Although there clearly exists a high frequency of ACVR1 somatic mutations in DIPG patients5,14,15 and the demonstrated preclinical potency of ACVR1 inhibitors at a μM level to inhibit DIPG cells with different biological backgounds2,12, we suggest that additional target validation and mechanistic studies of ACVR1 and associated pathways for DIPG therapeutics are required for the following reasons: First, the G328E/V/W mutations correlate with longer patient survival2,15. This evidence concerns the gene ACVR1 and diffuse intrinsic pontine glioma.