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 language | English |
|---|---|
| Pages (from-to) | 768-772 |
| Number of pages | 5 |
| Journal | Neurological Research |
| Volume | 27 |
| Issue number | 7 |
| DOIs | |
| Publication status | Published - Oct 2005 |
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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 journal › Article
}
TY - JOUR
T1 - Beneficial effects of intrathecal IGF-1 administration in patients with amyotrophic lateral sclerosis
AU - Nagano, Isao
AU - Shiote, Mito
AU - Murakami, Tetsuro
AU - Kamada, Hiroshi
AU - Hamakawa, Yoshiyuki
AU - Matsubara, Etsuro
AU - Yokoyama, Masataka
AU - Morita, Kiyoshi
AU - Shoji, Mikio
AU - Abe, Koji
PY - 2005/10
Y1 - 2005/10
N2 - 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.
AB - 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.
KW - Amyotrophic lateral sclerosis
KW - Insulin-like growth factor-1
KW - Intrathecal administration
KW - Norris scales
KW - Vital capacity
UR - http://www.scopus.com/inward/record.url?scp=27244455282&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=27244455282&partnerID=8YFLogxK
U2 - 10.1179/016164105X39860
DO - 10.1179/016164105X39860
M3 - Article
C2 - 16197815
AN - SCOPUS:27244455282
VL - 27
SP - 768
EP - 772
JO - Neurological Research
JF - Neurological Research
SN - 0161-6412
IS - 7
ER -