Mayo Clinic in Florida has received a grant from the National Institute of Neurological Disorders and Stroke (NIDS) to begin an innovative study that will provide hundreds of patients with amyotrophic lateral sclerosis (ALS) access to the experimental drug ibudilast over six months. The grant totals $22 million. Mayo Clinic will receive $12 million and manage the distribution of the remainder to its collaborators.
The U.S. Food and Drug Administration has granted Expanded Access Program (EAP) status to the study.
This program offers a pathway for patients with a serious or life-threatening disease or condition to gain access to an investigational medical product (drug, biologic or medical device) for treatment outside of a clinical trial when no effective therapy options are available. Björn Oskarsson, M.D.
, lead investigator for the project and director of Mayo Clinic's ALS Center of Excellence, says the primary 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 may indicate damage in neurons. About 200 patients in the program drawn from all three Mayo Clinic sites and other institutions will be able to see their own neurofilament light levels to determine if the drug is right for them.
This marks the first time that neurofilament light will be used in this way to guide patients and physicians to make decisions together. This trial will.
