2018•Proceedings for Annual Meeting of The Japanese Pharmacological SocietyOpen access
Intracerebroventricular 2-hydroxypropyl-β-cyclodextrin improves not only neurological symptoms but also hepatic abnormalities in Niemann-Pick disease type C model mice and patients
Madoka Fukaura, Yoichi Ishituka, Yuki Kondo, Ushihama Naoki, Toru Takeo, Naomi Nakagata, Takumi Era, Taishi Higashi, Keiichi Motoyama, Hidetoshi Arima, Yuki Kurauchi, Hiroshi Katsuki, Shunsuke Kamei, Tsuyoshi Shuto, Hirofumi Kai, Katsumi Higaki, Yoshio Sakiyama, Takafumi Sakakibara, Muneaki Matsuo, Tetsumi Irie
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Abstract
Niemann-Pick disease type C (NPC) is a fatal lysosomal lipid storage disorder. NPC patients show severe hepatosplenomegaly and progressive neurodegeneration, therefore the effective cure is needed. 2-Hydroxypropyl-β-cyclodextrin (HPBCD), a cellular cholesterol modifier, has been compassionately used to treat NPC patients by systemic or intracerebroventricular (icv) /intrathecal administration, but the optimal dosage regimen is not yet established. This study was conducted to investigate the effects of systemic or icv HPBCD treatment in Npc1 deficient (Npc1-/-) mice. In addition, we analyzed the clinical data from NPC patients to solve the clinical issues related to the icv-HPBCD therapy.
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