Innovative ALS study receives $22 million grant for experimental drug

The Mayo Clinic in Florida has received a grant from the National Institute of Neurological Disorders and Stroke (NIDS) to start an innovative study that will provide hundreds of patients with amyotrophic lateral sclerosis (ALS) access to the experimental drug ibudilast over a six-month period. The grant totals $22 million. The Mayo Clinic will receive $12 million and will distribute the rest to its partners.

The U.S. Food and Drug Administration has granted the study Expanded Access Program (EAP) status. This program provides patients with a serious or life-threatening disease or condition a way to access an investigational drug (drug, biologic, or medical device) for treatment outside of a clinical trial when no effective treatment options are available.

Björn Oskarsson, lead researcher on the project and director of the Mayo Clinic's ALS Center of Excellence, says the most important measure of the drug's effect on ALS progression will be a blood test that shows whether neurofilament protein levels have changed in patients with ALS. High levels of neurofilament proteins can indicate damage to neurons.

About 200 patients in the program from all three Mayo Clinic locations and other facilities will be able to see their own neurofilament light readings to determine if the drug is right for them. This is the first time neurofilament light has been used in this way to help patients and doctors make shared decisions.

This study will allow patients to monitor their own ALS condition and we hope to confirm that this is an effective way to assess progress in ALS patients. This is important because it is difficult to say whether the disease progression of ALS is accelerating or slowing down. Neurofilament light can be thought of as a kind of speedometer that can give an indication of whether a treatment is working or not.”

Björn Oskarsson, MD, principal investigator of the project and director of the ALS Center of Excellence at Mayo Clinic

ALS is a fatal motor neuron disease. People with ALS often die within three years of diagnosis. Although there are advances in the understanding of ALS, there is no cure or treatment that significantly improves the motor function of sufferers or prolongs their survival.

Under this program, physicians treating ALS patients will join a network managed by the Mayo Clinic and WideTrial, an organization dedicated to providing patients with access to experimental treatments through EAP. BioSEND, which stores biospecimens from studies supported by NINDS or conducted in collaboration with the institute, will store blood samples from the study for future research and help establish an ALS biobank.

MediciNova will provide ibudilast, an anti-inflammatory drug currently used in Japan. The drug's ability to penetrate the central nervous system, block harmful proteins and stimulate cell repair makes it a potential option for ALS treatment.

“It is unfortunate that we do not yet have highly effective treatments for most forms of ALS today,” says Dr. Oskarsson. “We urgently need to find such treatments, and this project could bring us one or more steps closer to that goal.”