Clive Svendsen, PhD

“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.

 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.

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. 

. Published online September 5, 2022. doi:10.1038/s41591-022-01956-3

Videos

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

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

“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.

 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.

Published online September 5, 2022. https://doi.org/10.1038/s41591-022-01956-3

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

Clive Svendsen, PhD

Articles

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

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