Targeted therapy extends progression-free survival in advanced RCC

March 13, 2008

An independent data monitoring committee stopped a major phase III clinical trial of the investigational drug everolimus (RAD001) after interim results showed significantly better progression-free survival in patients with advanced kidney cancer who received everolimus compared with placebo.

An independent data monitoring committee stopped a major phase III clinical trial of the investigational drug everolimus (RAD001) after interim results showed significantly better progression-free survival in patients with advanced kidney cancer who received everolimus compared with placebo.

Everolimus is a once-daily oral targeted therapy that inhibits the mTOR protein. The randomized, double-blind study included more than 400 patients in 12 countries, including patients who had their cancer worsen despite receiving approved treatments for renal cell carcinoma.

“Everolimus has the potential to greatly help patients with kidney cancer, especially those in advanced stages who up to now have had no treatment options, as patients in the clinical trial on everolimus experienced a significantly longer period of time during which their cancer did not progress,” said Daniel Vasella, MD, of Novartis, which expects to submit U.S. and European regulatory filings for the RCC  indication in the second half of 2008.

Safety findings in the study were manageable and consistent with those of prior phase II studies, according to a Novartis statement. Common adverse events included mouth ulcers, high blood lipids, elevated blood sugar, skin rash, low red blood count, low phosphate levels, and inflammation of the lungs.

Complete results of the RECORD-1 (REnal Cell cancer treatment with Oral RAD001 given Daily) trial will be submitted as a late-breaking abstract for presentation at the American Society of Clinical Oncology annual meeting in Chicago in May.