The U.S. Food and Drug Administration (FDA) on Wednesday gave a green light to Novartis AG's leukemia treatment, marking the first time that a highly anticipated new type of potent gene-modifying immunotherapy has gained approval in the United States.
Kymriah, a cell-based gene therapy known as a CAR-T drug, was approved to treat patients up to age 25 with what's called B-cell acute lymphoblastic leukemia.
Kymriah is a type of personalized immunotherapy known as CAR-T, or chimeric antigen receptor T-cell therapy.
"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", said FDA Commissioner Scott Gottlieb.
"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement.
What's perhaps most exciting about CAR-T is that it operates similarly to a vaccination, providing the patient with lifetime protection against the targeted cancer.
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When the study began, the participants completed a survey about their lifestyle, overall health, diet and coffee consumption. For the study, researchers investigated the association between coffee consumption and risk of death.
As such, each dose of Kymriah is customized to the individual patient's T-cells through genetic modification.
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. It is caused by overactive genetically modified immune cells.
"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER), in the FDA's post. "At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving". Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year.
The one-time treatment has a boxed warning for cytokine release syndrome or CRS, a life-threatening side effect that can cause blood pressure to drop dangerously low.
According to the study cited by the FDA, 83 percent of the 63 patients went into remission. The FDA also released a treatment for these side effects on Wednesday. Slightly fewer patients experienced neurological events, such as seizures and hallucinations.