I'll bet a story about drug companies keeping information from the public isn't most people's idea of exciting news. But most people aren't desperately trying to find the location of a clinical trial in the hopes that it might save their lives. After reading a recent newspaper account of how pharmaceutical companies routinely ignore a 1997 law designed to create a list of ongoing clinical trials for new drugs, I raced through the house, whooping ecstatically. "Vindicated!" I shouted.
The story about the Food and Drug Administration's registry of clinical drug trials appeared in The Post a week after my cousin Connie Ojile and I had returned to Washington from Houston. We'd gone there to see whether Connie qualified for a clinical trial for a new leukemia drug. Before we went, I'd tried my damnedest to get an answer to this question: Is the trial being held anywhere besides Houston?
It would be much easier on Connie to undergo a trial closer to home. But try as I did, I couldn't get the answer. Now I knew why. Not only were the companies flouting the law, but they weren't even required to register the specific trial Connie was looking at -- the very first, most preliminary trial to test a promising new experimental drug, the kind of trial that someone like Connie might be desperate to know something about.
The news -- both elements of it -- outraged me. But it also restored a bit of my dignity. I no longer felt inadequate for not getting the information Connie had wanted.
I cannot tell you how big a deal dignity is when you're dealing with cancer.
Connie and I, we know. We're a couple of old hands when it comes to leukemia. We know all about platelets and white blood counts and hemoglobin. If somebody forgets to give iodine before a certain test, we know to holler, "Hey, a little iodine over here." We check the date on the chemo to make sure it hasn't expired. That is, after we check the chemo label to make sure it's the right stuff in the first place.
I was diagnosed with leukemia after my gums started bleeding and my back radiated with pain so searing I couldn't sit up. That was 19 years ago, in 1985. Since then, I've had, let's see, a splenectomy, synthetic interferon, human interferon, something called 2-CdA (2-chlorodeoxyadenosine) and probably some stuff I'm just not remembering at the moment. Now I'm in remission. Remission, of course, is not cure, and I am proof of that: I've fallen out of remission three or four times, depending on how you define it.
Connie has it worse than I do. That she has the disease at all is something that, personally, I think is weird -- two first cousins with two different kinds of leukemia out of only eight on that side of the family. What are the odds? She was diagnosed eight years ago with chronic myelogenous leukemia. CML, for short. (I had hairy cell leukemia. It's an appropriate name; I'm a hairy guy.) Since then, she has undergone radiation, a bone marrow transplant, and more or less constant chemotherapy. She has yet to achieve remission.
Last month, Connie's New York oncologist recommended she go to Houston to see if she qualified for a trial of a brand-new drug that might put her into remission, or at least arrest her cancer's advance. The trial was due to begin in just two weeks. Overwhelmed by the details of preparing for a clinical trial, Connie failed to ask her doctor if it was being held in multiple locations. I figured, no big deal, I can get the information easily enough. As the days dragged on without success, Connie called her doctor but couldn't reach her.
The drug was called AMN107. Or maybe AMN-107. At the time, I didn't know how to spell it because I couldn't find out anything about it. With so little time before Connie had to decide whether to go to Houston, I was desperate to find any information I could. I searched for the drug under both spellings, with a hyphen and without one. Either way, it was as if it didn't exist.
I searched the Web site of the manufacturer, Novartis, to no avail. I called the customer relations division, but the rep I spoke to hadn't heard of AMN107 and couldn't find it in her computer. I sent e-mail to the company, asking for info. I called back and talked to a different representative. She didn't know anything about the drug, either. She referred me to the Web site, which I'd already tried, and to its oncology section, which I'd also tried, and then to another site which, well, I tried, too: www.ClinicalTrials.gov. That is where the Food and Drug Administration is supposed to keep a registry of the nation's clinical trials. Guess what? No trials for AMN107 or AMN-107 were listed there.
I tried Googling. I got a couple of things in German. A trial, it seemed, was also being conducted in Frankfurt. Not exactly the close-to-home location we were looking for. I tried the Web sites of the NIH, NCI -- oh, I'm sorry, the National Institutes of Health and the National Cancer Institute. Those are the two primary government organizations that oversee research on diseases like leukemia. They don't seem to have much idea of what clinical trials are going on in this country. At least, based on their Web sites, they didn't know a thing about this one. I called a few leukemia and cancer organizations. Again, no luck. Even the Web site of M.D. Anderson Cancer Center, the place conducting the trial Connie's doc had told us about, didn't have anything about it.
A rep at M.D. Anderson, though, did stay on the line with me and remained remarkably patient in the face of my increasing frustration. After first saying that perhaps I was mistaken about the name of the drug or the whereabouts of the trial, she discovered somewhere deep in the system, like going through the skin, the flesh, the bone and into the marrow to extract information about the progress of your leukemia, that, by golly, M.D. Anderson did have a clinical trial for AMN107 after all.
I finally found a little information about the drug (including the fact that its name didn't include a hyphen) at a Web site run by and for CML patients, www.CMLwatch.org. They knew about it because a few of them were in the trial. They didn't know, however, if a trial was being held anywhere other than Houston.
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.
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