Mayo Clinic investigates new experimental drug for ALS patients

ROCHESTER, Minn. (FOX 9) – Researchers at the Mayo Clinic have just received a new federal grant to begin studying an experimental drug in patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's syndrome.

What we know

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

The experimental drug is called Ibudilast. It is an anti-inflammatory drug manufactured by a Japanese pharmaceutical company and used in Japan to treat asthma. The drug is not approved in the United States, 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 whole point of this is to get people to test a product,” said Dr. Bjorn Oskarson, a neurologist and lead researcher at the Mayo Clinic in Jacksonville. “So we're hoping that for some people, taking the product will slow the progression of ALS.”

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Dr. Oskarsson says a small study done in the Carolinas about a decade ago showed that more than a third of patients who took the drug for six months did not appear to have worsening ALS symptoms. This new research will be conducted in parallel with a clinical trial of ibudilast that is more restrictive and includes patients in the early stages of ALS. Mayo's study under the EAP will also allow patients with advanced ALS to test the drug.

“It remains a deadly and difficult disease today,” said Dr. Oskarsson. According to the Centers for Disease Control, ALS affects the nerve cells that operate muscles in the upper and lower body. The nerves lose the ability to control muscles, leading to muscle weakness and paralysis. Most sufferers only survive two to five years after symptoms appear.

“We hope to eventually stop the disease altogether or at least push it back,” said Dr. Oskarsson about the hypothesis of his study on ibudilast. “And this is a step towards that. This drug alone probably won't get us there, but it could definitely help.”

What else

The study will involve 200 patients from Mayo Clinic's three locations in Florida, Minnesota and Arizona. The drug itself is taken orally in the form of a tablet. Each patient will undergo a series of blood tests to see if there are any changes in neurofilament protein levels. Dr. Oskarsson calls it a blood tachometer that will allow patients to monitor how the drug is working.

“We hope to start sometime in the next three to six months,” Dr. Oskarsson said of the Mayo study. “It's a tight schedule. Most projects take over a year to start, but we have strategies that I think will allow us to start sooner.”