Thalidomide's latest comeback

Thalidomide may be useful for the treatment of a hereditary condition that affects the blood vessels, suggests a report online in this week's Nature Medicine.

Hereditary hemorrhagic telangiectasia (HHT) is an inherited disorder characterized by malformations of the blood vessels. Many patients develop recurrent, difficult-to-treat nosebleeds, which can severely affect quality of life. Franck Lebrin and his colleagues report that treating HHT patients with thalidomide reduces the severity and frequency of nosebleeds. In an experimental mouse model of HHT, thalidomide treatment rescued vessel wall defects through a mechanism involving a growth factor known as PDGF. Biopsies of the nasal surface tissue from patients with HHT showed that similar mechanisms may explain the effects of thalidomide treatment in humans.

Thalidomide was originally used to treat nausea during pregnancy in the 1960s, but the drug was removed from the market after severe congenital defects appeared in newborns. More recently, thalidomide has experienced a revival and is being used to treat certain forms of cancer. Its potential use in people with HHT adds to the resurgence of this famous drug.