Positive Results Cut Short Everolimus Kidney Cancer Trial

A phase III clinical trial of everolimus (RAD001) was halted in late February after interim results showed significantly better progression-free survival in patients with advanced kidney cancer who received everolimus compared with placebo.

RECORD-1 is the largest phase III trial to investigate the potential of everolimus as a treatment option for patients with metastatic renal cell carcinoma that has progressed after prior targeted therapy. The results of this placebo-controlled trial, involving more than 400 patients and conducted in 12 countries, are being shared with investigators to allow them to offer the investigational drug to patients on placebo based on the progression-free survival improvement in patients taking the drug over placebo. Complete results of the trial will be submitted as a late-breaking abstract for presentation at the American Society of Clinical Oncology annual meeting in late May. The drug is expected to be filed for approval with the Food and Drug Administration later this year.

Everolimus is a once-daily oral drug that inhibits mTOR, a protein that acts as a central regulator of tumor cell division, cell metabolism, and blood vessel growth. The trial included patients whose cancer worsened despite receiving approved treatments, such as Nexavar (sorafenib), Sutent (sunitinib), or both.

In addition to RCC, everolimus is presently being evaluated in neuroendocrine tumors, lymphoma, melanoma, and other cancers as a single agent or in combination with existing cancer therapies. Everolimus is already approved as Certican for the prevention of organ rejection in heart and kidney transplant recipients.

Common side effects observed in the study included mouth ulcers, high blood lipids, high blood sugar, skin rash, low red blood count, low phosphate levels, and inflammation of the lungs.

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