July 13 (UPI) -- A first-ever treatment to genetically alter a patient's own cells, to fight relapsed B-cell acute lymphoblastic leukemia, or ALL, is one step closer to approval by the U.S. Food and Drug Administration.
A Food and Drug Administration panel on Wednesday unanimously recommended the agency approve the use of the treatment known as CTL019, or tisagenlecleucel, for use on patients with relapsing ALL.
The drug company Novartis presented its case for the treatment to be approved by the FDA for use on patients.
Approximately 620 pediatric and young adult patients with ALL relapse after achieving an initial response each year in the United States despite current treatments. The prognosis for these patients is very poor, especially in patients who have relapsed more than two times or who relapse following allogenic stem-cell transplants.
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CTL019 involves removing millions of a patient's T-cells and genetically engineering them to kill cancer cells by using a disabled form of HIV to carry new genetic material into the T-cells to reprogram them.
This super charges the T-cells to attack B-cells, which are immune cells that turn malignant in leukemia. The genetically-altered T-cells are then injected back into the patient so they can multiply and fight the cancer.
Each treatment is created for each individual patient by having their cells removed, frozen and shipped to a Novartis plant for processing.
One of the first patients to receive this type of treatment is Emily Whitehead, 12, who first received the treatment at age 6 in 2012 for ALL.
Pediatric oncologist Dr. Stephan A. Grupp, director of the Translational Research for the Center for Childhood Cancer Research at The Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania said the team reprogrammed the girl's own immune cells to attack an aggressive form of childhood leukemia.
"These engineered T-cells have proven to be active in B-cell leukemia in adults," Grupp said in a statement in 2012. "Our hope is that these results will lead to widely available treatments for high-risk B-cell leukemia and lymphoma, and perhaps other cancers in the future."
Novartis presented the findings from a study of 63 patients who received the treatment from April 2015 to August 2016. Approximately 82.5 percent of patients treated achieved remission.
