Introduction

Drug Repurposing To Find A Treatment For Neuroblastoma

Neuroblastoma is a type of cancer that develops from immature nerve cells found in several areas of the body. It’s most commonly found in and around the adrenal glands, situated in the abdomen just about the kidneys, but it can also develop in the neck, chest or spine.

Working with the European Neuroblastoma Association (ENEA), based in Italy, this is second repurposing project aPODD has undertaken. ENEA is focusing its research effort on this diseases and they have decided to partner with aPODD in order to identify new treatment opportunities for neuroblastoma patients

This project comprises of two stages, and with stage one just completed, the project is now at stage two and we are expecting some initial results by the end of the Summer 2019.

Stage 1

Identifying Which Drugs Can Treat Neuroblastoma

Working with a team of scientists at Healx, Cambridge UK, extensive research was initially undertaken to collect genomic information about this disease in children. This enabled the team to generate a profile of neuroblastoma that could be used in computer models. This profile was then used to match against thousands of drug profiles to see which drugs may have an effect against neuroblastoma. Using machine learning and artificial intelligence a shortlist of 10 drugs have been recommended for further investigation.

Stage 2

Laboratory Testing

Working with a team of investigators at Lund University in Sweden, the drugs identified are initially being tested in patient-derived cell lines. Dr Daniel Bexell’s group in Lund is ideally equipped to evaluate new drugs in neuroblastoma as they have developed over the years a wide array of laboratory models to study this disease. By looking at the effect of these drugs on cells that are representative of the most aggressive form of this disease, we are hoping to identify at least a couple of candidates for further evaluation. The goal would be to select the top candidates for further “in vivo” studies, meaning studies carried out in neuroblastom mouse models.

This work may ultimately lead to the selection of at least one drug, which would be immediately eligible, in theory, for clinical evaluation in children with neuroblastoma.

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