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 the Brain Tumor group at the Baylor College of Medicine, The Texas Children Hospital. This group is part of the Pediatric Preclinical Testing Consortium, funded by the National Cancer Institute (NCI) in the US. They are the specialist centre for evaluation of brain cancer drugs. The drugs identified at stage 1 are initially being tested in patient-derived cell lines. These are cells that are representative of the most aggressive genetic subtypes of medullobastoma. The scientists will look at which drugs are more effectively killing cancer cells in these “in vitro” experimental conditions. 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