A road map for the treatment of pediatric diffuse midline glioma

Cancer Cell. 2024 Jan 8;42(1):1-5. doi: 10.1016/j.ccell.2023.11.002. Epub 2023 Nov 30.


Recent clinical trials for H3K27-altered diffuse midline gliomas (DMGs) have shown much promise. We present a consensus roadmap and identify three major barriers: (1) refinement of experimental models to include immune and brain-specific components; (2) collaboration among researchers, clinicians, and industry to integrate patient-derived data through sharing, transparency, and regulatory considerations; and (3) streamlining clinical efforts including biopsy, CNS-drug delivery, endpoint determination, and response monitoring. We highlight the importance of comprehensive collaboration to advance the understanding, diagnostics, and therapeutics for DMGs.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural

MeSH terms

  • Biopsy
  • Brain / pathology
  • Brain Neoplasms* / genetics
  • Brain Neoplasms* / therapy
  • Child
  • Glioma* / diagnosis
  • Glioma* / genetics
  • Glioma* / therapy
  • Humans
  • Mutation