By Jessica Gorman
The Pathway to Better Care
How well-designed clinical trials aim to improve patient outcome
By Jessica Gorman
"My story probably is one of so many," says Tania Stutman, a recently retired cardiology technician and grandmother of five. During a routine gynecological exam, Stutman found out she might have a mass on her ovary. She went to the hospital for a hysterectomy, and the surgeon discovered that her ovaries were fine, but she had a tumor on her small intestine. It was a very rare cancer, and aside from surgery, she had no treatment options.
The doctor told Stutman's husband, Robert, that his wife would probably die within a year. Her husband broke the news. "That death sentence just blew me away," she says. "I was just crying and screaming ... No one is ever ready to die."
For more than a year after her diagnosis, Stutman searched in vain for a clinical trial—an experimental study in patients—that was testing any treatment for her cancer, known as GIST, or gastrointestinal stromal tumor. Assessing the odds at "less than one percent" that a clinical trial could help her, Stutman says she wanted any opportunity to try to treat her disease—and if not, to help find a treatment for someone else. "Maybe even if I don't survive, they'll learn something from it through me, as a patient," she remembers thinking. "I want to do something."
Today, Stutman's story is no longer one of so many stories, but one of the stories cancer patients and their doctors hope to hear more often. With the cancer metastasized to her liver, she enrolled in a study at last—and hit the jackpot: an extraordinarily successful trial of a new drug called Gleevec (imatinib mesylate). Eight years after Stutman's "death sentence," the drug is now the standard treatment for patients with her cancer.
Not many clinical trials have the same high payout as Gleevec's, and not many patients will hit the jackpot on a clinical trial the way Stutman, now 56, did. But the same process that made Gleevec the new option for GIST patients has improved the treatment of cancer throughout the last half-century. Like Stutman's "pot of gold," as she calls her saffron-colored Gleevec pills, other new cancer drugs and procedures have made their way out of the lab and through a system of clinical trials to ultimately change patient care.
Until the advent of modern clinical trials in the 20th century, most medicine was based on "anecdotalism," says Bernard Fisher, a biomedical researcher and surgeon at the University of Pittsburgh. Doctors used treatments that had worked for their earlier patients, and there was little science involved.
It's possible that the Bible's Book of Daniel contains the first record of an experiment resembling a clinical trial. King Nebuchadnezzar of Babylon, it says, ordered some Hebrew children to eat what he considered a healthful diet of meat and wine. Daniel, who didn't want to eat the king's food, suggested that he and three other children instead eat legumes (such as peas or beans) and water for 10 days, and then be examined. After the 10 days, the children consuming legumes and water looked healthier than those eating meat and wine, and they were allowed to continue their diet.
It wasn't a particularly sophisticated experiment. Well-designed modern clinical trials, in comparison, are meticulous processes, the best of which test whether a new option offers a true benefit over currently available drugs or procedures. They're not based on the whim of a king, a prophet, or even a doctor, but on scientific evidence from laboratory studies that have led logically to testing the new idea in people.
That drug or procedure often goes through several steps of study in patients. At each step, or phase, the trials test the safety, dosage, side effects or benefits of the treatment in a larger group of patients. Generally, in the most rigorous trials—which often grow out of more preliminary ones—researchers randomly assign large numbers of participants to a standard treatment or a newer option to see which works better.
"Clinical trials are the only way to know whether that new treatment really is better than the standard," says breast cancer survivor Peggy Devine, who is the founder and executive director of the Cancer Information and Support Network—an organization aiming to improve public understanding of cancer research and to help researchers, the public and pharmaceutical companies work together more effectively.