Duchenne’s Muscular Dystrophy, or DMD, has been in the news a lot recently. It’s the most common form of muscular dystrophy and was most recently featured in the Right to Try fight. The FDA also approved the first treatment of DMD’s symptoms in 2017, but a new paper published in Science points to a possible treatment of the root cause of the disorder. Scientists used a system-wide application of CRISPR to efficiently restore dystrophin expression in four dogs, a result that “exceeded [the lead author’s] most optimistic expectations.” There’s a ton of hurdles this treatment would have to pass to be a real therapy in humans, but this could be huge for DMD patients. Also, hey, it’s Labor Day weekend, maybe consider donating to the Muscular Dystrophy Association for Jerry’s Kids.
Hear InsightCity’s take on Right to Try with this short podcast.
Topics covered include: Right to Try explained, Compassionate Use programs, ambiguity in the legislation, and the role of insurers
The first case concerning the recent Right to Try legislation has come along and it involves Matt Bellina, one of the patients who pushed to pass the legislation in the first place. Before diving in this writer just wants to say—damn this guy is really a fighter. He’s not only a veteran battling ALS, but he also got legislation passed in freaking Congress, which isn’t easy. His case involves a cell therapy being developed by BrainStorm Cell Therapeutics, a biotech with no currently marketable treatments. It’s planning on addressing Right to Try applicants in a semicommercial model that they say won’t exploit patients. But since the company is pre-revenue there’s some big ethics concerns about how to charge vulnerable patients for the expensive treatments, which the law may not address.
Now we’re not going to begrudge anyone at the end of their rope looking for anything that can help, but we do have some strong opinions on the subject. Listen to our recent discussion on Right to Try here in our second InsightCity podcast.
The FDA’s Compassionate Use program helps patients that seek access to medications still in the development pipeline. But legislation signed into law this week allows patients to completely bypass the regulatory agency should they so choose. The “Right to Try” bill gives patients access to investigational drugs with the permission of just their physician and the drug manufacturer. It also shields those drug companies from the legal risks involved. Critics, including the American Cancer Society, say the bill gives false hope to patients, and champion the current process of compassionate use and clinical trials. After all, the FDA approves 99% of compassionate use applications, and can even do approvals over the phone. When asked for comment, some formerly prominent musicians said “You gotta fight for your right… to paaaaaaaaarty.”
If you were in the US House of Representatives (gross, right?), would you vote for the proposed “Right-to-Try” law?
Last week proposed legislation dubbed the “right-to-try” bill failed to pass the US House of Representatives, despite a lopsided 259-140 vote in favor of the law. Republicans who submitted the bill used a process that required a two-thirds majority to pass and the numbers fell just short of that. The new submission will require on a simple majority to pass—and it will. Here’s Austin Powers in a nutshell. More importantly, here’s the law in a nutshell… critically ill patients can gain access to experimental drugs if their physician and the drug company agree to it. The FDA would no longer need to sign off. Supporters say the law removes red tape. Critics say the FDA already approves 99% of requests and, according to Scott Gottlieb, FDA Commish, requests are usually approved immediately or within a few days anyway. So, where’s the beef?