IngentaConnect Beneficial effects of intrathecal IGF-1 administration in patient...

Authors: Nagano, Isao¹; Shiote, Mito¹; Murakami, Tetsuro¹; Kamada, Hiroshi¹; Hamakawa, Yoshiyuki¹; Matsubara, Etsuro¹; Yokoyama, Masataka²; Morita, Kiyoshi²; Shoji, Mikio¹; Abe, Koji¹

Source: Neurological Research, Volume 27, Number 7, October 2005 , pp. 768-772(5)

Publisher: Maney Publishing

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.

Keywords: AMYOTROPHIC LATERAL SCLEROSIS; INSULIN-LIKE GROWTH FACTOR-1; INTRATHECAL ADMINISTRATION; NORRIS SCALES; VITAL CAPACITY

Document Type: Research article

DOI: 10.1179/016164105X39860

Affiliations: 1: Department of Neurology, Graduate School of Medicine and Dentistry, Okayama University, 2-5-1 Shikata-cho, Okayama, 700-8558 Japan 2: Department of Anesthesiology, Graduate School of Medicine and Dentistry, Okayama University, 2-5-1 Shikata-cho, Okayama, 700-8558 Japan

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