Treatment with everolimus can significantly improve the progression-free survival of patients with advanced kidney cancer that has not responded to other treatments, according to a report in the online issue of The Lancet.

This finding stems from a study of 410 patients with kidney cancer that had spread, or “metastasized,” to other parts of the body, despite treatment with sunitinib, sorafenib or both drugs. The patients were randomly assigned to receive everolimus once a day or placebo, in addition to supportive care.

Everolimus has already been approved by the U.S. Food and Drug Administration under the trade name Certican for preventing organ rejection after a heart transplant. Approval as a cancer drug would widely expand the use of this agent.

Lead author Dr. Robert J. Motzer, from Sloan-Kettering Cancer Center in New York, and colleagues, initially planned to stop the study after 290 events occurred that indicated disease progression, but an interim analysis showed a clear advantage with everolimus therapy, so the trial was terminated after 191 events.

Overall, the rate of cancer progression in the everolimus group was 37 percent compared with 65 percent in the placebo group, a statistically significant difference. The average progression-free survival time was 4.0 months for the everolimus group and 1.9 months for the placebo group.

Mouth sores, rash and fatigue were more common in the everolimus group than in the placebo group, but were usually of mild or moderate severity, the researchers note. Twenty-two patients (8 percent) in the everolimus group developed inflammation of the lung, which became moderately severe in eight patients.

In a related editorial, Dr. Jennifer J. Knox, from the University of Toronto, comments that these study data support the choice of this treatment design for patients with advanced kidney cell cancer. “I would encourage international regulatory boards to accept these data as evidence of clinical benefit.”

SOURCE: The Lancet, online July 23, 2008.