Eighteen patients are about to receive an investigational stem cell/gene therapy combination that may stall progression of amyotrophic lateral sclerosis (ALS). Investigators at the Cedars-Sinai Board of Governors Regenerative Medicine Institute can test the drug thanks to recent approval from the Food and Drug Administration.

No effective treatment currently exists for ALS, which causes progressive paralysis and is ultimately fatal. This new combination approach involves engineering stem cells to produce an important protein called GDNF. It may protect the motor neurons that enable impulses to pass from the brain or spinal cord to a muscle. The investigators deduced that if motor neurons could be kept alive by adding supporting cells that make GDNF, then patients with ALS could retain the ability to move despite the disease’s encroachment.

“Any time you’re trying to treat an incurable disease, it is a long shot, but we believe the rationale behind our new approach is strong,” says Clive Svendsen, PhD, the institute’s director and the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine. Svendsen has spent a dozen years developing the treatment with funding from the California Institute for Regenerative Medicine.