Mini tools but maxi power to help kids with medulloblastoma!

This project was selected in the framework of our fourth European call: FIGHT KIDS CANCER 2023. It is jointly financed by Imagine for Margo, Kriibskrank Kanner Fondatioun, CRIS Cancer Foundation, KiKa and KickCancer.

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Brain tumours are among the most common and deadly solid tumours in children, adolescents, and young adults. Despite improvements in treatments such as chemotherapy, surgery and radiation therapy, the outlook for patients with a high-risk tumour is poor. In recent years, there has been little progress in the development of new drugs for brain tumours, including medulloblastoma.

This lack of new drugs is caused by the difficulty to screen new drugs preclinically as potential candidates for the clinic. The most commonly used technique for preclinical testing, two-dimensional culture models in petri dishes, poorly represents the complex behaviour of the drugs in patients; as a result, the results of such culture are not very reliable and there is a big discrepancy between the preclinical results and what is observed in the clinic.

Preclinical testing on mouse models is much more reliable (what is observed in a mouse is more likely to work in the clinic) but not very efficient in terms of number of drugs that can be tested.

Recently, the development of what is called “organoids” or miniature replication of human organs, has paved the way to a more reliable and faster technique to test new drugs preclinically as it can test up to thousands of drugs in one batch.

The “Medullodrug” team developed the first human organoid-based model for paediatric medulloblastoma and paediatric high-grade glioma opening the door for gaining unprecedented new knowledge into the development of paediatric brain cancer directly in a human system.

The aim of this project is to identify promising drugs, among drugs that have already been authorised, to initiate new clinical trials in Medulloblastoma.