FDA approves CAR T-cell therapy for treatment of acute lymphoblastic leukemia


Within the past few hours, the US Food and Drug Administration (FDA) has approved the very first form of chimeric antigen receptor (CAR) T-cell therapy for the treatment of cancer — and this is also the first true form of gene therapy to be made available here in the US.

Now this is going to have no immediate impact on the treatment of solid tumors like prostate cancer, but this is another real “first” in cancer therapy and it may well have implications for the treatment of solid tumors down the road.

The drug approved is known as tisagenlecleucel or Kymriah®, but you are more likely to have heard of it being referred to as CTL019. It is made by Novartis and it is exclusively being approved, initially, for the treatment of selected children and young adults with a particular form of relapsed or refractory blood cancer known as “relapsed or refractory B-cell precursor acute lymphoblastic leukemia”.

When this form of treatment works, it can work very, very well indeed, but it also comes with significant risk for a set of serious side effects called cytokine release syndrome or CRS that can rapidly become life threatening. However, luckily, it appears that CRS can be managed very effectively in about 70 percent of patients by using a drug originally developed for the treatment of rheumatoid arthritis that is known as tocilizumab (Actemra®). The FDA has approved the use of Actemra for the treatment of CRS today as well.

For additional information see the FDA’s media release. We understand that treatment with Kymriah is going to cost $475,000. However, it is said that Novartis “is working with Medicare on a system in which the government would only pay for CAR-T treatment if patients respond within a month.”

2 Responses

  1. For anyone ….. I realize this employs CAR cells, however is it similar in any way, and how does it differ to sipileucel-T (Provenge) and dendritic cells for solid tumors?

  2. Rick:

    This is a very different type of immunotherapy (although the process of treatment is similar). Here’s a link to an introduction.

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