A clinical trial with a super strong combo to KILL brain tumours!

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.

It is thanks to your participation in RUN TO KICK that this innovative project will be funded! Register now for RUN TO KICK 2023 and help us finance 9 new innovative research projects.

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.

Cancers are caused by abnormal genetic mutations that accumulate over time. These mutations lead to treatment resistance.

Poly (ADP-ribose) polymerase inhibitors (PARPi) are drugs developed to treat cancers caused by specific genetic mutations. PARPi work by preventing cells from repairing themselves, leading to those cancer cells death.

Combining PARPi with chemotherapy has been shown to improve outcomes in other cancers and is now being studied for use in paediatric cancers too, such as brain tumours.

The aim of this project is to study NIRAPARIB, a type of PARPi that can cross the ‘blood brain barrier’, in combination with IRINOTECAN, a chemotherapy used in several brain tumours. The first part of the study will focus on defining a well-tolerated dose and administration schedule. The second part of the study will evaluate the efficacy of this new treatment.

This study will be part of the platform study Access Secured - European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumours (AcSé-ESMART). The study plans to enrol 58 patients over 2 to 3 years in 7 European countries.

If this study shows encouraging results, NIRAPARIB with IRINOTECAN will be used in larger disease-specific studies to improve survival in patients with those specific brain tumours.