Gene therapy for advanced lymphoma gets FDA approval

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This week, Gilead Sciences became the second company to receive approval for a chimeric antigen receptor T cell (CAR T) therapy after the US Food and Drug Administration (FDA) gave Yescarta the nod for the treatment of adult patients with relapsed or refractory large B-cell lymphoma. Each treatment of the T-cell therapy is customized using a patient's own immune cells, starting with T cells collection via apheresis, shipment to a manufacturing facility, and genetic modification of the cells to express a auto specific to CD19, which is commonly over-expressed in B cell malignancies allowing the modified immune cell to target and kill the lymphoma cell. They are extracted, genetically engineered to recognize cancer cells, and infused back into the patient, where they form an army to attack and destroy malignant cells, potentially for years. The treatment, called Yescarta, is the first immunotherapy on the USA market for certain kinds of non-Hodgkin lymphoma (NHL), a cancer that attacks the lymph nodes. The FDA has linked a risk evaluation and mitigation strategy with the approval: hospitals and their associated clinics that dispense axicabtagene ciloleucel need to be specially certified.

The price of Yescarta is set below that of comparable treatments, according to Reuters, as Novartis AG's gene therapy costs $475,000. The FDA cautions that the drug is not indicated to treat patients with primary central nervous system lymphoma. Yescarta is also said to have a boxed warning on its product label regarding the risks of cytokine release syndrome and neurologic toxicities. It uses the same technology, called CAR-T, as the first gene therapy approved in the August, a treatment for childhood leukemia from Novartis Pharmaceuticals. "We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms".

All patients had chemorefractory disease and had received a median of 3 prior lines of therapy, with 54% refractory to 2 consecutive lines of therapy. "In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer", said FDA Commissioner Scott Gottlieb, MD.

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To start, a doctor removes some white blood cells, the part of our body's immune system responsible for combatting infections and foreign substances, from a patient.

Michaela Almgren, a clinical assistant professor at University of South Carolina College of Pharmacy, pointed out this will not be available in just any facility due to the many challenges surrounding extraction of a patient's T-cells, adjustment of genes, and reengineering the cells in sufficient quantity. "This approval would not have been possible without the courageous commitment of patients and clinicians, as well as the ongoing dedication of Kite's employees. Now, thanks to this new therapy many patients are in remission for months".

"The FDA approval of Yescarta is a landmark for patients" who qualify for the treatment, which is aimed at patients with the non-Hodgkin's form of the blood cancer who had other unsuccessful treatments, Kite founder Dr. Arie Belldegrun said in a statement.

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The treatment was first developed at the National Cancer Institute, led by Dr. Steven Rosenberg, who signed an agreement with Kite Pharma in 2012.

The FDA decision opens the door for a new gene therapy to treat adults with aggressive lymphoma, noted Maloney.

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