Ohio State involved in ‘milestone’ ALS study

Ohio State University’s Wexner Medical Center participated in a clinical trial that was shown to slow down the progression of ALS, also known as Lou Gehrig’s disease.

Massachusetts-based Amylyx Pharmaceuticals conducted the clinical trial evaluating AMX0035, an investigational neuroprotective therapy designed to reduce the death and dysfunction of motor neurons in people suffering from Amyotrophic Lateral Sclerosis.

AMX0035 won’t make ALS go away, but it was shown to slow the disease’s progression, said Dr. Adam Quick, a neurologist at Wexner Medical Center who helped with the study.

“We’re trying to find things that slow down the progression and make people stay at a higher level of function for a longer period of time,” Quick said. “That’s the cool thing about this medication is it really did do that.”

ALS affects some 16,000 Americans and usually leads to death within five years.

The disease causes the progressive degeneration and death of nerve cells in the brain and spinal cord that control voluntary movements such as chewing, talking, walking and breathing, according the National Institute of Neurological Disorders and Stroke. The cause of the disease is unknown and there is no cure.

There currently are only two Fdaapproved drugs – Radicava and Rilutek – to treat ALS, said Kuldip Dave, vice president of research at the ALS Association, based in Arlington, Virginia.

The hope is that AMX0035 will be approved as a third.

“We certainly think this is quite a milestone for the ALS community,” said Justin Klee, CO-CEO and cofounder of Amylyx Pharmaceuticals.

The function of ALS patients is measured by a tool called the ALS Functional Rating Scale. The 48-point scale measures aspects of physical function — ranging from a person’s ability to swallow and use utensils to climbing stairs and breathing. The lower the number on the scale, the more severe a person’s ALS symptoms.

ALS patients treated with AMX0035 scored an average 2.32 points higher on the scale after the 24-week trial, Dave said.

“This two-point difference is clinically meaningful,” Dave said. “It can translate for a patient the difference from being able to write versus being unable to write. Or being able to cut food versus needing to be fed.”

About 140 people with ALS from 25 medical centers across the country, including seven patients from Wexner Medical Center, participated in the trial. Two-thirds of patients were given the drug AMX0035 and the rest received a placebo in the double-blind study.

The results of the clinical trial were released Wednesday in the New England Journal of Medicine.

“It’s just really cool now that we have another medication that was shown to be beneficial,” Quick said.

AMX0035 still has to be approved by the U.S. Food and Drug Administration and isn’t currently available to the general public.

“I think it’s going to give a lot of people a lot of hope that it’s something else they can do to really help with this really terrible neurological illness,” Quick said. “So I’m hopeful that it will be available to everybody very soon.” mhenry@dispatch.com @megankhenry