Do you feel lucky, Matty?

I have always turned down opportunities to participate in phase I drug trials. This may be overstating it a bit, but phase I trials seem less interested in making you well than in making you ill.

You hang out in a hospital while researchers (I assume doctors and nurses) give you ever-increasing doses of the new drug, monitoring results, until you get really sick.

The American Cancer Society says, "The main reason for doing phase I studies is to find the highest dose of the new treatment that can be given safely without serious side effects." So basically what I said.

I have never been desperate enough. Note the past tense.

I am of an age and a disease progression that makes a treatment more necessary sooner. If I have to spend a few days barfing or bleeding out my eyes (probably not), it might be worth it.

I learned of several possible upcoming phase I's at my visit to the Children's Hospital of Philadelphia (more on the visit in coming posts). One sounded quite cool: gene therapy. It's not happening tomorrow and there are bigger risks, but recent research reports "rapid and complete reversal of sensory ataxia by gene therapy" in a mouse with Friedreich's ataxia.

Of course, a mouse isn't a person, as my doctor said, so we'll have to wait and see if it makes it to trial.

The main risk if it gets to phase I and I take part is that they guess wrong on the best dose in phase I.

One can only get gene therapy once, so if they come up with the winning dose in five years,  I can't get it.

But in five years, my body will be worse by five years.

So I have to ask myself: Do I feel lucky?