Beneficial effects of intrathecal IGF-1 administration in patients with amyotrophic lateral sclerosis.
OBJECTIVE
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 microg/kg of body weight) or low dose (0.5 microg/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.


CONCLUSIONS
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.
Nagano
Isao
I
Department of Neurology, Graduate School of Medicine and Dentistry, Okayama University, 2-5-1 Shikata-cho, Okayama, 700-8558 Japan. inagano@cc.okayama-u.ac.jp
Shiote
Mito
M
Murakami
Tetsuro
T
Kamada
Hiroshi
H
Hamakawa
Yoshiyuki
Y
Matsubara
Etsuro
E
Yokoyama
Masataka
M
Moritaz
Kiyoshi
K
Shoji
Mikio
M
Abe
Koji
K
D004305:Dose-Response Relationship, Drug; D006801:Humans; D007278:Injections, Spinal; D007334:Insulin-Like Growth Factor I / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D016472:Motor Neuron Disease / Q000188:drug therapy*; D018579:Patient Selection; D016896:Treatment Outcome
D007334:Insulin-Like Growth Factor I