Advance testing efficacy of albendazole in childhood acute myeloid leukaemia

This project is highly important for children with leukaemia. We have completed a lot of ground work in identifying a new drug that is effective in several types of childhood leukaemia, but has minimal effects in normal cells – the key characteristics of a new treatment for childhood cancer.

We now want to evaluate how effective ABZ is in advanced experiments in order to determine if it can be progressed to clinical trials. Specifically, this project is important for several reasons:

New, safe drugs for children with AML are urgently needed due to the poor survival outcomes and severe short term (e.g. vomiting and hair loss) and long-term side effects (e.g. bone pain, reduced lung function).

Our data to date suggests ABZ is very effective at low doses in a range of childhood AML cells and models. Our data demonstrates that ABZ is actually killing the leukaemia cells in less than 24 hours after treatment and this translates to greater survival in mice treated with the drug.

ABZ is a repurposed drug – therefore it is not expensive, safe (it has been used for many years to treat children with intestinal worms with no significant side effects) and has passed government approval – the point where the majority of new drugs fail. New drugs in cancer have the lowest likelihood of government approval, therefore our approach overcomes this challenge.

Genuine rapid clinical potential – as seen with the new repurposed drug dexamethasone to treat severe COVID-19 symptoms, repurposed drugs can be progressed to clinical trial and patient use quickly. We have the same objective for ABZ in childhood leukaemia.

We have very strong data on ABZ already. While like all science, there is some risk involved, we have shown in many experiments that ABZ is very effective at low doses which can be achieved by the current tablet form. We have also shown in a mouse experiment that ABZ is effective, a key achievement.

We are using the most innovative experiments to test ABZ – the design of the project to include determining how ABZ works and it’s activity in advanced mouse models should provide clear answers on this drug.

Recognition to date – this work has already been recognised by peers, doctors and other academics with several awards (Novartis Medal and Prize, Presentation Prize at the XXXVI World Congress International Society of Haematology, Early Career Research Excellence Award, etc.).

Working with the best people – we are collaborating with Professor Ross Levine in this project who is aworld leader in AML – both from a science perspective and as a doctor who treats patients with leukaemia. Professor Levine is based as one of the best cancer research institutes and hospitals globally – Memorial Sloan Kettering Cancer Center in New York.