Developing a new treatment and companion diagnostic to improve outcomes for glioblastoma patients

Objetivo

Glioblastoma (GBM) remains a devastating brain cancer with limited treatment advances and poor survival outcomes. The GLIOBREAK project aims to develop BEA-17, a first-in-class oral small molecule that degrades epigenetic regulators LSD1 and CoREST, thereby reversing GBM immune evasion and enhancing responsiveness to immunotherapy.

The main objectives are to advance BEA-17 and its biomarker-driven companion diagnostic from validated laboratory stage (TRL4) to early clinical readiness (TRL6). This will be achieved through scalable GMP manufacturing, GLP-compliant safety and pharmacokinetic studies, efficacy validation in preclinical models, biomarker discovery and validation, and regulatory preparation including orphan drug designation.

GLIOBREAK directly addresses key challenges outlined in the EICT work programme by proposing a novel immuno-epigenetic therapy targeting GBM’s immunosuppressive microenvironment, combined with precision biomarker-based patient stratification to improve clinical outcomes. This aligns with Europe’s Beating Cancer Plan and EU priorities for innovative, personalized oncology medicines.

By integrating fundamental biological insights with cutting-edge therapeutic and diagnostic development, GLIOBREAK aims to deliver a transformative new treatment option for GBM patients and establish a platform for expanding into other difficult-to-treat cancers, reinforcing Europe’s leadership in precision immuno-oncology.