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Sep 5, 2022

Stem cell-gene therapy shows promise in ALS safety trial

Posted by in categories: biotech/medical, life extension, neuroscience

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. 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.

In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans. The findings were reported today in the peer-reviewed journal Nature Medicine.

“Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that can’t otherwise get through the ,” said senior and corresponding author Clive Svendsen, Ph.D., professor of Biomedical Sciences and Medicine and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute. “We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect that die in ALS.”

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