The US FDA (Food and Drug Administration) has approved a new-generation blood cancer treatment.
Named Yescarta, the treatment is a cell-based gene therapy. It’s designed for diffuse large B-cell lymphoma (DLBCL) patients, of which the US has between 20,000 and 25,000.
To be eligible to receive the new drug, patients must be aged 18-plus. They must also have tried – and not benefitted from – two or more previous DLBCL treatments. Primary central nervous system lymphoma patients are specifically excluded from the approval.
A Living Drug
Yescarta is categorised as a CAR (chimeric antigen receptor)-T cell therapy and labelled a so-called “living drug.” Supplied as a one-time-shot, administered straight into the vein, it costs $373,000 a time. Each shot is produced specially for that patient, as it draws on his/her own immune system. T-cells (white blood cells) are harvested and genetically engineered, to give them a new lymphoma-fighting gene. Via infusion, these cells are then placed right back inside the patient and get to work.
Yescarta is produced by Kite Pharma, owned by pharmaceutical colossus Gilead. The parent firm’s shares leaped four per cent on the FDA approval news. Approval followed a clinical trial, in which some 100 adults participated. This Yescarta clinical trial’s overall post-treatment remission rate was 51 per cent.
Associated side effects for this “living drug” include weakened immune systems, reduced blood cell counts and the potential for serious infections to take hold. These tend to manifest within the first two weeks after infusion.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases”, states Scott Gottlieb, FDA Commissioner, in the administration’s Yescarta approval news release. "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.
“This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products”, Gottlieb continues. “We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies.”
“We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”