Biomarker study in the early phase patients with spinal and bulbar muscular atrophy

• Project/Area Number: 15K09311
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Neurology
• Research Institution: Nagoya University
• Principal Investigator: Hashizume Atsushi 名古屋大学, 医学部附属病院, 医員 (00637689)
• Co-Investigator(Kenkyū-buntansha): 祖父江 元 名古屋大学, 医学系研究科, 特任教授 (20148315) ; 勝野 雅央 名古屋大学, 医学系研究科, 教授 (50402566)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Project Status: Completed (Fiscal Year 2017)
• Budget Amount: ¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000); Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000); Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000); Fiscal Year 2015: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
• Keywords: 球脊髄性筋萎縮症 / 複合的臨床評価スケール / 運動ニューロン疾患 / 女性保因者 / X連鎖劣性遺伝 / 神経変性疾患 / バイオマーカー / レジストリ

Outline of Final Research Achievements

To develop new disease-modifying drugs for neurodegenerative diseases, a valid, reliable, and sensitive outcome measurement would be necessary. The present study aimed to develop a new composite measurement which reflects disease severty well. In this study, a total of 81 patients with SBMA and 23 normal contols were recruited. In the cross-sectional study, five components, tongue pressure, grip power, %PEF, 4.6 m timed walking test, and %VC, were appropriate to be combined with to develop a new composite mesurement.
We followed-up the subjects for at most 96 weeks to confirm the usefulness of this new measurement. The results of this longitudinal study indicated that the composite measurement was meaningful as for sensitiveness.

Research Products

• [Journal Article] Biomarker-based analysis of preclinical progression in spinal and bulbar muscular atrophy. (2018)
  • Author(s): Hijikata Y, Hashizume A, Yamada S, Inagaki T, Ito D, Hirakawa A, Suzuki K, Atsuta N, Tsuboi T, Hattori M, Hori A, Banno H, Sobue G, Katsuno M.
  • Journal Title: Neurology Volume: 90 Issue: 17 Pages: 1501-1509
  • DOI: 10.1212/wnl.0000000000005360
  • Peer Reviewed
• [Journal Article] Long-term treatment with leuprorelin for spinal and bulbar muscular atrophy: natural history-controlled study (2017)
  • Author(s): Hashizume Atsushi、Katsuno Masahisa、Suzuki Keisuke、Hirakawa Akihiro、Hijikata Yasuhiro、Yamada Shinichiro、Inagaki Tomonori、Banno Haruhiko、Sobue Gen
  • Journal Title: Journal of Neurology, Neurosurgery & Psychiatry Volume: 88 Issue: 12 Pages: 1026-1032
  • DOI: 10.1136/jnnp-2017-316015
  • Peer Reviewed
• [Journal Article] Quantitative Assessment of Swallowing Dysfunction in Patients with Spinal and Bulbar Muscular Atrophy (2017)
  • Author(s): Hashizume Atsushi、Banno Haruhiko、Katsuno Masahisa、Hijikata Yasuhiro、Yamada Shinichiro、Inagaki Tomonori、Suzuki Keisuke、Sobue Gen
  • Journal Title: Internal Medicine Volume: 56 Issue: 23 Pages: 3159-3165
  • DOI: 10.2169/internalmedicine.8799-16
  • NAID: 130006235519
  • ISSN: 0918-2918, 1349-7235
  • Peer Reviewed
• [Journal Article] 球脊髄性筋萎縮症の早期診断 (2017)
  • Author(s): 土方靖浩 橋詰 淳 勝野雅央 祖父江元
  • Journal Title: 神経内科 Volume: 86 Pages: 17-22
  • Peer Reviewed
• [Presentation] Development of a quantitative composite functional measurein spinal and bulbar muscular atrophy (2017)
  • Author(s): Tomonori Inagaki
  • Organizer: World congress of Neurology