Isofagomine (D-tartrate)

Isofagomine (D-tartrate) is a competitive inhibitor of human lysosomal β-glucosidase (K_i = 0.016-0.025 µM; IC₅₀ = 0.06 µM).¹ By interacting with the catalytic pocket of β-glucosidase it acts as a chemical chaperone that increases the amount of β-glucosidase by stabilizing and/or promoting the folding of the enzyme.² Isofagomine (D-tartrate) has been shown to increase lysosomal β-glucosidase activity by 2- to 3-fold in mutant N370S Gaucher fibroblasts.¹ This compound has been studied in the context of Gaucher disease, a lysosomal storage disorder resulting from substantial deficiency of β-glucosidase and recently identified as a parkinsonism risk factor.²

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1. Kuriyama, C., Kamiyama, O., Ikeda, K., et al. In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures. Bioorg. Med. Chem. 16(15), 7330-7336 (2008).

2. Witte, M.D., Kallemeijn, W.W., Aten, J., et al. Ultrasensitive in situ visualization of active glucocerebrosidase molecules. Nat. Chem. Biol. 6(12), 907-913 (2010).