The U.S. Food and Drug Administration on Wednesday approved the first gene therapy in the United States, to treat children and young adults with a particularly tough type of leukemia.

Kymriah (tisagenlecleucel) is for B-cell acute lymphoblastic leukemia (ALL), the agency said in a news release.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Dr. Scott Gottlieb. “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.”

Each dose of Kymriah is created using the person’s own immune system T-cells. These cells are lab-modified and then reinfused into the patient to target and kill leukemia cells, the FDA explained.

ALL is an aggressive cancer that affects some 3,100 people aged 20 and younger each year in the United States, making it the nation’s most common childhood cancer. Kymriah is intended for people with B-cell ALL whose cancer hasn’t responded to or has returned after treatment, the FDA said.

The therapy was evaluated in clinical testing involving 63 children and young adults with relapsed or refractory B-cell ALL. Some 83 percent of those given the therapy were in remission within three months of treatment, the agency said.

Potential side effects of Kymriah include cytokine release syndrome, a bodily response characterized by high fever and flu-like symptoms. Other possible severe side effects could include serious infection, low blood pressure, kidney injury, fever and decreased oxygen. Most of these symptoms should appear within three weeks of infusion with Kymriah, the FDA said.

Due to these and other potential risks, the agency said it is requiring hospitals that administer the therapy to be specially certified. The drug’s maker, Novartis, is required as a condition of approval to conduct a longer-term study of Kymriah’s use, the agency said.

Novartis is based in Switzerland.

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Source: HealthDay