Kymriah: First FDA-approved gene therapy for cancer

A medical breakthrough took place in the United States on Aug. 30, Wednesday. For the first time, the U.S. Food and Drug Administration approved a cell-based gene therapy treatment for cancer, which is called Kymriah (tisagenlecleucel).

Each dose of Kymriah is a customized treatment. The genetically-modified autologous T-cell immunotherapy uses an individual patient’s own T-cells or a lymphocyte, which is a type of white blood cell.

Kymriah is for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), a disease that progresses quickly. ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes, and is the most common childhood cancer in the U.S.

The cell-based gene therapy is approved in the U.S. for the treatment of patients aged 25 or younger with B-cell precursor ALL that is refractory or in second or later relapse. In a statement released by the FDA, FDA Commissioner Scott Gottlieb, M.D. 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 and create an inflection point in our ability to treat and even cure many intractable illnesses,” Gottlieb added. “At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”


The FDA’s Center for Biologics Evaluation and Research (CBER) director Peter Marks, M.D., Ph.D. explained that as a first-of-its-kind treatment approach, Kymriah “fills an important unmet need for children and young adults with this serious disease.” He added that aside from providing these patients with a new treatment option where very limited options existed, the cell-based gene therapy is also a treatment option with promising remission and survival rates in clinical trials.

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