A new treatment is giving hope to young people with a certain aggressive form of leukemia.
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The immunotherapy, called CAR T-cell therapy, was approved by the Food and Drug Administration last year for use in children and young adults up to age 25 who have relapsed or refractory B cell acute lymphoblastic leukemia (ALL).
“Relapsed or refractory” means that previous treatments either did not work on the cancer, or it worked but the cancer eventually came back. Relapsed ALL is among the leading causes of cancer death in children, according to the American Cancer Society.
“Up until now, we have had no other treatment to offer these patients who had grim prognoses,” says pediatric hematologist-oncologist Rabi Hanna, MD. “It is very exciting, and we see this as the first of many cellular therapies to come for specific types of leukemia.”
How CAR T-cell therapy works
The therapy involves removing certain immune system cells (T cells) from a patient’s blood and re-engineering those cells in a lab so that they can produce a gene that targets a particular receptor on cancer cells. Those engineered cells are then infused back into the patient’s blood through an IV, where they multiply and get to work finding and destroying cancer cells in the body.
“The key thing is that this treatment is personalized to the patient,” Dr. Hanna says. “We are able to obtain the T cell – the immune cell – the ‘soldiers’ of the body.”
Called Kymriah®, the treatment is the first of its kind available in the U.S. But it comes with significant risks and can only be administered by certain certified providers.
Patients may experience life-threatening side effects such as cytokine release syndrome (CRS), which can cause flu-like symptoms, shortness of breath, low blood pressure and rapid heartbeat. They might also lose their ability to produce immunoglobulin (proteins that fight off foreign cells) in the blood, leaving them at risk for infection, Dr. Hanna says. Some patients may need monthly infusions of intravenous immunoglobulin to counter this.
But CAR T-cell therapy lasts inside the body, so it only needs to be given once.
“It will save lives,” Dr. Hanna concludes. “Leukemia affects many organs and involves many specialists, and we believe this is going to be important to providing the very best care to our pediatric patients moving forward.”