The Food and Drug Administration — the American agency in charge of ensuring the safety of most of the things we ingest — has approved a radical new therapy for fighting cancer called Yescarta. It is a type of CAR T-cell therapy, a technique that genetically modifies the subject’s immune cells so that they can attack cancer (specifically, in this case, non-Hodgkin lymphoma) directly. This is only the second time it’s approved such a treatment. The first, back in August, was for leukemia that was non-responsive to more traditional drugs.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” FDA Commissioner Scott Gottlieb said in a statement. “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.”
Indeed, these new treatments could be boons for cancers that are traditionally stubborn or nearly impossible to treat. Diffuse large B-cell lymphoma is among the most common forms of non-Hodgkin lymphoma, and it can also be tough to treat. After a time, these cancers can become resistant to chemotherapy and other avenues of treatment.
With CAR T-cell, each patient gets a customized dose, designed to re-tool their immune cells. T-cells, which form one of the backbones of the immune system are collected and modified by a lab to target and attack cancerous lymphoma cells. After that, these cells are put back into the patient, where they work their magic.
“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 added. “We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine.”
That too, is important to note. While CAR T-cell could already save tens of thousands each year, we are getting into some potentially dicey territory. This marks some of the very first broadly approved genetic treatments in humans. We’ve taken those nascent steps into the world of modifying our own internal mechanics to cure and treat aggressive diseases. That’s not to say that it’s bad, just something to be aware of.
Like any tool of science, these mechanisms can be used for good or ill, and when you start tinkering with genes, it’s important that we get those steps absolutely right. It’s not any more or less natural than a transplant or crutch, even. Humans have been modifying anything and everything we can to combat medical maladies since time immemorial, but in an era where just about everything is hackable, it does raise questions about how secure all this is.
And that doesn’t even touch the expense of the treatment, which can run $373,000 for a round. Even so, Yescarta was able to bring complete remission for 51 out of 100 studied patients — a staggering result. It obviously carries some major risks, and, given the complexity, the FDA requires that hospitals that offer it be specially trained. What a world.
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