Development of effective treatment by immune tolerance induction in enzyme replacement therapy for lysosomal storage diseases

2015 Fiscal Year Final Research Report

• Project/Area Number: 24591526
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Jikei University School of Medicine
• Principal Investigator: OHASHI TOYA 東京慈恵会医科大学, 医学部, 教授 (60160595)
• Co-Investigator(Kenkyū-buntansha): SHIMADA Yohta 東京慈恵会医科大学, 医学部, 助教 (20560824) ; HIGUCHI Takashi 東京慈恵会医科大学, 医学部, 助教 (30595327) ; KOBAYASHI Hiroshi 東京慈恵会医科大学, 医学部, 准教授 (90266619)
• Project Period (FY): 2012-04-01 – 2016-03-31
• Keywords: ライソゾーム病蓄積症 / ファブリー病 / ポンぺ病 / 酵素補充療法 / 抗体 / 免疫寛容

Outline of Final Research Achievements

Lysosomal storage diseases are characterized by genetically deficient activity of lysosomal enzyme, resulting in intracellular accumulation of various compounds. This causes various clinical symptoms. So far, about 40 lysosomal storage disease are known. As a treatment, enzyme replacement therapy is approved for 7 lysosomal storage diseases. However, development of antibody against infused enzyme negatively impact on therapeutic efficacy. From our study, antibody not only inhibit the enzyme activity but also cellular uptake of enzyme. Oral administration of enzyme prevent the antibody formation in enzyme replacement therapy for lysosomal storage diseases.

Free Research Field

小児科学、先天代謝異常症、遺伝子治療