How Drugs Get From the Test Tubes to Us, Part 1

With all the advances that have been made so far, we’ve come to expect that medical science can successfully treat much of what ails us. And if there isn’t a solution at the moment, we like to think that one is just around the corner.

But getting from point A, the disease, to point B, the treatment, takes a giant step. And that includes doing tests on human beings.

Such tests, called clinical trials, are the only way to advance medicine, said Dr. Stefan Madajewicz, director of clinical trials in oncology at the State University of New York, University Hospital and Medical Center at Stony Brook, N.Y. “There are many ideas in the medical and paramedical communities. But the only proof of value in a treatment is through clinical trial. It’s not based on individual or group beliefs. It’s based on hard, cold mathematical data.”

For some, the idea of doing experimentation that could put people at risk smacks of human beings turned guinea pigs. And some recent experiments, including one in gene therapy that resulted in the death of a participant, have raised fears anew of the dangers of testing out treatments on people.

However, said Robert Finn, author of “Cancer Clinical Trials,” (O’Reilly, 1999, $14.95) “Participating in a clinical trial is the opposite of being a guinea pig. Experimental animals have no choice about whether to participate in an experiment and no ability to leave the it.”

Humans, on the other hand, decide which trial to participate in and, once enrolled, can leave at any time, he said. And that means whether you don’t like the side effects, you didn’t get into the study arm you preferred, you’ve found a better treatment option, or you just want out.

But the tougher decision for some is not when to stop, but whether to begin. And since there are thousands of clinical trials going on at any given time, you may well have to make that decision for yourself or for a family member.

“I believe in the value of clinical trials, and I believe in the family’s right to make their own decision, without pressure,” said Nancy Keene, who enrolled her young daughter in a clinical trial for children with leukemia. “Doctors and parents should work together, with the understanding it’s a free choice.” If a clinical trial is an option, it has to be weighed carefully against the choice of continuing with the standard treatment.

Keene, the co-author of “Childhood Cancer,” (O’Reilly, 1999, $24.95) went through an intense process of uncovering information as she made her decision. She said she talked so much that the doctor wrote in her daughter’s chart, “Mother asks innumerable questions.”

Still, with only 72 hours to decide, she felt she didn’t have enough information. The particular trial she was evaluating was to find out whether you could treat leukemia as well and do less damage to the brain by eliminating radiation and giving a child more chemotherapy.

She said she signed the consent form in time to enroll her child, knowing that in the first month of the trial there would be no differences in treatment and she could continue to do research. “My family and friends helped me do a huge literature search. I read and read and read.” By the time the randomized part of the study began — some children getting radiation and some getting more chemotherapy — Keene said she decided to go ahead. “If I didn’t think it was in my child’s best interest, I would have withdrawn.”

One of the biggest challenges for patients who are contemplating a clinical trial is not to get lulled into wishful thinking and practice selective hearing, listening for the positive possibilities and not evaluating the risks sufficiently.

What can happen, Finn said, is that a person has a serious illness, is very worried and hears that there is an experimental treatment under investigation. Handed an informed consent form, the patient may be quick to say, “OK, doc, where do I sign?” Finn said.

Instead of rushing into it, he said to read the informed consent form very carefully, ask lots and lots of questions, think it over, talk to knowledgeable family members and friends, and consider getting a second medical opinion.

In the pull between the needs of research and those of the patient, you are still your own best advocate. Given the available options, being wary and well-informed can keep you on a decision-making course that is best for your own health.