DIPG DMG Drugs & Treatments - expert opinions

This blog post will be an archive for various data points and expert opinions on drugs not commonly used or in clinical trials for DMGs. Parents consider these based on hypothesized effects or pre-clinical data.

Image source: https://www.koschmannlab.org/precision-medicine

• Salinomycin People have published that Salinomycin targets cancer stem cells  there are invitro studies on its use in GBM cells. Salinomycin (Sali) is an anticoccidial polyether antibiotic. 20240329 (pre clinical DIPG experts) The drug seems to  be toxic and does not cross the BBB https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2258498/

• TIL Therapy 20240329 (pre clinical DIPG experts) DMGs are pretty immunologically cold / quite but I wonder if we should try such for a complicated cancer such as DMG

• Disulfram 20240330 (pre clinical DIPG experts) Disulfiram alone is not effective in DMGs.

  In combination with ONC201 (dordaviprone, commercial name Modeyso), effects appear additive but limited based on preclinical data. The magnitude of benefit appears modest and not clearly clinically meaningful at this stage.

• ONC201 / dordaviprone (Modeyso) – clinical update (2025)

  ONC201, now known as dordaviprone (brand name Modeyso), received accelerated approval from the U.S. Food and Drug Administration in 2025 for recurrent H3 K27M-mutant diffuse midline glioma.

  This makes it one of the first approved therapies targeting this disease biology.

  While many combinations are still being explored, it is no longer purely experimental.

• VENCLEXTA® (venetoclax tablets) 2023 pre clinical data showed good results. But BBB penetration levels are to too low for any theraputic levels. Venclexta and Venclyxto, is a medication used to treat adults with chronic lymphocytic leukemia, small lymphocytic lymphoma, or acute myeloid leukemia.