CAR T cell therapy selectively depletes disease-driving mutant calreticulin cells in xenotransplants and human organoid models of myelofibrosis

Alexandros Rampotas, Zoë C. Wong, Isaac Gannon, Charlotte K. Brierley, Yuqi Shen, Camelia Benlabiod, / science - Science Translational Medicine, Volume 18, Issue 856, July 2026.

AI Summary: Preclinical studies show CAR‑T cells engineered to target mutant calreticulin selectively eliminate disease‑driving clones in xenografts and human organoid models of myelofibrosis. The results reveal a potentially curative approach for a previously intractable mutation‑driven disease, though safety, on‑target effects and clinical translation remain to be rigorously tested.