DuchenneUK Combination Therapies




Combination therapies of FDA-approved drugs

FED is working together with the DuchenneUK foundation to bring combination therapies using FDA-approved drugs to improved health in DMD patients.  The rationale is that DMD involves a number of tissue problems for muscle, including inflammation, degeneration/regeneration, metabolic failure, and fibrosis.  There are existing FDA-approved drugs that target many or all of these different pathological pathways.  Can we learn which existing FDA-approved drugs show benefit in DMD, and how to use them in the best possible combinations?

This route towards combination therapies has been well-proven in HIV/AIDS, where the standard of care is multiple drug therapy.  In the trials to define the combination therapies for HIV/AIDS, the biomarker of ‘blood viral load’ was critical, both to see which drugs worked, and then to see how combinations could be optimized.

The combination therapies of FDA-approved drugs in DMD seeks to repeat this successful path for Duchenne boys, similarly using blood biomarkers as potential sensitive, reliable, and acute (rapid) read-outs of the effects of drugs, used singly and in combinations.

FED has funded the first biomarker-focused pre-clinical mdx trial of combination therapies, expected to be complete by end of 2016.   The goals are to transition to short biomarker-focused clinical trials in DMD in the next year.

This effort is integrated with a similar interest and focus of Duchenne UK in the United Kingdom.