Immunotherapy Pioneered at MSK Receives FDA Approval for Advanced Kidney Cancer

Robert J. Motzer

Medical oncologist Robert J. Motzer led the trial that underpinned the FDA’s approval of nivolumab for advanced kidney cancer.

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 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.


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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.

Recently I've been dx with a metastatic renal cell tumor which is on my left adrenal gland. I had my right kidney removed in June of 2009 and was in a clinical trial including Sunitinib and Sorafenib for one year. Ever since receiving these medications (which I believe I did receive because of the numerous side effects) I have been suffering with terrible leg cramping. My Doctor now at fox Chase wants me to use Chemo again to shrink this newest tumor before removing it with the hope that we are able to transplant my left kidney successfully into my pelvic area. Do you believe Nivolumab would be a good choice?
Thank you,

Dear Alex, we are sorry to hear about your diagnosis. We can’t offer a treatment recommendations without knowing more about you and your cancer. If you would like to make an appointment with one of our specialists to discuss possible treatment options, including immunotherapy, please call our Physician Referral Service at 800-525-2225. Thank you for reaching out to us.

I was diagnosed with stage 4 RCC within the last week. I have a 4cm tumor on my left kidney, which is not effecting the kidney's function, and a 4cm tumor in my left upper chest cavity, no other organs seem to be effected right now. I just started Sutent yesterday and my Dr wants me to do two cycles and see how the tumors react. Is Nivolumab something I should look into if my tumors don't respond to Sutent or is if I end up experiencing some of the more suvere side effects of Sutent.

Dear Joe, we are sorry to hear about your diagnosis. We recommend that you follow up with your oncologist, who is best equipped to answer specific questions about your treatment plan. If you would like to consult with one of our specialists for a second opinion or about possible next steps in your care, please call our Physician Referral Service at 800-525-2225. Thank you for reaching out to us.

God Bless this work you are doing. I was diagnosed with Stage IV RCC in May 2016. After two other targeted therapies, I began nivolumab on January 12. After just three cycles, the CT scans show considerable improvement with many nodules being completely resolved. I hope this success continues. I did have a major side effect--went into DKA and now have Diabetes Stage 2 which doctor hopes may go away with time. Again thank you for saving my life!

Dear Austin, we are so glad to hear that you’re doing well. Thank you for your comment! Best wishes to you.

Has there been any progress on the acceptance of dual immunotherapy for the treatment of RCC? My husband is stage 4 and has been told by 4 oncologist's that this is the treatment he needs but insurance is still calling the treatment "experimental"

Dear Candace, we’re sorry to hear about your husband’s diagnosis. We recommend that you discuss this with his healthcare team and your insurance company. Thank you for your comment, and best wishes to you and your husband.

Is this treatment still considered experimental? My Mom has renal cancer and I would like to learn more about this. Thank you

Dear Michelle, this treatment is FDA approved for certain patients. We recommend that you and your mother discuss it with her medical team. Thank you for your comment and best wishes to both of you.