Autoimmune disease
Mouse studies suggest stimulating cholesterol efflux in phagocytes by promoting LXR expression could help treat MS. In a mouse model of lysolecithin-induced MS, cholesterol accumulation in the membranes of lesion-infiltrating phagocytes was higher in aged mice than in young mice. Also in the aged mouse model, stimulation of cholesterol efflux with an LXR agonist tool compound or 2-hydroxypropyl-β-cyclodextrin (HPBCD) -- which promotes LXR expression -- decreased cholesterol accumulation in phagocytes in lesions and increased the number of myelinated axons in the lesion compared with vehicle. Next steps include validating the cholesterol efflux mechanism in postmortem patient tissue samples and induced pluripotent stem (iPS) cell-derived cell cultures.
Sucampo Pharmaceuticals Inc. has VTS-270, an undisclosed formulation of HPBCD, in Phase III testing to treat Niemann-Pick disease type C1 (NPC1)...
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