Development of peptide-drug conjugates for the treatment of Chronic Myelomonocytic Leukaemia (CMML)

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Chronic Myelomonocytic Leukaemia (CMML) is a type of blood cancer for which is no generally effective treatment. For the majority of people diagnosed with CMML the prognosis is dismal, with 50% of patients dying within 15-20 months of diagnosis.Chemotherapy approaches in CMML frequently fail due to a lack of selectivity for the cancerous cells relative to the rest of the patient's body, or lack of activity against the cancerous cells in the bone marrow that cause the disease to recur. We are seeking to address this shortfall by developing a new class of drug that selectively targets and kills the cancerous cells through the patient's body, with little or no impact on healthy cell types. We will achieve this by attaching a highly toxic compound to a protein that is taken up only by the type of cells that have become cancerous in patients with CMML.We have demonstrated that a prototype of these drugs can selectively target and kill CMML cells in a test tube and that this activity and selectivity is maintained in animal experiments, which are needed to model the complex requirements of treating a patient with CMML.In this project we will conduct further studies to make as sure as possible that these compounds will be useful for treating CMML, whist also developing these prototypes to ensure they have all the required properties for testing in patients. If successful this will give us all the information we need to take one of these drugs into final toxicity tests and then clinical trials in CMML patients.