Development of biomarker and therapy for amyotrophic lateral sclerosis targeting cystatin C

2015 Fiscal Year Final Research Report

• Project/Area Number: 15K15337
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Neurology
• Research Institution: Nagoya University
• Principal Investigator: Katsuno Masahisa 名古屋大学, 医学(系)研究科(研究院), 教授 (50402566)
• Co-Investigator(Kenkyū-buntansha): Sahashi Kentaro 名古屋大学, 医学部附属病院, 医員 (90710103) ; KONDO Naohide 名古屋大学, 医学部附属病院, 医員 (20725527)
• Co-Investigator(Renkei-kenkyūsha): Sobue Gen 名古屋大学, 大学院医学系研究科, 特任教授 (20148315) ; Okada Yohei 愛知医科大学, 医学部, 准教授 (30383714)
• Project Period (FY): 2015-04-01 – 2016-03-31
• Keywords: 運動ニューロン疾患 / シスタチンC / バイオマーカー / 筋萎縮性側索硬化症

Outline of Final Research Achievements

We investigated the serum levels of cystatin C in the subjects with sporadic ALS, both cross-sectionally and longitudinally, to clarify whether serum cystatin C is a potential biomarker reflecting the severity of motor dysfunction and predicting prognosis of this disease. The results were compared with those with healthy controls (HC) and spinal and bulbar muscular atrophy (SBMA). Baseline levels of serum cystatin C in the subjects with ALS were significantly higher than those of HC and SBMA. As for the relationship between disease severity, a strong correlation was found between baseline levels of serum cystatin C and ALSFRS-R in the subjects with ALS. Furthermore, baseline levels of serum cystatin C were correlated with the 24-week change of %FVC and that of appendicular lean soft tissue mass, as an index of skeletal muscle mass, in ALS. In conclusion, serum cystatin C may be a potential biomarker which reflects the disease progression of ALS.

Free Research Field

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