title: Nerve growth factor for the treatment of spinocerebellar ataxia type 3: an open-label study.
abstract: BACKGROUND
  Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA worldwide, and runs a slowly progressive and unremitting disease course. There is currently no curable treatment available. Growing evidence has suggested that nerve growth factor (NGF) may have therapeutic effects in neurodegenerative diseases, and possibly also in SCA3. The objective of this study was to test the efficacy of NGF in SCA3 patients.
  
  
  METHODS
  We performed an open-label prospective study in genetically confirmed adult (>18 years old) SCA3 patients. NGF was administered by intramuscular injection (18 μg once daily) for 28 days consecutively. All the patients were evaluated at baseline and 2 and 4 weeks after treatment using the Chinese version of the scale for assessment and rating of ataxia (SARA).
  
  
  RESULTS
  Twenty-one SCA3 patients (10 men and 11 women, mean age 39.14 ± 7.81 years, mean disease duration 4.14 ± 1.90 years, mean CAG repeats number 77.57 ± 2.27) were enrolled. After 28 days of NGF treatment, the mean total SARA score decreased significantly from a baseline of 8.48 ± 2.40 to 6.30 ± 1.87 (P < 0.001). Subsections SARA scores also showed significant improvements in stance (P = 0.003), speech (P = 0.023), finger chase (P = 0.015), fast alternating hand movements (P = 0.009), and heel-shin slide (P = 0.001).
  
  
  CONCLUSIONS
  Our preliminary data suggest that NGF may be effective in treating patients with SCA3.
authors: Song
authors: S
authors: Tan
authors: Rui-Hao
authors: RH
authors: Wang
authors: Hui-Xia
authors: HX
authors: Niu
authors: Chang-He
authors: CH
authors: Shi
authors: Cheng-Yuan
authors: CY
authors: Mao
authors: Rui
authors: R
authors: Zhang
authors: Bo
authors: B
authors: Song
authors: Shi-Lei
authors: SL
authors: Sun
authors: Xin-Jing
authors: XJ
authors: Liu
authors: Hai-Man
authors: HM
authors: Hou
authors: Yu-Tao
authors: YT
authors: Liu
authors: Yuan
authors: Y
authors: Gao
authors: Hui
authors: H
authors: Fang
authors: Xiang-Dong
authors: XD
authors: Kong
authors: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan 450052, China.
authors: Yu-Ming
authors: YM
authors: Xu
mesh_terms: D000328:Adult; D000818:Animals; D005260:Female; D006801:Humans; D007273:Injections, Intramuscular; D017827:Machado-Joseph Disease / Q000188:drug therapy*; D008297:Male; D051379:Mice; D008875:Middle Aged; D020932:Nerve Growth Factor / Q000008:administration & dosage / Q000627:therapeutic use*; D011446:Prospective Studies
chemical_list: D020932:Nerve Growth Factor
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