Research Identifies Potential Treatment Targets for Rare Childhood Brain Tumors

Pediatric neuro-oncologists at Hackensack Meridian Children’s Health published research on embryonal tumor with multilayered rosettes (ETMR) and atypical teratoid/rhabdoid tumor (ATRT), identifying potential new treatment targets for these aggressive pediatric brain cancers with poor prognoses and no standard treatments.

In a case study of a five-year-old female with ETMR published in Cancer Reports, the team presented a case with prolonged survival after post-treatment differentiation in the patient’s pathology — supporting the development of novel therapeutics with a lower toxicity profile than chemotherapy that could be used to cause cell changes that lead to long-term remission.

In multiple papers published in Neuro-Oncology Advances and Neuro-Oncology, the team offered new insights into ETMR biology and identified potential targets for novel therapeutic approaches, including:

• Signaling pathways that regulate ETMR vascular and tumor cells,
• Modulation of the cardiolipin acyl chain structure to prevent tumor growth.
• Lipidomic pathways within the microenvironment.
• Enzymes that process cardiolipin to reduce tumor growth.

An additional paper published in Neuro-Oncology identified a gene-silencing target that could potentially modulate factors within the tumor and the immune microenvironment that enhance tumor growth in children diagnosed with ATRT.

“This research is focused on identifying potential targets to treat rare, aggressive childhood brain tumors for which there are currently no targeted therapies,” said Derek R. Hanson, M.D., pediatric neuro-oncologist at Hackensack Meridian Children’s Health. “Through our research, our goal is to advance the field’s understanding of the mechanisms that cause tumor development and growth so we can offer new hope for children diagnosed with rare brain tumors.”