A novel approach to realize target-specific, noninvasive and highly efficient delivery of macromolecular drugs to the central nervous system

• Project/Area Number: 15K12549
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Biomedical engineering/Biomaterial science and engineering
• Research Institution: National Institute of Advanced Industrial Science and Technology
• Principal Investigator: Kondo Tetsuro 国立研究開発法人産業技術総合研究所, 生命工学領域, 主任研究員 (30344229)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Project Status: Completed (Fiscal Year 2017)
• Budget Amount: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000); Fiscal Year 2017: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000); Fiscal Year 2016: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000); Fiscal Year 2015: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
• Keywords: 脳 / 高分子タンパク医薬 / 抗体医薬 / 進化工学 / DDS / 指向性進化工学

Outline of Final Research Achievements

Antibody drugs have increasingly occupied a high share of the global pharmaceutical market in the past decade. Brain-targeted antibody drugs and macromolecular drugs are expected to be next generation biodrugs to have direct effects on brain tumors and neurodegenerative diseases such as Alzheimer's disease. However, so far, many of brain-targeted antibody drugs have resulted in clinical trial failures, possibly due to the inefficiency of CNS delivery by systemic injection and the difficulties of crossing the blood-brain barrier. In the present study, to realize noninvasive, highly efficient and target-specific delivery of macromolecular drugs to the CNS, a new in vivo screening method based on directed evolution was established in mice.