MDA funds and coordinates a worldwide program of ALS research, providing support for investigations ranging from early-stage science, to preclinical testing and therapy development, to human clinical trials.
The Association manages a fluid and diverse research portfolio, with support extended to projects in key areas of ALS research, including: the identification and characterization of ALS-associated genes; the health of cellular energy factories called mitochondria; stem cell therapy; the use of “antisense” therapy to block toxic genes; immune system modulation; and the development of therapies that nourish and protect motor neurons.
In addition, MDA is at the forefront of advocacy efforts to speed up the federal approval process in the development of ALS therapies.
MDA funding for ALS research includes:
Building on MDA-supported studies, riluzole (Rilutek), a glutamate inhibitor, was developed and approved by the U.S. Food and Drug Administration in 1995. It remains the only FDA-approved drug therapy for ALS.
In 2007, through its Augie’s Quest research initiative, MDA joined forces with the ALS Therapy Development Institute (ALS TDI) of Cambridge, Mass., to launch the largest privately funded ALS drug development project in history. Today, this multimillion-dollar collaboration continues to combine the passion and dedication of nonprofit-fueled research with the entrepreneurial spirit and scientific resources of a biotechnology company.
A collaboration forged in 2013 between MDA and the U.S. National Institutes of Health aims to generate exome sequencing information on samples taken from 1,000 people with ALS. Data generated by the project is expected to accelerate the pace of ALS research by helping scientists identify genes associated with the disease.