AN INFAMOUS drug which left more than 10,000 babies severely handicapped could be redeveloped to safely treat HIV and leprosy following a historic breakthrough by Scottish scientists.
For the first time, experts at Aberdeen University have uncovered how and why limbs are targeted by thalidomide, which caused serious physical defects in infants whose mothers had taken it to relieve morning sickness.
In Third World countries where thalidomide is used to treat leprosy, it continues to leave babies handicapped.
But the Aberdeen discovery could save thousands of lives by removing the component from the drug which stunts the growth of new blood vessels in developing babies.
By isolating the component, experts say that the drug’s anti-inflammatory abilities and its effects on the immune system could be harnessed to treat serious and life-threatening illness such as HIV and Crohn’s disease.
The research was hailed as a “major discovery” last night – but a former Scottish Government minister warned that the public still fears the infamous drug.
Thalidomide was available to pregnant women in the late 50s but was taken off the UK market in 1961 after it was linked to babies born with serious defects. Some had stunted arms or legs and in some cases no limbs.
In a paper published today in the prestigious Proceedings of the National Academy of Sciences journal, the research team at Aberdeen demonstrates not only which part of the drug causes the limb defects, but also how limb defects then arise.
Dr Neil Vargesson, lead researcher and lecturer in developmental biology at the university, said: “We have put to rest a 50-year puzzle in finally deducing how thalidomide triggers limb defects and why it appears to target limbs preferentially.
“This has become more urgent since the drug is now used around the world to treat leprosy and multiple myeloma, due to the drug’s anti-inflammatory abilities and its effects on the immune system. Tragically, some children are still being born today with thalidomide-induced limb defects in South America and Africa, where it is used as a treatment for leprosy.
“Now we understand which property of the drug causes limb defects, it remains possible that we could make a safer form of the drug that has clinical benefits for sufferers of leprosy but does not cause limb defects.”
Sufferers of HIV and Crohn’s disease could also benefit, he said.
Thalidomide has several active components. The team found only one active element causes abnormalities. It has “antiangiogenic” properties, which means it inhibits the growth of new blood vessels.
Dr Vargesson added: “Thalidomide is a complicated drug with many actions, but which of these cause limb defects has eluded researchers for many years.
“Many theories have been put forward but this is the first paper to conclusively show it is the antiangiogenic property of the drug that is to blame for the defects.”
The drug’s ability to stop blood vessels growing has already been harnessed to treat cancerous tumours and cancer of the bone marrow.
Dr Vargesson said a new version of the drug could now be developed to treat early forms of cancer.
Thalidomide Trust director Dr Martin Johnson was sceptical, saying he would wait to see the full report before welcoming the findings.
Scottish Lib Dem health spokesman Ross Finnie said the researchers had made a “significant discovery” – but warned that the public still fear thalidomide. He said: “Due to the high levels of public concern over thalidomide, there must be extensive testing to reassure an understandably nervous public any redevelopment of this drug has been made safe.”
