February 16, 2023

Developing Personalized Treatments of Pediatric Aggressive Gliomas

5th Call, iDoc

Short Summary

This project includes research on Diffusive Intrinsic Pontine Gliomas (DIPGs) by engineering a microfluidic platform to search for sonosensitizers while reducing time and amount of patient material needed for drug screens. The proposed microfluidic translational in vitro platform is aimed at providing time-sensitive data to the clinic, offering clinicians the possibility to make informed decisions for personalized treatment strategies. Our proposed platform will allow to tackle two major current limitations of in vitro testing with primary patient material, namely the throughput caused by the low cell number and the ability of testing non-pharmacological treatment options. Miniaturization of the testing volume will enable to increase the number of tests with small sample volumes, and the compatibility of the platform with ultrasound treatment will enable to test complementary and combined therapeutic strategies on primary cells.

Goals

Our goal is to develop a microfluidic system that provides clinically relevant information by adapting modalities to specific patients and to identify treatments with high efficacy against cancer cells and low toxicity to healthy tissue.

Significance

The possibility of testing drug compounds and treatment options in vitro using primary patient material prior to beginning a therapeutic cycle represents a major advancement of personalized treatments. Along with genomic testing of biopsy cells, in vitro testing of different compounds and treatment combinations on primary cells will allow to timely identify promising treatment strategies.

Background

DIPGs are deadly pediatric aggressive gliomas occurring in midline structures of the brain with less than 10% of the very young patients surviving 2 years from diagnosis. Despite increased understanding of DIPG biology, there are no effective treatments, with radiotherapy remaining the standard of care despite offering only transient relief.