In what feels like back-to-back news, the U.S. Food and Drug Administration (FDA) has approved two gene therapies for cancer. The actual announcements happened seven weeks apart, but for patients, manufacturers, investors and shareholders, it’s like Christmas – over and over again.
As you probably know, Novartis’ Kymriah was approved at the end of August to treat pediatric patients with acute lymphoblastic leukemia (ALL). On Wednesday (October 18), Kite’s Yescarta was given the green light to treat adults with relapsed or refractory large B-cell lymphoma, including aggressive non-Hodgkin lymphoma, if they have failed two or more traditional treatments, such as chemotherapy or a bone marrow transplant. (Kite is now owned by Gilead Sciences.)
(On October 12, the FDA looked likely to approve Luxturna, from Sparx Therapeutics Inc., a treatment that could stop patients from going blind. It would be the first gene therapy approved to treat an inherited condition. The FDA has until January 12, 2018, to make a final decision. You can read about it here.)
Both of the approved treatments rely on Chimeric Antigen Receptor modified T cells (CAR-Ts), which BioCanRx explains here. At Signals, we’ve been paying attention to CAR-Ts for years, along with the companies in this space. One of them, Kite, has always looked promising. Bluebird bio is another company we’ve written about often.
Nick Leschly, CEO bluebird bio, is featured in a TEDx Talk on gene therapies and how they are being used to treat patients with specific diseases, like Adrenoleukodystrophy (ALD) – the childhood disease that usually affects males and was featured in the movie Lorenzo’s Oil. Watch this compelling talk to learn more about gene therapies and how they work, and to hear about reimbursement/payer issues (a topic for another time).
Here’s a short video on how gene therapies work, from bluebird. A longer version is included in the TEDx Talk, above.
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