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Thalidomide treats blood disorder

6th April 2010

Thalidomide may be useful in treating a rare blood disorder affecting one person in every 5,000, according to a recent French study.

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Though the drug was once prescribed in order to treat morning sickness in pregnant women, the drug was eventually banned for its high risk of birth defects.

However, the sedative can be used to treat hereditary haemorrhagic telangiectasia (HHT), which causes persistent nosebleeds in sufferers who have inherited the disorder.

The drug can stop such nosebleeds by stabilising blood vessels, as it did in test rats whose genetic makeup had been engineered to mimic HHT.

The stabilising influence of the drug is due to its ability to increase PDGF-B, one of the body's proteins.

Thalidomide was sold between 1957 and 1961, during which time about 10,000 babies were born with birth defects caused by the sedative.

After the countries where the babies were born linked the effect of the drug in the mother to birth defects in children, the drug was banned.

While the drug is still strictly controlled and only sold to researchers, other studies have shown that it can treat leprosy and certain cancers such as multiple myeloma.

Study leader Franck Lebrin, of the National Institute for Health and Medical Research (INSERM) in Paris, said that HHT repaired blood vessels and caused cell growth, which alleviated the effects of HHT in the test rats.

He said that biopsies of the nasal surface tissue from patients with HHT showed similar mechanisms at work, which may explain the effects of thalidomide treatment in humans.

Patients with HHT have a very low quality of life, due to the fact that their recurrent nosebleeds are very difficult to stop.

Paul Oh, an experimental geneticist at the University of Florida, Gainesville, said that he believed it was possible for thalidomide to help all HHT sufferers, and that thalidomide therapy was the first major therapy the HHT community had ever seen.

Rosemary Akhurst, a molecular biologist at the University of California in San Francisco, said that she believed researchers should look for a drug with similar effects and a safer reputation.

She said that, ultimately, she did not want anyone to be using a drug as hazardous as thalidomide, but that the researchers might point toward a drug that benefited patients without causing any dangerous side-effects.

 

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