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The regional University of Malaga treats patients with haemophilia A with gene therapy for the first time in Spain


Posted 17.1.2019 6:08:00 PMCET


The University Regional Hospital in Malaga treated for the first time in Spain patients with hemophilia A gene therapy, the first time the molecule is studied globally, as assured the principal center researcher and member of the Spanish Society of Thrombosis and Hemostasis (SETH) , María Eva Mingot.

The treatment is part of a Phase I international clinical trial for patients with severe haemophilia A who do not have immunity to the vector virus. Currently, this technique is not available for all patients, only for those who meet the criteria for inclusion of the clinical trial and none for exclusion, Mingot said.

It is a drug administered only once in the hospital, but requires strict monitoring in the center. The start of the study, which began in November last year at the Malaga Regional Hospital, represents a very important advance for haemophiliacs, since this type of treatment can be the final cure of their disease.

"Gene therapy aims to cure congenital diseases caused by the alteration of one or more genes, such as hemophilia, by introducing the healthy gene into the genetic material of a known (modified) virus and then inoculating it into the patient.

When the virus "infects" the cells that produce the abnormal protein, it introduces the healthy gene and replaces the pathology so that the patient is partially or totally healed. "The healing or near normalization of factor VIII allows the affected person to live independently of the treatment," hematologist stressed.

However, he warned that the results should be assessed with "caution", although he said it was a "great option" if the response was maintained over time with a good safety profile.

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