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USF helps reach milestone for treatment of ALS patients

A clinical trial found success with a new drug to slow progression of the disease.
Dr. Tuan H. Vu performs a procedure called electromyography on a patient in the ALS clinic at USF Health. Vu led USF's portion of a major national study on a possible third drug to treat the illness, commonly known as Lou Gehrig's disease.
Dr. Tuan H. Vu performs a procedure called electromyography on a patient in the ALS clinic at USF Health. Vu led USF's portion of a major national study on a possible third drug to treat the illness, commonly known as Lou Gehrig's disease. [ USF Health ]
Published Sep. 2, 2020

TAMPA — In the 10 months since he was diagnosed with Lou Gehrig’s disease, few things have brought Ziggy Grajzer hope like the University of South Florida.

There he has found support, relief and reason to believe he and others might get better. He’s feeling that again now as results of a clinical trial show success with a new drug to treat the disease, also known as amyotrophic lateral sclerosis, or ALS.

The university’s ALS clinic is one of 25 medical centers in the United States chosen to participate in the 24-week trial that experts are holding up as groundbreaking for patients like Grajzer, 69, of Clearwater. It’s the first successful trial his physician, Dr. Tuan Vu, a professor of neurology and director of the USF Health ALS Clinic, has seen in years.

“We’re actually starting to make a difference,” Vu told the Tampa Bay Times. “After many failed trials, this is a successful one.”

The findings are being published this week in the New England Journal of Medicine.

Dr. Tuan Vu, MD, professor of neurology and director of the USF Health ALS Clinic.
Dr. Tuan Vu, MD, professor of neurology and director of the USF Health ALS Clinic. [ University of South Florida ]

About 140 adult patients participated in the trial, with four at USF. A third were given a placebo pill twice daily while others were given AMX0035, a pill combining two medicines already in use: sodium phenylbutyrate and taurursodiol.

Vu and others in the trial found that patients who received the drug saw it slow the pace of their loss of daily functions, like the ability to walk, dress, eat, speak and breathe. There were minimal adverse effects, according to trial results.

“We basically believe this drug decreases the stress on the mitochondria and the endoplasmic reticulum,” Vu said, referring to two kinds of specialized structures in a living cell. “If you lessen the stress on those organs, they can retain energy and retain survival.”

In other words, the drug serves as a neuroprotectant, or a shield for the central nervous system. It blocks the damage ALS causes, preventing patients’ cells from dying as quickly.

Though more study is needed, AMX0035 is poised to become the third medication approved to treat ALS patients. It’s the first trial drug to show significant benefits since 2017, when the U.S. Food and Drug Administration approved edaravone. The only other drug approved to treat ALS is riluzole, which came on the market more than two decades ago.

Because the trial drug comes from two existing drugs already approved by the federal government, some patients have started making their own version, Vu said. “Some people look at it and say, I don’t want to wait for the FDA.”

Zbigniew "Ziggy" Grajzer was diagnosed with ALS in November 2019 and has been a patient at the USF ASL Clinic since.
Zbigniew "Ziggy" Grajzer was diagnosed with ALS in November 2019 and has been a patient at the USF ASL Clinic since. [ University of South Florida ]
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Since his diagnosis, Grajzer has lost use of his left hand. His legs don’t work as well, he has very little energy and he’s losing weight all the time. He spends most days at home, missing all the traveling he used to do and thinking about the role he can play in finding a cure for ALS.

“It’s hard to say it, but everything has changed,” he said. “Nothing matters except finding a cure so maybe you can live longer.”

In the meantime, the physicians and fellow patients at the USF clinic have become his second family, Grajzer said. He didn’t participate in the AMX0035 trial, but he’s been involved in two others.

Being there makes him feel “like a regular person, not a sick person,” he said. And it reminds him to look toward the future. To hope.

“Even if it doesn’t help me, it could help somebody else,” Grajzer said. “That means a lot.”