Clive Svendsen, PhD, on Astrocyte Progenitor Cell Therapy in ALS


The director of the Regenerative Medicine Institute at Cedars Sinai discussed results from a phase 1/2a study of CNS10-NPC-GDNF.

“This product is very simple. It's a progenitor cell that turns into an astrocyte when you transplant it and it gets across the blood brain barrier. We engineered it with a lentivirus to secrete GDNF. It’s a combined stem and gene cell therapy product which can deliver both an astrocyte to the diseased area in the brain, and then when it gets there, it releases GDNF around the dying dopamine or motor neurons.”

CNS10-NPC-GDNF, an allogeneic human neural progenitor cell therapy transduced with glial cell line-derived neurotrophic factor (GDNF) and differentiated to astrocyte cells, well-tolerated and produced GDNF in participants with amyotrophic lateral sclerosis (ALS) according to recent data from a phase 1/2a study (NCT02943850). No adverse events (AEs) were observed due to surgery or cell transplantation and treated legs showed promising trends of greater limb isometric strength compared to untreated legs of patients with ALS.

CGTLive spoke with principal investigator Clive Svendsen, PhD, professor of medicine and director, Regenerative Medicine Institute, Cedars Sinai, to learn more about the study’s findings and next steps in assessing CNS10-NPC-GDNF. He discussed advantages in administrating a therapy that can be precisely targeted to diseased area in the brain.

Baloh RH, Johnson JP, Avalos P, et al. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial. Nat Med. Published online September 5, 2022.
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