Insulin-like Growth Factor-1 (IGF-1) is an important 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 that binds to growth hormone receptors on liver and other cells, where it stimulates the cellular production and secretion of IGF-1 into the bloodstream.
Any defect in the above mentioned pathway can lead to growth failure and short stature. Supplementation with human growth hormone is the currently available method for management of the condition. However, patients who are deficient in IGF-1 and resistant to the effects of growth hormone do not respond well to the treatment.
Primary IGFD afflicts an estimated 30,000 children evaluated for short stature in the United States. A child with short stature is defined as being shorter than 97.5 percent of all children the same age and gender.
If untreated, primary IGFD may lead, in children and adults, to a range of other metabolic disorders, including lipid abnormalities, decreased bone density, obesity, and insulin resistance.
A clinical trial aiming at replacing IGF-1 directly rather than using growth hormone to stimulate the production has been initiated. 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).
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.
Ongoing clinical trails would be directed towards assessment the efficacy, safety and tolerability of the drug over the conventional treatment.