Chimeric antigen receptor T-cell (CART) therapy: current data, future potential


An article in today’s issue of the New England Journal of Medicine reports data from a Phase II trial of CART therapy in the treatment of patients with acute lymphoblastic leukemia or ALL (an aggressive and difficult to treat form of cancer). This is arguably one of the most exciting papers on the treatment of cancer in the past 50+ years.

We have mentioned the development and evolution of CART therapy a few times before, but these are the first data from a carefully structured clinical trial designed to test for effectiveness and safety. The results clearly confirm the earlier experience.

Maude et al. report that

  • 30 children and adults received the experimental form of CART therapy known as CTL019.
  • 27/30 patients (90 percent) responded with a complete remission of their cancer.
  • The overall survival rate was 78 percent at up to 24 months.
  • At 6 months of follow-up, 68 percent of patients continued to exhibit the presence of CTL019 in their blood stream (which is a good thing).

However, from a side effect perspective,

  • All patients exhibited the cytokine-release syndrome.
  • 27 percent of patients exhibited severe cytokine-release syndrome, which was associated with a higher disease burden before infusion but could be effectively managed with the anti–interleukin-6 receptor antibody tocilizumab.

This level of response to CART among patients who had all received aggressive, prior forms of treatment for their ALL is unprecedented. However, it is clear that treatment does come with risks for serious side effects.

The key questions now are going to be: (a) whether we will see true long-term survival of these patients; (b) whether we can appropriately manage and minimize the risks for side effects in the use of CART; and (c) whether disease-specific forms of CART can be engineered and shown to be effective and safe in other forms of cancer — high-risk forms of prostate cancer included.

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