New Study has Potential to Improve Lives of ALS Patients

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.”

Contract No: W81XWG1910471

The views expressed here are those of the author and may not reflect the official policy or position of the Department of the Army, Department of Defense, or the U.S. Government. In conducting research using animals, the investigator(s) adheres to the laws of the United States and regulations of the Department of Agriculture.

About the Center

The Center for Innovation in Brain Science (CIBS) at the University of Arizona Health Sciences is addressing the challenge that, in the 21st century, there is not a single cure for a single neurodegenerative disease. The CIBS team is focused on four age-associated diseases, Alzheimer's, Parkinson's, Multiple Sclerosis and ALS. With expertise spanning discovery, translational, regulatory and clinical science, CIBS is shifting the research paradigm as one of the nation's leading research centers, pioneering patient-inspired, data-driven approaches to find cures for Alzheimer's and other neurodegenerative diseases. The CIBS mission is to create innovations in brain science of the future for those who need a cure today. Find out more about how CIBS is achieving the vision of vibrant brains that last a lifetime.  cibs.uahs.arizona.edu. (Follow us: Twitter | Facebook)

About the University of Arizona Health Sciences

The University of Arizona Health Sciences is the statewide leader in biomedical research and health professions training. The UA Health Sciences includes the UA Colleges of Medicine (Tucson and Phoenix), Nursing, Pharmacy, and the Mel and Enid Zuckerman College of Public Health, with main campus locations in Tucson and the growing Phoenix Biomedical Campus in downtown Phoenix. From these vantage points, the UA Health Sciences reaches across the state of Arizona and the greater Southwest to provide cutting-edge health education, research, patient care and community outreach services. A major economic engine, the UA Health Sciences employs nearly 5,000 people, has approximately 900 faculty members and garners $200 million in research grants and contracts annually. For more information: uahs.arizona.edu (Follow us: Facebook | Twitter | YouTube | LinkedIn | Instagram)