Beverly Grove-based Cedars-Sinai Medical Center announced June 23 that it has been awarded nearly $12 million from California’s stem cell agency to launch a clinical trial for a potential gene and stem cell therapy for amyotrophic lateral sclerosis, or ALS.
Commonly known as Lou Gehrig’s Disease, ALS is a progressive neurodegenerative disease that damages nerve cells known as motor neurons in the brain and spinal cord that control voluntary muscle movement.
According to the Cedars-Sinai announcement, ALS affects an estimated 16,000 people in the U.S. and is usually fatal within five years of diagnosis. There is no known cure and only limited treatments that slow progression in some patients.
The $11.99 million award from the California Institute for Regenerative Medicine builds on earlier support from the stem cell agency enabling Cedars-Sinai to investigate the use of stem cells delivered to the spinal cord to treat ALS patients.
Research into this latest stem cell therapy is being led by Cedars-Sinai researcher Clive Svendsen, who is executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and also is a professor of biomedical sciences and medicine.
“We are extremely grateful for CIRM’s support of our ALS work over many years,” Svendsen said. “This disease remains a remarkably stubborn and heartbreaking disorder. The new grant allows us to follow the science wherever it takes to best serve our patients.”
The therapy Cedars-Sinai is developing is designed to transplant neural progenitor cells secreting a powerful growth factor into the brain cortices of ALS patients. The aim is to slow the progression of ALS.
CIRM has helped fund ongoing work with this same therapy that is currently being tested by the same Cedars-Sinai regenerative medicine institute in a U.S. Food and Drug Administration-approved clinical trial
This stem cell therapy builds on previous research of a similar therapy that successfully slowed the progression of ALS in rodent models.
The latest CIRM grant allows Cedars-Sinai to enroll 16 patients in a clinical trial to demonstrate the safety and preliminary effectiveness of the stem cell therapy.
The trial’s clinical lead researchers are Adam Mamelak, professor of neurosurgery, and Richard Lewis, professor of neurology. The project leader is Pablo Avalos, a research scientist in Svendsen’s laboratory; Avalos is the inventor on a patent application filed by Cedars-Sinai Medical Center involving this stem cell therapy.