University of South Florida

USF Health was among top U.S. medical centers to test new ALS treatment shown to significantly benefit patients

This week’s New England Journal of Medicine reported results of the pivotal CENTAUR clinical trial; the USF Health ALS Clinic was one of only two sites in Florida for the study

Tampa, FL (Sept. 2, 2020) – Despite two approved medications and at least 100 clinical trials later, an urgent need for new treatments to improve the lives of patients with amyotrophic lateral sclerosis (ALS), a relentlessly progressive and fatal disease, continues to exist.

This week’s New England Journal of Medicine published results of the highly anticipated CENTAUR clinical trial, and included among the study’s coinvestigators was Tuan Vu, MD, professor of neurology at the Morsani College of Medicine and director of the USF Health ALS Clinic.

CENTAUR was a 24-week randomized, double-blind trial Phase 2/3 trial of 137 adults with ALS conducted across 25 top medical centers in the U.S. through the Northeast ALS (NEALS) Consortium. USF Health was one of two Florida sites to evaluate the safety and effect on disease progression of Amylx Pharmaceuticals’ investigational neuroprotective therapy, known as AMX0035. The oral therapy combines two different medicines: sodium phenylbutyrate and taurursodiol.

Using the Revised ALS Functional Rating Scale (ALSFRS-R) to measure daily functions such as the ability to walk, dress independently, self-feed, speak and breathe, the CENTAUR investigators demonstrated that treatment with AMX0035 decreased the rate of decline in patients with ALS compared to those with ALS receiving a placebo. The therapy was generally well tolerated, with similar rates of adverse events reported in the AMX0035 and placebo groups. For more details on the CENTAUR trial, including the open label extension study, and next steps, click here. 

“ALS is likely the end result of several different pathological processes leading to the death of nerve cells in the brain and spinal cord that control voluntary muscle movements — so there is no one treatment solution. To combat this complex neurodegenerative disease we need to target more than one process at the same time,” Dr. Vu said.  “This investigational drug protects motor neurons in a different way than the two currently approved ALS medications (riluzole and edaravone). It was beneficial in slowing disease progression, allowing patients to remain functional for a longer period of time.”

Only two drugs to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, have been approved by the FDA over the last 25 years.

“CENTAUR met its prespecified primary outcome, showing a clinically meaningful and statistically significant treatment benefit on the ALSFRS-R, the most commonly used scale in clinics worldwide to measure function in ALS,” said Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General and assistant professor of physical medicine and rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.”

AMX0035 was the first investigational therapy to demonstrate statistically significant benefit on the prespecified primary outcome in patients with ALS since edaravone, an intravenous drug approved by the U.S. Food and Drug Administration (FDA) in 2017.  The first and only other commercially available drug to treat ALS, riluzole, was FDA approved 25 years ago.

The NEJM study authors concluded that sodium phenybutyrate/taurursodiol (AMX0035) resulted in slower progression of ALS over 24 weeks as measured by the ALSFRS-R total score, but recommended longer and larger trials to further evaluate the therapy’s effectiveness and safety.

Tuan Vu, MD

Dr. Tuan Vu, professor of neurology, directs the USF Health ALS Clinic. Below: Dr. Vu performs an electromyography (EMG), one of the tests used to help diagnose ALS.

Zbigniew (Ziggy) Grajzer of Clearwater, Fla, was diagnosed with ALS in November 2019 and began treatment at the USF Health ALS Clinic in February 2020. He takes both edaravone and riluzole and remains hopeful about any steps forward to find new ALS treatments to improve quality of life and maintain independence.  While he was not enrolled in CENTAUR, Grajzer, 69, participates in other ALS clinical trials at the USF Health whenever he can. “It is the only way to find a cure,” he said, “if not in time for me, then to help others in the future.”

Grajzer said he feels fortunate that his ALS appears to be progressing slowly, but acknowledges the struggles of living with a disease that gradually robs an individual’s ability to control muscle movement. He has lost use of his left hand, his legs are weaker, and he tires easily, even walking from one room to another.  “I enjoyed going everywhere, to the beach and the mountains. Now most of what I do is around the house. But at least I can still move, speak, eat, breathe and think, and I’m grateful for that,” he said.

Zbigniew (Ziggy) Grajzer

Zbigniew (Ziggy) Grajzer began treatment at the USF Health ALS Clinic earlier this year.

CENTAUR, the recipient of an ALS Accelerated Therapeutics grant, is supported by The ALS AssociationALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS ConsortiumHealey Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge.

The USF Health Amyotrophic Lateral Sclerosis (ALS) Clinic in Tampa, Fla., offers the latest treatment, clinical research opportunities, supportive care and educational resources to help patients, their families and caregivers manage the effects of ALS. The clinic has been recognized by The ALS Association as a Certified Treatment Center of Excellence.

ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The vast majority of patients with ALS (>90%) have sporadic disease, showing no clear family history. Approximately 6000 people are diagnosed with ALS in the United States every year with an approximately similar number of deaths every year.

AMX0035 is an investigational neuroprotective therapy designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

-Video by Allison Long, USF Health Communications and Marketing

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