Immunotherapy Pioneered at MSK Receives FDA Approval for Advanced Kidney Cancer

By Matthew Tontonoz,

Friday, December 4, 2015

Robert J. Motzer

The US Food and Drug Administration recently approved an already existing immunotherapy drug, nivolumab, for people with advanced renal cell carcinoma (a type of kidney cancer). This means that patients will now have access to the drug outside of clinical trials. MSK’s Robert J. Motzer led a large trial that informed the FDA’s decision.

  • The FDA has approved nivolumab for people with advanced kidney cancer.
  • This is the first approval of an immune checkpoint inhibitor for the disease.
  • Many patients have tolerated the drug better than existing treatments.

On November 23, the US Food and Drug Administration approved the immunotherapy drug nivolumab (Opdivo®) for the treatment of advanced renal cell carcinoma, a form of kidney cancer. Nivolumab belongs to a class of immunotherapies called checkpoint inhibitors that release the brakes on the immune system, allowing it to mount a stronger attack against cancer. This approach, which Memorial Sloan Kettering physician-scientists played a major role in developing, is producing stunning results against many cancers.

Although nivolumab is already used to treat melanoma and lung cancer, this marks the first time the FDA has approved a checkpoint inhibitor for the treatment of kidney cancer.

To learn more about what this approval means for kidney cancer patients, we spoke to MSK medical oncologist Robert Motzer, who led the clinical trial that supported the FDA’s decision.

Who will benefit most from this new FDA approval of nivolumab?

This approval is for patients with metastatic renal cell carcinoma who have been previously treated with other medications and whose disease is now getting worse. In other words, as second-line or third-line therapy following standard drugs such as sunitinib (Sutent®), pazopanib (Votrient®), or axitinib (Inlyta®).

The clinical trial supporting this approval compared nivolumab with another drug called everolimus (Afinitor®) in people whose disease had progressed on other therapies. Everolimus is one of the most widely used kidney cancer drugs worldwide. It is often used as second- or third-line therapy after a patient stops responding to other drugs.

Based on this new approval, patients will be eligible to receive nivolumab as second-line or third-line therapy instead of everolimus. Nivolumab is well tolerated by patients and has better quality of life compared with everolimus. I think most people should get this as their second-line treatment. 

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How have your patients fared on treatment with nivolumab?

One of the questions that I would get from people time and again was, “Am I getting a placebo?” They didn’t really believe they were getting a drug because they felt so good. I think the limited side effects of this drug are one of its strong points.

What was really noticeable with nivolumab was how well my patients tolerated the drug.
Robert J. Motzer
Robert J. Motzer Kidney cancer specialist

One of the problems with other therapies, including everolimus, is the chronic, ongoing side effects that can interfere with daily life. What was really noticeable with nivolumab was how well my patients tolerated the drug. It was quite striking right from the beginning.

Still, some people do have side effects on nivolumab, including certain tricky immune-related adverse events that require steroids and can sometimes be difficult to diagnose. But many patients don’t have any side effects at all.

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How well does immunotherapy work for kidney cancer compared to, say, melanoma?

Learn more about the diagnosis and treatment options for kidney cancer.

I’ve been taking care of kidney cancer patients since the 1980s. Even back then, the two cancers that were always linked to the immune system were melanoma and renal cell cancer. The belief that the immune system played a role in these cancers was largely based on the observation that these cancers sometimes regressed spontaneously, and also that available immunotherapies at the time — interferon and interleukin-2 — really only worked in renal cancer and melanoma.

For a while, interest in immunotherapy for renal cancer faded, but it’s almost like it’s been resurrected with this trial. We now have proof of principle that these new modern immunotherapies benefit patients with renal cancer. It’s set a path for further development of this class of drugs in this disease.

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Did this clinical trial benefit from other immunotherapy research happening at MSK?

Absolutely. [Cancer immunologist] Jedd Wolchok’s pioneering work on immunotherapy for melanoma really opened the door to this approach in other cancers, including kidney cancer. It also set the stage for the pivotal role MSK played in conducting these studies. That’s been good for us as physicians because when we first started using these drugs, we had questions about how to best manage the immune-related side effects in some patients. Dr. Wolchok’s group was extremely helpful.

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This approval is for advanced kidney cancer following progression on one or two prior therapies. Are there studies looking at the use of nivolumab in earlier settings?

Yes, but the question is how well nivolumab will perform compared to existing drugs in that context. The drugs that make up the mainstay of initial treatment for kidney cancer, sunitinib and pazopanib, are given as pills and are quite effective. For nivolumab to move into first-line therapy, it would either have to be in a situation where a biomarker existed that allowed us to target the therapy to patients most likely to respond, or it would have to be used in combination with other drugs. We’re conducting some of those combination studies here at MSK.

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What would you recommend to a patient who is interested in nivolumab but who doesn’t meet the current qualifications for the drug?

The current evidence says that nivolumab may benefit patients whose disease has progressed on prior therapy. I don’t think it should be offered to patients as first-line therapy outside a clinical trial. But clinical trials can be a good option for patients and we encourage our patients to consider them.

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How does this trial relate to other work you’ve done in kidney cancer?

I believe this represents another important step in our clinical trial program here at MSK. Our efforts in developing several new drugs have impacted patients with kidney cancer around the world. This most recent approval means that a new drug with a whole different mechanism of action from those approved previously is now available to patients being treated for kidney cancer.

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The phase III study of nivolumab was supported by Bristol-Myers Squibb.


Thanks for the amazing work guys.

Thank you for the work you are doing. Thanks to you I have the second stage drug Opdivo in the arsenal if Sutent doesn't work well for me. Me and my wonderful wife are very grateful everyday for the work you are pioneering in kidney cancer.

Dear Mike, thank you so much for sharing your thoughts and experience on our blog. We wish you all our best.

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