Clinical evaluation of ex vivo drug sensitivity profiling in the pediatric precision oncology program INFORM

INFORM is an international precision oncology program enrolling pediatric patients with relapsed cancer. In 2019, the program expanded its comprehensive molecular analysis to include ex vivo drug sensitivity profiling (DSP). DSP is conducted on multicellular fresh tumor spheroid cultures in 384-well plates, with an average processing time of three weeks before presentation to the INFORM board. In selected patients, notable parallels between DSP results and clinical outcomes have been observed. To systematically evaluate the predictive power of DSP, we are analyzing a cohort of 40 patients with clinical follow-up data and available DSP results. This test cohort will identify the most relevant parameter(s) for predicting clinical response. The accuracy of these predictors will be validated in a second cohort. So far, we have found that, besides classical parameters such as drug response, some other factors may be important as well, such as the number of drugs a patient has been received during one treatment regimen. Our analyses will support the development of prospective clinical trials and may eventually lead to the incorporation of DSP into personalized treatment decisions.

Publication History

Article published online:
09 May 2025