Research press release


Nature Medicine

Thalidomide's latest comeback


遺伝性出血性毛細管拡張症(HTT)は血管の異形成を特徴とする遺伝性疾患で、多くの患者は鼻血を繰り返し出すが、この鼻出血は治療が難しく、QOL(クオリティ・オブ・ライフ)に大きく影響する。F Lebrinたちからの報告によれば、HHT患者にサリドマイドを投与すると鼻出血の程度が軽くなり、回数も減るという。HHTの実験マウスモデルでは、サリドマイド治療により血管壁の異常が回復し、それには成長因子PDGFが関与していた。HHT患者の鼻腔面組織の生検によって、ヒトのサリドマイド治療の効果も、同じ仕組みで説明できることがわかった。


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.

doi: 10.1038/nm.2131


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