August 7, 2012

NEALS research site Cedars-Sinai Medical Center Regenerative Medicine Institute has been awarded a $17.8 million grant from the California Institute for Regenerative Medicine (CIRM) to develop a stem cell treatment for patients with ALS.  NEALS will provide project management, data management, systems support, outcome measure training and validation for a future Phase 1 clinical study.

“We are thrilled to work with Clive Svendsen and his team to help bring the application of this technology as a potential therapeutic treatment to patients with ALS,” says Dr. Merit Cudkowicz, NEALS Co-Director and Neurology Chief at Massachusetts General Hospital, “This is an important step forward in developing cell-based therapies in ALS.”

Clive Svendsen, PhD, Director of the Regenerative Medicine Institute and NEALS Researcher, will lead the effort, which involves a combination of implantation of neural stem cells and gene transfer of a protein called GDNF that promotes neuron survival.  Delivering GDNF to the brain or spinal cord previously has been unachievable because it does not cross from the blood to the tissue of the spinal cord.  Stem cell implantation, on its own, has the potential to protect damaged motor neurons; therefore, the potential should further increase when stem cell implantation is combined with GDNF.
 
“We’re looking at a novel and exciting way of using stem cells as ‘Trojan horses’ that arrive at the sick motor neurons and deliver the protein exactly where it’s needed,” says Dr. Svendsen.  “Our early laboratory studies indicate this approach has significant potential and we’re excited to bring this treatment a step closer to helping ALS patients.”

Svendsen’s team at Cedars-Sinai will collaborate with research teams at two other NEALS sites, Emory University in Atlanta, GA and California Pacific Medical Center in San Francisco, to take stem cells from pre-clinical studies to running the 18-patient clinical trial for people with ALS in four years.

“With this CIRM funding, we can now move the development effort forward,” says Dr. Svendsen.

Clive Svendsen, PhD
Director of the Regenerative Medicine Institute
at Cedars-Sinai Medical Center