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UCI opens new stem cell clinical trial for ALS patients

Neurodegenerative disease has no treatment or cure

April 06, 2018

The University of California, Irvine Alpha Stem Cell Clinic and the UCI School of Medicine's Department of Neurology will be conducting a randomized phase 3 trial to determine whether an experimental stem cell-based treatment can slow, and potentially even stop, the progression of amyotrophic lateral sclerosis (ALS).

ALS, also known as Lou Gehrig’s Disease, attacks motor neurons, the cells in the brain and spinal cord that control our muscles and movement. Over time the disease destroys a person’s ability to speak, to swallow, to move, even to breathe.

“This disease has no effective treatment and no cure,” said neurologist Namita A. Goyal, MD, co-director of the UCI Health ALS & Neuromuscular Center and the trial’s principal investigator at UCI. “The early phases results have been promising and many ALS patients are eager to enroll.”

Nearly 50 other clinical trials for ALS have shown little to no effect on slowing the disease’s progression, she said.

The NurOwn therapy, developed by BrainStorm Cell Therapeutics, uses mesenchymal stem cells that are taken from the patient’s own bone marrow. These stem cells are then modified to boost their production of neurotrophic factors, which are known to help support and protect neurons, the cells destroyed by the disease.

In an earlier phase 2 clinical trial, involving 48 people with ALS, NurOwn was shown to be safe and well-tolerated. The trial also showed it could halt or reverse disease progression over six months, compared to a placebo.

The phase 3 trial will seek to enroll 200 eligible subjects at UCI and five other U.S. centers. It is co-sponsored by the California Institute for Regenerative Medicine.

“CIRM is partnering with BrainStorm to follow up on the company’s promising phase 2 trial in ALS,” said Maria T. Millan, MD, CIRM’s interim President & CEO. “CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurologic condition for which there is currently no cure.”

The UCI Alpha Stem Cell Clinic is the clinical trials program for the UCI Sue & Bill Gross Stem Research Center. Established in 2010, the research center is a leader in regenerative medicine and is pioneering research into stem cell therapies for neurodegenerative diseases such as, ALS, Huntington’s and Alzheimer’s diseases, spinal cord and traumatic brain injury, the degenerative eye disease retinitis pigmentosa, multiple sclerosis and other debilitating conditions.

To learn more about this ALS trial or other stem cell research at UCI, please call 949-824-3990 or email stemcell@uci.edu.  

About the University of California, Irvine: Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 30,000 students and offers 192 degree programs. It’s located in one of the world’s safest and most economically vibrant communities and it is Orange County’s second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit www.uci.edu.

Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.

Media contact: John Murray, 714-456-7759, jdmurray@uci.edu