Ohio State professors involved in study to get ALS drug approved speak on success and impact

Katherine Simon
Fri, 11 Nov 2022 01:25:43 +0000
Campus, Instagram Page, News, als, ALS/MND Multidisciplinary Clinic and Translational Research Program, amyotrophic lateral sclerosis, Buckeyes, Dr. Adam Quick, Dr. Stephen Kolb, Edaravone, food and drug administration, Lou Gehrig’s disease, neurology, ohio state, Relyvrio, Riluzole

Sandra Abrevaya helps her husband, Brian Wallach, walk at their Kenilworth home on Feb. 13, 2022. Wallach was diagnosed with ALS, or amyotrophic lateral sclerosis, in 2017 at the age of 37 and was told he wouldn’t live past his infant daughter’s first birthday. He’s still here, although the disease progresses every year. Credit: Courtesy of Erin Hooley

For the first time since 2017, the Food and Drug Administration approved a new medication to treat patients with amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.?

Dr. Adam Quick, associate professor of neurology at Ohio State, said the new drug, Relyvrio, is the third approved medication for ALS, a progressive nervous system disease that causes cells in the nervous system to degenerate, which can lead to the loss of muscle control. The medication slows down the progression, prolonging a person’s “life expectancy by about six to six and a half months” past the three or fours years typically expected after symptoms first appear.??

“What the new medicine does is it basically prolongs survival in people with ALS,” Quick said. “It doesn’t stop it, unfortunately. It would be great if we were at that point, but it does slow it down, so it prolongs the process of how long it takes to get sick from ALS.”

Quick said ALS is difficult to treat because clinicians don’t know what causes the cells to die.?

“The challenge with neurological disease is that you’re really taking what were normal healthy cells within the body that are sort of getting sick and dying for reasons that we don’t totally understand, and trying to sort of support those and prevent that process from happening,” Quick said.??

Dr. Stephen Kolb, director of the ALS/MND Multidisciplinary Clinic and Translational Research Program and a professor in neuroscience and neurology, said Relyvrio is a combination of two different drugs thought to help slow the rate of decline in patients.?

Quick said to test the effectiveness of the drug, research institutions and universities with ALS clinics participated in a clinical trial in which patients took either the medication or a placebo. Researchers then observed results over six-month periods, he said. The study was sent to databases and analyzed by sites nationwide to measure effectiveness.?

Ohio State was one of the highest patient-enrolling sites for both the first and second trials, Quick said.?

Quick said for FDA approval, typically a medicine must show effectiveness in two separate placebo-controlled studies, but because ALS has so few treatment options, the FDA approved the drug before the second clinical trial —?which is currently ongoing — is over. He said the procedure is a 48-month study rather than six.

According to the FDA, the most common negative reactions to the drug are diarrhea, abdominal pain, nausea and upper respiratory tract infections.?

“It’s really not a toxic medication,” Quick said. “It doesn’t have detrimental effects on other organs like the liver or the kidney or anything like that, which some medications can.”

Kolb said the other two drugs to treat ALS are Riluzole and Edaravone, FDA approved in 1995 and 2017, respectively.?

“They’re thought to have somewhat different mechanisms from a cellular, basic science side, but they are all, in general, trying to calm down the kind of neuronal excitement of toxicity and free radicals and other things that are going on in different ways,” Kolb said.?

Kolb said the drug moving so quickly from an idea, to trials to being available to patients is a hopeful sign for the future of ALS.?

“I think this was just one of the first of many things that are going to be coming for ALS patients down the road, and I think it’s a really exciting time in the field,” Kolb said.?