Repurposing alcohol-abuse medicines to treat an incurable childhood brain tumour

Project title: Repurposing the alcohol-abuse drug disulfiram to target a key epigenetic hallmark of diffuse midline glioma

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Dr David Michod, University College London
Award: £200,980.16
Awarded July 2022

Diffuse midline glioma is a very aggressive tumour that mostly affects young children and is the main cause of brain tumour related death in children. It has a zero percent survival rate and, on average, children diagnosed with this cancer only survive for nine months. Use of chemotherapy to treat diffuse midline glioma does not work and so standard radiotherapy remains the only proven treatment. Unfortunately for most patients, this treatment is often palliative. Therefore, there is an urgent need for new treatment options.

Dr David Michod works at University College London. In previous work, his team has found that an existing medicine called disulfiram is very good at killing diffuse midline glioma cells. They also showed that the medicine targets known weaknesses of diffuse midline glioma cells. Disulfiram is a drug that has been used to support the treatment of long-term alcohol addiction for the past sixty years and therefore already approved, meaning it could be introduced as a cancer treatment faster.

The key goals of this project are to understand how disulfiram attacks diffuse midline glioma cells, and to gather data to show whether it is an effective treatment. If the researchers have enough evidence to show disulfiram effectively kills the cancer cells, it could help advance the treatment into clinical trials. Dr David Michod hopes that, if his theories are proven correct, disulfiram could be introduced for use in real patients faster than usual because disulfiram has been already shown to have only minor side effects.