Path for Lenalidomide Approval Long but Rewarding

Pictured: Director of the Blood and Marrow Transplant Center at Roswell Park, Philip McCarthy, MD.

On Feb. 22, 2017, the U.S. Food and Drug Administration approved a new indication for the oral drug lenalidomide (brand name Revlimid). While lenalidomide was previously approved for multiple myeloma patients with relapsed or newly diagnosed disease, this new approval makes the drug available as maintenance therapy for multiple myeloma patients following autologous hematopoietic stem cell transplant (ASCT), also known as autologous blood and marrow transplant (BMT).

I am very excited about this approval. I had the privilege of leading the major U.S. cooperative-group study of lenalidomide as maintenance therapy following transplant for patients with newly diagnosed multiple myeloma. This research effort (CALGB 100104) involved more than 450 patients treated at 46 institutions in the U.S. and, along with two other large cooperative studies (IFM 2005-02 from France and RV-MM-PI-209 from Italy), established clear evidence that lenalidomide maintenance therapy prevents the recurrence or progression of multiple myeloma following autologous stem cell transplant. Of note, our U.S. study showed a significant improvement in overall survival for those patients receiving maintenance lenalidomide.

The approval by the FDA, which follows a similar decision last month from the agency’s European counterpart, is very important because it makes it easier for patients around the country to access and get insurance coverage for a beneficial therapy.

Clinical cancer research sometimes takes a long time. We started working on this effort all the way back in 2001, when we submitted the first research concept to use lenalidomide as maintenance therapy. It had not been FDA-approved for any clinical use at that point but had shown efficacy in phase I and II clinical trials led by Dr. Kenneth Anderson at the Dana-Farber Cancer Institute. We developed a phase III placebo-controlled clinical trial to examine lenalidomide maintenance following transplant with Dr. Charles Linker, who has since retired from the University of California at San Francisco but was then chair of the transplant committee for CALGB, the cooperative group now known as the Alliance for Clinical Trials in Oncology.

I’m very grateful to the Alliance for Clinical Trials in Oncology for sponsoring this work, to everyone at Roswell Park who played a role in our study and to Drs. Linker and Anderson. I am indebted to the 461 patients who volunteered to be a part of our study and to all patients, families and clinicians who participated in the earlier-phase studies that preceded it. Thousands of other people with multiple myeloma have benefitted because they chose to participate in a study of an agent whose effectiveness had not yet been demonstrated.

There’s still no cure for multiple myeloma, and lenalidomide isn’t appropriate for all patients. We continue to see more and better treatment options. In 2015 alone, there were five new multiple myeloma drugs approved, which was very exciting. As one of my colleagues, Dr. Khalid Al Hashmi, a senior hematologist from Oman, has noted, “Before the new drugs, treating multiple myeloma was like waiting for a taxi and none would come. Then all of a sudden, five come at once.” The challenge now is to figure out what is the best way to use these new drugs – when to give them, what combinations work best and who are the patients who benefit most from particular therapies.

I’m eagerly looking forward to the next big treatment breakthrough. I am optimistic that I’m going to see a cure for at least some multiple myeloma patients while I’m still practicing. I hope I’m right.

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