Biotech Gets $15.4 Million for DMD Drug Work

The National Institutes of Health (NIH) has awarded a five-year, $15.4 grant to the biopharmaceutical company PTC Therapeutics for the development of small-molecule drugs to treat Duchenne muscular dystrophy (DMD).

PTC Therapeutics of South Plainfield, N.J., specializes in exploiting “post-transcriptional control” mechanisms for the development of treatments for disease. Post-transcriptional control refers to altering the cell’s interpretation of genetic material (DNA) after it has been transcribed into a closely related molecule, RNA, but before a protein has been constructed from the DNA instructions.

In 2005, MDA gave the company $1.5 million for the development of PTC124, an experimental compound that causes cells to ignore erroneous stop signals in DNA and construct normal protein molecules. Some 15 percent of boys with DMD are thought to have this type of genetic error (called a premature stop codon or nonsense mutation) in the gene for the muscle protein dystrophin.

The new NIH funding will go toward development of additional gene-modifying constructs to treat DMD. Among these are compounds to increase production of utrophin, a protein that can to some extent compensate for a deficiency of dystrophin; of agrin, another protein that may be helpful in DMD; and of IGF1, a growth factor protein that researchers hope will help offset muscle cell destruction in this disease.

H. Lee Sweeney, a professor of physiology at the University of Pennsylvania in Philadelphia, is the principal investigator on the new NIH grant. Sweeney has also worked closely with MDA and held several MDA grants between 1992 and 2006.

In a PTC Therapeutics press release, John Porter of NIH said this grant is the type of translational (lab to clinic) research his group wishes to support. Porter is program director of the Neurogenetics Cluster and Technology Development Program at the NIH Institute of Neurological Diseases and Stroke. He’s also a member of MDA’s Translational Research Advisory Committee.