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