Late last month, Jurassic Park actor Sam Neill put the treatment in the spotlight, revealing his stage three cancer was in remission after undergoing CAR T-cell therapy as part of a clinical trial in Sydney. He stopped short of describing his remission as a miracle – the success, he said, was “science at its best”

The history of CAR (for “chimeric antigen receptor”) T-cell therapy is one of small discoveries accumulating over decades, leading to major advances in patient care. Pioneered in the 1990s, the therapy has exploded in the past decade. Four CAR T-cell therapies have been approved by the Therapeutic Goods Administration for use in Australia since 2018. All are for blood cancers.

The success of those therapies is increasing enthusiasm among researchers and clinicians that CAR T-cell therapies will soon become a major weapon in the battle against cancer. It is now being tweaked to combat solid tumours, with promising early signs of success tempered by the difficulties in tailoring T-cells to find their target. The future may even see it become an injectable.

Scientists from the German Cancer Research Center (DKFZ) and the HI-STEM Stem Cell Institute have deciphered a key mechanism that contributes to treatment failure in acute myeloid leukemia (AML). They show that there are not just one, but four different subtypes of leukemia stem cells. This diversity could explain why one of the most important AML drugs does not work sufficiently in some patients or loses its effectiveness over time—resulting in the return of leukemia.

This discovery lays an important foundation for more precise and long-term successful treatment strategies that could specifically overcome resistance mechanisms. The findings are published in the journal Cell Stem Cell.

Acute myeloid leukemia (AML) is an aggressive form of blood cancer that primarily affects older people and often has a poor prognosis despite improved therapies. In recent years, the targeted drug venetoclax has significantly improved treatment. In combination with other drugs, venetoclax often shows good therapeutic success in AML and will, at least in part, replace highly aggressive chemotherapy in the future. However, AML returns in nearly all patients—usually because individual cancer stem cells become resistant to the drug.