Minoxidil Ameliorates Myelodysplastic Syndrome by Targeting Wnt4 While Sparing Normal Hematopoiesis

The study investigates the potential of minoxidil, a common hair growth treatment, as a therapeutic agent for myelodysplastic syndrome (MDS) by targeting the Wnt4 signaling pathway. Using zebrafish models, minoxidil was found to suppress the proliferation of hematopoietic stem and progenitor cells (HSPCs) without causing apoptosis, alleviating MDS-like symptoms while minimizing adverse effects. The effects were consistent in vitro with human malignant hematopoietic cells and in vivo in mice, where lower doses minimized hematopoietic toxicity. The study highlights Wnt4 as a novel target for MDS treatment and supports the repurposing of minoxidil for hematopoietic disorders, suggesting its potential to selectively modulate abnormal progenitor proliferation while sparing normal hematopoiesis.