I don’t remember her name anymore. She was 8 years old: blonde, pretty, smiling politely. I remember her parents, older than most, looking concerned. She looked too healthy to be the source of their concern, but as I moved close to her, I could see the petechiae on her cheeks. In three weeks, she was dead.

ALL (acute lymphoblastic leukemia) has come a long way since then. About 80% of children respond to chemotherapy (induction, consolidation, and maintenance). But nonresponders and those who relapse have a 2-year survival rate of only about 15%. Stem cell transplantation may be curative, but comes with its own difficulties in implementation and severe toxicity.

Now these patients have another option. Tisagenlecleucel (Kymriah) is not really a drug. It is a form of immunotherapy that uses the patient’s own genetically modified T cells to attack their leukemic cells. In a clinical trial in children, adolescents, and young adults with refractory or relapsed ALL, half of whom had had stem cell transplants, 83% achieved a complete remission, and some had no signs of residual disease. This unique treatment often causes a severe reaction that has its own unique antidote, the IL-6 inhibitor tocilizumab (Actemra).

There is, of course, much more to say about this exciting treatment, which may also have applications in other types of malignancies. A review will appear in the next issue (1532; October 23, 2017) of The Medical Letter.

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  1. When I was an intern, one of my first patients had ALL, and I was told at the time that he would likely die soon. He did. I’m delighted that half a century later, we have drugs and methodology to use against this and so many other diseases.

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