PATIENTS NEEDED TO SAVE LIVES–MAYBE THEIR OWN

For those facing chronic or potentially terminal illnesses, the choices for treatment can be as confusing as they are daunting: Switch doctors? Change hospitals? Increase a drug dosage? Opt for surgery?

Yet most patients would agree on one thing: They want state-of-the-art care that will give them the greatest odds to survive.

What many don’t know is that often the best chance for getting such care is through a poorly understood and underutilized process: the clinical trial, in which typically an experimental treatment is compared with the current best available treatment.

The problem is that even as new, promising drugs and treatments are emerging at a rapid rate, researchers are having trouble finding enough volunteers for trials, slowing medical advances and limiting patients’ options.

The complicated factors behind the shortage of trial participants range from doctors’ lack of knowledge about ongoing trials or reluctance to refer patients, to lack of insurance coverage or patients’ own misconceptions about the trials.

The issue will be one focus of an unprecedented march Friday and Saturday in Washington, D.C., where tens of thousands of cancer patients and their families, medical experts and politicians are expected. Leaders plan to call for the need to increase access to clinical trials and to make cancer the nation’s top research priority.

In Chicago, a candlelight vigil in Grant Park on Friday will be tied to the national event, called “The March: Coming to Conquer Cancer.”

In the U.S., only about 3 percent of adult cancer patients participate in clinical cancer trials, according to experts. The number of patients participating in cooperative group treatment trials, which involve research institutions throughout the country, dropped to 16,000 in 1995 from 21,000 in 1988, said Mary McCabe, director of the office of clinical research promotion for the National Cancer Institute.

At the same time, the field of childhood cancers is a good example of how clinical trials have become the gold standard for care.

About 95 percent of children up to age 14 who develop cancer are evaluated at an institution involved in a cooperative group trial that is being run nationwide. Of these children, 70 percent participate in one or more clinical trials.

“Our search was to get the best care for our child,” said John Mussman, 46, of Chicago’s Northwest Side.

Mussman and his wife, Kimberly, enrolled their then 17-month-old son, J.P., in a clinical research trial at Children’s Memorial Hospital in Chicago after doctors found a grapefruit-sized tumor at the base of the boy’s spine that had spread to other parts of his body. The trial randomly assigned patients to the standard dosage of chemotherapy drugs or to a higher dosage.

The study, now complete, showed that the higher dosage– which J.P. received–produced significantly superior cure rates, said Dr. Sharon B. Murphy, chair of the pediatric oncology group at Children’s. Today, J.P. is in 3rd grade and cured.

“Not only do you want the latest treatment, but there is some awareness that even if you die or your child does, you’re part of something,” said Mussman, a Loop attorney “Most people grappling with the loss of their son won’t think about the altruism. There were kids put on trials five years ago that didn’t work.” The resulting knowledge contributed to the more successful treatment for his own child, he said.

“The best available treatment for curing cancer at this time in history is not necessarily what a doctor in Chicago thinks is the best treatment,” he added, “but it will be what the clinical trials are.”

The advantages of clinical trials are that participants receive high-quality care and will be among the first to benefit if a new approach is proven to work. However, there is no guarantee the new treatment will be superior to the current standard, and it may have unexpected side effects too.

While the National Institutes of Health hasn’t tracked the rising number of clinical trials over the years, the agency is increasing the number of clinical trials it sponsors as well as its financial support for them, said Dr. William Harlan, associate director for disease prevention at the NIH.

As part of its budget request for the coming fiscal year, the National Cancer Institute announced that it hopes to achieve a five-fold increase over the next five years in enrollment in cooperative treatment trials. If the aim is met, more than 1 million patients each year would participate in such clinical trials.

Also, with people living longer, research is now focusing on prevention of chronic conditions, paving the way for massive trials that recruit thousands of patients.

An expert at Loyola University Medical Center in Maywood, where about 450 research studies involving patients are under way, echoed the sentiment about the search for trial participants held at many Chicago-area research institutions: “We could always do better (in recruitment), but the numbers speak for themselves. If we present a protocol in an honest fashion and give a patient time to think about it . . . we have done our job,” said Dr. Ken Micetich, chair of the board that reviews research projects involving humans.

At the same time, many patients have misconceptions about trials. Contrary to popular belief, patients can quit a trial once they have started one, and if early trial results obviously demonstrate that one treatment is superior or inferior to the other, the trial will be stopped. Also, relatively few trials involve a placebo.

Another obstacle is that many insurers don’t pay the costs of clinical trials, and in fact, coverage may vary from day to day and depending on the individual case, even at the same insurer.

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“It’s arbitrary and capricious. There is nothing a patient can depend upon. All we’re asking is that routine patient care costs are covered,” said Ellen Stovall, president of The March and executive director of the National Coalition for Cancer Survivorship.

Most health-care insurers won’t pay for the care of patients enrolled in clinical or experimental trials, said Chip Kahn, CEO of the Health Insurance Association of America. However, exceptions generally are made for trials that involve treatments that have already been federally approved or that are in common use.

Beyond trying to cooperate more closely with community physicians, Chicago-area researchers are also recruiting patients directly: One trial at Rush-Presbyterian-St. Luke’s Medical Center sent out more than 50,000 letters about the study, and a program at the University of Illinois at Chicago is working with local organizations to recruit minorities to trials.

After Edward Smith, 71, was diagnosed with acute leukemia last year, he quickly left his community physician in Florida and headed for Loyola, where he joined a clinical trial introduced to him by his physician, Dr. John Godwin.

For Smith, whose tall, broad-shouldered frame was down to 139 pounds, there was no hand-wringing when Godwin approached him.

“Without hesitation I said, `What have I got to lose?’ I’m here with my life in everyone else’s hands,” said Smith, who is now in complete remission. “I have to selfishly say I was thinking of myself at first, and thought whatever can be offered that’s going to make me well, that’s what I want. Then, after I got into it, I thought if this offers help to someone else, then good.”