A Phase III Trial of IGF-1 in Patients with ALS
Study Complete
Disappointing Results from IGF-1 Clinical Trial
Subcutaneous (under the skin) delivery of insulin-like growth factor 1 (IGF-1), known as the drug Myotrophin, does not benefit people with ALS at a dose of 0.5 milligrams per kilogram of body weight, according to a large clinical trial whose results were released today.
IGF-1 is a substance the body produces to sustain motor neurons, the nerve cells that die in ALS. Experiments in animal models of the disease suggested IGF-1 treatment may delay death of motor neurons. IGF-1 was tested in ALS a decade ago in two trials, but the results of the two were inconsistent, with one suggesting treatment was beneficial, and the other showing no benefit.
“These results are deeply disappointing to all of us in the ALS community,” said Lucie Bruijn, Ph.D., senior vice president, research and development, The ALS Association. “The subcutaneous delivery route may be the key problem, or it may be that IGF-1 alone is not sufficient to rescue motor neurons.”
The current trial involved 330 people with ALS from 20 ALS treatment centers across the United States. Patients were randomly assigned to receive either IGF-1 or a placebo, injected under the skin twice a day, for two years. The dose used was the highest tolerated dose from previous studies. Neither doctors nor patients knew which treatment the patient had received until the end of the study.
At the end of the two-year treatment period, there were no differences between people with ALS who received IGF-1 and those who received placebo in muscle strength, the need for a tracheostomy for breathing, or survival, indicating that IGF-1 provided patients no benefit.
The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information. The Association is currently exploring multiple new avenues for treatment through its TREAT-ALS (Translational Research Advancing Therapies for ALS) drug discovery program and clinical trials process.
For more information about The ALS Association’s research program, please visit the research section of our web site.
