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.

Leadership

Board of Directors

Joel Wood, President | Scott Sinder, Secretary | James Wood, Treasurer | Dan Carson, Director | Dave Heil, Director | Brad Todd, Director

Advisors

Dr. Eric Hoffman Dana Wood Monica Heil Tina Carson

Staff

Michelle Payne, Executive Director

Financial & Tax Information

FED strives to be transparent in our work. Here are links to our most recent IRS form 990 and audited financial statements. If you have questions, please do not hesitate to contact us.

What is Duchenne Muscular Dystrophy?

The words “incurable” and “untreatable” have been closely associated with Duchenne. It is an X-chromosome disease that afflicts boys, and it will never be eliminated through genetic screening because the dystrophin gene is the largest gene in body – times ten – and thus much more prone to have flaws that are not inherited. The trajectory of this disorder is insidious. Duchenne boys typically are not diagnosed until they are four years old or older. They tend to get better in the so-called “honeymoon” stage, which usually lasts until the early teenage years. But their muscles are deteriorating and cannot regenerate. Dystrophin is part of the chemical glue that holds muscle cell walls together, and those walls eventually collapse and are filled with collagen that causes fibrosis. First the legs give out, slowly, then, the arms and upper body. The disease moves from skeletal muscle to smooth muscle tissue. Lungs and hearts deteriorate. Corticosteroid treatment forestalls the muscle deterioration, but steroids have terrible side effects such as making bones brittle. Unlike many single-gene-defect disorders that only strike at certain ethnicities or demographics, Duchenne is a disease that does not discriminate – it is the world’s leading lethal inherited disease, afflicting one in 3,500 boys. The incidence rate among all races is identical. As the world becomes more conscious of issues of racial disparity, it is clear that standards of care are resulting in disparate quality of life and certainly life expectancy among sufferers of different races. You get the picture. Duchenne Muscular Dystrophy is excruciating, ruthless, sadistic, and painful. More than anything, it is cruel. The FED’s mission is to identify, leverage, and bring to market treatments that will benefit ALL sufferers of this disease.