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A new therapy could improve the response to childhood acute lymphoblastic leukaemia

The Development and Lymphocytic Diseases group at the Josep Carreras Leukaemia Research Institute, led by Dr. Maribel Parra, has identified a new drug combination that can improve the prognosis of a highly aggressive type of paediatric leukaemia characterized by a poor response to conventional therapy. The therapeutic strategy proposed by the research team reduces the aggressiveness of leukemic cells and increases their response to glucocorticoid-based chemotherapy.

A new therapy could improve the response to childhood acute lymphoblastic leukaemia
A new therapy could improve the response to childhood acute lymphoblastic leukaemia

Pro-B-cell acute lymphoblastic leukaemia (pro-B-ALL) is the most common type of leukaemia in infants. It is characterized by the presence of immature B lymphocytes in the bone marrow. Although survival is generally high, one in ten cases presents a chromosomal abnormality, coded as t(4;11), which causes patients to relapse and survival rates to fall below 40%. While the incorporation of new therapeutic strategies based on immunotherapy has improved the prognosis, we are still far from offering an effective solution for the 500 children under one year of age who suffer from this disease each year in Europe.

The research, led by Dr. Oriol de Barrios with the collaboration of research groups from the Netherlands, Italy, Germany, and the group of Dr. Pablo Menéndez, also from the Josep Carreras Institute, has just demonstrated that insufficient amounts of the HDAC7 protein in leukemic cells are a key factor in the poor prognosis of this disease. Thus, they propose a new therapy based on the drugs MI-538 and chidamide, capable of increasing HDAC7 expression within leukemic cells. Such treatment improves their maturation, reduces their aggressiveness, and increases their sensitivity to conventional chemotherapy in the laboratory.

The results, recently published in the specialized journal Biomarker Research, were obtained using cells derived directly from patients with pro-B-ALL t(4;11) leukaemia and in mouse preclinical models. Under these conditions, when MI-538 and chidamide was added to the treatment, the leukemic potential of the cells was reduced and the onset of potential relapse, delayed.

Dr. de Barrios emphasizes that "the results of the study may contribute to improving the prognosis of infants diagnosed with pro-B-ALL t(4;11)" and adds that "by combining these drugs in the first line of treatment, the doses of conventional therapy could be reduced, thus reducing the potential side effects, improving the quality of life of patients and their families."

This study has received funding from the Ministry of Science and Innovation, the Generalitat de Catalunya, the Deutsche José Carreras Leukämie Stiftung, the European funding program Fight Kids Cancer, and the Cris Cancer Foundation. No generative AI tools have been used in the preparation of this article.

Reference article: de Barrios O, Ocón-Gabarró I, Gusi-Vives M, Collazo O, Meler A, Romecín PA, Martínez-Moreno A, Tejedor JR, Fraga MF, Schneider P, Bardini M, Cazzaniga G, Marschalek R, Stam RW, Bueno C, Menéndez P, Parra M. “HDAC7 induction combined with standard‑of‑care chemotherapy provides a therapeutic advantage in t(4;11) infant B‑cell acute lymphoblastic leukemia”. Biomark Res 2025; 13:99 doi:10.1186/s40364-025-00810-1.



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