Longevity & AgingNew Tumor Organoid Biobank Exposes MCL-1 as a Drug Target in Rare Sarcomas
Investigators at the Princess Máxima Center established a first-of-its-kind biobank of patient-derived tumor organoids (tumoroids) from small round cell sarcomas (SRCS), rare and highly aggressive cancers affecting mostly children and young adults. The biobank includes tumors harboring EWSR1::FLI1, EWSR1::ERG, EWSR1::FEV, CIC::DUX4, and BCOR-rearrangements. Tumoroids faithfully retained the histology, chromosomal translocations, marker gene expression, and cellular heterogeneity of the original patient tumors over long-term culture. Drug screening across the biobank revealed subtype-specific drug sensitivities, most notably that CIC::DUX4 sarcomas are selectively vulnerable to MCL-1 inhibitors, a class of targeted anti-apoptotic agents, offering a potential new treatment avenue for a sarcoma subtype that responds poorly to standard chemotherapy.
