A Patient's Painful Search for a Clinical Trial

Connie didn't want to go to Houston. She doesn't know a soul there. Connie is 53 years old. When she was younger, she was astonishing. She danced -- so well that she was accepted into Eliot Feld's American Ballet Company in New York City. But shortly after fleeing her home town of Flint, Mich., she twisted her ankle. Dance career over. She regrouped, earned advanced degrees in anthropology and women's studies, and traveled the world -- Asia, Europe, all over.

But nowadays, Connie can't work. Eight years of constant testing has made her an extremely unstable employee. She is wiped out financially. She does have Medicare and private insurance, but she's been told that neither cover investigational trials. The trial would last at least four weeks, maybe as long as eight. Typically, a manufacturer picks up the tab for the drug it is testing. That's it. Otherwise, a trial participant is responsible for all the costs: housing, food, travel and the many medical tests involved. How would Connie pay for it all?

It would be hell, going to Houston in the summer, alone, not knowing anybody, and getting long needles shoved through her bone and into her marrow. It would be hell, too, asking for money from her friends and family to help pay for the trip. But that is what she would have to do. She'd have to sell some of her stuff on consignment, and go with some stalwart hope that the clinical trial would have her.

I felt stupid not being able to find any answers for Connie. I knew the leukemia ropes and I'd spent a lifetime researching all sorts of stories and issues as a freelance writer. How could I not find anything? This was, after all, a clinical trial for a potentially significant new drug. As time went on and the answer remained elusive, I began to wonder if it was subversive of me to even ask the question. Was it wrong for me to expect that drug companies might divulge information about their clinical trials so that desperate cancer patients might avail themselves of what could be their last chance?

The story I eventually read in The Post about the FDA's clinical trial registry showed me there was nothing wrong with my efforts. The story said that although drug companies conduct 80 percent of the trials going on in the country, they represented only 13 percent of the trials listed on ClinicalTrials.gov. That means that countless industry-sponsored trials weren't listed. And that meant that it was improbable that I'd find an answer to my question. But beyond that, the law as written turned my search from difficult to more or less impossible. That's because it only covers Phase 2 through 4 trials. It exempts Phase 1 trials. That is the category AMN107 falls into.

A Phase 1 is known as a "safety" trial, while Phases 2 through 4 are considered "effectiveness" trials. What safety means, in this case, is that they keep jacking up the dosage to gauge your tolerance. They want to see how much you can take before you throw up. Or spike a fever. Or get the chills. Or suffer some combination of all three. It also means, more significantly, that they want to see if this stuff might actually work. And sometimes it does.

A Phase 1 trial requires a lot of testing to monitor the patient's response to the drug. It could mean lots of painful intrusions, such as bone marrow biopsies. Yet it turns out that the very people who would submit to the expense and horrors of a Phase 1 trial -- that is, the people in the worst shape -- are, by law, the ones least likely to find out about them. People like Connie.

I have no idea why the law would exempt Phase 1 trials. Maybe it's a concern about their risky nature. Maybe it's a bow to the industry's fear of "poaching" -- company B finding out what company A is doing and, um, borrowing it. But that can happen at any stage of testing. Whatever the reason, that Phase 1 trials aren't even included is, to me, a disgrace.

Connie went down to Houston on a Saturday. I followed the next day, so that she wouldn't have to be alone at least for this first trip. When I arrived, she was pale. She told me she had had nonstop diarrhea and stomach cramps throughout the night. "I always get really anxious before big test days," she said apologetically.

The next day, she endured a battery of tests just to see if she qualified to take the actual tests to get into the trial. It was like trying to get into Harvard or something. The day after that, Tuesday, she met with a doctor who ran the clinical trial. He finally answered our question: Yes, M.D. Anderson is the only place in America conducting the trial for AMN107. (Later, Novartis came through with that info, too.)

Now, Connie could decide. Should she gamble that she wouldn't get any worse any time soon, save her money, and hope that AMN107 went into the less intensive Phase 2, which might be held at hospitals around the country, reasonably soon? But what if her condition, which is already in the accelerated stage, shot into "blast" while she was waiting for Phase 2? The blast stage is not good. Coming back from blast is a little like raising Lazarus. It can be done, but it isn't an everyday occurrence.

She called me after we returned home. "What do you think I should do?" she asked. "I don't know," I replied. "What do you think you should do?"

She mentioned the high cost, the constant testing, the uncertain dosages and coping with the unknown side effects on her own. "I think I'll wait," she said. "I think I will be okay."

We're both holding our breath a little. I hope she's right. It's hard to tell, even for a couple of old leukemia hands like us.

But I hope, too, that she and the millions of others like her might someday have a registry that meets the spirit and the letter of the law, that enforces compliance and includes Phase 1 trials. For I do know that, when it comes to clinical trials, what you don't know can't help you.

Author's e-mail: jimshahin@aol.com

Jim Shahin is a freelance writer who lives in Silver Spring.