New use for once-cursed drug Thalidomide?

Apr 04, 2010

Thalidomide, the sedative blamed for tragic birth defects half a century ago, treated a rare inherited blood disorder, according to lab experiments reported on Sunday.

Around one person in 10,000 has a disorder called hereditary haemorrhagic telangiectasia, or HHT, which causes frequent, hard-to-treat nosebleeds.

French doctors, experimenting on mice genetically engineered to have HHT, found that reduced the risk of haemorrhage and stabilised blood vessels.

The drug works by boosting a protein called PDGF-B, the team from the National Institute of Health and Medical Research (Inserm) report in the journal Nature Medicine.

The work is experimental, aimed at showing the pathways by which HHT could be cured.

Thalidomide was sold between 1957 and 1961 until it was found that pregnant women who took the drug for morning sickness were at high risk of having a child missing a limb or having stunted arms.

More than 10,000 babies, especially in Germany, Britain, Australia and Canada, were affected.

The scandal led to a tightening of vetting procedures for drug prototypes and scrutiny of claims by pharmaceutical giants.

Thalidomide remains outlawed for general distribution.

In recent years, though, interest in it has revived as a research tool but also as a treatment for a form of cancer called and side-effects from . It is prescribed to patients only in very tightly-controlled conditions.

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