Thalidomide IS safe… but only for treating rare blood vessel disorders

Thalidomide could save the lives of patients suffering from a rare blood vessel disease, research suggests.

The notorious drug killed and injured up to 100,000 babies in the 1950s, leaving thousands severely disabled with 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.

The condition, which can occur anywhere in the body, can prove unbearable, causing bleeding and even triggering strokes.

Current treatment options include surgery to remove the tangled vessels and injections to block blood flow through them.

Thalidomide was hailed as a “miracle drug” for morning sickness after it was developed by German pharmaceutical giant Grünenthal Group in the 1950s. But it was blamed for the deaths of 100,000 babies and left 10,000 severely disabled – such as with missing or deformed limbs – when it was withdrawn in 1961

Thalidomide was hailed as a “miracle drug” for morning sickness when it was developed by German pharmaceutical giant Grünenthal Group in the 1950s.

But it was soon withdrawn after a doctor in Australia reported a link between the drug and birth defects such as deformed hands, facial disfigurement and brain damage.

Charities blame the drug for the deaths of up to 100,000 babies worldwide and say it left 10,000 severely disabled – with missing or deformed limbs, for example.

Experiments later showed that it caused birth defects by stopping blood vessels from forming in babies.

The same mechanism is why it works in treating AVM, according to the Belgian researchers who made the discovery.

The results showed that all of the participants experienced reduced pain, stopped bleeding, and had ulcers heal.

Study author Professor Miikka Vikkula said: “The results are compelling and we hope they will be confirmed by larger studies.

“We hypothesized that thalidomide should work in these patients, so our results were not surprising.

“But it was great to get clinical confirmation that we were right.

Professor Vikkula of the de Duve Institute added: ‘From our point of view, this is a groundbreaking finding.’

About 14 out of every million people suffer from AVM, which is believed to be caused by a failure in the formation of blood vessels in early pregnancy.

If the involved 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 developing 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 with small AVMs don’t need treatment, others may need surgery to remove the tangles.

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, the abnormal nodes are at risk of rupturing and triggering a stroke.

The complication affects 1 percent of patients each year.

Previous research by the Belgian team found that AVM is caused by mutations in cells found in the walls of blood vessels that promote abnormal formations.

Professor Vikkula said the discovery “led us to consider the possibility of using thalidomide to inhibit their formation”.

About 14 out of every million people suffer from AVM, which is believed to be caused by a failure in the formation of blood vessels in early pregnancy. If the involved 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 developing 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 with small AVMs don’t need treatment, others may need surgery to remove the tangles

Thalidomide interferes with a protein called vascular endothelial growth factor, which promotes the growth of new blood vessels.

AVM patients have high protein levels which are thought to promote tangles in their vessels.

Eighteen patients, ranging in age from 19 to 70, were involved in the study, which will be presented at a medical conference this week.

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

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

Patients also agreed to use contraception for at least one month before the start of the study and for four weeks after the end of the study, with men having to promise to wear condoms during sex.

The results, previously published in the journal Nature Cardiovascular Research, show that all patients experienced rapid pain relief, stopped bleeding and ulcers healed.

Heart failure patients recovered and one “appears to be fully healed,” the researchers said.

The team also found that patients taking thalidomide who were undergoing injection treatment were able to reduce their dose from 100 mg or 200 mg to 50 mg.

Stopping the dose reduced side effects such as fatigue and numb limbs.

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

The researchers found that the cost of treating AVM patients with thalidomide is up to 12 times lower than other drugs being tested for the condition.

The research results will be presented at the annual meeting of the European Society for Human Genetics in Vienna on Sunday.

Professor Alexandre Reymond, chair of the conference, said: “This study shows not only the health and economic benefits of reusing medicines – even the most maligned ones – but also how genetic research is leading to real breakthroughs in therapies for difficult-to-treat, burdensome drugs can lead to diseases.’

Although thalidomide was pulled for morning sickness in 1961, it is still used today. However, there are strict rules for women of childbearing age using the drug.

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