Thalidomide was once a dreaded name in the Pharmaceutical industry. It was initially marketed in some European countries nearly 5 decades back as a sedative and after millions of pregnant women took it for morning sickness it had devastating results on the babies. The babies were born with flippers instead of limbs a condition known as phocomelia. Since then it has been under strict control.
Now reports have emerged this Sunday of its utility in a rare hereditary blood disorder called hereditary haemorrhagic telangiectasia, or HHT, which causes frequent, hard-to-treat nosebleeds. The occurrence of this disease is really rare with only one in 10,000 getting it.
Research on Thalidomide has for many years been concentrated on its utility in other diseases like Amyotrophic lateral sclerosis, Behcet's syndrome, Brain, Breast and Colorectal cancer, Congestive heart failure, Crohn's disease, HIV infections, Hodgkin's disease, Kaposi's sarcoma, Leukaemia, Myelofibrosis, Myeloid leukaemia and Non-Hodgkin's lymphoma.
Since then a drug with similar chemical structure called Revlimid was made by US ‘pharma’ company Celgene and approved for use in multiple myeloma.
Researchers in France have shown that genetically engineered mice who had HHT showed beneficial results when given Thalidomide. There was a reduced risk of bleeding and blood vessels were better stabilized with Thalidomide. According to the scientists from the National Institute of Health and Medical Research (INSERM) as they report in the journal Nature Medicine, Thalidomide acts here by improving a protein called PDGF-B.
Franck Lebrin, one of the lead researchers of this study said "Biopsies of the nasal surface tissue from patients with HHT showed that similar mechanisms may explain the effects of thalidomide treatment in humans."
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