Since the discovery in 1986 of the defective gene that causes Duchenne muscular dystrophy, research strategies have focused principally on correcting the genetic flaw by manipulating the genetic code in each muscle cell, or by alleviating the effect of the genetic flaw by re-engineering the muscle cell.
While Gene therapy and Cell therapy research strategies appear promising, we need to also focus on keeping the boys healthy today, so they too can benefit from the cure.
Scientist are now looking to a third, alternative therapy, a Pharmacological approach that may not cure or entirely alleviate the effects of Duchenne, but may result in viable therapies that would extend the integrity of muscle function today (short term) until a cure is available.
The Pharmacological approach seeks to treat the symptoms of Duchenne without necessarily addressing the cause, and therefore side stepping the most daunting obstacles that face gene therapy and cell therapy.
Scientists agree, that given the technology of high through put screening (sifting through millions of molecular compounds) we have the ability to isolate a drug that will slow the deterioration of muscle fibers or increase the muscle integrity.
Compound Quest Goal: Identify drug treatments that will result in preserving as much muscle integrity as possible in boys with Duchenne.
The Jett Foundation has recently invested significant resources in a drug screening company, Myomics, that is on the cutting edge of some very exiting discoveries! Together, and in conjunction with the world’s leading expert on Duchenne, Dr. Brian Tseng of Massachusetts General Hospital, we have identified a new technology that can measure the function of all muscle fibers when compounds/supplements are added. The technology is quick – and considerably less expensive than any other strategies that exist today for Duchenne. These already affordable, accessible compounds, will be combined with supplements, giving us a cocktail of sorts, to slow the progression of this disorder. Already, we have identified several positive “hits” that have proven to strengthen muscle function in vitro. We hope to move these discoveries into clinical trials next year at MGH. Funding this research will not only help extend the life for boys affected with Duchenne but also work as a springboard to all other muscle related issues that impact recoveries from cancers, heart attacks, and strokes. Finally, after nine years, the opportunity to positively impact the lifespan of all boys afflicted with Duchenne is at our fingertips. Research has and continues to be the most important means we have in finding appropriate treatments – and of the tools necessary to continue we have all but one; adequate funding.