So… we’re done with the raw notes for now, and we’re back to the writing! Believe me, you do NOT want to miss the info in today’s installment. If you’ve ever wondered whether or not you should keep your investments in a certain biotech company, for instance… the answer’s here. Enjoy!
Dr. Nanette Joyce stood up to speak. The title of her talk certainly caught my attention right away. Stories from the Trenches: 365 Days In the Life of a Doctor.
Apparently, she’d won a CIRM stem cell grant to develop an ALS mESC treatment. The procedure altered cells so that they produced a protein which helped ALS in some important way. As part of the grant, she’d had to outline an ethics plan, and I wondered if it was because she worked with terminal patients.
She also talked about stem cell tourism, but she underlined a fact that Tim Caulfield hadn’t really addressed: more and more people were going to clinics right in the U.S. Most were in California; one in Roseville was actually approved by a UC-Davis doctor. That struck me as a pretty horrifying fact. Dr. Joyce’s alma mater college had sent out a program to her including a workshop on regenerative medicine, options for osteoarthritis, and how to use unproven therapies. Oh, dear God, I thought.
Stem cell clinics almost all seemed to be using a technique that sounded a lot like the punchline of a bad joke about weight loss. Fat was liposuctioned out of a patient and put through some kind of test-tube process that I totally admit went right over my head. The MSC cell was neatly extracted (remember how 1% of all the cells in the human body are actually adult stem cells?) Then it was… well… put back in. This could be done in a variety of ways, but the one I knew I would never forget was the inhaler I pictured it. Fat liposuctioned out of your butt, liquefied, and the stem cell pellet extracted, put into an inhaler, and then you stuck the end in your mouth and took a big whiff. (And if you mixed it up with your asthma medication, God only knew where it would all end.) Those originating- from-your-butt stem cells then somehow magically knew where to go in order to offer any of the zillion or so cures that had been promised. It sounded like a bizarre parody of stem cell therapy. And yet… and yet, even a parody is always based on something real.
My mind was still spinning on that one when Alison Sorkin took the stand. She worked at the University of Colorado hospital and discussed the recent Right to Try law that had passed the year before in Colorado, one of six states as of February 2015. Like all the rest, it proclaimed that patients now had the right to use unapproved drugs that had passed FDA’s Phase I, which only established safety, not efficacy. In other words, a study trying out aspirin for colon cancer could pass Phase I. There’s nothing dangerous about it, but the question of whether or not it works is a very different one indeed. What really raised the flags for me was the fact that nobody was obligated to pay for anything except for the patient.
There was zero requirement for any insurance company to cover anything, which I guessed was natural enough, but the next provision was the real stunner. Insurance wouldn’t be required to pay for any condition– related to the treatment or not– for six months after the experimental treatment was tried. Typical of those vultures, I thought, not feeling very guilty about not being very charitable towards insurance companies after all of my ordeals with Humana.
The key, however, was that although Right to Try was a legislative declaration which had passed unanimously, it did not trump federal law. Alison correctly pointed out that because of the rather insanely lengthy list of exclusions and eligibility, exactly zero patients had as yet qualified for anything at all under Colorado’s law. But exactly one day after the RTT law had passed the Colorado legislature and gone into effect, the calls had started pouring in.
The most memorable had been the man who called and announced that he was on his way from Pueblo to pick up experimental medication for his mother. No, he couldn’t bring her in. He’d just pick up the med, thank you very much. When Alison tried to gently explain that it just didn’t work that way, he apparently grew quite irate and insisted that the law was supposed to guarantee that anyone could just waltz in and snatch up any experimental drug that happened to be lying around. The phone call didn’t end well, from what I could infer.
A lot of people in the room smiled and shook their heads, but I had to wonder if it was really all that funny; there was something almost mean about being so amused. How were patients and their relatives supposed to know what was and wasn’t possible when it was so difficult to learn anything new? I could remember my own struggles for information all too well. She herself said that nobody who called knew anything about the existing compassionate use laws, which was they often ended up actually using to get experimental drugs. Why blame people for ignorance when it was so hard to be anything but ignorant?
As skeptical as I was when it came to Right to Try, I knew, knew in my bones, why patients were grasping at that desperate straw, just as I understood why people with terminal illnesses went to dubious stem cell clinics.
I looked down at the day’s schedule and saw that Paul Knoepfler would speak next. Paul has to be the most qualified scientist writing a stem cell blog today, and he’s kept it up since 2010. He has never shied away from unpopular topics, and he’s frequently gone against the crowd to point out problems in stem cell techniques that everyone else was uncritically accepting. He was the first scientist in the world to raise serious questions about the STAP scandal in Japan, back when everyone else was falling over themselves to provide fawning coverage. He’s written a prediction post at the end of every year for the biggest developments in stem cell research and development in the next year, and he has very rarely been wrong. Virtually everything he’s ever predicted has come true. For the stem cell investor crowd: Paul Knoepfler invests in one biotech company. Exactly one. Guess which one it is?
Do I hear “Ocata”?
We have a winner!
In fact, the only area of disagreement I’ve ever really had with him is that I think he’s too uncritical when it comes to the subject of iPSC clinical trials in Japan under Dr. Takahashi. But a huge part of the reason why I have that opinion is that Dr. Knoepfler himself was so critical of these trials rushing to the clinical phase too soon, and that he had this point of view for such a long time. I never really saw any convincing reason for him to do a 180 on the topic, which is why I could never support the frantic rush to the clinic with insufficiently tested iPSC cells, which is what has ended up happening. But I never would have come to that conclusion in the first place without the evidence that was published on Paul Knoepfler’s blog.
His topic was listed as: FDA Approval of Stem Cell Therapies: Does It Really Matter? I already knew that the answer would undoubtedly turn out to be yes; that wasn’t the first rhetorical question posed during the conference. But the question was why. I knew that whatever reason Paul Knoepfler had would turn out to be important.
To be continued….
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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