Cancer drug trial provides lessons for future

A cancer drug was found to be ineffective in preventing recurrence of kidney cancer in patients who recently underwent tumor removal surgery, according to a clinical trial published in the Journal of Clinical Oncology.

More than 80,000 new cases of kidney cancer are diagnosed each year, according to the American Cancer Society, and roughly 14,000 will die from the disease.

In the study, investigators sought to test the effectiveness of pazopanib, a cancer drug which works by decreasing blood supply to tumors, versus placebo, said David Cella, Ph.D., professor of Medical Social Sciences, who was a co-author of the study.

"There are very few clinical trials in oncology like this one, where we have a placebo arm," said Cella, who is also associate director of Cancer Prevention and Control at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

"Usually there's an active comparator that's the standard-of-care, and that is compared against an experimental therapy. With this placebo treatment, we had a nice group of comparison that's not getting toxicity or side effects from any other treatments."

Of 129 kidney cancer patients who showed no signs of the disease following tumor removal surgery, half were randomly assigned to receive either pazopanib or a placebo. After three years of treatment, patients who received a placebo were more likely to be cancer-free (27%) versus the pazopanib group (21%), but the difference was not statistically significant.

Using a questionnaire developed by Cella, study participants on pazopanib experienced worse quality-of-life outcomes, likely due to side effects of the drug, according to the study.

The findings highlight the potential risks of experimental cancer treatments, Cella said, and provides more information for future kidney cancer therapies.

"Everybody wants effective treatments," Cella said. "A study like this does help us to understand the risks that patients take when they're trying to improve their lives with cancer. Because unfortunately, sometimes the treatments can make things worse."

The study and patient quality-of-life questionnaire responses offer important insights for the design of future clinical trials, Cella said.

"For me, the next steps are to take these symptom and quality of life tools to future kidney cancer research, and apply the information that we learned here about what's a meaningful difference to patients," Cella said.