臨床神経学

第50回日本神経学会総会

<50周年記念シンポジウム―5>〜飛躍の未来に向けて〜
トランスレーショナルリサーチに向けての発展神経変性疾患の病態抑止治療(分子標的治療)の開発に向けて

祖父江 元

名古屋大学大学院医学系研究科神経内科学〔〒466-8550 名古屋市昭和区鶴舞町65〕

Neurodegenerative diseases have been construed as incurable disorders. However, therapeutic development for these diseases is now facing a turning point: analyses of cellular and animal models have provided insights into pathogenesis of neurodegenerative diseases, and have indicated rational therapeutic approaches to them. Therefore, how to realize molecular targeted therapy for neurodegenerative diseases is becoming one of the most challenging issues in the clinical neurology. Primarily, pathophysiological understanding of the disease from basic science is the first step. For the successful clinical trials, effective trial design, sufficient economic and social support, and education are indispensable. The development of androgen deprivation therapy for spinal and bulbar muscular atrophy (SBMA) is a representative study in this field. SBMA is a hereditary neurodegenerative disease caused by expansion of a trinucleotide CAG repeat in the first exon of the androgen receptor (AR) gene. There is increasing evidence that testosterone, the ligand of AR, plays a pivotal role in the neurodegeneration in SBMA. The striking success of androgen deprivation therapy in SBMA mouse models has been translated into phase 2, and then phase 3, clinical trials.
Full Text of this Article in Japanese PDF (227K)

(臨床神経, 49:747−749, 2009)
key words:分子標的治療, 臨床試験, 神経変性疾患, 球脊髄性筋萎縮症(SBMA), リュープロレリン酢酸塩

(受付日:2009年5月20日)