How are children’s lives affected now?

In children, teenagers and young adults with T-ALL, the bone marrow makes too many white blood cells called T-cells, which prevents the production of healthy blood cells. Treatment usually involves chemotherapy and aims to destroy the cancer cells and restore normal bone marrow function.

“Thankfully, most children are cured after treatment,” says Dr van Delft. “But unfortunately, the outlook for children whose cancer does not respond to treatment, or comes back later, is not as positive.”

Using higher doses of chemotherapy could potentially help cure more children with T-ALL, but this would greatly increase the risk of life-threatening side effects. An alternative approach is to develop new targeted drugs, such as those that work by blocking key proteins that help cancer cells survive treatment.

“Unfortunately, a drug that targets these survival proteins and has shown promise in clinical trials is no longer available for children with T-ALL,” says Dr van Delft. “The absence of this leaves doctors with even fewer treatment options and has slowed progress in developing new therapies.”

How could this research help?

“Our goal is to quickly identify a suitable replacement for this drug – for use in current and future clinical trials aimed at improving survival for children with the most aggressive forms of T-ALL,” says Dr van Delft.

The researchers have already identified some existing drugs that work in a similar way and could serve as potential alternatives, but these require further laboratory testing to confirm their safety and effectiveness.

“We will carry out a series of experiments in our established laboratory models of T-ALL to evaluate these drugs, both alone and in combination with existing treatments,” says Dr van Delft.”

The team hopes this project will allow them to select the most promising drug candidate to take forward into the clinic to benefit young patients as quickly as possible.

References

1. Cancer Research UK. Acute lymphoblastic leukaemia (ALL) statistics: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/leukaemia-all/incidence#heading-One [website accessed 06 January 2025].
2. Buckley, M. et al. T-cell acute lymphoblastic leukaemia: subtype prevalence, clinical outcome, and emerging targeted treatments. Leukemia. 2025;39:1294-130.
3. Amaral, P. et al. Underlying biology, challenges and emergent concepts in the treatment of relapsed and refractory pediatric T-cell acute lymphoblastic leukemia. Leukemia. 2025; 39(11):2575-2589.

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