Potential Gene Therapy Trials in Autosomal Dominant Progressive Sensorineural Hearing Loss

alexandrossfakianakis shared this article with you from Inoreader Attitudes of Potential Participants Towards Potential Gene Therapy Trials in Autosomal Dominant Progressive Sensorineural Hearing Loss Via Otology & Neurotology - Published Ahead-of-Print Background: Advances in gene therapeutic approaches to treat sensorineural hearing loss (SNHL) confront us with future challenges of translating these animal studies into clinical trials. Little is known on patient attitudes towards future innovative therapies. Objective: We aimed to better understand the willingness of patients with progressive SNHL and vestibular function loss of autosomal dominant (AD) inheritance to participate in potential gene therapy trials to prevent, stabilize, or slow down hearing loss. Methods: A survey was performed in carriers of the P51S and G88E pathogenic variant in the COCH gene (DFNA9). Various hypothetical scenarios were presented while using a Likert scale. Results: Fifty three participants were included, incl. 49 symptomatic patients, one presymptomatic patient, and three participants at risk. Their attitude towards potential trials studying innovative therapies was overall affirmative, even if the treatment would only slow down the decline of hearing and vestibular function, rather than cure the disease. Among the different potential scenarios, the less invasive and less frequent treatments increased the likelihood to enroll. Daily oral medication and annual intravenous infusion were awarded the highest scores. The more invasive, more frequent, and more at-risk treatments were still likely to be accepted but decreased the willingness to participate. The presence of a placebo arm was met with the lowest scores of willingness to participate. Conclusions: Overall, most symptomatic DFNA9 patients would likely consider participation in future innovative inner ear therapy trials, even if it would only slow down the decline of hearing and vestibular function. Address correspondence and reprint requests to Camille Levie, M.D., Department of Otorhinolaryngology and Head & Neck surgery, Antwerp University Hospital, Wilrijkstraat 10, 2610 Edegem, Belgium; E-mail: camille.levie@uza.be Disclosure of funding: The authors received no specific funding for this work. The authors disclose no conflicts of interest. Copyright © 2020 by Otology & Neurotology, Inc. Image copyright © 2010 Wolters Kluwer Health/Anatomical Chart Company View on the web Inoreader. Take back control of your news feed. Follow us on Twitter and Facebook.