U.S. Department of Defense Earmarks $758,121 to Develop RASRx1902 as Potential ALS Therapy

The U.S. Department of Defense (DoD) has issued a two-year research grant totaling $758,121 to support a group of scientists 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).

The project, led by Kathleen Rodgers, PhD, and Kevin Gaffney, PhD, will focus on evaluating the effects of the compound in cells from ALS patients cultured in a lab dish. The main goal of these experiments is to determine first if RASRx1902 can improve the overall health of patients’ cells, and then see at which stage of the disorder the treatment is most effective.

“The goal of our research is to develop tomorrow’s cures for patients that need them today,” Gaffney, an assistant research professor at CIBS, said in a press release. “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.”

RASRx1902 is an investigational oral drug that has been shown to reduce inflammation and oxidative stress (cellular damage that occurs as a consequence of high levels of oxidant molecules), improve cognitive function, and stimulate muscle regeneration in previous studies.

Because of its muscle regenerative properties, RASRx1902 has been explored as a potential treatment for Duchenne muscular dystrophy (DMD), a genetic disorder that gradually leads to muscle deterioration. Studies in animal models of DMD have shown the compound increased muscle strength and regeneration, reduced muscle inflammation, degeneration and cell death, without posing any toxic side effects.

Based on those promising findings, the U.S. Food and Drug Administration (FDA) granted the designation of orphan drug to RASRx1902 for the treatment of DMD in 2017.

In the new project, Rodgers and Gaffney will attempt to gather pre-clinical evidence demonstrating the effectiveness of RASRx1902 in ALS. If they are successful, RASRx1902 then may be tested in ALS patients participating in clinical trials.

“This is a critical stage of discovery in our pursuit of a cure for ALS,” said Rodgers, associate director of translational neuroscience at CIBS and 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,” Rodgers said.