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. 2019 Sep 16;116(40):20097–20103. doi: 10.1073/pnas.1912108116

Fig. 5.

Fig. 5.

Acid ceramidase as a target for substrate reduction therapy. (A) Carmofur efficiently inhibits acid ceramidase activity against galactosylceramide in vitro. (B) Carmofur (3 μM, C-3) decreases psychosine accumulation in human fibroblasts from a Krabbe patient compared to vehicle (V). (C) Carmofur increases ceramide accumulation in human fibroblasts from a Krabbe patient. (D) Acid ceramidase activity is decreased in the liver of Twi/FDH mice treated with carmofur. (E) Psychosine is decreased in the livers of Twi and Twi/FDH mice following twice-daily i.p. injections of carmofur (C) at 30 mg/kg compared to vehicle (V). (F) Ceramides are not elevated in the brains of the same mice shown in E. Twice-daily i.p. injections of carmofur significantly increases the median life span of Twi/FDH mice (G) compared to vehicle controls. *P ≤ 0.05, **P ≤ 0.01, ***P ≤ 0.001; ns, P > 0.05.