Hemangiomas -- strawberry-like birthmarks that commonly develop in early infancy -- are generally harmless, but up to 10 percent cause tissue distortion or destruction and sometimes obstruction of vision or breathing.
Since the 1960s, problematic hemangiomas have been treated with corticosteroids such as dexamethasone or prednisone. But steroids have considerable side effects, don't always work, and their mechanism of action in hemangioma has remained a mystery.
Researchers at Children's Hospital Boston recently discovered that infantile hemangiomas originate from stem cells, and have used these stem cells to better understand this tumor in the laboratory. In the March 18 issue of The New England Journal of Medicine, they show that steroids target hemangioma stem cells specifically, reveal their mechanism of their action and suggest other possible ways to halt and shrink hemangiomas.
Steroids usually result only in stabilization of hemangioma growth, and about 30 percent of hemangiomas don't respond to steroid treatment. Steroids also have side effects including facial swelling, hyperactivity, growth retardation and increased blood pressure. Although the effects on appearance may seem minor, research indicates that a baby's physical appearance can interfere with maternal bonding.
"My dream has always been to give a drug to stop hemangioma at its first appearance," says Children's plastic surgeon John Mulliken, MD, co-director of Children's Vascular Anomalies Center and a co-author on the study.
VEGF-A was detected in actively growing hemangiomas, but not in regressing (involuting) hemangiomas.
"Steroids are inhibiting expression of a central regulator of blood vessel growth: VEGF-A," says Bischoff. "But we'd like to target the stem cell itself - stop its proliferation, prevent it from differentiating into unwanted blood vessels and, at the same time, eliminate the cellular source of VEGF-A."
The study was funded by the National Institutes of Health, the Translational Research Program at Children's Hospital Boston, a Harvard Skin Diseases Pilot Study Grant, Sheba Medical Center (Israel), and the John Butler Mulliken Foundation.Citation: Greenberger S, Boscolo E, Adini I, Mulliken J and Bischoff J. Corticosteroid suppression of VEGF-A in infantile hemangioma-derived stem cells. N Engl J Med 2010 Mar 18; 362(11):30-38.
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