Today, a phase III trial of the immunotherapy drug nivolumab (Opdivo®) in patients with advanced kidney cancer was stopped early because results showed a clear benefit to patients: those receiving nivolumab lived longer than those receiving the standard-of-care drug everolimus. Patients in the trial who were receiving everolimus will now be eligible to receive nivolumab instead.
Nivolumab, made by Bristol-Myers Squibb (BMS), is a member of a class of immune drugs called checkpoint inhibitors. These drugs work by “releasing the brakes” on the immune system, allowing a stronger immune attack against cancer. The particular braking molecule targeted by nivolumab is called PD-1.
The trial enrolled 821 patients with advanced or metastatic renal cell carcinoma, the most common type of kidney cancer. Half of the patients received nivolumab, the other half received everolimus. The primary endpoint of the study was overall survival—meaning a statistically significant difference in the length of time patients survived between the two treatment groups. An independent Data Monitoring Committee determined that the nivolumab group had longer overall survival than the everolimus group, although the complete results of the trial have not yet been released by BMS.
This large phase III trial of nivolumab is one of several that have been stopped early because the interim results showed greater effectiveness of nivolumab compared to the standard-of-care drug. The most recent was a trial of nivolumab in non-squamous non-small cell lung cancer, which was stopped in April of this year. The kidney cancer trial was scheduled to end in February 2016.
Clinical trials are stopped early when results are so clear that it would be unethical to continue treating patients on the control arm with the less effective drug. Phase III trials are the last stop on the road to drug development before FDA approval. Nivolumab is already FDA approved to treat melanoma and squamous cell non-small cell lung cancer.
Kidney cancer accounts for more than 100,000 deaths wordwide each year. Globally, the 5-year survival rate for those diagnosed with advanced kidney cancer is 12 percent, and better treatments are badly needed. Immunotherapies like nivolumab hold great promise for treating kidney cancer and many other cancers.
For a personal view of what nivolumab has meant to patients with kidney cancer, see Richard C.’s story here.