Hope for ALS Patients

Researchers in the University of Arizona Health Sciences Center for Innovation in Brain Science are investigating a drug that may benefit people with Lou Gehrig’s disease.

Fall 2022
Female researcher talking with two other researchers

Kathleen Rodgers is developing a novel therapeutic for ALS, also known as Lou Gehrig’s disease.

/ Chris Richards photo

When Lou Gehrig’s disease begins, it always progresses. People with the disease eventually lose what most of us take for granted — the ability to walk, dress, write, speak and even swallow and breathe. 

The disease, also called amyotrophic lateral sclerosis or ALS, a progressive neurological disease, causes neurons in the brain and spinal cord to stop working and die. As neurons lose the ability to trigger specific muscles, the muscles weaken and can become paralyzed. 
Researchers at the University of Arizona Health Sciences Center for Innovation in Brain Science (CIBS) are developing a novel therapeutic to curb the disease’s acceleration with the help of a $1.5 million grant from the U.S. Department of Defense. Researchers will examine whether an investigational drug has the potential to reduce inflammation and increase the regeneration of nerve cells in people with ALS.
Research has shown that people who served in the military are twice as likely to be diagnosed with and die from ALS as those with no history of military service.

Kathleen Rodgers, associate director of translational neuroscience at CIBS, is using the two-year grant to study the drug, called CAP-1902 or RASRx1902. Research in models of Duchenne muscular dystrophy, another disease that causes progressive muscle degeneration, has shown that CAP-1902 reduces inflammation and oxidative stress, improves cognitive function and stimulates muscle regeneration.
“CAP-1902 has a very promising profile that warrants further development as a treatment for ALS patients,” says Rodgers, who also is a professor of pharmacology in the UArizona College of Medicine – Tucson. 

“This funding will enable us to produce CAP-1902 and determine its efficacy as a treatment for ALS, as well as to identify blood-based biomarkers of the response to treatment that will help us quickly advance this novel ALS therapeutic to the clinic, where it is needed most.”
There are two forms of ALS: sporadic and familial. Sporadic ALS is the most common form in the U.S., accounting for 90-95% of all cases. The cause of ALS is not well understood, but it is known that inflammation is a key driver of neuronal death and accelerates the disease.

The study will target the renin-angiotensin system, a hormone system that regulates blood pressure and inflammatory responses, among other things. The protective arm of the system, which Rodgers hopes to activate with CAP-1902, is known to reduce inflammation and oxidative stress. 

The goal is to increase the regeneration of nerve cells and reduce the inflammation that causes nerve cell death and accelerates ALS disease progression.

“Dr. Rodgers’ research is advancing a congressionally directed medical research program to develop a novel therapeutic for ALS, which is critical for our active military and veterans,” says Roberta Diaz Brinton, director of CIBS, Regents’ Professor of pharmacology and a member of the BIO5 Institute. 

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