The pull for an ALS drug, and because a FDA has such a tough job
April 10, 2015 - als
BioFlash Editor- Boston Business Journal
Yesterday we spoke by phone with a lifelong Franklin proprietor with amyotrophic parallel sclerosis named Melissa King. Actually, we talked to her iPad.
King was diagnosed with ALS final year and now can't speak, eat, splash or take medicine on her own. She spoke to me regulating an app on an iPad she got by a ALS Association so she could still communicate. The subject was a petition seeking to vigour a Food and Drug Administration to conditionally approve an early-stage drug by a California association named Genervon that appears effective in crude course of a illness in 12 hearing subjects.
I was put in hold with King by studious advocates who contacted final week me due to my stating on Sarepta Therapeutics (Nasdaq: SRPT), that is building a drug for Duchenne robust dystrophy. In some ways, what ALS patients wish is a same as what families of Duchenne patients are seeking; Namely, redeeming capitulation of a drug formed on a tiny trial, tentative a outcome of a incomparable trial. The disproportion is that in Sarepta’s case, a 12 patients have been holding a drug now for some-more than 3 years with no reserve issues, and continued justification that it works. In Genervon’s case, a 12 patients were on a drug for 3 months — most shorter than a FDA typically requires even in vast trials.
In King’s eyes, a tiny hearing distance and brief generation are not current arguments for a FDA’s not permitting patients entrance to a drug, called GM604. The life outlook for people with ALS is dual to 5 years. Her symptoms emerged dual years ago. In a really genuine sense, she has small to lose.