Saturday, March 28, 2009

Clinical Trials and the End Game

Clinical trials of new treatments are of the utmost importance. Without them, doctors would still be bleeding us for the ague. A proper clinical trial provides a bit of unshakable truth in the struggle against cancer. Participation in clinical trials is often presented to the newly diagnosed as if it were a routine or natural thing to do. There are even those who will suggest that there is a duty to science or humanity to be part of a process that might lead to cures for presently fatal diseases.

If Christopher Columbus had said, “Oh, I don't think I will try to discover a New World because the voyage will be dangerous and we may not make it,” he never would have had a day named after him!!!!!!!!!
— A fellow myeloma patient
Is having a day named after you worth your untimely death? I strongly recommend that most patients stay the hell away from clinical trials.

I believe that volunteers in the best sense of the word participate in clinical trials as a way of doing good. While I admire the motive, I think they are nuts to do it. Let some other, more desperate person take the pill and the associated risk. It isn't the patient's job to advance the cause of medical research; his job is to fight his own cancer as effectively and courageously as he can.

"No bastard ever won a war by dying for his country. He won it by making the other poor dumb bastard die for his country.”
— Attributed to General George Patton
If someone is in reasonable physical shape, has recently been diagnosed or is responding to routine first-line therapies, and, therefore, the disease is not progressing out of control, then one doesn't actually need to participate in clinical trials and, therefore, shouldn’t.

You may have read of a relatively-recent clinical trial of a genetic-level cure that in a matter of minutes destroyed the livers of the participants, a problem that did not show up in animal testing. I personally had a friend who, although an excellent candidate for a conventional transplant (listen to me call the most punishing procedure in all of medicine “conventional”), saw the seductive promise of cure in a trial of Holmium-16.

The idea was that a small dose of Holmium, a naturally-radioactive substance, would home in on the marrow, help Melphalan kill the old marrow by radiation, and then be flushed from the body in less than an hour, making it harmless. If transplants failed because of incomplete marrow ablation, with Holmium there was the promise of cure. Cure, so achingly seductive, so necessary!

She died a few months later. Actually, her bones died first from the “small” dose radiation. As someone who has studied delayed radiation in Hiroshima and Nagasaki, I should have remembered that there is no established dose of such substances low enough to be considered safe. I too was blinded by the promise of cure. I encouraged her. I feel guilty about doing so to this day; she might otherwise still be alive. I am.

Yet there is a time to consider participation in clinical trials. For me it came with the End Game milestone. Participation makes sense when you are clinically desperate. If a patient has relapsed after responding to one of our usually-reliable therapies or become refractory (failed on more than one) or is demonstrably belongs to a very high risk category—and the remaining choices are unlikely to stabilize the disease for any meaningful length of time—then looking for a promising clinical trial is better than looking for a hospice.

The downside of this high-level of prudence is that many early clinical trials only accept volunteers who are recently diagnosed and/or previously untreated. Therefore many that seem promising will not be open. However, new treatments seldom come from these early clinical trials. There are always more advanced trials, where the risks and benefits are better known, and others designated for the relapsed/refractory category (or are open to all). Here is a link to the national data base of clinical trials.

In my opinion, fear (or its more handsome twin, hope) is the biggest risk to us, whether we are among the newly diagnosed or the veterans. With a handful of exceptions, multiple myeloma is incurable and universally fatal. This fact cannot be wished away. Fear of death can drive us toward any promise of escape, however foolish: inadvisable gambles seem attractive; denial stops decision making because there is no need for it; fortunes are spent on "alternative" treatments; the good in present life is degraded by obsessing on the future bad. This blog is indirectly devoted to providing tools to help neutralize anxiety, regardless of the kind of cancer you may be fighting, so it never drives or degrades treatment decisions. All of my big medical mistakes have stemmed from yielding, consciously or unconsciously, to fear.


  1. Lon--

    Absolutely agreed about the alleged duty to further medical science! One other population for whom trials make sense, though: those for whom established and appropriate treatments are unavailable for financial reasons (e.g., government rationing or lack of insurance).

    Thank you very much for this blog (which I only recently discovered). I will read it faithfully as my wife does battle with her plasma-cell leukemia (a rare, aggressive, variant of MM). And I will be thinking of you and wishing you the very best.

    One trivial request: Any chance you could slow down the animation of that fascinating chess game? I can't quite take it in at its current speed...

    Craig Stinson

    1. Finally, years later, I can reply! I don't know how to slow it down but I can take a look at the HTML to see if there's something there I can change....

  2. You are right about the additional population, much to our national shame. Hopefully, some kind of universal health care will be available to all Americans soon.

  3. Lon -- like the rest of the general population, I thought the phrase "clinical trial" meant the newest, the best information, offering the greatest hope. As I have been following various MM blogs, yours included, I have become increasingly certain that clinical trials are just advanced rat studies. What disturbs me the most is that when the 'victim' is at his or her most vulnerable, having just heard the diagnosis and extraordinarily fearful of what lies ahead, that is when some soft-spoken, very good salesperson, quietly says, "We can probably get you into a clinical trial that will offer the most hope for your situation."
    And I know someone who has been convinced (first diagnosis) that this is the best route... and so far, I am disturbed by the results.
    As long as insurance won't pay for everything, as long as people who are scared and scrambling for financial support of their decisions are being driven into potentially dead-end canyons of clinical trials, there will be 'rats' for their experiments.
    So I sound angry. I am. And because I am not the direct 'next-of-kin' in this situation, I have no voice - had no voice that could be heard in the beginning when I suggested doing more research on places outside the family's existing medical exposure, however renowned it appeared to be. I don't propose to know how to solve the 'clinical trial carrot' but the medical field is complicit with the researchers, not separated from them.

  4. Sandy, you are right on the mark. I've had to resurrect this post for a friend's mom. I suspect the standard treatment would be likely to succeed, but the lure of something more (I don't know yet what, keeping the breast?) is pulling the woman toward the trial.


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