Breakthrough in Childhood Leukemia Treatment: New Drug Combination Shows Promise

Researchers at the University of Cambridge have developed a promising new treatment approach for B-cell acute lymphoblastic leukemia (B-ALL), the most prevalent form of childhood cancer. This innovative therapy involves the combined use of two oral drugs, venetoclax and inobrodib, which together could significantly reduce the reliance on traditional chemotherapy, known for its toxic side effects.

Currently, treatment for B-ALL in children requires over two years of intensive chemotherapy, which can lead to severe side effects such as risk of infections, hair loss, nausea, bleeding, and long-term organ damage. Although many younger patients are cured, outcomes for older children and adults are often less favorable. Existing alternatives like bone marrow transplants and immunotherapies like CAR-T cells also pose challenges due to their side effects and cost.

The new approach, detailed in the journal Nature Communications, aims to be more effective across all age groups and less harmful. B-ALL is characterized by an overproduction of primitive B-cells that originate in the bone marrow, invade other parts of the body, and hinder normal blood cell production. These malignant cells can hide within the body, making treatment difficult.

The research team discovered that shutting down a gene called CREBBP—which often mutates during disease progression—rewires B-cell metabolism. When combined with venetoclax, which targets the BCL2 protein to induce cell death, this strategy triggers ferroptosis, a form of programmed cell death caused by lipid membrane damage. This dual therapy effectively kills off early-stage B-cells, including those resistant to single-drug treatments, in both human and mouse models.

Experts believe this drug combination could offer a safer, more cost-effective alternative to current therapies. Since both drugs are already in use or in trial phases for other cancers, the team is optimistic about initiating clinical trials in teenagers and adults with B-ALL soon. Dr. Simon Richardson emphasized that this approach could revolutionize treatment by minimizing long-term side effects while improving outcomes.

The study highlights the importance of targeting specific genetic and metabolic pathways in cancer treatment, paving the way for personalized and less toxic therapies for children and adults battling leukemia.

Source: https://medicalxpress.com/news/2025-05-scientists-treatment-potential-tackle-common.html