As the most common childhood cancer in the United States, leukemia affects about 3,100 patients under the age of 20 each year. Fortunately, that number could decrease very soon, thanks to the Food and Drug Administration’s recent approval of a groundbreaking new cancer treatment.
The immunotherapy, also called CAR T-cell therapy, Kymriah, or CTL019, treats patients with pediatric acute lymphoblastic leukemia. By modifying a person’s cells, it can direct the body’s immune system to attack the cancer cells. (You shouldn’t ignore these silent signs of leukemia.)
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “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.”
A three-month trial of the treatment produced remission in 83 percent of 63 pediatric and young adult patients, prompting FDA approval. They intend for the drug to be used for patients under age 25 who do not respond to standard treatment or have suffered relapses. However, the treatment does risk causing a dangerous overreaction from the patient’s immune system, known as cytokine-release syndrome. The FDA has required strong warnings for the treatment as a result.
At the moment, the treatment will only be available at 32 hospitals and clinics that have received special training to deliver the therapy. A one-time treatment would cost $475,000 for patients who respond, but patients who do not respond to the treatment within a month will not be charged. The company is looking into making the drug affordable to everyone who needs it, NPR reported.
Regardless of its limitations at the moment, the development looks promising for the future of cancer treatment. “I think this is the most exciting thing I’ve seen in my lifetime,” said Dr. Tim Cripe, an oncologist who was part of the FDA advisory committee panel that voted in July to recommend the drug’s approval.
Hopefully these hopeful cancer statistics will spread some good cheer, as well.