Abstract
Diffuse midline glioma(DMG), H3 K27M-mutant is an infiltrative midline high-grade glioma with predominantly astrocytic differentiation and a K27M mutation in either H3F3A or HIST1H3B/C. It is commonly located in the brain stem, thalamus, and spinal cord. DMG is predominant in children but can occur in adults. Brain stem disease, known as diffuse intrinsic pontine glioma(DIPG), is the representative: -80% harbor the H3 K27M mutation. Generally, the prognosis of DMG is poor: the 2-year survival rate is < 10%, despite intensive research and therapies. Consequently, radiation is the only treatment and there is no effective chemotherapeutic regimen. The recent findings concerning the genetic profiles of DMG shed light on precision medicine. Until today, approximately 250 clinical trials with molecular targeted therapy as a strategy have been conducted for different biological pathways in DMG. Unfortunately, none of them has shown significant efficacy for DMG. One of the problems in these clinical trials is insufficient knowledge of whether the used molecular targeted agents penetrate the blood-brain barrier. Continuous efforts to develop effective precision medicine against DMG should pave the way for overcoming DMG in the future.
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