New Use Identified for Birth Deformity Drug

According to a study just published in Nature Medicine, thalidomide, a drug found to cause birth defects when it was launched as a morning sickness pill half a century ago, may be useful for treating a condition that affects blood vessels.

French researchers found giving thalidomide to patients with hereditary hemorrhagic telangiectasia (HHT) reduced the severity and frequency of nosebleeds, one of the main symptoms.

HHT affects about one in 5,000 people. Many patients develop recurrent, difficult-to-treat nosebleeds which can significantly harm their quality of life.

Researchers from the National Institute for Health and Medical Research (INSERM) in Paris, said experiments on mice with HHT showed that thalidomide treatment increased platelet-derived growth factor-B (PDGF-B) expression in endothelial cells and appeared to repair blood vessel walls.  Additionally, biopsies of the nasal surface tissue from patients with HHT suggested that similar mechanisms may explain the effects of thalidomide treatment in humans.

Thalidomide was sold as a treatment for morning sickness 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 with severe congenital defects, notably missing or stunted limbs.

The drug was taken off the market, but more than 10,000 babies, especially in Germany, Britain, Australia and Canada, were affected.

The scandal led to a tightening of the approval procedures for new drugs in several countries, including the US.
Although thalidomide remains outlawed for general distribution, in recent years, it has experienced a revival, being used under very tightly-controlled conditions to treat certain forms of cancer such as multiple myeloma and side-effects from leprosy.

Word on Health wonders how many more uses will be found for this drug that was once considered so disastrous.

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