Researchers linked to the University of Surrey report that forskolin, a plant-derived compound, can slow the growth of KMT2A‑rearranged acute myeloid leukaemia cells in the lab and increase their sensitivity to the chemotherapy drug daunorubicin. The findings, from a study published in the British Journal of Pharmacology, suggest a possible way to make existing treatments more effective, though further research is required before any change to clinical practice.
A laboratory study involving scientists from the University of Surrey and other institutions reports promising results for treating KMT2A‑rearranged acute myeloid leukaemia (KMT2A‑r AML), an aggressive form of the disease.
The research, led by Yoana Arroyo‑Berdugo and colleagues and published online ahead of print in the British Journal of Pharmacology in August 2025 (DOI: 10.1111/bph.70158), examines the effects of forskolin, a natural compound derived from the plant Coleus forskohlii, on KMT2A‑r AML cells.
According to the study and a summary from the University of Surrey, forskolin activates Protein Phosphatase 2A (PP2A) and reduces the expression of several cancer‑linked genes, including MYC, HOXA9 and HOXA10, in KMT2A‑r AML cells. This combination of actions was found to arrest proliferation and induce cell death in these leukaemia cells, indicating direct anti‑leukaemic activity.
The researchers also identified a notable additional effect. In KMT2A‑r AML cell lines and patient‑derived xenograft models, forskolin increased sensitivity to daunorubicin, a standard chemotherapy drug used to treat AML. The study reports that this chemosensitising effect did not depend on PP2A activation or on increased cyclic AMP levels. Instead, forskolin was found to raise the intracellular accumulation of daunorubicin by inhibiting the drug efflux pump P‑glycoprotein 1 (also known as a multidrug resistance protein), which cancer cells use to expel chemotherapy drugs.
In comments released by the University of Surrey and reproduced by outlets including Medical Xpress and ecancer, Dr Maria Teresa Esposito, Senior Lecturer in Biochemistry at the University of Surrey and a co‑author of the paper, said: "Our findings have highlighted an exciting dual mechanism of action for forskolin. Not only does it have direct anti‑leukaemic effects, but it also acts as a powerful enhancer to conventional chemotherapy. Combining forskolin with daunorubicin could lead to a more effective treatment strategy, potentially allowing for lower doses of chemotherapy and reducing the severe side effects often associated with AML treatments."
The work was supported by funding from Leukaemia UK, as noted in the journal article and institutional summaries. Additional support came from the Institute of Biomedical Science, the University of Roehampton and the University of Surrey. Collaborators on the study are affiliated with institutions including the University of Roehampton, the University of West London, Brunel University London, the Centre for Genomic Regulation in Barcelona, CEINGE Biotecnologie Avanzate in Naples, Pompeu Fabra University in Barcelona, the Great Ormond Street Institute of Child Health at UCL, Barts Cancer Institute at Queen Mary University of London and the University of Surrey.
In a statement carried by Medical Xpress, Dr Simon Ridley, director of research and advocacy at Leukaemia UK, described the potential implications of the research. He said that acute myeloid leukaemia is one of the most aggressive and deadly cancer types and that studies such as this deepen understanding of KMT2A‑rearranged AML while opening the door to potentially kinder, more effective treatments. He linked work of this kind to Leukaemia UK's goal of helping to double the five‑year survival rate for AML over the next decade.
The authors and the University of Surrey emphasise that the current findings come from preclinical experiments in cell lines and animal models. While the results support further investigation of forskolin, particularly in combination with daunorubicin, additional studies, including clinical trials, would be needed to determine whether this approach is safe and effective for patients with AML.
