The FDA caused major controversy by approving the drug Exondys 51 from Sarepta Therapeutics for treating Duchenne muscular dystrophy over the recommendations of a scientific panel it convened that had voted 7-3 against approval.
Is this contentious Sarepta approval serve as a sign of how the FDA will deal with stem cells moving forward?
Will the FDA approval, which was based on very limited, unclear data and in large part due to pressure from some patient advocates, serve as a warning to those of us hoping the agency will not yield to political or other pressure in weakening oversight of investigational stem cell therapies?
Newly released internal FDA emails show that Sarepta approval was enormously controversial within the agency itself. A top FDA scientist challenged the agency’s decision in strong terms, noting that “Sarepta was unable to reproduce its own findings and there was “no way to reach a rational conclusion” a “reasonably likely” clinical benefit could be predicted.”
I wonder if similar debates within the FDA could be going on right now related to its four draft guidances on stem cells and regenerative medicine. Ultimately could the agency leadership make judgment calls without data in effect green lighting stem cell clinics to continue offering unproven stem cell therapies and putting patients at grave risk?
If I had to guess I would say optimistically that this is unlikely to happen, particularly given the disastrous case studies the FDA heard at the meetings it called on stem cells. For instance, we heard more about the patient Jim Gass developing a spinal tumor and the breaking story of at least three women reportedly being blinded by a stem cell clinic in Florida. A man in the audience reportedly wore a sign around his neck saying he was blinded by a stem cell clinic. I have heard through the grapevine that more bad news is coming from Florida on the stem cell clinic front.
One way or another the FDA should say something about its draft guidances on stem cells by early 2017. The number of stem cell clinics appears to be continuing to shoot up. The FDA can be more efficient in reviewing stem cell products in development, but it all has to remain data-centric.
“I’ve heard through the grapevine.” That sounds likes real “science” to me.
Really?
Haha you may not intend it but you really come across like a jackass, Bill. You’re posting on a stem cell scientist’s blog…one where emotional dissent from all those who eschew concepts such as science-based medicine and rational review is freely allowed, yet you must resort to snide comments like that?
Dr. Knoepfler probably speaks to more people that are actually in this field before 10 am than you do in an entire year.
“I’ve heard through the grapevine” sounds like Marvin Gaye to me, not science. If I made a statement like that, I’d have probably been asked to present some facts to back it up. Just sayin’…
And read your own post before you deride me for making snide comments, jackass.
No more “jackass” stuff from either of you.
Miranda, I didn’t mention therapeutic efficacy. You did and you missed my point entirely. Read my comment again and if you still can’t figure it out, let me know.
Of course you did, you said, “How many patients do you estimate have received stem cell TREATMENTS…”
Any treatment must include efficacy unless you are speaking about the non-efficacious treatments that some unregulated stem cell clinics cheat people with?
If your point was different then sorry, I’m afraid I wasn’t able to find it.
The point is that according to @admin (somewhere in another post if memory serves me) there are as many as tens of thousands of stem cell treatments being performed in the US each year. And yet, we’ve seen less than a dozen “disastrous case studies” like Jim Gass. The point was safety. If it’s so dangerous, where are all the casualties? Where are all the complaints to the FDA and FTC? They don’t exist.
…”How many patients do you estimate have received stem cell treatments in the US over the past 6 years?”
The number treated is not a measure of therapeutic efficacy Bill – robust clinical data is. It would be like claiming to have good financial prospects after buying ten thousand lottery tickets
That’s 5 patients out tens of thousands, one of whom was treated with fetal stem calls abroad and others who were not even treated by a medical doctor. That’s the best you can come up with? How many patients do you estimate have received stem cell treatments in the US over the past 6 years?
“I’ve heard through the grapevine.” That sounds likes real “science” to me. Really?