The study suggests that thalidomide could save the lives of patients struggling with a rare blood vessel disease.
The notorious drug killed and injured as many as 100,000 children in the 1950s, and severely maimed thousands, injuring their limbs, ears, and eyes.
But scientists now offer a “breakthrough” in the treatment of severe arteriovenous malformations (AVMs) – abnormal blood vessel nodes.
The condition, which can occur in any part of the body, can be unbearable, causing bleeding and even paralysis.
Current treatment options include surgery to remove the circulating blood vessels and injections to keep blood from flowing through them.
Thalidomide was hailed as a “miracle cure” for morning sickness after it was created in the 1950s by German pharmaceutical giant Gruenenthal Group. But when he retired in 1961, he was blamed for the deaths of 100,000 children and the death of 10,000 more severely – including missing or deformed limbs.
Thalidomide was hailed as a “miracle cure” for morning sickness when it was developed by German pharmaceutical giant Gruenenthal Group in the 1950s.
But it was withdrawn soon after an Australian doctor reported a link between the drug and birth defects such as deformed hands, facial disfigurements and brain damage.
Charities blame the drug for the deaths of up to 100,000 children worldwide and say it has left 10,000 seriously disabled, such as missing or deformed limbs.
Experiments later revealed that it inhibited the formation of blood vessels in infants, causing birth defects.
According to the Belgian researchers who made the discovery, the same mechanism works in the treatment of AVM.
The results showed that pain was reduced, bleeding stopped, and ulcers healed in all participants.
Study author Professor Miikka Vikkula said: “The results are convincing and we hope that they will be confirmed by larger studies.
“We thought thalidomide would work in these patients, so our results were not a surprise.
“But getting clinical confirmation that we were right was great.
Professor Vikkula of the De Duve Institute added: “For us, this is a revolutionary discovery”.
About 14 people per million suffer from AVM in early pregnancy, which can be caused by an error in blood vessel formation.
If the tangled blood vessels are close to the skin, the condition can cause blue, purple, or red discoloration and swelling. The skin can also be sensitive and prone to sores.
AVM patients’ hearts have to work harder to keep up with the extra blood flow that can lead to heart problems.
People with small AVMs do not need treatment, but others may need surgery to remove the nodes.
Other treatment options include embolization, which is an injection that destroys blood vessels. However, it is not always effective.
Although most AVM patients lead relatively normal lives, there is a risk that abnormal tangles can burst and cause paralysis.
The complication affects 1% of patients each year.
Previous research by the Belgian team found that AVM is caused by mutations in cells in blood vessel walls that promote abnormal formations.
Professor Vikkula said the discovery “causes us to question the possibility of using thalidomide to prevent their creation.”
About 14 per million people suffer from AVM in early pregnancy, which can be caused by an error in blood vessel formation. If the tangled blood vessels are close to the skin, the condition can cause blue, purple, or red discoloration and swelling. The skin can also be sensitive and prone to sores. AVM patients’ hearts have to work harder to keep up with the extra blood flow that can lead to heart problems. People with small AVMs do not need treatment, but others may need surgery to remove the nodes.
Thalidomide interferes with a protein called vascular endothelial growth factor, which stimulates the growth of new blood vessels.
AVM patients have high levels of protein in their arteries, which is thought to be a mess.
Eighteen patients aged 19 to 70 participated in the study, which will be presented at a medical conference this week.
Participants took 50 mg, 100 mg, or 200 mg of thalidomide daily for two months to four years.
All were followed for four and a half years after they stopped taking the medication.
The patients also agreed to use birth control for at least one month before the start of the study and for four weeks after the end of the study, and required men to pledge to wear condoms during sex.
The findings, previously published in the journal Cardiovascular Research on Nature, show that all patients experienced rapid pain relief, stopped bleeding and healed ulcers.
The patients with heart failure recovered, and one “recovered completely,” the researchers said.
The team also found that when patients received thalidomide and embolized with injection therapy, their dose could be reduced from 100 mg or 200 mg to 50 mg.
Reducing the dose reduces side effects such as fatigue and numbness in the limbs.
All eight patients had stable AVMs five and a half years after the study.
The researchers noted that the cost of treating AVM patients with thalidomide was up to 12 times cheaper than other drugs tested for the condition.
The research will be presented Sunday at the annual conference of the European Society for Human Genetics in Vienna.
Professor Alexandre Reymond, chair of the conference, said: “This study demonstrates not only the health and economic benefits of drug reuse – even the most underrated – but also how genetic research can lead to real breakthroughs in therapies to use”. .treatment, uncomfortable conditions.’
Although taken for morning sickness in 1961, thalidomide is still used today. However, there are strict guidelines for women of childbearing age taking the drug.
It is given to patients with myeloma, a cancer that starts in the bone marrow, and to treat Hansen’s disease (also known as leprosy), an infection caused by slow-growing bacteria.
Source: Daily Mail
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