Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, affecting vital functions such as speech, swallowing and breathing. Commonly known as Lou Gehrig’s disease, the disorder is characterized by muscle weakness, stiffness and, eventually, paralysis. 

As part of the international effort to combat this devastating disorder, researchers at the University of Arizona Health Sciences Center for Innovation in Brain Science (CIBS) have been awarded $758,121 for a two-year grant from the U.S. Department of Defense (DoD) to advance the study of a novel therapeutic, RASRx1902, which in early studies has been shown to reduce inflammation and oxidative stress, improve cognitive function and regenerate muscle.  

Led by Kathleen Rodgers, PhD, associate director of translational neuroscience, and Kevin Gaffney, PhD, assistant research professor, the study will test isolated cells from ALS patients to determine whether cell health can be improved and at what stage of the disease the therapeutic is most effective. Extensive pre-clinical work has  been conducted on this novel therapeutic for Duchenne muscular dystrophy (DMD) and has been shown to be safe and efficacious, with no toxic side effects. Moving forward, Drs. Rodgers and   Gaffney will work to collect the pre-clinical efficacy data that would allow RASRx1902 to move toward clinical studies in ALS patients. 

“This is a critical stage of discovery in our pursuit of a cure for ALS,” said Dr. Rodgers, principal investigator of the study. “We have an urgent need for treatments that will have immediate and long-term benefits for individuals with this devastating disease. RASRx1902 has the potential to improve the treatment, quality of life and long-term outlook for ALS patients and their families,” Dr. Rodgers said.

ALS usually strikes people between the ages of 40 and 70 and estimates indicate at least 20,000 Americans have the disease at any given time, according the ALS Association. For unknown reasons, military veterans are about twice as likely to be diagnosed with the disease as the general public.

“The goal of our research is to develop tomorrow’s cures for patients that need them today,” Dr. Gaffney said. “We are excited for this opportunity to assess the potential of RASRx1902 to treat ALS. We truly appreciate the DoD’s investment in this important research.”