JCMM Annual Review on Advances in Biotechnology for the Treatment of Haematological Malignancies: A Review of the Latest In-Patient Developments 2024–2025

Abstract

Advances in biotechnology are set to revolutionise the treatment of haematological malignancies. Current treatments have limited efficacy due to their high toxicity and poor long-term outcomes, particularly in acute myeloid leukaemia (AML). In 2024–2025, emerging targeted therapies promise improved results with reduced adverse effects. This review is the first of an annual series exploring the latest in-patient developments in several key areas, focusing on mechanisms. We present advances in the use and action of several therapies including epigenetic modulators, protein degraders, apoptotic inducers, gene editing, and immunotherapy. We discuss the individual molecular mechanisms and clinical findings of these developments, emphasising their potential to improve survival and offer renewed hope for individuals with blood cancer. Importantly, such novel approaches with testing in patients should lead to further intensive efforts both at basic and translational research levels aiming at effective targeted and immunotherapy in blood cancers.