Diagnosis
Diffuse brainstem tumor (diffuse midline glioma). Condition following radiation therapy and chemotherapy.
Patient:
Child, 7 years old, Russia
Physician:
Prof. Dr. med. Gudrun Fleischhack, Head of Pediatric Oncology, University Hospital Essen
Parents’ Inquiries:
Are there alternative treatment options for diffuse brainstem tumors? We have heard about the experimental drug ONC201 currently undergoing clinical trials. Is it possible to use this medication in our daughter's case?
Expert Opinion by Prof. Gudrun Fleischhack:
Based on the medical records provided, your daughter has a diffuse midline glioma of the brainstem. Regrettably, complete or partial surgical resection of such tumors is impossible due to their critical location and infiltrative, diffuse growth pattern within the brainstem structures. The MRI scans show pronounced tumor infiltration into the pons, where the borders are particularly diffuse, leaving no possibility for surgical removal.
In this clinical scenario, radiation therapy remains the primary standard treatment. Since high-dose radiotherapy has already been completed, re-irradiation cannot be safely performed at this time. Furthermore, various chemotherapy regimens were previously administered but unfortunately yielded no long-term disease control.
Targeted therapy approaches are currently being actively researched worldwide. In France, relevant clinical trials are conducted under the leadership of the Gustave Roussy Institute.
Italian and Dutch colleagues are investigating whether re-irradiation can improve overall prognosis when administered at least 3 to 6 months following initial radiotherapy.
Dr. Maura Massimino from the National Cancer Institute in Milan utilizes a combination of Nimotuzumab and Vinorelbine, which has demonstrated extended survival progression, though not a definitive cure.
At our university hospital, clinical trials involving ONC201 are not currently conducted, and we do not have direct clinical experience with this compound. For detailed insights into trial findings regarding this targeted therapy, you may refer to the study: "Chi, A.S., Tarapore, R.S., Hall, M.D. et al. Pediatric and adult H3 K27M-mutant diffuse midline glioma treated with the selective DRD2 antagonist ONC201. J Neurooncol 145, 97–105 (2019)" (https://doi.org/10.1007/s11060-019-03271-3).
Ongoing and future clinical trials will determine whether the encouraging responses observed in a select subgroup of patients can be consistently replicated across broader patient cohorts. Currently, ONC201 remains an unapproved investigational drug.
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