Safety trial on stem cell-gene therapy for ALS shows promise
Cedars-Sinai researchers have developed a therapy that uses support cells, a protective protein and can pass through the blood-brain barriers. This stem cell and gene combination therapy could potentially protect the diseased motor neurones in the spinal chord of patients with amyotrophic lateral sclerosis (ALS), a fatal neurological disease known as Lou Gehrig’s Disease or ALS.
The Cedars-Sinai researchers conducted the first clinical trial of this kind to show that the delivery of the combined treatment was safe for humans. The findings have been published in Nature Medicine, a peer-reviewed scientific journal.
\”Using stem cell therapy is an effective way to deliver proteins that cannot otherwise pass through the blood-brain-barrier to the brain and spinal cord,\” said Clive Svendsen Ph.D. Clive Svendsen is professor of Biomedical Science and Medicine at Cedars-Sinai Board of Governors Regenerative Medicine Institute and executive director. We were able show that the engineered product could be safely implanted into the human spinal chord. After a single treatment, the cells survive and continue to produce an important protein that protects motor neurons in ALS for more than three years.
Source:
https://medicalxpress.com/news/2022-09-stem-cell-gene-therapy-als-safety.html
