Despite Flat Budget, NIH’s TRND Program Tries to Spark Interest in Rare and Neglected Diseases

Posted on January 31, 2012 by

With 14 approved programs in total, TRND has progressed two into clinical development.

For the past two years NIH has been trying to double its funds for developing orphan drugs. But the FY 2012 budget offered more of the same, as in the same $24 million of annual funding set aside for NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program since its creation in 2009. The institute had sought $50 million for TRND this year and last.

Genetic Engineering & Biotechnology News, January 31, 2012 (Alex Philippidis)

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Dr. Eric Hoffman Dana Wood Monica Heil Tina Carson

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Michelle Payne, Executive Director

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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.