Ines Lohse*, Hassan Al-Ali, Giselle Dutcher, Warren Alperstein, Donald W. Coulter, Matteo Trucco, Jonathan C. Trent and Claes Wahlestedt
Precision medicine approaches using ex vivo Drug Sensitivity Testing (DST) have recently received attention in the cancer research community as a means to improve treatment stratification in populations where multiple treatment attempts are not feasible, or no standard treatment exists. This is particularly relevant for ultra-rare cancers such as osteosarcoma where population sizes preclude traditional prospective randomized clinical trials that can yield statistically meaningful data. DST has the potential to supplement existing patient stratification approaches by providing tumor-specific response data to aid in treatment selection at the time of treatment decision.
Here we present the case of a pediatric osteosarcoma patient who was evaluated using DST at the time of standard of care treatment. The DST screen indicated significant treatment sensitivity toward anthracyclines and methotrexate, consistent with the first-line standard of care therapy MAP. Consistent with the DST results, clinical follow up showed treatment sensitivity towards standard of care MAP treatment and pathology results of 90% necrosis.
The present case shows that DST screening is feasible from a technical standpoint, can be performed in a clinically relevant time frame that does not delay treatment start, provides personalized drug sensitivity information on clinically available agents and the DST results align with the clinical treatment response. Further evaluation of drug sensitivity testing is warranted to supplement current used methods for the treatment stratification of osteosarcoma patients.