TUCSON, Ariz., Dec. 19, 2011 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) has awarded $750,000 to Summit PLC, a UK biotechnology company, to continue development of an experimental drug that may become a viable treatment for boys with Duchenne muscular dystrophy (DMD).
The funding comes from MDA Venture Philanthropy (MVP), a part of MDA's translational research program.
DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily affecting voluntary muscles. Caused by mutations in the gene that makes dystrophin, a protein that normally protects muscle cells and keeps them intact, DMD eventually affects all voluntary muscles, and the heart and breathing muscles. DMD affects 1 in 3,500 boys with an estimated patient population of 50,000 in the developed world.
Summit's SMT C1100 is an experimental compound designed to increase the muscle protein utrophin, which is a naturally-occurring protein that has a similar function to dystrophin. Investigators are confident that if utrophin could be upregulated sufficiently, it could act as a substitute for the missing dystrophin in DMD to help maintain healthy muscles.
"A long-time champion for utrophin upregulation as a promising therapeutic strategy for treating Duchenne muscular dystrophy, MDA is pleased Summit soon will be testing a new formulation of SMT C1100 in a human clinical trial," said Valerie Cwik, M.D. Executive Vice President Research and Medical Director for the Muscular Dystrophy Association.
"This is a vital step toward knowing definitively whether utrophin can successfully stand-in for the missing dystrophin protein that MDA-funded investigators identified as the underlying cause for this form of muscular dystrophy," Cwik added.
Barry Price, Ph.D., Executive Chairman of Summit said: "The new clinical study to be conducted on SMT C1100, a drug discovered and developed by our scientists is an important initiative. We believe SMT C1100 has the potential to treat all DMD patients and provide benefit for them and their families."
A previous trial of an earlier SMT C1100 formulation on healthy volunteers by another biotech firm in California deemed the compound to be safe and well-tolerated. However, variable blood concentrations were generated with only some individuals achieving expected therapeutic levels in the bloodstream.
About Utrophin Upregulation
Utrophin is a naturally-occurring protein that has a similar function to dystrophin. Utrophin is produced during fetal development but its production is switched off in adults. If utrophin production could be maintained, it could act as a substitute for the missing dystrophin to maintain the healthy function of muscles. One method of turning utrophin production back on is through pharmacological means. Utrophin upregulation has the potential to be beneficial to all DMD patients regardless of their specific genetic mutation and is also expected to be complementary to other therapeutic approaches in development.
About Muscular Dystrophy Association
MDA is the nonprofit agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive healthcare and support services, advocacy and education.
Long the world's leading non-governmental funder of muscular dystrophy research and health care services to affected individuals, MDA has played a pioneering role in utrophin-related research since 1996 (investing nearly $16 million to help develop this promising therapeutic pathway). Presently, MDA active grants include 102 focused on Duchenne muscular dystrophy, representing a multi-year investment of close to $35 million.
In addition to annually funding some 300 research teams worldwide, MDA maintains a national network of some 200 hospital-affiliated clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides extraordinary local summer camp opportunities for thousands of youngsters fighting progressive muscle diseases. Known globally for its Labor Day Telethon, MDA is the first nonprofit to receive a Lifetime Achievement Award from the American Medical Association "for significant and lasting contributions to the health and welfare of humanity." To learn more, watch the award-winning "Make a Muscle, Make a Difference" PSA, visit mda.org or follow MDA on Facebook or Twitter.
Summit is an Oxford, UK based drug discovery Company with an innovative Seglin™ technology platform for the discovery of new medicines and a portfolio of drug program assets. Seglin™ technology is using new chemistry to access biological drug targets that cannot be exploited by conventional drug discovery approaches and it is expected to have broad use in major disease areas. Summit's program portfolio consists of a number of drug programs targeting areas of high unmet medical need including Duchenne Muscular Dystrophy and C. difficile infection.
Summit is listed on the AIM market of the London Stock Exchange and trades under the ticker symbol SUMM. Further information is available at summitplc.com.
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SOURCE Muscular Dystrophy Association