Category: Pediatric High-Grade Glioma

M4K Pharma, founded by Aled Edwards and Owen Roberts from University of Toronto, is an open source pharmaceutical company dedicated to accelerating drug discovery and development for rare childhood cancers (e.g. DIPG) through public data-sharing and effective collaboration. Since it is unique for a pharmaceutical company to share their thoughts/on-going research and internal/external meetings with the public, I have been largely inspired by the successful impacts their open approach to drug development; the best drug candidates have passed in vivo PK and tolerability tests and are currently being tested for in vivo efficacy! Working with M4K Pharma to initially validate new drug candidates for ALK2 target have been a huge privilege.

Within the K27M altered tumors, one of my main interest lies on a specific tumor type called Diffuse Intrinsic Pontine Glioma (DIPG). DIPG is a rare, aggressive, and malignant brain tumor which develops in a part of the brainstem called the pons and more than 80% of DIPG patients are known to harbor H3 K27M-alterations. Due to its locational characteristic, DIPG is non-surgically resectable which hugely limits its treatment options. Most children with DIPG do not live longer than 2 years after diagnosis.

Published in 2021, the fifth edition of the WHO Classification of Tumors of the Central Nervous System highlights the role of advances in novel molecular profiling approaches in identifying new CNS tumor types and subtypes.

The changes in CNS tumor classification have been impacted by the improvements in novel diagnostic technologies and the recommendations of the Consortium to Inform Molecular and Practical Approaches to CNS Tumor Taxonomy (clMPACT-NOW).

Developing on from the fourth edition in 2016, where nucleic acid-based methodologies* clearly contributed towards a shift in CNS tumor classification and diagnosis, the fifth CNS WHO Classification (WHO CNS5) incorporates the powerful impact of recent emergence of DNA methylome profiling* into the classification system.