Navigating the Upside Down

Being Yr Guide to clinical trials, experimental treatment, and how to make informed decisions when there is no real information

The other day (yesterday, it was yesterday), I mentioned to several friends, in various places, what life was like for cancer patients prior to Roe v Wade establishing bodily autonomy and patients’ rights to informed consent. You may not be familiar with cancer survival prior to the 70’s. That’s largely because it didn’t exist, but I’m sure that’s a total coincidence and the two points in history are unrelate. So, we all used to go to Mexico for treatment. I should point out that wasn’t some sort of informed, preferred choice, it was sheer desperation. We went to foreign countries because physicians in this country felt no financial or political pressure to treat us competently. The moron I was discussing this with likened my participating in a clinical trial for a repurposed chemo agent with him taking horse paste. For some reason, when I pointed out that he was taking veterinary meds, he bristled. I don’t think he’ll appreciate the mail-order hay I’ve sent. However, it does kind of bring to the fore the incredible lack of scientific literacy in the American public that equates “experimental” or “unproven” treatment with “witchcraft and Joe Rogan.” BTW, if you’re in that latter group, call me, because I have some great “Male enhancement” pills that I’m selling at a steep discount.

So, when you have a rare condition and the science and medicine are a few decades behind you, how do you navigate a world where the pseudoscience, quackery, and emerging science/data start to blur together? Well, here are some fast rules I used.

1. Experimental treatment is absolutely no substitute for established medicine, it’s only an acceptable substitute when the alternatives are “thoughts and prayers.” As I pointed out, if the only possible treatment for COVID was horse medication (again, if that label bothers you, facts don’t care about your feelings, and you should feel free to seek medical attention that isn’t in a barn). When I got into treatment, overall five-year survival for ATRX mutants like me was 50% (that statistic cited by my first neurooncologist, and it might be outdated). When I started treatment with my current oncologists (lovingly dubbed, “The Warlocks), they told me that I would absolutely receive Standard of Care for glioblastoma (that’s 6 weeks of chemoradiation, followed by 48 weeks of on-again, off-again oral chemo), HOWEVER, if I was interested, my pathology and tumor markers made me a candidate for additional, experimental treatment. When it comes to an incurable, rare cancer, your primary treatment choice is, “More treatment, please.” There are a ton of moving parts involved in participating in a clinical trial, so I can’t really point to one and say, “That’s it. The single thing that got me to the 54-month mark,” but I can conclude that there was nothing I did that was fatal. I encounter a lot of people under the delusion that experimental treatment is an acceptable alternative to standard care — I made that one, as well, when my glioma was a minor recurrence. Any time there is an established treatment protocol, that should be incorporated into the experimental treatment. This is especially true with cancers, because they can progress past the point where any competent oncologist would recommend treatment (at which point, you’re back at Cabo with margaritas and back-room medicine). As an aside, I’ve head of glioma patients who think the COC protocol can be used as a “natural alternative” to chemoradiation, when, right on the front page, the protocol tells you that it’s to be used WITH standard chemoradiation.
2. There’s actual, peer-reviewed science to back up claims. This is the critical point my horse pill lovin’ friend made. “Scientific evidence” is not “A Youtube Channel where a schlubby white guy in Oakleys rambles about lizard people.” It’s peer-reviewed studies in scientific journals. My broad, general rule is, you should be able to find this (or supporting evidence) on Pubmed or Clinicaltrials.gov. In the case of my chemo, there were numerous tests in animal models showing it demonstrably increased radiotherapy effectiveness. And it was a registered trial with the FDA. In the case of neurofeedback (a specialized form of biofeedback that I used infrequently to recover from previous neurosurgeries), my provider was a licensed psychologist, and he had a lot of preclinical data correlating forms of his treatment with neurological recovery. It convinced me, at the time, however, he hasn’t kept up with FDA licensing and testing (and, hey, clinical trials are expensive, but I’m not going to risk my well-being and life on a treatment that isn’t updated with further data and testing), so, I’ve quietly stopped looking into that as a recovery tool.
3. This is being administered in a formal, controlled environment. The very weird guy defending horse medication tried to frame his choice of apple-flavored medication (no, I’m not going to back down on that because, again, if you’re getting your health regimen from a farm supply store, you’re an idiot who deserves scorn and chronic pulmonary issues) as an experimental treatment. To be fair, I can’t argue with that, but it’s an experiment in exactly the same way that “Are rocks magnetic” at a middle school science fair is an experiment. It’s not going to prove much, and there are a ton of variables (I absolutely support the conclusion, “An excellent large animal vet and never being exposed to COVID” is correlated with not getting COVID, but, that doesn’t mean much). When I was in the clinical trial, I was one of 70-odd patients there; I was in the center during treatment and for an hour or two post-treatment so no life-threatening acute side effects hit me. And, for the day or two after, I adhered to a set of (admittedly broad and negotiable) behavioral guidelines my oncologists and I agreed to (no drinking, no non-pharmaceutical drugs that they didn’t know about, no unmentioned chemo or immunotherapy). This is different than, “I woke up in the morning, gargled with horse paste, lived my life in an under-reported, unaccountable fashion, and chose to believe it made me better.” And, let me be clear, this sort of loose, “anything goes, we’re just havin’ fun with cancer” approach is the nightmare future I’m trying to avoid by endorsing bodily autonomy and informed consent for everyone.
4. The numbers back it up. I recently had a very disturbing series of exchanges on Instagram with a Spanish glioma survivor who was convinced that a clinical trial in German was just the thing. Her source was three other people on IG who claimed to have used this treatment. I haven’t heard back from her after I pointed out that I could readily find three people who were misdiagnosed and got famous on it. In the Clinical Trial I participated in, there were 71 other patients (I was #72), and there were portions of the clinical trial that had completed. In the case of neurofeedback, I tracked down several other people who’d been successfully treated, and asked my GP about it (he didn’t exactly endorse it, but neurological recovery is such a rare thing that you’re operating more like an investigative journalist than a researcher, so, there wasn’t a whole lot to deter me).
5. The science is successful enough to warrant further testing and investigation. I’m not big on the idea of capitalism and completely free markets, because they have, historically, underserved orphan diseases and drugs. However, when you see the price tag attached to running a clinical trial (something like seven million dollars just for each phase of the trial), you know there’s enormous economic pressure to write off the cost and petition for a tax rebate. So, whenever you see that a treatment has advanced in testing, or similar experiments are receiving funding, it’s a good bet that someone in a suit has crunched the numbers, and decided this project is worth a few more million dollars. Which means it’s probably a decent bet for you, the patient. Again, what kind of turned my thinking on neurofeedback was that specialist didn’t fund or conduct further research on his program, so, even he thought it wouldn’t be profitable. Marizomib was in Phase 3 testing in the EU, last time I checked (actually, that’s a great idea, because most European regulatory apparatus have more-stringent standards than US regulatory agencies, so it will be legally safe and effective everywhere), so, Pfizer thought it was worth pursuing at least that far. As far as I know, no one is funding a trial to repurpose Ivermectin (although, it is on the WHO’s list of essential medications for antihelminth infections; if you have worms, it’s just the thing). As Deep Throat told us, “Follow the money.”

And, in closing, I’d like to remind everyone of what I was told in the first month of grad school, “First-time-published data/findings are the least reliable, then secondary studies that correlate or repeat those findings, then tertiary reviews of literature, then textbooks.” The downside, as they also pointed out, is that, by the time a textbook or review is ready for publishing, a fair amount of that data might be obsolete or misleading. In surviving a rare cancer, there are no textbooks or reviews of literature available in your projected life expectancy, so, I used primary findings, secondary findings, and anecdotal evidence — that’s the guy with a Youtube channel. I’d point out that I approached anecdotal evidence just like an investigative reporter or intelligence analyst, and looked for multiple confirmations or similarities in everyone’s stories before I acted on it.

Nothing in life is to be feared, it is only to be understood. Now is the time to understand more, so that we may fear less. — Marie Curie