Monday, April 7, 2014

Week 33: Lessons learned

One lesson that I've learned since starting my new job is that it is almost always worth going to Grand Rounds.  Even if the title doesn't particularly strike me, there's inevitably some nugget of information that I find useful in thinking about our campaign against CF.  Take last week, for instance--a talk about the history of bone marrow transplant.  How could that be relevant?

Well, it turns out that women with life-threatening breast cancer are part of a vocal, determined, and proactive group of patients, loved ones, and caregivers--much like the CF community.  And, like the CF community, they are desperate for new treatments that may keep them alive just a little bit longer.  In the late 1980's and early 1990's, such a treatment seemed to have emerged: massively high doses of chemotherapy followed by a bone marrow transplant to replace the essential blood-forming cells that were also killed by the chemo.  Patients demanded the treatment, which was presumed to be better than anything else available, and sued their insurance companies to cover the cost even though it was still considered "experimental."  Some insurance companies eventually began to cover the procedure rather than risk lawsuits.

As it turned out, this treatment (which was horrible for patients to endure and expensive to boot) was actually no better at prolonging survival than the standard of care at the time.  The first studies on the treatment had been conducted without controls, so there was no way of knowing if the new treatment was better than the standard of care. And, when the proper trials were finally run, it was apparent that the new treatment was no better than the old, and it gradually fell out of use.  But, this all unfolded over the course of about a decade, during which many millions of dollars were spent on the procedure, and patients were subjected to awful treatments that in all likelihood did them not much good.

All of this makes me think about the use of new, experimental therapies for CF and its complications.  It will be no surprise to all of you that I am a huge fan of research, and am so hopeful that in time science will yield the answers we need to cure this disease.  But, how much time is reasonable?  I have a child who has the disease NOW.  A phase I trial that won't have results until 2018 seems way too slow, especially since I am listening to my poor kid cough and cough as I type this.  If something new is available that could help, shouldn't he be able to have access to it right away?  But, on the other hand, without careful studies, how would I know that a new treatment was any better than what I already have?  Or, for that matter, worse?  It's a conundrum. 

To me, the lesson from our fellow travelers in the breast cancer sphere is that it may be wiser to use our voices, our determination, and our dollars to push aggressively for research studies, to encourage patients to enroll, to encourage the FDA to consider intermediate endpoints along the way rather than waiting until the very end of the study to determine whether a new treatment shows enough promise to be made available to the community.  Hard as it is to be even a little bit patient, I hope the CF community will manage to avoid the trap that the breast cancer community found itself in 20 years ago.

Well, this post turned out a little darker than I intended--since Lemon has a bad cough right now I think I am in a gloomier state of mind than usual.  So, to end on a lighter note, although Lemon can neither crawl nor walk just yet, the kid can dance!

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