In the February 20th edition of the New York Times, Reed Abelson reports a "New Tack on a Muscle Disease." The "new tack" is, in fact, a scaling back of ambition in muscular dystrophy (MD) research. Since identification of various genetic mutations that cause muscular dystrophies, gene transfer has been a logical goal. But because the gene is so large, and delivery to all of a patient's muscles so difficult to achieve, gene transfer against MD has presented a formidable challenge to researchers.
What is so striking about Abelson's article, in my view, is his suggestion that the ambition of a one-off cure has clouded recognition of interventions that extend life and improve its quality for MD patients. Abelson writes "the lack of interest in the mundane has... slowed progress in knowing what available therapies are the most useful."
One researcher stated "for too long, I was stuck on [Duchenne] as the home run or nothing." Though speculative, "home run" research deserves sanctuary in biomedical research, I regard a critical role for disease advocates, caregivers, and bioethicists to advocate on behalf of the quotidian needs of present day patients (and, I should add, gene transfer will likely play an important role in serving these quotidian needs). (photo credit: Evamol, "Still Life," 2007)