Wednesday, December 9, 2009

My MS Story Chapter 21- A good friend turns into a cold hearted sonofabitch.

Progression types of Multiple sclerosis
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There are two obstacles to finding a treatment for Primary Progressive Multiple Sclerosis (PPMS). First, it appears that nothing works. Second, even if something were to work, I doubt that the scientists would notice it.

Here’s the problem. With the more common form of MS, Relapsing Remitting Multiple Sclerosis (RRMS), there are some clinical measurements that correlate with disease activity: the number of enhancing lesions measured by MRI and the number of relapses experienced over a given period of time. Because people with PPMS don't have enhancing lesions and don't have relapses, all we are left with is trying to quantify the change in overall level of disability. This is accomplished by measuring patients’ ability to perform certain tasks, as I described in My MS Story #19.

It's much more challenging, from a statistical analysis point of view, to draw conclusions from these PPMS tests than from the RRMS tests. To make matters worse, I don't think they were even measuring the correct tasks during my trial.

When I was participating in the clinical trial I never had any vision or cognitive problems, so measuring those types of tasks was meaningless for me. I didn’t have any disability in my hands during the trial, so the test where I placed pegs in holes didn't yield any interesting results. The only part of my body that was affected at that time was my legs. Even then, the tests that they did on my legs didn’t actually measure what was wrong with them.

If I came to the doctor’s office using my scooter, so that my legs were not tired, I demonstrated near normal strength in all my leg muscles. I could walk across the examining room, albeit a bit like a drunk. That's how they tested me- always fresh and well rested (no, they didn’t test me drunk). However, at this point in my disease progression the defining characteristic of my disability was not how strong my legs were when I was well rested, but rather how quickly my leg strength deteriorated during exertion.

These are some of the reasons why I wonder if researchers would notice an effective treatment for PPMS, even if it was staring them in the face. PPMS progression is just so difficult to measure.

Throughout the trial my “official” performance data on the various tests indicated that the disease was no longer progressing. In fact, on good days, I was supposedly getting ever so slightly better. For the first year or so, I agreed with this assessment.

Unfortunately, in the summer of 2006, a little over a year into the two-year trial, I noticed that my legs were becoming fatigued more quickly than they used to. But still this change in my condition did not show up in the trial data. At work and at home I started using two crutches all the time, instead of sometimes one and sometimes two. I was just too unstable on one crutch. This indicated definite disease progression, but still at a much slower pace than before the trial.

At this point I had received three of my four courses of Rituxan (six of my eight infusions). In November of 2006 I was scheduled for my last pair of infusions. Both the doctor and I considered this trial to be a success for me personally. We’d have to wait a while to see if the larger population of trial participants also benefited. By his measures my progression had stopped. By my measures it had slowed down significantly. But either way, the trial had been worth it for me.  Naturally, I began to wonder about how to transition from getting Rituxan in the trial to getting Rituxan outside the trial, ideally without skipping a beat.

I was very pleased with my trial experience. The doctors, the staff, and the facilities were first rate.  I was lucky enough to be receiving the trial drug instead of the placebo (a correct assumption as it turned out). These clinical trials, I thought, are a good thing both for the participants and for medical science in general. The results, although imperfect, were much better than anything else I had ever tried. The trial had become like an old friend to me.  I didn’t want the relationship to end.

But soon enough I would learn that clinical trials can be an uncaring, coldhearted sonofabitch.

For the next installment in this series, click here.
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  1. Is it possible, too, that PPMS is an entirely different illness that RRMS? If so, the testing paradigm might be inherently flawed.

  2. Hi Mitch

    Just read your experience of the drug trial. Thanks for posting it very informative


  3. Judy,

    Thanks for your comment.

    You may be right. What we call two strains of one disease may actually be two separate diseases. I guess there are semantics at work here. What criteria determines if two conditions are to be considered separate diseases or not? I'd say two things: do they have the same cause and do they have a similar pathology?

    Since we don't know the root causes of either PPMS or RRMS, this question cannot be answered today. How about pathology? Well, that's a tough one too. It's not like all RRMS people have a similar disease course, or all PPMS people follow a common recipe for progression. It's almost like everyone's disease course is unique. It's almost like there are a million diseases all referred to by the name of "MS".

    But from one point of view, I do think of PPMS as its own disease- everything that works for RRMS does not work for PPMS. I have to deal every day with the fact that the level of optimism associated with RRMS (which is not high, but much higher than zero) does not apply to me.

  4. Andrew,

    Thanks for stopping by and leaving a comment. I'll get to the story about what went wrong at the end of my trial soon. It was quite a learning experience for me.