USA regulators on Wednesday approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults. The T cells are filtered from a patient's blood, reprogrammed to target and kill cancer cells, and then hundreds of millions of copies are grown. These are then sent to a facility in El Segunda, California - which Kite opened in June past year - where they are stimulated to proliferate and transduced with a retroviral vector to introduce the auto sequence into the patient's T cells before being propagated in cell culture bags.
CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer can often evade. Hospitals that provide the therapy will have to be specially certified, the FDA said.
The treatment is based on CAR-T therapy, which was developed by Israeli researchers at the Weizmann Institute of Science and the Tel Aviv Sourasky Medical Center. To further evaluate long-term safety, FDA is also requiring that the manufacturer conduct a post-marketing observational study on patients treated with the therapy.
Yescarta has a boxed warning regarding the risks of cytokine release syndrome (CRS) and neurological toxicities.
Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, said in a statement, "The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options-those adults with certain types of lymphoma that have not responded to previous treatments". An estimated 72,000 new cases are diagnosed each year in the US.
Diffuse large B-cell lymphoma is the most common aggressive non-Hodgkin lymphoma, roughly three out of every five cases.
"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", FDA Commissioner Dr. Scott Gottlieb said in a statement.
U.S. regulators have approved a second form of gene therapy to fight cancer.
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.
Dr. Arie Belldegrun, founder of Kite, recognized "the FDA for their ability to embrace and support transformational new technologies that treat life-threatening illnesses". "We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms". The therapy was evaluated in a multicenter clinical trial involving more than 100 adults, where the complete remission rate after treatment with Yescarta was 51 percent, the FDA said. In the United States each year, approximately 7,500 patients with refractory DLBCL are eligible for CAR-T therapy.
Almgren said the approval of Yescarta is "certainly very exciting".
People with advanced lymphoma now have another type of treatment to consider. The therapy is not indicated for treating primary central nervous system lymphoma.