Clive Svendsen, PhD, on Unlocking the Potential of Astrocyte Cell Therapy

“All your neurons were born before you were because during development, they're all laid down. So, to try and transplant a new neuron in a 6-year-old patient and get it to function normally is really a big challenge. But if you can protect the ones we have, retain your memories, retain those dopamine circuits, retain those motor circuits, that's going to give you a huge boost and maybe prevent degradation.”

The allogeneic human neural progenitor cell therapy CNS10-NPC-GDNFwas well-tolerated and produced GDNF in participants with amyotrophic lateral sclerosis (ALS) according to recent data from a phase 1/2a study (NCT02943850). The therapy is transduced with glial cell line-derived neurotrophic factor (GDNF) and works by differentiating to astrocyte cells to support neurons. No adverse events (AEs) were observed due to surgery or cell transplantation, and preliminary efficacy analyses showed that 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 potential of astrocyte cell therapy in ALS and other conditions it is being evaluated in, including retinitis pigmentosa, stroke, Alzheimer disease, and Huntington disease.

REFERENCE

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. doi:10.1038/s41591-022-01956-3