Thalidomide: the ‘cursed’ drug that could help those affected by vascular malformations


A study published in the journal ‘Nature Cardiovascular Research’ indicates that use of the drug shows a significant reduction in symptoms and an improvement in quality of life.

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its effects thalidomide They were destructive. In the late 1950s and early 1960s, more than 12,000 babies were born with serious birth defects from this drug.

What happened was because of tough drug safety controls. However, years later it began to be used again – under strict control and for very specific indications – upon discovering its usefulness against certain tumors, graft-versus-host disease, Crohn’s disease and some types of arthritis.

To this list can now be added its usefulness as a medicine. aneurysm malformationsAccording to a study which will be presented this Sunday at the Congress of the European Society of Human Genetics which is being held in Vienna these days and is published this Friday in the journal. Nature Heart Research,

According to the data of this investigation, the use of the drug shows that significant reduction in symptoms and improve the quality of life of those affected by this problem.

The mechanisms explaining the usefulness of the drug in this vascular disorder are precisely those that caused devastation in the fetus: The property of the drug to prevent the formation of blood vessels,

Arteriovenous malformation can be very painful for the sufferer and can cause deformity, bleeding and heart problems. Although they are often congenital, they show their face only in adolescence or adulthood, when the individual becomes older. The treatment available so far is surgery or embolization, although this is not always effective.

Some people can lead relatively normal lives, the authors of the work explain, although even in less severe cases there is always a risk that the vessels rupture and cause a stroke. One out of every 100 patients with this condition suffers from a cerebral infarction every year.

research authorThey have been studying this type of pathology for 30 years And after discovering the genetic causes that lead to the abnormal formation of blood vessels (angiogenesis), he realized that thalidomide, precisely because of Known Antiangiogenic Properties could be an option.

They first tested the effect in a mouse model and then recruited 18 patients with vascular problems aged between 19 and 70 years And with traditional medicine good results were not obtained. Participants signed an agreement to use contraception for at least four weeks before starting thalidomide treatment and continued to use contraception for four weeks after completion. Both men and women had to sign a commitment, as the drug is also present in semen.

patients Received doses of 50 mg, 100 mg, or 200 mg for 52 months, The researchers noted that all patients experienced rapid reduction in pain, cessation of bleeding, and healing of ulcers in the cases in which they were present. Three patients with heart failure also improved, and one experienced complete remission after 19 months of treatment and eight years of follow-up.

The authors acknowledge that this is a small study that needs to be confirmed by a larger investigation, but emphasize that their results are promising.

Any treatment with thalidomide is absolutely contraindicated in pregnant women and should be accompanied by Pregnancy prevention program in all patientsWhether they are female or male, among other requirements, are explained in this article by Javier García Pellisar, Section Head of the Hospital Pharmacy Service of La Fe University Hospital in Valencia and a member of the Spanish Society of Hospital Pharmacy.

Its antiangiogenic properties were first described in 1994, leading to its use in certain types of cancer. The first favorable results were obtained in multiple myeloma and, in fact, in 2008 the European Medicines Agency authorized the use of the drug in some of these patients, Garca Pellisar reported.

Avoidance or repurposing of drugs is an increasingly popular trend in pharmacology, a strategy that allows finding new uses for existing or even discarded drugs.

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