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First NIH-funded personalized drug development center in US will focus on muscle disease

First National Institutes of Health-funded personalized drug development center in the U.S. will focus on muscle disease New Center of Research Translation awarded to Children's National Medical Center, University of Pittsburgh School of Medicine and Carolinas Medical Center to accelerate R&D

Washington, DC The first Center of Research Translation (CORT), focused on personalized drug development for Duchenne muscular dystrophy (DMD), has been created through a $7.9 million grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health. The 5-year grant was awarded to a consortium of academic laboratories at Children's National Medical Center in Washington, DC, University of Pittsburgh in Pittsburgh, PA, and Carolinas Medical Center in Charlotte, NC.

The Center's personalized drug development will focus on a series of small molecule drugs, called antisense oligonucleotides (AO), which target specific mutations in the dystrophin gene to partially correct the DMD genetic defect at the mRNA splicing level. This approach is called "exon skipping," and is intended to help DMD patients produce dystrophin, the protein that is missing in their muscles. Exons are those segments on a gene that contain the necessary genetic information to produce a protein.

"While there has been very promising clinical trial data recently reported for specific exon 51 AO drugs in DMD, the Center of Research Translation will lay the groundwork for the next series of exon-specific drugs," said Eric Hoffman, PhD, director of the Center for Genetic Medicine Research at Children's National Medical Center and the CORT's co-principal investigator. "Patients with DMD have different mutations on various exons, so finding a treatment that is effective for the majority of patients involves looking at multiple defects simultaneously, which is what the Center aims to do."

Pursuit of AO drug development extends more than 20 years, particularly focused in cancer and inflammatory disease, although success in clinical trials has been limited. "We are seeing this type of therapeutic approach working better in DMD than other indications because of improved drug delivery, and more effective molecular targeting," noted Paula Clemens, MD, associate professor of neurology at the University of Pittsburgh and co-principal investigator of the new center. Dr. Qi Lu, director of McColl Lockwood Laboratory at Carolinas Medical Center and co-investigator of the CORT, provided additional rationale for improved efficacy in DMD: "We may need to reach only 20 percent of RNA targets in a successful 'rescue' application such as in DMD, whereas the previous 'knock-down' approaches in cancer and immunological disorders had to hit 90 percent of molecular targets." Dr. Lu's research in the new CORT will help optimize drugs to achieve the highest therapeutic potential for the majority of applicable DMD patients.

Research projects sponsored by the Center will include the first natural history study of Becker muscular dystrophy, a milder form of disease that is caused by mutations in the dystrophin gene. By studying specific mutations that cause Becker muscular dystrophy, CORT researchers will gain insights into the therapeutic potential of exon-skipping approaches. A second project will look at the biochemical function of the dystrophin proteins produced through application of the different exon-skipping drugs, which will help researchers determine the most effective AO drug candidates. The Center also includes tissue and cell culture banks which will allow for more accurate assessments of the individual response to selected AO drugs, fulfilling the need for unique personalized medicine.

"If we can show solid evidence of efficacy and safety for two or three exon-specific drugs simultaneously and these drugs behave similarly, then we are hopeful that this can facilitate regulatory assessment of subsequent AO drugs of this type," said Edward Connor, MD, director of the Office of Innovation Development and Investigational Therapeutics at Children's National Medical Center. "The new Center will serve as an important catalyst for advancing AO drugs for DMD and will be at the forefront of clinical and translational science in this field."

"The NIAMS Centers of Research Translation grants represent a significant investment in the most promising translational research," said Dr. Glen Nuckolls, NIH Program Officer for the new Center. "I am pleased to see this new Center join our other new CORTs in scleroderma, osteoarthritis and Sjogren's syndrome."


Contact: Emily Hartman
Children's National Medical Center

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