Rush University Medical Center is participating in a clinical trial to evaluate the potential benefit of the first major innovation in 20 years for the treatment of growth failure.
The drug, called Increlex, was approved by the FDA August 31 for the most severe form of short stature due to a deficiency of Insulin-like Growth Factor-1 (IGF-1). Ongoing trials will determine if the drug may be used for less severe growth disease.
Insulin-like Growth Factor-1 (IGF-1) is the proximate hormone necessary for statural growth and must be present in order for children's bones, cartilage and organs to grow normally. In healthy individuals, growth hormone is secreted into the bloodstream by the pituitary gland and binds to growth hormone receptors on liver and other cells, where it stimulates the cellular production and secretion of IGF-1 into the bloodstream.
"For decades, the only available drug treatment for short stature has been shots of growth hormone. However, patients who are deficient in IGF-1 and resistant to the effects of growth hormone do not respond well, if at all, to the shots," said Dr. Richard Levy, a pediatric endocrinologist at Rush. "Instead of using growth hormone to stimulate the production of IGF-1, the goal is to replace the IGF-1 directly."
Increlex is a genetically engineered copy of IGF-1. The purified protein has been shown to be structurally and functionally identical to natural human IGF-1. It is injected daily before a meal to provide the catalyst the body needs to grow.