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).

ALS TDI, located in Cambridge, Mass., screens at least 25 potential ALS therapeutics every year.

“MDA has been, and continues to be a key partner in providing us the resources needed to move more ideas and projects forward faster than before,” said Steve Perrin, ALS TDI CEO and chief scientific officer. “MDA support has enabled many discoveries and advancements which hold great potential for people diagnosed with 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.

Data generated by the first-of-its-kind project will be made publicly available online and are expected to accelerate the pace of ALS research by helping scientists identify genes associated with the disease.

The exome-sequencing project, which is expected to be completed within 12 months, will produce genetic information for:

• 360 deceased individuals who had the sporadic (without any family history of the disease) form of ALS, for whom postmortem tissue samples are available; and
• 640 samples stored at the Coriell ALS Repository from people (both living and deceased) with sporadic ALS.

Exome sequencing data from a large number of people unaffected by ALS will be used for comparison in analysis.

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.

MDA has awarded $1 million to biopharmaceutical company ARMGO Pharma for development of a new strategy for treating Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

The award supports laboratory work that will enable the company to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration. (An IND application is a step a company must take before it can test a new drug in humans.) Receiving an IND approval will allow ARMGO Pharma to prepare for clinical trials with ARM210 in healthy volunteers and for subsequent clinical studies in people with DMD.

The experimental class of compounds — which ARMGO calls Rycal drugs — is designed to normalize the way skeletal muscle fibers and heart muscle cells handle calcium.

The lead compound, ARM210, is aimed at treating DMD or BMD, which are caused by mutations in the dystrophin gene. However, the strategy is not dependent on the specific disease-causing genetic mutation and potentially could have application to other muscle disorders.

The Muscular Dystrophy Association has awarded $1,195,762 over two years to biotechnology company Valerion Therapeutics (formerly 4s3 Bioscience) for development of a treatment for myotubular myopathy (MTM).

The Concord, Mass., company is developing a therapy for MTM that involves delivering myotubularin — the protein that is deficient in MTM — to muscle fibers using an experimental cell-penetrating strategy.

The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA's translational research program.

It will allow the company to continue testing its experimental compound3E10FV-MTM1 in mice and to begin producing a pharmaceutical-grade drug that potentially can be tested in people with MTM.

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.

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.

Connolly will conduct this phase 2 trial through the five centers that make up the MDA Duchenne Clinical Research Network.

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.

In an attempt to balance the side effects and benefits of corticosteroids for DMD, multiple dosing regimens have been in use. This multinational trial, known as FOR-DMD, will compare the three most widely used regimens of two corticosteroids to determine the optimal dosing schedule.

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).

Penton's grant was given through MDA's Bridge-to-Industry program, which connects promising new researchers to mentors in academia and industry. He'll work with one mentor at the University of Arizona in Tucson, Ariz., and another at the Oro Valley, Ariz., site of Sanofi, a multinational pharmaceutical company.