Thalidomide is an effective treatment for abnormal angioplasty

The same properties of thalidomide that caused birth defects when given to pregnant women-inhibition of angiogenesis (anti-angiogenesis) -leaded to interest in the therapeutic utility of thalidomide in other areas. I am. At the annual meeting of the European Society of Anthropology on Sunday, Professor Miikka Vikkula of the de Duve Institute at the University of Louvain in Brussels, Belgium, presents the results of a study on the use of salidamide in patients with severe arteriovenous malformations. .. (AVM).These results were published today Nature Cardio Vascular ResearchShows a marked reduction in symptoms and subsequent improvement in quality of life.

AVM is an abnormal entanglement of blood vessels that connect arteries and veins, altering normal blood flow. They are very painful and cause bleeding and deformity of the affected area and heart problems. It is usually congenital and often only stands out in adolescence or adulthood as a person grows up. Treatment of severe cases is usually done by surgery or embolization (injection of a drug that locally destroys blood vessels and induces scar tissue), but this is rarely completely effective and makes things worse. May cause you to.

Some people with AVM can lead a relatively normal life, but even if it is not so serious, an abnormal entanglement of blood vessels ruptures and causes a stroke while in the brain. There is always a risk of causing it. About 1 in 100 AVM patients suffers a stroke each year.

“Our group has been studying the causes of vascular abnormalities for 30 years,” says Professor Vikkula. “We have identified some Genetic cause We were able to show that certain mutations activate signal transduction within the parietal cells of blood vessels, which promotes abnormal formation of blood vessels (angiogenesis). This made me wonder about the possibility of using thalidomide to suppress abnormal angioplasty. “

After showing that vascular malformations can be corrected Mouse modelProfessor Lawrence Boon of the Center for Vascular Abnormalities at Cliniques University Hospital in Brussels has been working with Professor Vicla for 30 years to recruit 18 patients with AVM to study their use in the state of salidamide. They were 19 to 70 years old and all had severe malformations that could not be treated with conventional approaches. Patients had to agree to use contraception for at least 4 weeks before starting thalidomide and continue for 4 weeks after discontinuation of treatment. Because thalidomide is contained in semen, men also had to agree to use condoms during sex.

Patients received 50 mg, 100 mg, or 200 mg of thalidomide daily for 2-52 months. Eight AVMs were stable after a 54-month mean thalidomide discontinuation, and four relapsed after a mean 11.5-month period. Combined treatment with embolization, in which the arteries or veins in the AVM are blocked by drugs that destroy vessel parietal cells, was able to reduce the dose of salidamide to 50 mg per day in 5 patients. It was important to reduce the dose as much as possible, says Professor Vikkula. This is because higher doses cause side effects, especially fatigue and peripheral neuropathy, damage to nerves outside the brain and spinal cord, especially weakness and numbness in the hands and spinal cord. Feet.

“All patients experienced rapid pain relief, along with bleeding arrest and ulcer healing where the ulcer was present,” says Professor Vikkula. “Three patients with heart failure also saw the problem resolved, and one AVM appeared to be completely cured after 19 months of thalidomide and 8 years of follow-up.

“We have thalidomide Vascular Endothelial Growth Factor (VEGF) Signal transduction protein that promotes new growth Blood vessels.. Because VEGF levels are high in vascular abnormalities such as AVM, thalidomide may reduce signaling through angiogenesis-promoting pathways. Our study is small, but we hope the results are compelling and confirmed in larger trials. “

Another benefit of using thalidomide to treat AVM is one of the costs. Recently developed for use in oncology and tested to treat AVM, the other two drugs are up to 12 times more expensive and have many side effects.

“We assumed that thalidomide Our results weren’t surprising as they should work in these patients, but it was great to be able to clinically confirm that we were right, “Concludes Professor Vikkula. Development of molecular therapy for AVM. “

Professor Alexander Raymond, chair of the conference, said: Economic benefits Not only about diversion of drugs-even the most malicious ones-but how Genetic research It can bring true breakthroughs to the treatment of difficult and painful conditions. ”

Don’t sleep with the possibility of thalidomide hypnosis

For more information:
A case report study of thalidomide therapy in 18 patients with severe arteriovenous malformations, Nature Cardio Vascular Research (2022). DOI: 10.1038 / s44161-022-00080-2

Provided by the European Society of Human Genetics

Quote: Thalidomide was obtained from on June 10, 2022 for abnormal angiogenesis (June 10, 2022). It is an effective treatment method.

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Thalidomide is an effective treatment for abnormal angioplasty

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