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

Video

The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.

“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

Recent Videos
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Related Content
© 2025 MJH Life Sciences

All rights reserved.