The basic story is this. A boy, now 19, suffers from bone cancer (described in the article as "osteosarcosis", but I'm virtually certain they mean osteosarcoma, and Google suggests the same substitution). From the description, it is clearly metastatic and like many cases of metastatic cancer it is failing to respond to chemotherapy or radiation. The prognosis for such patients is grim. The family has racked up $250K in "medical bills", and a reality TV star had publicized their crisis. Someone stepped forward claiming they would cover the bills, but it turned out to be a hoax apparently aimed at meeting the reality TV star. All in all, a depressing story of how some people have no scruples when it comes to pseudo-celebrity.
But, how did the family rack up the $250K? Well, the article hints that much of cost was 'alternative therapy'. And while it doesn't detail everything, the family's blog (referenced in the article) describes needing $40K for two weeks at a 'cancer center', whose website touts therapies around modifying your body's pH.
Which is, a sad vein mined by quacks and not serious biologists. I've had a lot of courses, attended more than a few seminars and have read stacks and stacks of papers, and have yet to hear any serious scientist pursue such a theory in cancer. I'll be the first to admit that what I don't know about the biology of cancer far outweighs what I do know, and that the field is still sometimes grasping for answers. But, that doesn't excuse putting a bunch of scientific-sounding vocabulary around some far-out idea with no hard evidence to back it up.
Of course, those who believe in such therapies are quick to tout 'evidence', but unfortunately to anyone with a scientific training it simply doesn't count. A key tactic is offering testimonials, but of course these are simply anecdotes with a horrendous reporting bias; those for whom the 'therapy' has failed rarely give their side. If pharma companies could launch products based only on Phase I anecdotes, there would be a lot more drugs on the market; seemingly every oncology drug that fails in Phase II had at least one strong responder in Phase I; perhaps in some cases we'll figure out a rationale for it but with the numbers so small, such cases will rarely be convincingly solved.
I must confess that I've generally steered away from pseudoscience and quackery in this space. It's not that I have any tolerance for it; it's simply that it's too stressful and exhausting to deal with. Most people who believe in irrational ideas are well trained in defending them; to properly address their claims one must do a lot of research. It's also hard to discuss real science with them, because the sorts of arguments that they aren't prepared to deal with involve all sorts of important technical details that are challenging to explain clearly. If you botch an attempt, you look silly and undermine your credibility, which perversely raises your attackers credibility. And for me, the worst part is it all puts me in a foul mood. So, for my own sanity I generally don't poke these hornets' nests. But, this time I felt compelled.
If you go to the website for the 'cancer center' and discard the fancy wordage, what you are really left with is a form of palliative care. I can certainly believe that a desperate patient racked with pain could benefit psychologically from massages and a lot of positive attention. As a parent myself, I can only begin to imagine what straws I might grasp at if I had the same horrible situation. But for $40K for two weeks? Yikes!!! It's amazing all the vitriol that is dumped on the biopharma industry for charging similar amounts for a year for a product which has undergone extensive (and expensive) clinical testing, and which involved a bunch of people staking the project with huge sums. It's also amazing the vitriol dumped on biopharma for their profits; if this spa can average an occupancy of 12 patients, that's a cool $1M per month. For far less than $20K a week, a patient could have regular massage treatments locally.
Perhaps what makes me sadder is that it is the continuing limited success of evidence-based medicine to address cancer. Cancer, particularly when metastatic, is a difficult beast and in only a few rare cases have cures really been possible. In many of cases, months or years of time can be purchased for patients, albeit with steep side effects. Alas, there are far too many cancers in which current therapies barely budge the dial. Given this, the allure offered by quacks of certain cure, no side effects and the rest could be awfully enticing. It's easy to tout that your treatments have no side effects; unfortunately too many people are not suspicious that something with no side effects may simply be biologically inert.
When I left Infinity almost exactly a year ago, one of my regrets is that I was leaving an exciting clinical program called IPI-926 (aka saridegib), an inhibitor of the hedgehog pathway. Here was an opportunity to participate in moving an entire new class of compounds into the clinic (though Genentech was ahead in some areas) with possible application to a wide range of cancers. Indeed, sarcomas were under study, with much in vitro, in vivo and 'Phase 0' work suggesting a role for hedgehog signalling in sarcomas. One of the three trials Infinity had underway was in chondrosarcoma, and osteosarcoma had been explored as well (in dogs!). There were also hints from the literature of the possibility of distinct genomics alterations in these tumors, spurring my notions for pharmacogenetic experiments.
Alas, after my departure things went very badly for this program. First the pancreatic adenocarcinoma trial was stopped after an interim analysis was sour. Then, the chondrosarcoma trial also had a disappointing interim analysis and was also halted, with the entire program being shut down. Well-designed clinical trials are a brutal standard, and unfortunately IPI-926 did not pass. That does not mean it might never pass or that hedgehog pathway inhibition can't be used successfully in oncology (indeed, Genentech has approval for their compound in certain rare cancers). But for now, it turned out to be too risky a bet for a company the size of Infinity. Sadder still, it means that yet another dry well has been drilled in several cancers for which there simply are no good therapies. Yet another hope for delivering hope to patients has been thwarted by our still fuzzy understanding of the biology of cancer.
That's the sort of news that is routine in the pharmaceutical industry, but sorely lacking elsewhere. In medicine, if you haven't failed then you truly aren't trying hard enough -- at least not trying hard enough to determine if you've failed. Perhaps the worst taught lesson in school science classes is this: one of the easiest things to do in life is to fool yourself. Good scientific enterprises bend over backwards to detect self-deception, and if you're not looking for it you are almost certain to fall for it. Particularly, when there are emotions involved.
Hi Dr Robison
It seems like there is a growing consensus that cancer should be treated with a cocktail of targeted therapy drugs that are tailored to the omics profile of one's tumor cells. What do you think about this?
It seems to me the personalized nature of this kind of therapy runs against the current FDA's one-drug-at-a-time approval process. What can we do about this?
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