Identifying and prioritizing therapeutic targets for Duchenne

Who determines what drug(s) should be tested in the clinic and what are the ‘rules’ for making decisions about trials?

PPMD has requested Treat-NMD organize this committee to evaluate the therapeutic potential of approved drugs with preclinical data and develop a priority list for clinical trials. There is an immediate need to develop an impartial committee with the capability and expertise to evaluate the case for moving these drugs toward clinical trial. Evaluation must be accomplished in an unbiased way. The committee developed must include expertise in preclinical, toxicology, regulatory issues, clinical trials, ethics; and advocacy.

Because new chemical entities are moving into human studies, it is critical that approved drugs suggested to have benefit on Duchenne pathology are systematically reviewed. We have a limited patient population and cannot afford to waste time and precious funds on trials that have little chance of success.

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Comment by Pat Furlong on January 13, 2010 at 11:51am
Hello Paul,
I do not think Hopkins will be involved in the Losartin trial. Rather MDA is funding the Losartin trial in their 5 clincal network sites:
Washington University in St. Louis;

Boston Children's Hospital;

Nationwide Children's Hospital in Columbus, Ohio;

University of California Davis in Sacramento, Calif.; and

University of Minnesota in Minneapolis
Comment by Paul Cliff on January 13, 2010 at 9:20am

Is there any word on the Losartan trials for DMD? I had thought that they to begin at Johns Hopkins this year, but I have only heard that suspicious silence I associate with FDA created obstacles.

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