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 journalArticle

78 Citations (Scopus)

Abstract

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
Volume27
Issue number7
DOIs
Publication statusPublished - Oct 2005

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Amyotrophic Lateral Sclerosis
Somatomedins
Vital Capacity
Extremities
Body Weight
Transgenic Mice
Disease Progression
Therapeutics
Clinical Trials
Pharmacology
Survival

Keywords

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

ASJC Scopus subject areas

  • Clinical Neurology
  • Neuroscience(all)

Cite this

Beneficial effects of intrathecal IGF-1 administration in patients with amyotrophic lateral sclerosis. / Nagano, Isao; Shiote, Mito; Murakami, Tetsuro; Kamada, Hiroshi; Hamakawa, Yoshiyuki; Matsubara, Etsuro; Yokoyama, Masataka; Morita, Kiyoshi; Shoji, Mikio; Abe, Koji.

In: Neurological Research, Vol. 27, No. 7, 10.2005, p. 768-772.

Research output: Contribution to journalArticle

Nagano, I, Shiote, M, Murakami, T, Kamada, H, Hamakawa, Y, Matsubara, E, Yokoyama, M, Morita, K, Shoji, M & Abe, K 2005, 'Beneficial effects of intrathecal IGF-1 administration in patients with amyotrophic lateral sclerosis', Neurological Research, vol. 27, no. 7, pp. 768-772. https://doi.org/10.1179/016164105X39860
Nagano, Isao ; Shiote, Mito ; Murakami, Tetsuro ; Kamada, Hiroshi ; Hamakawa, Yoshiyuki ; Matsubara, Etsuro ; Yokoyama, Masataka ; Morita, Kiyoshi ; Shoji, Mikio ; Abe, Koji. / Beneficial effects of intrathecal IGF-1 administration in patients with amyotrophic lateral sclerosis. In: Neurological Research. 2005 ; Vol. 27, No. 7. pp. 768-772.
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