The Haematological Malignancies Focus Group is focusing on the Myeloid Malignancies spectrum of diseases which includes Myelodysplastic Syndromes (MDS), Myeloproliferative Neoplasms (MPN), Acute Myeloid Leukaemia (AML), and Chronic Myeloid Leukaemia (CML).
A significant percentage of patients with MDS, MPN or CML will eventually transfrom into an AML type disease. AML, MDS and MPN are predominantly a disease of the elderly and unsatisfactory outcomes persist for the majority of these patients. Therefore, novel and less intensive therapies are required for this group of patients. Around 35 genes have been shown to be mutated across the myeloid malignancies spectrum. These mutations often occur in combination negating or amplifying any individual prognostic benefit. This molecular complexity means that targeted therapies to an individual gene may be less effective and combination therapies or more system target therapies may be required.
The Haematological Malignancies Focus Group is using different research strands to identify and understand how novel therapies could be used in combination with other novel or cytotoxic therapies. These strands include using in vivo models of AML particularly to assess the role of the HOX-TALE axis in disease initiation and maintenance; characterising the molecular responses to epigenetic, proteasome and other novel agents; using integrated analysis of markers of disease progression to re-purpose therapeutic agents against the myeloid malignancies; and identifying the molecular basis for a rationally designed epigenetic priming therapeutic strategy.
The focus group involves a wide range of translational and clinical scientists, bio-informaticians, medicinal chemists, pathologists and academic clinicians from the CCRCB and the Belfast Trust. The laboratory approaches are complemented by national and international trials for CML, MPD and AML supported by the Leukaemia and Lymphoma Research Therapy Acceleration Programme (TAP) portfolio within the Experimental Cancer Medicine Centre.