Beneficial effects of intrathecal IGF-1 administration in patients with amyotrophic lateral sclerosis

Isao Nagano, Mito Shiote, Tetsuro Murakami, Hiroshi Kamada, Yoshiyuki Hamakawa, Etsuro Matsubara, Masataka Yokoyama, Kiyoshi Morita, Mikio Shoji, Koji Abe

Research output: Contribution to journalArticlepeer-review

86 Citations (Scopus)


Objectives: There is currently no effective pharmacological treatment for amyotrophic lateral sclerosis (ALS). In a transgenic mouse model of ALS, intrathecal infusion of insulin-like growth factor (IGF)-1 showed a promising increase in survival. We performed a double-blind clinical trial to assess the effect of intrathecal administration of IGF-1 on disease progression in patients with ALS. Methods: Nine patients with ALS were randomly assigned to receive either a high dose (3 μg/kg of body weight) or low dose (0.5 μg/kg of body weight) of IGF-1 every 2 weeks for 40 weeks. The outcome measurements were the rate of decline of bulbar and limb functions (Norris scales) and forced vital capacity. Results: The high-dose treatment slowed a decline of motor functions of the ALS patients in total Norris and limb Norris scales, but not in bulbar Norris or vital capacity. The intrathecal administration of IGF-1 had a modest but significant beneficial effect in ALS patients without any serious adverse effects. Discussion: Intrathecal IGF-1 treatment could provide an effective choice for ALS although further studies in more patients are needed to confirm its efficacy and optimize dosages of IGF-1.

Original languageEnglish
Pages (from-to)768-772
Number of pages5
JournalNeurological Research
Issue number7
Publication statusPublished - Oct 2005


  • Amyotrophic lateral sclerosis
  • Insulin-like growth factor-1
  • Intrathecal administration
  • Norris scales
  • Vital capacity

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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