More than 20 years ago, for a high school science project, I gave a presentation to my biology class on gene therapy. To me, the idea of editing a person's genes to cure disease seemed wildly futuristic, but by the time I'd finished my project, I was convinced it would someday be possible.
In an important respect, that "someday" arrived this week when, on Aug. 30, the U.S. Food and Drug Administration approved Kymriah, the first gene-therapy treatment to reach this milestone. Kymriah will be used to treat acute lymphoblastic leukemia, the most common pediatric cancer, in patients whose disease returns after they complete conventional chemotherapy. In the past, these patients have usually died.
The treatment's approval was heralded with excitement from experts, including those at Stanford, as the Los Angeles Times reported:
FDA Commissioner Scott Gottlieb, himself a survivor of blood cancer, predicted that this new approach to cancer treatment will 'change the face of modern medicine.'
Cancer researchers and physicians outside the agency shared Gottlieb’s enthusiasm.
Dr. Crystal L. Mackall, associate director of Stanford University’s Cancer Institute, called Kymriah 'a transformative therapy. … It represents an entirely new class of cancer therapies that holds promise for all cancer patients.'
Kymriah is a therapeutic approach where some immune cells, called T cells, are removed from the patient's blood, genetically engineered to recognize cancer cells, and returned to the patient to selectively bind to and kill the cancer. The LA Times piece includes a great infographic that explains the process. The story also reports that Novartis, Kymriah's manufacturer, has announced its intention to make the treatment available in 32 certified treatment centers by the end of 2018.
Several Stanford scientists are also doing research in gene therapy and cancer immunotherapy to refine and expand the ways these strategies be used. When I heard Mackall describe her work at a scientific meeting at Stanford earlier this year, she talked about the shift that this approach to cancer represents: “Historically, we would go after tumors with poisons or targeted agents... What has changed is that we can also go after the host and change the way the host responds to the tumor.”
Previously: Stanford scientists describe stem cell and gene therapy advances at scientific symposium, New center to advance cancer cell therapy and Exploring the promise and challenges of cancer immunotherapy
Scanning electron micrograph of a human T cell by NIAID