The U.S. made the first gene therapy available in the country on Wednesday, a historic move that could benefit treatment for cancer and other serious diseases.
“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,” FDA Commissioner Dr. Scott Gottlieb said in a statement.
The drug in question — Kymriah — is a cell-based gene therapy that is customizable based on a patient’s T-cells, a type of white blood cell called a lymphocyte. It’s approved for pediatric and young adult patients up to age 25 who have a form of acute lymphoblastic leukemia, the most common U.S. childhood cancer.
These T-cells, once taken from a patient, go to a manufacturing center where they are genetically modified with a new gene that instructs the T-cells to kill specific leukemia cells. The modified T-cells then go back into the patient to prepare to fight the cancer.
The treatment will cost a seemingly hefty $475,000, though it’s not as expensive an analysts anticipated, reports STAT News.
Researchers performed a clinical trial on 63 pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The trial found that 83 percent went into remission within three months of treatment. The Associated Press notes certain patients relapsed months after.
“A far higher percentage of patients go into remission with this therapy than anything else we’ve seen to date with relapsed leukemia,” Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites, tells the Associated Press.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.
The FDA cautions “severe” side effects are possible. It’s adding a boxed warning to watch for development of cytokine release syndrome, “a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events,” according to a news release. These could both prove life-threatening.
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First Gene Therapy a ‘Historic’ Move for United States Cancer Treatment originally appeared on usnews.com
