Drug repurposing via ultra-high throughput 3D spheroids screening in rare cancer treatment

Human SCG tissue samples and population-level datasets assessed translational relevance. uHTS screening identified Clofoctol as a lead compound with potent glioma growth inhibition in both primary SCG cells and xenograft models. Mechanistic studies revealed pleiotrophin (PTN) as a key target, with confirmatory expression patterns in human SCG samples, establishing translational validity. Integration of large-animal cancer models with drug-repurposing pipelines and uHTS platforms is feasible and effective for rare cancer drug discovery.

This approach successfully identified Clofoctol, an FDA-approved compound with therapeutic potential for spinal cord gliomas and establishes a scalable model applicable to other orphan cancers.