Thalidomide could be a life-saver for patients battling deadly blood vessel condition — and scientists say it’s down to the same mechanism that causes birth defects

Research suggests that thalidomide may save the lives of patients battling a rare blood vessel condition.

The infamous drug killed and injured 100,000 children and severely disabled thousands in the 1950s, causing damage to their limbs, ears, and eyes.

But it offers a ‘breakthrough’ in the treatment of severe arteriovenous malformations (AVMs) – abnormal tangles of blood vessels, scientists now say.

This condition, which can occur anywhere in the body, can prove excruciating, cause bleeding and even lead to stroke.

Current treatment options include surgery to remove the clogged vessels and injections that block blood from flowing through them.

Thalidomide is touted as a ‘wonder drug’ for morning sickness, created in the 1950s by the German pharmaceutical giant Gruenthal Group. But it was blamed for 100,000 infant deaths and 10,000 severely disabled—such as missing or deformed limbs—by the time it was withdrawn in 1961.

How did the Thalidomide scandal unfold?

1953: Drug made by Gruenthal Group in Germany

1958: Thalidomide is licensed for use in the UK for the first time

1961: Australian doctor William McBride reported an increase in births of malformed babies to mothers taking thalidomide at his hospital. The drug was withdrawn later that year.

1968: UK manufacturer Distillers Biochemicals Ltd (now Diageo) reached a compensation settlement after a legal battle by the affected families

2005: Diageo increases its compensation payments from £2.8m to approximately £6.5m a year

2008: Drug approved by European Medicines Agency to treat multiple myeloma – bone marrow cancer

2009: The UK Government agrees a £20m grant to be paid over three years to the Thalidomide Trust

2010: Health Minister Mike O’Brien made a formal apology on behalf of the government to thalidomide victims

Thalidomide was touted as a ‘wonder drug’ for morning sickness when it was created by the German pharmaceutical giant Gruenthal Group in the 1950s.

But it was soon pulled down after a doctor in Australia reported a link between the drug and birth defects, such as deformed hands, facial deformities, and brain damage.

The charity blames the drug for the deaths of 100,000 children worldwide, and says it left 10,000 seriously disabled – such as those with missing or deformed limbs.

Later experiments showed that it caused birth defects in babies by preventing blood vessels from forming.

According to the Belgian researchers who made the discovery, the same mechanism works in the treatment of AVMs.

The results showed that pain decreased, bleeding stopped, and ulcers healed in all participants.

Study author Professor Mikka Vikula said: ‘The results are reassuring, and we hope to confirm them with larger trials.

‘We hypothesized that thalidomide should work in these patients, so our results did not come as a surprise.

‘But it was great to have the clinical confirmation that we were right.

Professor Vikkula from the De Duve Institute said: ‘In our view, this is a successful discovery.’

About 14 people per million suffer from AVMs, which are caused by an error in the formation of blood vessels in early pregnancy.

If the clogged vessels are near the skin, the condition can cause blue, purple or red discoloration and swelling. The skin can also be sensitive and prone to ulcers.

The heart of AVM patients has to work harder to keep up with the extra blood flow, which can lead to heart problems.

People who have small AVMs do not need treatment but others may need surgery to correct complications.

Other treatment options include embolization — an injection that destroys blood vessels. However, it is not always effective.

While most AVM patients lead relatively normal lives, there is a risk that abnormal entanglements may burst and trigger a stroke.

The complication affects 1 percent of patients each year.

Earlier research by the Belgian team found that AVMs are caused by mutations in cells located in the walls of blood vessels, which encourage abnormal formations.

Professor Vikkula said the discovery ’caused us to think about the possibility of using thalidomide to prevent their manufacture’.

About 14 people per million suffer from AVMs, which are caused by an error in the formation of blood vessels in early pregnancy. If the clogged vessels are near the skin, the condition can cause blue, purple or red discoloration and swelling. The skin can also be sensitive and prone to ulcers. The heart of AVM patients has to work harder to keep up with the extra blood flow, which can lead to heart problems. People who have small AVMs do not need treatment but others may need surgery to correct complications.

Thalidomide interferes with a protein, called vascular endothelial growth factor, which encourages new blood vessels to grow.

AVM patients have higher levels of the protein, which is believed to encourage tangles in their vessels.

The study involved eighteen patients aged 19 to 70, which will be presented this week at a medical conference.

Participants took 50 mg, 100 mg or 200 mg of thalidomide per day for between two months and four years.

They were all monitored for four and a half years after they stopped taking the drug.

Patients agreed to use contraception for at least one month before the start of the study and for four weeks after the end of the trial, in which men were promised condoms during sex.

Results already published in the journal Nature Cardiovascular Research showed that all patients experienced a rapid reduction in pain, their bleeding stopped and ulcers healed.

The heart failure patients recovered and one made a ‘complete recovery’, the researchers said.

The team also found that when patients took thalidomide and had injection treatment embolization, they were able to reduce their dosage from 100mg or 200mg to 50mg.

Lowering the dose reduces side effects, such as fatigue and numb limbs.

Eight patients also had stable AVMs after five and a half years of study.

The researchers noted that the cost of treating AVM patients with thalidomide is 12 times cheaper than other drugs being 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 Raymond, chair of the conference, said: ‘This study not only shows the economic benefits of repurposing health care and drugs – even the deadliest ones – but also how genetic research can help to find a cure. can lead to real successes in difficult, troubling situations.’

Despite being pulled down for morning sickness in 1961, thalidomide is still used today. However, there are strict rules on women of child-bearing age using the drug.

It is given to patients with myeloma, a type of cancer that begins in the bone marrow, and for the treatment of Hansen’s disease (also known as leprosy), which is caused by slow-growing bacteria. infection occurring.