MDA and the nonprofit biotech ALS Therapy Development Institute (ALS TDI) have extended their strategic research partnership through 2013. With the extension comes a $3.2 million MDA grant to help support the nonprofit biotech's continued progress toward developing treatments for amyotrophic lateral sclerosis (ALS).
MDA has awarded $400,000 to National Institutes of Health (NIH) Laboratory of Neurogenetics researchers to perform exome sequencing on samples taken from 1,000 people with sporadic amyotrophic lateral sclerosis (ALS). The project will be led by neurologist Bryan Traynor, head of the Neuromuscular Diseases Research Group at the NIH in Bethesda, Md.
Anne Connolly, a pediatric neurologist at Washington University in St. Louis, was awarded an MDA human clinical trial grant totaling $343,787 over a period of nearly three years (through April 30, 2016) to conduct a trial of prednisone in boys with Duchenne muscular dystrophy (DMD) who are younger than 2½ years old.
Christopher Penton, who recently earned his doctorate in integrated biomedical sciences at Ohio State University in Columbus, was awarded an MDA research grant totaling $180,000 over three years to study ways to improve muscle regeneration and decrease muscle scar tissue formation in Duchenne muscular dystrophy (DMD).
MDA awarded a $500,000 grant to biotechnology company Halo Therapeutics for development of a treatment for Duchenne muscular dystrophy (DMD). The two-year grant begins in November 2013 and will run through Nov. 15, 2014. The Newton, Mass., company is developing HT-100, an experimental compound designed to combat excessive inflammation and formation of scar tissue in muscle and promote muscle regeneration. It has the potential to be used alone or in conjunction with other therapies and is now being tested in a 30-person clinical trial in boys and young men with DMD.
Robert Griggs, a professor of neurology at the University of Rochester in New York state, received an MDA grant of $237,316 over three years to cover travel costs for North American participants in a clinical trial of prednisone and deflazacort, two corticosteroid drugs used to treat Duchenne muscular dystrophy (DMD). The average participant and his parent or guardian will have to stay overnight near the trial site to complete all procedures at each visit — a considerable expense for many families.
Jeffrey Chamberlain, a molecular biologist at the University of Washington, Seattle, was awarded an MDA grant of $328,628 over one year (through Jan. 31, 2014) to develop gene therapy delivery vehicles ("vectors") for gene transfer in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). Chamberlain will develop gene transfer vectors derived from adeno-associated viruses (AAVs). They'll be designed to carry miniaturized versions of the gene for the dystrophin protein, which is missing in DMD-affected muscles and deficient in muscles affected by BMD. The studies are directly related to moving gene therapy for DMD/BMD into clinical trials.