Subcutaneous IGF-1 is not beneficial in 2-year ALS trial.
BACKGROUND
Previous human clinical trials of insulin-like growth factor type I (IGF-1) in amyotrophic lateral sclerosis (ALS) have been inconsistent. This phase III, randomized, double-blind, placebo-controlled study was undertaken to address whether IGF-1 benefited patients with ALS.


METHODS
A total of 330 patients from 20 medical centers were randomized to receive 0.05 mg/kg body weight of human recombinant IGF-1 given subcutaneously twice daily or placebo for 2 years. The primary outcome measure was change in their manual muscle testing score. Secondary outcome measures included tracheostomy-free survival and rate of change in the revised ALS functional rating scale. Intention to treat analysis was used.


RESULTS
There was no difference between treatment groups in the primary or secondary outcome measures after the 2-year treatment period.


CONCLUSIONS
Insulin-like growth factor type I does not provide benefit for patients with amyotrophic lateral sclerosis.
Sorenson
E J
EJ
Department of Neurology, Mayo Clinic, 200 1st St. SW, Rochester, MN 55905, USA. sorenson.eric@mayo.edu
Windbank
A J
AJ
Mandrekar
J N
JN
Bamlet
W R
WR
Appel
S H
SH
Armon
C
C
Barkhaus
P E
PE
Bosch
P
P
Boylan
K
K
David
W S
WS
Feldman
E
E
Glass
J
J
Gutmann
L
L
Katz
J
J
King
W
W
Luciano
C A
CA
McCluskey
L F
LF
Nash
S
S
Newman
D S
DS
Pascuzzi
R M
RM
Pioro
E
E
Sams
L J
LJ
Scelsa
S
S
Simpson
E P
EP
Subramony
S H
SH
Tiryaki
E
E
Thornton
C A
CA
D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000503:physiopathology*; D002491:Central Nervous System Agents / Q000008:administration & dosage* / Q000009:adverse effects; D003679:Deglutition; D004311:Double-Blind Method; D005260:Female; D018737:Hand Strength; D006801:Humans; D007279:Injections, Subcutaneous; D007334:Insulin-Like Growth Factor I / Q000008:administration & dosage* / Q000009:adverse effects; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D012107:Research Design; D013923:Thromboembolism / Q000139:chemically induced; D013997:Time Factors; D014139:Tracheostomy; D017211:Treatment Failure
D002491:Central Nervous System Agents; D007334:Insulin-Like Growth Factor I