sábado, junio 15

Gene therapy to help hearing, a small revolution

A PEA hearing test, called “auditory evoked potential”, is carried out on a newborn, at the Saint-Vincent-de-Paul hospital, in Lille, in April 2012.

It’s a story that made headlines across the New York Times. On January 23, the American newspaper devoted a long article to Aissam Dam. This young boy, aged 11, born in Morocco, regained almost normal hearing thanks to a gene therapy treatment carried out in a Philadelphia hospital. The next day, researchers from Fudan University (Shanghai) published in The Lancet the results of a clinical trial of the same nature carried out on six children and adolescents. Five of them also partially regained their hearing.

All suffered from a very rare congenital profound deafness. Called “DFNB9”, it concerns between 3% and 8% of cases of congenital genetic deafness (i.e., in France, around ten children per year). It is characterized by a mutation in the Otof gene, which partially or completely alters the production of a protein, otoferlin, essential for the transmission of sound information at the synapses of auditory sensory cells.

Gene therapy to help hearing? It’s quite simply a small revolution. Until now, there was no treatment to treat this deficiency. For around thirty years, the treatment of this deafness has been based on the placement of an implant in the cochlea, a small organ of the inner ear which plays a major role in the transmission of sound. This device helps restore hearing, but it is not perfect. And this technique doesn’t work on everyone.

Also read the archive (2017) | Article reserved for our subscribers Deafness: the key to implant success

France is in turn embarking on the adventure. The first clinical trial of gene therapy aimed at testing a drug intended to restore hearing in at least twelve children aged 6 to 31 months, Audiogène, will be launched before the summer. It was developed in partnership with research teams from the Hearing Institute (Institut Pasteur), the ENT department and the pediatric audiology research center at the Necker-Enfants Malades hospital (AP-HP) as well as of the Hearing Foundation and Sensorion. This French biotech, based in Montpellier, has developed the gene therapy drug (Sens-501). The clinical trial must be carried out in several recruitment centers (France, Germany, Italy, United Kingdom and Australia). “Since 2020, Sensorion has worked with ten centers in Europe where doctors systematically genotype babies who are born deaf to identify the mutation”specifies Nawal Ouzren, its general director.

Twenty years of research

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