Optimisation of novel GATA2 inhibitors as targeted therapeutics for therapy-resistant acute myeloid leukaemia

This project aims to improve outcomes for patients in Wales diagnosed with acute myeloid leukaemia (AML), an aggressive blood cancer with poor survival rates and limited treatment options, particularly when the disease becomes resistant to therapy. Despite intensive chemotherapy, many patients relapse because a small population of leukaemia stem cells survives treatment and drives disease recurrence.

This project will address this major cause of treatment failure by advancing a first-in-class targeted therapy aimed at leukaemia stem cells. The project builds on Cancer Research Wales–funded discoveries of small-molecule inhibitors of GATA2, a protein that is abnormally overexpressed in therapy-resistant AML and critical for disease persistence. By optimising these compounds and testing them in patient-derived samples, the project will aim to accelerate their progression towards preclinical development and future clinical translation.

For patients in Wales, this research offers the potential for more effective and better-tolerated treatments, reduced relapse rates, and improved survival. In the longer term, it supports the development of innovative, Wales-led cancer therapies, strengthens local research expertise, and increases the likelihood that patients in Wales will benefit early from new clinical advances.