Drs. Silvia Stacchiotti, Sandro Pasquali, and Nadia Zaffaroni (IRCCS – INT, Milan, Italy) and colleagues published their promising findings in Clinical Cancer Research, September 16, 2024. This study, funded in part by The EHE Foundation in partnership with EHE Rare Cancer Charity (UK), aimed to assess the efficacy of sirolimus for the treatment of EHE and to identify biomarkers to predict EHE aggressiveness. 

Results of the study show that:

• In the two models used, a patient-derived xenograft (PDX) and paired EHE cell line, EHE cells produce and release growth differentiation factor-15 (GDF-15). GDF-15 is a cytokine that cancer cells produce and is used as a biomarker for the severity of many diseases.
• These results indicate that GDF-15 could be a useful novel biomarker to predict EHE aggressiveness and disease progression.
• In the EHE models used, sirolimus was shown to reduce GDF-15 expression.
• Additionally, sirolimus had a higher anti-tumor activity than doxorubicin.

For EHE patients, these findings give hope for further preclinical drug development using human-derived EHE models, better inform treatment decisions, and monitor disease progression, all with the goal of improving patient outcomes. The EHE Foundation is grateful to the research team at Fondazione IRCCS Istituto Nazionale dei Tumori and their colleagues for this publication and their commitment to EHE research.