USF Health plays role in emerging treatment for ALSTrio of drugs could work together

A quarter century after researchers identified two drugs to improve the lives of those with amyotrophic lateral sclerosis (ALS), a therapy is emerging from the CENTAUR clinical trial as a precious third. USF Health ALS Clinic Director Dr. Tuan Vu worked with co-investigators to test and evaluate Amylx Pharmaceuticals’ AMX0035, a neuroprotective therapy. USF Health was one of just two Florida sites involved; 25 top U.S. medical centers participated.

Identifying treatment modes for ALS has long been an arduous task. Numerous trials have been conducted to no avail; up until now, advancements were confined to just the two approved medications. It took CENTAUR’s 24-week randomized trial of AMX0035, an oral therapy comprised of sodium phenylbutyrate and taurursodiol, to change that.

The drugs act upon different pathways and can therefore be taken together as complimentary therapies. AMX0035 joins the ALS fight alongside Riluzole, the first drug approved for ALS, that acts to block the neuroexcitatory toxic effect. The second, Edaravone, serves as a free radical scavenger. Now, the additive effect of AMX0035 makes it a likely candidate to be added to patient treatment protocols.

“AMX0035 acts as a neuroprotectant at the endoplasmic reticulum and mitochondrial level,” Vu says.

ALS robs the stricken of the ability to move and speak. The progressively relentless disorder involves nerve cell death of the brain and spinal cord; AMX0035 protects motor neurons differently than the other two approved ALS medications. No, it is not a cure. Still, study data indicates it possesses an inherent ability to slow disease progression.

This is no small benefit. Amyotrophic lateral sclerosis (ALS) is progressive and fatal. The neurogenerative disorder leads to muscle deterioration, robs the patient of the ability to speak or move, and eventually causes death. Every year, approximately 6,000 people in the United States are diagnosed with ALS. More extensive trials are recommended to gain a better understanding of the efficacy and safety of the new medication, but for now, optimism reigns. 

“It’s another step to slow the disease while we are waiting to find a cure,” Vu says.

Postscript: Remember the ALS Ice Bucket Challenge? CENTAUR was funded partly by that, so anyone who braved an icy deluge in the name of philanthropy should feel proud.

Discover more about the USF Health ALS Clinic.