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FDA Approves Landmark New Gene Therapy For Non-Hodgkin’s Lymphoma

October 26, 2017

The Food and Drug Administration last week approved the second revolutionary gene-altering treatment that turns cells into “living drugs” to kill cancer.

The FDA has approved Yescarta ™ (axicabtagene ciloleucel) developed by Kite Pharma, A Gilead Company, for the treatment of adults with aggressive forms of non-Hodgkin’s lymphoma after progression of their disease through other treatments.  Earlier this year, The FDA approved Kymriah (tisagenlecleucel) developed by, Novartis, Inc., for pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia.

“This latest FDA approval of Yescarta is a landmark moment in cancer care and for cancer immunotherapy,” says Omar Eton, MD, FACP, Medical Director of the Phase 1 Clinical Trials Unit for the Hartford HealthCare Cancer Institute. “This approval is the culmination of efforts of dedicated scientists with great vision and perseverance in designing a first-in-class biologic treatment tailored for each individual patient using their own modified immune cells.  This treatment can, in one dose, reduce some of the most deadly relapsed and refractory lymphomas which ordinarily carry a dismal prognosis.” 

Under the study, 72 percent of patients treated with just a single infusion of Yescarta responded to treatment, including 51 percent of patients who had no detectable cancer remaining, representing a complete remission rate between 41 – 62 percent, says Doctor Eton.

During the treatment, millions of a patient’s own T-cells are removed and frozen.  The frozen T-cells are then sent off to Kite Pharma and are reengineered to allow the patient’s own immune system to detect and kill cancer cells.  After being reprogrammed, the genetically modified T-cells are again frozen and sent back to the cancer center where they are infused back into the patient.

There have been some serious side effects in some patients including high fever, headache, tremor, dizziness and crashing blood pressure.

“There are unique side effects that were predicted and are apparent with Yescarta™ which unleashes the power of a patient’s own immune system against the cancer,” says Eton. “The treatment must be done by oncologists who have been specially trained and certified to manage the potential side effects of this novel form of immunotherapy.”

Peter Yu, MD, Physician-in-Chief, Hartford HealthCare Cancer Institute, points out that CAR-T cell treatments are complex cell-based treatments that are in many ways similar to bone marrow transplants. He says while this particular treatment is not yet available to Hartford HealthCare Cancer Institute patients, the Institute’s membership in the Memorial Sloan Kettering (MSK) Cancer Alliance will mean access to these cell-based treatments  in the future.

“Through our partnership with Memorial Sloan Kettering Cancer Center, we are able to identify which of our Hartford HealthCare patients would most likely benefit from cell-based therapies, connect them to the MSK team and facilitate their earlier return back to Connecticut where we will continue to provide follow-up care in close collaboration with MSK providers,” says Yu.  “With our partners at MSK, we are working on the technology transfer that will allow us to deliver cell based-therapies here at Hartford HealthCare in the future, much as we already are doing with our Phase 1 Clinical Trials Unit which brings the latest new drugs in the development pipeline to our patients right here in Hartford.”

The projected list price of the tailored treatment is $373 thousand. While the treatment might seem quite expensive, Eton says it’s a “bargain” when compared to the costs of less effective and even more toxic approaches currently used to manage patients with relapsed or refractory non-Hodgkin’s lymphoma.

“This is just the beginning, as treatments like this are constantly being refined. CAR-T cell treatments will become ever more effective with less projected toxicity and cost and will prove to be applicable against a whole host of hematological malignancies,” says Eton. “Hopefully, dedicated scientists will be able to overcome certain challenges to be able to one day similarly help patients suffering from the more common solid tumors. We are closing in, at an ever faster pace, on better and better ways to harness a patient’s own immune response against cancer.”

Eton says oncologists at the Hartford Health Cancer Institute are already routinely treating patients with the latest immunotherapies, with the latest CAR-T therapies to come on line in the future thanks to the Institute’s affiliation with MSK.