Thursday, November 5, 2009

My MS Story Chapter 16- The Clinical Trial Begins

(Photo credit: jeff_golden)
In early 2005 I was accepted into the Rituxan clinical trial, or so I was told. I kept waiting for word on when I should show up in Worcester for my first appointment, but the months kept going by. The start of the trial was perpetually imminent.

Finally, in May of 2005 I got the call instructing me to report to the UMass Memorial Hospital in Worcester, Massachusetts for my first appointment. I was excited about the trial, but not in the way that some people get excited about prospects like this. Let me explain. Oftentimes, when people who are in dire straits see a rare opportunity, they invest emotionally in a positive outcome. They seize hope and hold on tightly.

“I know this will work.”

“Fate has brought me here. Everything happens for a reason.”

“This is the answer to my prayers.”

I'm not finding fault with people who operate in this manner. It's just not me. I had a modest level of optimism because the pharmaceutical company was investing a lot of money in this trial. Some project manager or product manager or vice president had stuck his neck out to advocate for this very expensive experiment. The fact that a board of directors was allowing the expenditure of precious corporate assets made me feel that this trial had a better than average chance of success.

I was enthusiastic and cautiously optimistic, but I knew that no trial in the history of clinical trials had ever produced a treatment for PPMS.

The protocol for this trial was double-blind, placebo-controlled. The placebo-controlled part, at least in this case, meant that two thirds of the participants would receive Rituxan and one third would receive a placebo. The double-blind part meant that both the patient and the treating physician (and his/her staff) were blind to whether the patient was receiving Rituxan or a placebo. Only a few nameless, faceless (at least to me) data analysts would know who was who.

Each trial participant was making a 2½ year commitment that we would not undergo any other MS treatments and that we would show up in Worcester between one and four times a month for treatments and examinations. For me, it was a two hour drive to the trial hospital, each way. This was a huge commitment.

It bothered me that I might forego all other treatment options, as unpromising as they might have been, while receiving nothing but a placebo for 2½ years. This was a frightening prospect, especially since I had a progressive disease. Left untreated, I could only guess what my condition would be 2½ years down the road. Those of us who participate in clinical trials are often heralded for our unselfishness. We risk our health for the cause of science, but let's examine that concept a little more closely.

If I knew that a certain placebo-controlled clinical trial would produce results beneficial to mankind, then that would be one deeply personal decision for me to make. However, in the clinical trial world it is very unlikely that patients’ sacrifices will contribute to significant medical progress. Most clinical trials fail, especially clinical trials for PPMS. Scientists are quick to point out that even failed trials yield important scientific and medical information. Okay, I'll concede that point, sort of.

My health and well-being may be worth sacrificing for significant medical progress. However, there is no way that my health is worth sacrificing for tidbits of scientific information that may or may not ever make a difference to anyone. Furthermore, my health is clearly not worth sacrificing simply for the financial gains of a pharmaceutical company.

But evaluating all of this from the front end of a trial is daunting. You don’t know anything. You don’t know if this trial could be THE trial. You can’t possibly know if you’re making a huge mistake. You just can’t anticipate what your world will look like 2½ years down the road, with or without the trial.

There was one fact that made my decision to go forward easier, though. I simply had no better option.

So I came up with a plan. I decided to break the rules and make every reasonable effort to ascertain whether I was on the placebo or the trial drug. If I determined that I was actually getting Rituxan, I would stick with the trial until the end. If I determined that I was on the placebo, that would be another story. I would secretly reserve the right to withdraw from the trial if I felt that I was missing out on some other opportunity to fight the disease.  This might mean that I would be a quitter, something Mom and Dad and the President and Jesus told me I shouldn’t be. Nevertheless, this was the only plan I could live with.

So how in the world could I outsmart these brilliant doctors and figure out if I was on the trial drug or a placebo? I couldn't figure it out with certainty, but I had some ideas.

Because Rituxan was already being used for cancer patients there was a lot of documentation about its side effects. I learned that when somebody gets their first infusion of Rituxan their blood pressure typically drops and their core body temperature typically rises. Sometimes they have a more severe respiratory reaction that forces the infusion to be temporarily stopped. On subsequent infusions there are usually no side effects at all. So my plan was to see if I had the side effects on infusion one, but not on infusion two, which would come only two weeks later.

As a friend of mine said to me, “You need to be in this trial because it might make Mitch better, not because it might further scientific knowledge. If you happen to contribute to a medical science in the process, then that’s just a bonus.”

Selfish? Perhaps. Do I regret taking this position? Not one bit.

In fact I did withdraw from the trial before its completion- not because I thought I was on the placebo, but for a completely different reason.

For the next installment in this series, click here.
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  1. Hello! I am glad I found your blog!

    I don't think it is selfish at all. I probably would have done the same thing... =)

  2. Welcome "Have Myelin?". I'm glad you found me as well. Thanks for the support.

    Love your name. I have myelin, but not enough.