Home Science & Technology Thalidomide is an effective remedy for the treatment of abnormal blood vessel...

Thalidomide is an effective remedy for the treatment of abnormal blood vessel formations. – Researcher


The same properties that caused birth defects when given to pregnant women – inhibition of blood vessel formation (anti-angiogenesis), led to interest in the therapeutic utility of thalidamide in other areas. At the annual conference of the European Society of Human Genetics on Sunday, Professor Mike Vickula of the Institut de Duve, Université Catholique de Louvain, Brussels, Belgium, will present the results of a study of thalidamide use in patients with severe arteriovenous malformations. (AVMs). These are the results released today (Friday) The nature of cardiovascular researchdemonstrate a dramatic reduction in symptoms and a subsequent improvement in quality of life.

AVMs are abnormal lumps of blood vessels that connect arteries and veins that alter normal blood flow. They are very painful, causing bleeding and deformity of the affected part of the body, as well as heart problems. Usually they are congenital, they are often only noticeable in adolescence or adulthood when a person is growing up. Treatment of severe cases is usually performed by surgery or embolization (injection of an agent that locally destroys blood vessels, thus causing scar tissue), although this is rarely completely effective and can worsen the situation.

Some people with AVM may live a relatively normal life, but even in less severe cases there is always a risk that abnormal lumps of blood vessels can rupture and cause a stroke. Approximately every hundred patients with AVM suffer a stroke each year.


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“Our group has been studying the causes of vascular abnormalities for 30 years,” says Professor Vickula. “We have identified several genetic causes and have been able to show that some mutations activate signaling within blood vessel wall cells, and this contributes to the pathological formation of blood vessels (angiogenesis). This made us think about the possibility of using thalidamide to inhibit the pathological formation of blood vessels.

Demonstrating that vascular defects can be corrected on a mouse model, Professor Lawrence Boone of the Center for Vascular Anomalies at St. Luke’s University Hospital in Brussels, who has worked with Professor Vickula for 30 years, recruited 18 patients with AVM to study their thalidamide use. becomes. They ranged in age from 19 to 70, and all had severe malformations that could not be treated with conventional approaches. Patients had to agree to use contraception for at least four weeks before starting thalidamide and to continue for four weeks after stopping treatment. Because thalidamide is present in semen, men also had to agree to use condoms during sex.

Patients received either 50 mg, 100 mg, or thalidamide 200 mg daily for two to 52 months. Eight AVMs were stable after a mean discontinuation of thalidamide at 54 months, and four recurred after a mean duration of 11.5 months. Combination therapy with embolization, when arteries or veins in the AVM are blocked by an agent that destroys vascular wall cells, has reduced the dose of thalidamide to 50 mg per day in five patients. Dose reduction where possible was important, says Professor Vickula, because the higher dose was associated with side effects, especially fatigue and peripheral neuropathy, damage to nerves located outside the brain and spinal cord that causes weakness and numbness, especially in arms and legs.

“All patients experienced a rapid reduction in pain as well as cessation of bleeding and healing of ulcers where they were present,” says Professor Vickula. “Three patients with heart failure also disappeared, and one AVM was completely cured after 19 months of taking thalidamide and eight years of follow-up.

“We know that thalidomide acts through vascular endothelial growth factor (VEGF), a signaling protein that promotes the growth of new blood vessels. VEGF levels are high in vascular abnormalities such as AVM, and thus it is likely that thalidamide reduces signaling through pathways that promote angiogenesis. Although our research is only small, the results are convincing, and we hope that they will be confirmed by larger tests. “

An additional advantage of using thalidamide in the treatment of AVM is the cost. Two other drugs, recently developed for use in oncology and being tested for the treatment of AVM, are twelve times more expensive and also have many side effects.

“We suggested that thalidamide should work in these patients, so our results were not a surprise, but it was great to have clinical evidence that we were right,” concludes Professor Vickula. “In our view, this is a groundbreaking finding and provides a solid foundation for the development of molecular treatments for AVM.”

Professor Alexander Raymond, chairman of the conference, said: “This study shows not only the medical and economic benefits of drug reprofiling – even the most broken – but also how genetic research can lead to real breakthroughs in difficult-to-treat, painful conditions. ».

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