Critical Path Institute Forms 10th Consortium for Duchenne Muscular Dystrophy

by Margarida Azevedo, MSc | August 5, 2015

Arizona-based Critical Path Institute (C-Path), a leading non-profit organization committed to accelerate and reduce developmental costs of medical products, recently announced it has formed its 10th consortium: The Duchenne Regulatory Sciences Consortium (D-RSC). Together with Parent Project Muscular Dystrophy (PPMD), the new consortium will utilize C-Path’s tested consensus science model to find a cure for DMD. Founding members of the D-RSC include Santhera Pharmaceuticals, PTC Therapeutics, and Dr. Brenda Wong from Cincinnati Children’s Hospital Medical Center. Sarepta Therapeutics, Inc. is expected to join the D-RSC, once C-Path has signed an agreement with the company. More members will be added to the consortium in the months to come.

The D-RSC will be responsible for collating and storing clinical information for easy access to its members, with the primary purpose of developing a disease progression model. “New drug development tools can increase the efficiency of clinical trials,” said Tim Franson, MD, a C-Path board member with consulting experience in Duchenne.

C-Path will soon pursue US and European regulatory endorsement for these tools. “Using our unique model and working directly with regulators, we aim to reduce the amount of time to get treatments to patients for this devastating disease,” explained Martha Brumfield, PhD, C-Path’s President and CEO.

A recent study recently published in the journal ACS Nano revealed a new technique to trigger muscle cell differentiation from adult stem cells using a genetic approach. The study is entitled “Inducing Stem Cell Myogenesis Using NanoScript” and was conducted by researchers at Rutgers, The State University of New Jersey in the United States and the Kyoto University in Japan.

Researchers used the NanoScript to mimic the body’s natural transcription factor that induces muscle-cell growth (myogenesis) – myogenic regulatory factors (MRFs). The generated NanoScript-MRF was found to be stable in physiological environments and to localize within the nucleus. Interestingly, the NanoScript-MRF induced the differentiation of adipose-derived adult stem cells into mature muscle cells in 7 days, so that fat tissue actually became muscle.

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About the Author

Margarida Azevedo, MSc Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.