Gene Therapy a New Milestone in Immunotherapy

CAR T-cell therapy is a new type of immunotherapy that uses the patient’s genetically modified immune T-cells to attack cancer cells. In 2017, the FDA approved the first 2 CAR T-cell therapies for several types of blood cancer.
December 2017 Vol 3 No 6
Dana Taylor

New developments in immunotherapy in the past 2 decades have transformed the treatment of cancer. In the words of Bruce Gershenhorn, DO, Medical Director of the Lung Center and Medical Oncologist, Cancer Treatment Centers of America, “Immunotherapy harnesses the power of the body’s own immune system, teaching it how to identify, recognize, and kill cancer cells that are otherwise overlooked and not treated.”

CAR T-cell therapy is a new type of immunotherapy that uses the patient’s genetically modified immune T-cells to attack cancer cells. In 2017, the FDA approved the first 2 CAR T-cell therapies for several types of blood cancer.

First Gene Therapy

In August 2017, the FDA approved Kymriah (tisagenlecleucel; from Novartis) for young patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), a CAR T-cell immunotherapy. Kymriah was the first gene therapy ever to receive approval by the FDA, ushering in a new type of immunotherapy for patients with cancer.

Kymriah is the first drug that uses the patient’s genetically modified immune T-cells to target the leukemia cells associated with CD19 antigens. These reprogrammed cells are then put back into the patient to fight cancer cells.

Commenting on this new milestone, FDA Commissioner Scott Gottlieb, MD, said, “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer. New technologies such as gene and cell therapies hold out the potential to transform medicine.”

Peter Marks, MD, PhD, Director of the FDA’s Center for Biologics Evaluation and Research, noted, “Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease.”

In the clinical trial that led to its approval, 83% of patients who received Kymriah showed positive response to therapy within 3 months.

Although highly effective, like most immunotherapies, Kymriah is associated with serious side effects, including cytokine-release syndrome and neurologic effects, which could be life-threatening.

Second Gene Therapy

A few weeks later, in October 2017, the FDA approved the second gene therapy, Yescarta (axicabtagene ciloleucel; from Kite Pharma), another CAR T-cell immunotherapy, for the treatment of adults with several types of relapsed or refractory large B-cell lymphoma (after 2 or more lines of therapy), including unspecified diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,” Dr. Gottlieb said.

“The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options––those adults with certain types of lymphoma that have not responded to previous treatments,” added Dr. Marks.

As with Kymriah, Yescarta may cause serious, potentially life-threatening side effects, including cytokine-release syndrome and neurologic toxicities that must be reported immediately.

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Last modified: January 29, 2018

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