Researchers identify a new therapeutic vulnerability in one of the most aggressive forms of childhood leukemia

An international research team led by the Josep Carreras Leukaemia Research Institute and the Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENyO) has identified a new therapeutic vulnerability in one of the most aggressive subtypes of childhood leukemia. The findings, published in the journal Blood, open the door to the development of future treatment strategies for patients who currently have very limited therapeutic options.

The research focuses on KMT2A-rearranged B-cell acute lymphoblastic leukemia (B-ALL), a particularly aggressive disease that mainly affects infants and very young children. Although childhood leukemia generally has high survival rates, this subtype remains associated with a high risk of relapse and a significantly poorer prognosis than other forms of the disease.

The study identified a new weakness in these leukemic cells: the loss of the HDAC7 protein, which is essential for the normal development of B lymphocytes. Researchers demonstrated that restoring its activity through a combination of experimental drugs and conventional chemotherapy promotes the maturation of cancer cells, reduces their ability to grow, and increases their sensitivity to treatment.

The findings, obtained using cell models, animal models and patient samples, also showed a reduced capacity of leukemic cells to trigger relapse, one of the greatest clinical challenges in this type of leukemia. Although further research will be needed before this strategy can be translated into clinical practice, the results represent an important step towards more precise and effective treatments for patients with the poorest prognosis.

This work once again highlights the crucial role of research in advancing the fight against leukemia. Every new scientific discovery brings us closer to more effective and personalized treatments and, above all, represents new hope for children and families living with this disease. Because behind every scientific breakthrough lies a shared goal: making leukemia a curable disease for every patient, one day.

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