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Mayo Clinic to study new experimental drug for ALS patients

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Published  September 25, 2024 6:33pm CDT
Health
FOX 9

Mayo Clinic’s studying experimental ALS drug

Mayo Clinic researchers have just been awarded a new federal grant to start an investigation of an experimental drug on patients with amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig’s disease.

The Brief

    • Mayo Clinic received a federal grant to investigate ibudilast, an experimental drug for ALS (Lou Gehrig's disease).
    • The drug, approved in Japan for asthma, is being tested in the U.S. under the Expanded Access Program, which allows its use in patients with life-threatening conditions without a cure.
    • The study will enroll 200 patients across Mayo's Florida, Minnesota, and Arizona campuses, with hopes to slow ALS progression.

ROCHESTER, Minn. (FOX 9) - Mayo Clinic researchers have just been awarded a new federal grant to start an investigation of an experimental drug on patients with amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig’s disease.

What we know

The grant from the National Institute of Neurological Disorders and Stroke totals $22 million. Mayo Clinic’s Florida campus in Jacksonville will receive $12 million and manage the distribution of the remaining grant to its collaborators in the study.

The experimental drug is called ibudilast.  It is an anti-inflammatory drug made by a Japanese pharmaceutical company and used in Japan to treat asthma. The drug is not approved in the U.S. but has been granted Expanded Access Program (EAP) status.  The EAP classification allows researchers to test the drug on patients with life-threatening diseases or conditions for which there is no cure or approved therapy.

"The core of this is to let people test a product," said Dr. Bjorn Oskarson, a neurologist and lead investigator at Mayo Clinic in Jacksonville.  "So we are hoping to see that some people have a slowing of the ALS disease when they're taking the product."

Context

Dr. Oskarsson says a small study done in the Carolinas about ten years ago showed upwards of a third of the patients who took the drug for six months did not seem to have their ALS symptoms worsen.  This new investigation will run in parallel with a clinical trial of ibudilast that is more restrictive to include patients at the beginning stages of ALS.  Mayo’s study under the EAP will allow patients with advanced ALS to also test the drug.

"The illness today remains a deadly and difficult disease," said Dr. Oskarsson. According to the Centers for Disease Control, ALS affects the nerve cells that make muscles work in both the upper and lower portions of the body.  The nerves lose the ability to trigger muscles, which causes the muscles to weaken and lead to paralysis.  Most people live only two to five years after symptoms develop.

"We're hoping to slow the disease down eventually to a complete standstill or start pushing back," said Dr. Oskarsson of his hypothesis for his study of Ibudilast. "And this will be a step on the way. This drug in of itself probably won't take us all the way there, but it could definitely help."

What else

The study will include 200 patients from across Mayo Clinic’s three campuses in Florida, Minnesota, and Arizona.  The drug itself is a pill taken orally.  Each patient will have a series of blood tests to show whether neurofilament protein levels change.  Dr. Oskarsson calls it a blood speedometer that allows patients to monitor how the drug may be working.

"We are hoping to launch somewhere in the next 3 to 6 months," said Dr. Oskarsson of the Mayo study. "That's an aggressive time schedule. Most projects take over a year to start, but we have strategies that I think will allow us to start sooner."