Studies in mice and humans conducted by an international group of investigators indicate that thalidomide may be an effective treatment for hemorrhagic telangiectasia (HHT), an inherited disorder that is characterized by vascular malformations (Lebrin F et al. Nat Med. 2010;16:420-428). Many with the disease develop recurrent nosebleeds that can severely affect their quality of life and are difficult to treat.
In a mouse model of HHT, thalidomide treatment rescued vessel wall defects, increased platelet-derived growth factor B expression in endothelial cells, and stimulated activation of cells involved in the formation of normal vasculature. The effects were partially reversed by pharmacological or genetic interference with platelet-derived growth factor signaling.
Hampton T. Thalidomide for Vascular Disorder. JAMA. 2010;303(21):2129. doi:10.1001/jama.2010.688