Developing better and safer medicines for children with leukaemia

Despite a high cure rate, treatment of children’s leukaemia is often harsh and toxic to patients, with many long-term side effects. A lot of focus is directed towards replacing current toxic chemotherapy with more targeted therapies (“chemo-free” therapy) that specifically targets the leukaemia cells but spares normal tissues.

As with current chemotherapy treatments, several new drugs (novel agents) will need to be combined to avoid the development of resistance to the drugs. This is known as combination therapy.

This study will look at different combinations of novel agents and assess which drugs act best with each other and are most efficient at the lowest dose. The objective is to develop better and safer medicines for children with leukaemia.

One major barrier to developing novel combination therapies has been our inability to cultivate leukemic cells in the laboratory. This is even a bigger problem as there are a very limited number of patients available for clinical trials. We have managed to improve the culture conditions for human leukaemia and can now grow cells from individual leukaemia patients. We have shown that the cancer cells preserve their initial leukemic characteristics, meaning they behave in our laboratory in a very similar way as they would behave in the patient.

We will now use this technology to establish a platform of leukaemia samples to test novel drug combinations. Selection of the different novel agents will be informed by our molecular studies and aimed at specifically targeting pathways that promote survival and growth of the leukemic cells. The ultimate aim is to generate data for new clinical trials with the ultimate goal to develop more efficient and less toxic therapies for children with leukaemia.