To elucidate the mechanism of actions of new drug candidate of Niemann-Pick disease type C and search for new candidate substance

2017 Fiscal Year Final Research Report

• Project/Area Number: 16K19054
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Pathological medical chemistry
• Research Institution: Kumamoto University
• Principal Investigator: SOGA Minami 熊本大学, 発生医学研究所, 助教 (80768002)
• Project Period (FY): 2016-04-01 – 2018-03-31
• Keywords: 疾患由来iPS細胞 / ニーマンピック病C型（NPC） / コレステロール / ライソゾーム病 / 代謝性疾患 / 神経障害 / 肝脾腫 / シクロデキストリン

Outline of Final Research Achievements

The aim to this study is to elucidate the mechanism of ations at the molecular level of 2-Hydroxypropyl-gamma-cyclodextrin (HPGCD), develop a new drug, and develop an HPGCD administration method effective for Neurological disorder. I used microarray analysis to characterize the NPC-derived neural progenitors (NPs). A large number of genes were differentially expressed in each NPC-derived NPs line compared with healthy donor-derived NPs. Further I identified a gene sets whose expression varied in HPGCD-treated NPs.
Intraventricular administration of HPGCD significantly prolonged survival time as compared with subcutaneous administration, suppressed Purkinje cell loss, and delayed the onset of neurological disorder. These results revealed that Intraventricular administration of HPGCD is an effective dosing method for suppressing neuropathy.

Free Research Field

再生医学