The U.S. Department of Defense (DoD) issued a two-year grant for $758,121 to support investigators from the University of Arizona Health Sciences Center for Innovation in Brain Science (CIBS) working on the development of RASRx1902, a potential treatment for amyotrophic lateral sclerosis (ALS).
ALS News Today reports that the study focuses on the effects of RASRx1902 in cells from ALS patients cultured in a lab dish. The main goal of these studies is to determine if the compound can improve the overall health of patients’ cells and assess at what stage of ALS this investigative treatment could be optimal.
What is RASRx1902
RASRx1902 is an investigational oral drug that shows promise for the treatment of Duchenne muscular dystrophy (DMD), a genetic disorder that leads to muscle deterioration.
Preclinical Animal Studies
Preclinical animal models of DMD reveal increased muscle strength and regeneration—Additionally, researchers have found that the compound can reduce muscle inflammation, degeneration, and cell death, without posing any toxic side effects.
FDA Grants Orphan Status
Based on preclinical animal model studies, the U.S. Food and Drug Administration (FDA) designated RASRx1902 the designation of the orphan drug for the potential treatment of DMD in 2017.
Assuming the two investigators can build the case from preclinical research evidence, the next steps will introduce RASRx1902 to be tested by ALS patients participating in clinical trials.
Kathleen Rogers, Ph.D.
Kevin Gaffney, Ph.D.
Call to Action: If you are interested in tracking new amyotrophic lateral sclerosis treatments in the preclinical state sign up for our newsletter as we will track this effort.