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partnership to apply gene therapy to Dravet syndrome

Scientists from Tel Aviv University, the high school de Genetics Molecular de Montpellier, the Cima and the Clínica Universidad de Navarra are investigating the safety and efficacy of this treatment in an experimental model of the disease.

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International scientific team of project CureDravet. PHOTO: Manuel Castells
22/02/19 16:50 Miriam Salcedo

Scientists from the University of Tel Aviv (Israel), the high school de Genetics Molecular de Montpellier (France), the Cima and the Clínica Universidad de Navarratogether with Innomedyx (Spain), are meeting in Pamplona to advance the European project CureDravet, research which aims to demonstrate the safety and efficacy of gene therapy for the treatment of Dravet Syndrome.

Dravet syndrome is a severe form of childhood epilepsy caused by a mutation in the SCN1A gene, which is involved in controlling the spread of nerve stimuli in the brain. It is a rare disease Genetics that affects 1 in 20,000 people and, in most cases, has no family history.

The lack of effective medication currently available makes the latest advances in gene therapy an alternative treatment. "Gene therapy could bring about a lasting improvement in the lives of patients with monogenic diseases such as Dravet Syndrome, since it affects the basic alteration that causes all the clinical manifestations," says Dr. Rubén Hernández, researcher of the Cima University of Navarra and participant in the project. The final goal of CureDravet, which is currently investigating an experimental model of the disease, "is to try to prevent, stop or reverse the symptoms of this disease," says Dr. Moran Rubinstein, of Tel Aviv University and coordinator of project.

Social awareness and promotion of knowledge Dravet clinical

From the medical perspective, Dr. Rocío Sánchez-Carpintero, manager of the Neuropediatrics Unit of the Clínica Universidad de Navarra, points out that, "until we achieve a cure with financial aid from scientists, our role is to care for children with Dravet with all the means at our disposal". According to the expert, this disease "causes a lot of suffering to families because of the great ignorance that surrounds them". Along these lines, the European project CureDravet, promoted by the Dravet Syndrome Foundation, seeks to increase social awareness and clinical knowledge about this rare disease. To this end, throughout the project Innomedyx will carry out various promotional activities on the possibilities of gene therapy aimed at health professionals and the general public.

Solidarity Gala 'Abrazos

The research in Dravet Syndrome of Cima, partner of project CureDravet, has been selected to star in the solidarity campaign 'Abrazos', an initiative promoted by Innomedyx. With the slogan "Cure Dravet", the campaign will consist of different actions of social responsibility with companies and institutions to raise awareness and raise funds. The proceeds will be announced at the 'Gala Abrazos', which will take place in November in Valencia, and will go to support the continuity of the research in Dravet that performs the Cima. This action is in addition to those carried out by families of patients, associations and companies in support of the research in Dravet of the research center Navarra.

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