Development of a novel therapy for neurodegenerative disease using tacrolimus derivative

2016 Fiscal Year Final Research Report

• Project/Area Number: 15K19482
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Neurology
• Research Institution: Nagoya University
• Principal Investigator: TOHNAI Genki 名古屋大学, 医学系研究科, 特任助教 (00748353)
• Project Period (FY): 2015-04-01 – 2017-03-31
• Keywords: 神経変性疾患 / 球脊髄性筋萎縮症 / タクロリムス

Outline of Final Research Achievements

To clarify the effect of medical-induction of proteolytic machinery in the spinal and bulbar muscular atrophy (SBMA), a neuromuscular disease caused by the CAG repeat expansion in the androgen receptor (AR) gene, we investigated effect of tacrolimus derivative in the SBMA model. Tacrolimus derivative increased the expression of proteolytic machinery-related molecule in cell models of SBMA. Mutant AR was reduced in the presence of tacrolimus derivative. Administration of the tacrolimus derivative ameliorated behavioral parameters in SBMA Tg mice. These observations suggest that tacrolimus derivative is a promising therapeutic candidate for motor neuron diseases including SBMA.

Free Research Field

神経内科学