New data show RAD001 (everolimus) may provide an important new treatment option for patients with advanced kidney cancer who have failed standard therapies.
The interim study findings demonstrated that RAD001 significantly extended the time without tumour growth from 1.9 to 4 months and reduced the risk of cancer progression by 70 per cent. The study, RECORD-1 (REnal Cell cancer treatment with Oral RAD001 given Daily), will be presented at the 44th annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago, Illinois, US on Saturday, May 31, 2008.
Earlier this year, an independent data monitoring committee stopped the RECORD-1 trial after interim results showed that patients receiving RAD001 experienced a significantly longer time without their cancer worsening compared to patients receiving placebo. The trial included patients whose cancer had stopped responding to approved treatments for renal cell carcinoma (RCC), such as Nexavar (sorafenib) or Sutent (sunitinib), or both.
RAD001 is a once-daily oral therapy that may offer a new approach to cancer treatment by continuously inhibiting the mTOR protein, a central regulator of tumour cell division and blood vessel growth in cancer cells.
"This is the first study to show clinical benefit in patients with advanced kidney cancer who have experienced treatment failure with the most commonly used first-line therapies," said Robert J. Motzer, MD, attending physician, Memorial Sloan-Kettering Cancer Center, New York, and principal investigator of the RECORD-1 trial. "The results show RAD001 extended progression-free survival in patients regardless of their prior treatments, risk status, age, or gender".
During the second half of 2008, the interim results from RECORD-1 will be used to submit a new drug application for RAD001 as a treatment for metastatic renal cell carcinoma.
"As we will see in presentations at the upcoming meeting, RAD001 has the potential to benefit patients living with a variety of cancers including neuroendocrine, breast, gastric, and lung," said David Epstein, CEO and President of Novartis Oncology. "We look forward to updates from trials in pancreatic neuroendocrine tumours before year-end."
RECORD-1 results
RECORD-1 is the largest phase III clinical trial investigating the effects of an oral mTOR inhibitor in metastatic RCC. It is a randomized, double-blind placebo-controlled multicenter trial of more than 400 patients with RCC whose cancer worsened despite prior treatment, including Nexavar or Sutent, or both. In addition, prior therapy with Avastin, interferon, and interleukin-2 was allowed.
The primary endpoint of RECORD-1 was progression-free survival (PFS) assessed via a blinded, independent central review and defined as the amount of time between randomization and first documented disease progression or death due to any cause. Results of the study demonstrated a statistically significant improvement in PFS for RAD001 compared to placebo.