As much of the DMD community is already aware, yesterday Sarepta Therapeutics went in front of the FDA, seeking approval to sell Eteplirsen within the US.
Unfortunately, the FDA voted against this 7-3, with 3 votes abstaining.
If approved, Eteplirsen would have been able to treat ~13% of the DMD population, based on specific mutations. Despite 12+ hours of hearings, including patient and advocate testimonials, the FDA found the trial to be too poorly designed with not enough concrete outcome evidence, thus the denial. The FDA went so far to state that “anecdote and emotions don’t change the data.”
While blame is easy to turf, and hindsight is 20/20, I think we (as advocates and health care professionals), have a lot we can learn from this to better prepare for the next proposal. I believe that there is a fair amount of short-sightedness in the clinical trial design (with understandable reasoning) in that many companies hope and expect a drug to work in 6-24 months (all companies included), while these diseases take a lifetime to affect boys. In this limited timeframe, mitigation of disease is subtle with current outcome measures. Overall, it would be more cost efficient to run a single successful large randomized control trial for a longer time, rather than multiple failed trials.
In all, it makes me appreciate what ImagingDMD does, using quantitative, longitudinal, and objective outcome measures like MRI, to assess the state of health.
Just the other night, our mentors, Drs. Walter and Vandenborne, hosted a going away party for me with our lab. I have been quite fortunate to be around a scholarly group of researchers, but more than scientists and physical therapists, they're simply good people. They're good people, motivated to help accelerate the finding of a cure for the spectrum of muscular dystrophies, from basic benchtop findings to logistical planning of the clinical trials.
As a student, my perspective of the lab's operation is narrow, but I've been able to have a glimpse into what it takes to run and lead a lab of this magnitude through the leadership of Drs. Walter and Vandenborne, and for that, I'm grateful.
Beyond professional connections, I've been fortunate enough to make some life long friends through this journey, and look forward to future professional collaborations with these friends! Thanks!
Acute heart failure with cardiomyocyte atrophy induced in adult mice by ablation of cardiac myosin light chain kinase
In a collaboration with Dr. Hideko Kasahara and colleagues, we recently published in Cardiovascular Research. It was found that using a new mouse model to mimic the acute reduction of cardiac myosin light chain kinase, we were able to observe sarcomeric disorganization and functional losses as hearts transitioned from compensated to decompensated hypertrophy.
I'm a current MD-PhD candidate, working hard to help treat and manage muscular dystrophies