Introduction

Drug Repurposing To Find A Treatment For Medulloblastoma

Medulloblastoma is the most common type of paediatric brain cancer and accounts for 15-20% of all childhood brain tumours. Most commonly found in children between the ages of three and eight, this cancer has a higher occurrence in boys.

The first drug repurposing project aPODD has undertaken, Medulloblastoma was chosen for investigation due to its relatively high incidence and unmet medical need.  The project comprises of two stages, with stage one completed, the project is now at stage two and we are expecting the final results to be received at the end of 2018.

Stage 1

Identifying Which Drugs Can Treat Medulloblastoma

We have chosen to partner with Healx, an award-winning technology company based in Cambridge UK. Healx’s expertise lies in using technologies such as genomics, computational biology and machine learning to identify new uses for known drugs.  This comprehensive approach was used to screen thousands of drug profiles to see which drugs may be effective against medulloblastoma. A shortlist of 10 drugs, have been recommended for further investigation.

Stage 2

Laboratory Testing

As testing partner we have chosen Associate Professor Beth Coyle at The University of Nottingham. Professor Coyle joined the University of Nottingham Children's Brain Tumour Research Centre (CBTRC) in 2002 as an independent Research Fellow, based in the School of Biology. The CBTRC are committed to developing new and better methods of treating childhood brain cancer and improving the quality of life of those who are cured.

The team have developed medulloblastoma assays which will be used for the first round of testing. The goal would be to select the best 2-3 candidates for further “in vivo” studies, meaning studies carried out in medulloblastoma mouse models. 

It is hoped that this work will lead to the selection of at least one drug which would be immediately eligible, in theory, for clinical evaluation in children with medulloblastoma.

The advantage of drug repurposing is that we are working with drugs that are already in clinical use, therefore we may safely bypass many of the conventional evaluation steps in drug development, thus making these potential new treatments available to patients much more quickly