Innovative ALS study receives $22 million grant for experimental drug

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.

ALS is a fatal motor neuron disease. People with ALS often die within three years of diagnosis. While there have been advances in understanding ALS, there is no cure or treatment that significantly improves motor function or extends survival in those with the disease.

In this program, physicians treating ALS patients will join a network managed by Mayo Clinic and WideTrial, an organization focused on expanding patient access to experimental treatments through EAP. BioSEND, which houses biospecimens collected through studies supported by or conducted in collaboration with NINDS, will hold 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 possible option for ALS treatment.

"It is unfortunate that today we do not yet have highly effective treatments for most forms of ALS," Dr. Oskarsson says. "We urgently need to find such treatments, and this project could get us one or more steps closer to finding our way there."

Source:

Mayo Clinic