Hey, all! Yep, today our story continues. When we left off at the end of Part 5, UC-Davis scientist and stem cell blogger extraordinaire, Paul Knoepfler, was about to take the stand to speak… and here he is. There’s also a teaser at the end for a BIG surprise tomorrow, in Part Seven…
“The FDA’s mission is to assist safety and efficacy and speed innovations,” Paul Knoepfler said in a soft voice. “The issue is getting the two to mix. Believe me, I do realize that a lot of people see this as an issue. But the thing is, no other regulatory body oversees stem cell medications in the U.S. And in the past, various drug mistakes have hurt or killed people. There’s no way around that. Thalidomide is a good example.”
Oh, please, please don’t show any slides of deformed babies, I silently prayed.
Whether it was because he’d read my mind or not, none of those were shown. I knew what had happened in the late 1950’s with thalidomide, a drug originally used as a mild sedative and sleeping pill. It was also given to pregnant women to lessen morning sickness, with tragic, unforgivable results. But what I hadn’t known was that the FDA had not approved the drug in the US, despite considerable business pressures at the time. Thalidomide had been marketed and sold in the rest of the world, starting in Germany. It was one of the most widely prescribed drugs of any kind in that era. If I hadn’t already been listening intently—which I was, of course, because this was Paul Knoepfler—then that was a fact that would have made me sit up and pay attention.
Gene therapy was another example, which was something I knew a fair amount about—more, in fact, than I wanted to. In the 1990’s, the trials had gone forward prematurely, pushed by the eager, the greedy, and the unrealistic. And people had died as a result.Jesse Gelsinger was the most well-known example, but he was far from the only one. The resulting negative publicity had hurt the gene therapy field considerably.
I privately thought that this was a good reason to not rush induced pluripotent stem cells to the clinic. The first IPSC trial had already begun in Japan in September 2014 in Japan with Dr. Takahashi, long before it was ready for the jump from mice to humans, in my opinion. But that’s another post.
Paul went on to say that a huge controversial current issue was the so-called “three person cloning,” an IVF technique that would produce embryos with genetic material from three people. The UK Parliament and Science and Technology Committee was in the middle of rushing through that technology at that very moment. He paused. “This technology conclusively does create genetically modified human embryos and hence it would produce human beings that are GMOs. They would literally be GMO babies.”
That was a disturbing idea if I’d ever heard one, I thought. Of course, what I didn’t know at the time was that human germline genetic modification was about to burst onto the scene literally a few weeks later, and that it would be far more extreme than the three-person IVF technique. Considering that the room was full of a good chunk of the top scientific experts in the stem cell field, I really think that somebody had to know something about the gene editing technology going on elsewhere at that very moment. Still, I’m not so sure that too many of them understood that it was about to hit the field with the force of a nuclear-powered runaway train.
But to get back to the FDA. Paul stated that a faster review process really was necessary. In terms of the way that a drug would normally go through the process, there were a couple of options for expedited technique. These included fast track, accelerated approval, breakthrough designation (no stem cell therapy has had that yet), and priority review. It wasn’t so easy to tell the difference between the four.
In the bench to FDA to bedside track, compassionate use spanned the three… theoretically, at least. The reality was that stem cell clinics were currently hopping over the process and illegally inserting themselves at the end. The FDA might actually crack down on them at some point, but it hadn’t really done it yet. Baby steps had been taken in the form of new draft guidelines that applied to what stem cell clinics were doing. For instance, the FDA had ruled that adipose tissue was a drug, and so the same surgical procedure or minimal manipulation exceptions did not apply (remember that liposuction stem cell technique?) Places like Cell Surgical Network( the franchising chain in the U.S.,) exclusively used the SVF fat stem cell product to offer therapy for more than a dozen conditions. None of them had FDA approval of any kind, Gordie Howe being a good example.
I was struck by the fact that every researcher and scientist over the past two days had spoken about illegal stem cell clinics.
“But the FDA itself has some issues right now,” said Paul Knoepfler. “There have been some real criticisms that it’s not reporting known research misconduct to anyone in the medical community. Overall, yes, reforms are needed. The FDA needs to be more clear and open, especially with research communities.”
(Here’s the cite showing the FDA’s knowledge of widespread research misconduct. It’s actually an article in the Journal of Internal Medicine: “Research Misconduct Identified by the US FDA.”
And here’s the evidence that the FDA knows about it and isn’t telling.)
It was getting close to lunchtime by then. This had been a day of academics, and in all honesty, I was starting to get a little restless with them. I couldn’t help thinking that almost none of them had a personal stake in any of this. Understand, I wasn’t being completely fair, and I knew it. Maybe the subject had come up in my mind because Paul Knoepfler was the last speaker, and I knew that he’d survived prostate cancer, that he’d crossed over that line and come back. I didn’t think that any of the other academic speakers could say the same thing. Not to say that they didn’t have passion for what they were doing, but there was no way around the fact that, as Dr. Freischlag had said, you became a different person when you crossed over into patient-land. As vicious and crazy as the fights for grants and publication could be, there was still something essentially intellectual about it all, nothing raw and real that cut to the hearts of these people. Where were those patients reps I’d heard about?
The question was answered pretty fast.
The bouncy-ball man in front of me rose from his seat and strutted up to the podium. I had to smile. He seemed so full of self-confidence, taking so much joy in the simple act of walking, as if every step were a miracle to him. It didn’t take long to find out why.
TOMORROW… The identity of the mystery man, revealed at last!! Stay tuned for his incredible story.
I survived the disease, and I now have the only remission on record. But a remission is not a cure. The stem cell drug I need for a real cure is now rapidly moving towards approval in the rest of the world, but in the USA, it—and all other cellular therapies-- are still stuck behind prejudice, ignorance, and lack of funding. Hundreds of millions of desperate patients with incurable diseases need these drugs.
That’s why I’ve gone on to fight for greater public education on stem cell drugs, knowing that our laws must be changed so that all of us can get access to the best treatments instead of our health and our lives being held hostage by special interests.
I’m now a patient advocate whose work on stem cells and patients’ rights has been published in outlets such as the Oregonian. I am a manager at popular science and financial blog www.stemcellinvestor.com and a frequent speaker at many venues across the spectrum, including churches, scientific conferences, and atheist groups, and everything in between. I’m also an advocate for Right to Try laws that would allow access to experimental medication for terminal patients at the state level. Read the entire story in my upcoming book, And the Blind Shall See: A Skeptic Patient Surprised by Faith, Science, Family, and Miracle Cures.
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