Dianne Harris Wright didn't know what treatment options she faced after her 2002 surgery for stage III ovarian cancer at VCU Massey Cancer Center. She only knew she wanted to be healthy again. When she learned about a clinical trial at Massey that might benefit her, she was hopeful, enrolling in the study that year.
"My tumor had been an explosion in my system," Wright explains. "My doctor told me that the clinical trial [designed for women with stage III or IV ovarian cancer] was in my best interest."
Clinical trials help researchers find better ways to prevent, diagnose or treat disease by using new drugs or devices. Trials are designed to answer specific questions about both established and novel therapies. Typically, people enrolled in these studies are monitored more closely by doctors and nurses than patients cared for in clinical practice; the structure of a trial requires regular check points.
"Controlling and curing diseases is a huge, international scientific endeavor," says Dr. John Roberts, associate director of clinical research at Massey. "In the U.S. alone, more than 6,000 [current] cancer-related clinical trials are aimed at preventing cancer, reducing mortality and improving quality of life for people who suffer from this disease." He adds, "Massey is part of the nationwide network of 64 research centers designated by the National Cancer Institute to advance this research and make the latest discoveries available to people in our community first."
Clinical trials are offered in almost any field of medicine. For instance, across the Central Virginia HCA network at any given time, physicians are conducting between 10 and 20 trials in the areas of cardiology, neurology, oncology, orthopedics and wound healing.
Trials are the only way to determine if a new drug or treatment will work in humans, and most of them progress in phases. Phase I trials are the first studies to evaluate how a new drug should be administered, how often and in what dosage. Phase II provides information on how well the new drug works. In Phase III, researchers compare a new drug, procedure or combination of drugs with a current standard of treatment. Finally, Phase IV offers continuing evaluation of the drug or treatment after it has received approval from the Food and Drug Administration and is on the market.
Safety is always top-of-mind in any clinical trial. "The studies are carefully monitored," explains Dr. Mark Newton, a cardiologist with Virginia Cardiovascular Specialists and medical director of the cardiac catheterization lab at Levinson Heart Hospital at Chippenham Hospital, which conducts clinical trials. "To ensure safety, there are three layers of oversight. They include the FDA and a data monitoring and safety board, as well as an institutional review board at the hospital."
The first people to benefit from a trial are the ones who participate in it; however, the trial's findings benefit future generations of patients, too. As medical director of research at Virginia Cancer Institute, Dr. James Khatcheressian works with both Phase II and Phase III studies. "When we do a Phase III trial, we are trying to take whatever treatment is standard of care and improve upon it," he explains. "We always make it a point when we are treating patients to tell them what the goal of therapy is. If they have an incurable cancer, the goal is to help them live longer and feel better."
Massey is the only cancer center in the region to offer early-phase studies that have just emerged from the lab. Dr. Paul Fisher, chairman of VCU's department of human and molecular genetics and a leading researcher at Massey, says it can take 15 years for a discovery made by a researcher to become applicable in the clinic. "That's why clinical trials are important," he says. "It's the only way to know if an agent has a beneficial effect in a person."
As part of the monitoring and data-sharing process, clinical researchers can identify any irregularities in symptoms. If a problem arises, the trial can be altered or stopped. Notably, a patient may opt out of the trial at any time, for any reason. "No one has a crystal ball," Fisher says. "You can get surprises when the drug [is administered] to people."
Even though they are not risk-free, clinical trials can yield great gains. "There are some devastating diseases for which there is no treatment," Fisher says. "You never want to use a patient as a test subject, but if you didn't test, you wouldn't have the benefit of knowing if a drug or new therapy would be worth expanded testing."
In his cardiology work, Newton deals with Phase III and Phase IV clinical trials geared toward new medicines and devices that relate to the heart, giving patients the early use of something that may be advantageous to them. Newton's medical group was selected as one of the test sites for the drug Plavix, which helps prevent blood clots, in the late 1990s.
The clinical trial showed a 20 percent improvement in patients who used a combination of Plavix and aspirin. "The trial was a great benefit for patients," he says. "Now the drug is part of the standard guidelines for treating unstable angina or in patients with chest pain due to heart artery blockage."
Patients may have concerns about clinical trials that relate to risks and treatments. Wright admits she worried that she'd be given a placebo instead of the new medicine and not get the benefit of the drug. But Roberts says placebos are far from the norm. In fact, placebos are used only when there is no documented, current standard of care for a specific type of cancer. In those cases, patients are informed they may be receiving a placebo.
Participating in a clinical trial worked for Wright, who went into remission after only three months in the program. By the sixth month, her tumor marker — a substance from the tumor that's released into the blood — had dropped from high to normal. "My body responded very well," she says. "I was able to tolerate the drug. I was fortunate that I didn't experience any nausea or sickness."
She remained in remission for two years after the trial. Since then, she's had recurrences of cancer in other parts of her body. Those too have mostly gone into remission. "I might not be here today if I hadn't entered the program," she notes.