Statins give bone growth a boost

Abstract

The development of stem-cell-based models of two diseases that cause dwarfism reveals that statins — drugs that are used to treat high levels of blood cholesterol — may also promote cartilage formation and bone growth. See Article p.507 Some of the most common causes of dwarfism or skeletal dysplasia in humans are the result of gain-of-function mutations in the fibroblast growth factor receptor 3 gene (FGFR3). Noriyuki Tsumaki and colleagues have reprogrammed fibroblasts from patients with two such conditions — thanatophoric dysplasia type 1 (TD1) and achondroplasia (ACH) — to produce induced pluripotent stem cells (iPSCs). Chondrogenic differentiation of TD1 iPSCs resulted in the formation of degraded cartilage. A screen for molecules with the ability to rescue chondrogenically differentiated TD1 iPSCs from the degraded cartilage phenotype identified statins — drugs normally used to reduce blood cholesterol levels — as the most effective. Statins were included as candidate molecules because they have been reported to have anabolic effects on chondrocytes. In addition, statin treatment led to significant recovery of bone growth in a mouse model of ACH. These findings suggest that statins may have potential as a medical treatment for infants and children with TD1 and ACH.