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

• Pharmacology 'Zombie' drugs arrive

• Repositioning Old drugs, new uses: this is how the hidden potential of medicines is discovered

The effects of

were catastrophic.

More than 12,000 children were born with serious birth defects from this drug in the late 1950s and early 1960s.

What happened led to tougher pharmacological safety controls.

However, years later it began to be used again -under strict controls and for very specific indications- upon discovering its usefulness against some tumors, graft-versus-host disease, Crohn's disease and some types of arthritis, among others.

To this list can now be added its usefulness as a therapy against

, according to a study that will be presented this Sunday at the Congress of the European Society of Human Genetics that is being held these days in Vienna and is published this Friday in the journal

.

According to the data from this research, the use of the drug shows a

and an improvement in the quality of life among those affected by this problem.

The mechanisms that explain the usefulness of the drug in this vascular disorder are precisely the same ones that caused the catastrophe in the fetuses:

.

Arteriovenous malformations can be very painful for those who suffer from it and cause deformation, bleeding and heart problems.

Although they are often congenital, they only show their face in adolescence or adulthood, when the person grows up.

The treatment available so far is surgery or embolization, although it is not always effective.

Some people can lead a relatively normal life, the authors of the work point out, although even in less severe cases there is always the risk that the vessels will rupture and cause a stroke.

The authors of the research

and after finding genetic causes that cause the abnormal formation of blood vessels (angiogenesis), they realized that thalidomide, precisely because of its

, could be a option.

They first tested the effect in mouse models, and then recruited

and had failed conventional therapy.

Participants signed an agreement to use contraception at least four weeks before starting thalidomide treatment and continue to use contraception for four weeks after completion.

Both men and women had to sign the commitment, since the drug is also present in semen.

Patients

.

All patients, the researchers note, experienced rapid reduction in pain, cessation of bleeding, and healing of ulcers where present.

Three patients with heart failure also improved, and one experienced a 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 larger investigations, but stress that their results are promising.

"All treatment with thalidomide is totally contraindicated in pregnant women and must be accompanied by a

, whether they are female or male, among other requirements," explained Javier García Pellicer, head of section of the Hospital Pharmacy Service of the La Fe University Hospital in Valencia and member of the Spanish Society of Hospital Pharmacy.

«In 1994 its antiangiogenic properties were described for the first time, which led to its use in some 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”, pointed out García Pellicer.

The rescue or repositioning 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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