2017 IFOS meeting
Final program
Bridging the gap from basic to translational sciences in otology
Organized by the Fondation pour l’Audition
Tuesday June 27, 2017; 11 am – 12:30 pm – Room Maillot
Moderated by Karen B. Avraham and Lawrence R. Lustig
The French Hearing Institute: from basic to translational research
Christine Petit, Pasteur Institute, Paris, France
Paul Avan, University of Clermont-Auvergne, Clermont-Ferrand, France
Developing molecular therapeutics for human inner ear disease
HinrichStaecker
University of Kansas School of Medicine, KS, USA
Early phase trials of novel hearing therapies; challenges and opportunities.
AnneSchilder
UCL Ear Institute, London, UK
---
Abstracts
The French Hearing Institute: from basic to translational research
Christine Petit, Pasteur Institute, Paris, France
Paul Avan, University of Clermont-Auvergne, Clermont-Ferrand, France
Hearing loss is one of the major health concerns affecting 360 million people worldwide.
Scheduled to open in 2018 in Paris, the French Hearing Institute, known in French as the ‘Institut del’Audition’, aims to advance our knowledge in Hearing Sciences, from perception to cognition, and to promote translational research in the field by developing innovative diagnostic and therapeutic strategies with the ultimate goal of treating hearing disorders.
The Institutdel’Audition will bring together dedicated scientists and clinicians working on basic and clinical aspects of hearing from biophysics, acoustics, neurobiology, molecular and cellular biology, genetics to engineering, bioinformatics and computational neuroscience, all highly committed to interdisciplinary research.
TheInstitutdel’Auditionwill closely work with a hearing clinical research center, that will launch its activities at the same time in Paris, in order to foster translational applications arising from the scientific and medical community locally, nationally and internationally. Both entities, supported by the Fondation pour l’Audition, will benefit from state-of-the-art technological platforms and with the Vision Institute, just nearby, they will give rise to a unique sensory campus of excellence in France.
The structure, the scientific and clinical objectives of the Institutdel’Audition and the hearing clinical research center will be presented.
---
Developing molecular therapeutics for human inner ear disease
HinrichStaecker
Department of Otolaryngology Head and Neck Surgery
University of Kansas School of Medicine
Rainbow Blvd, MS 3010
Kansas City KS 66160
The last 30 years have seen an explosion in our understanding of the molecular basis of hearing loss. The pathways underlying the development of the ear as well as the numerous molecular components that make hearing work have been extensively explored. So far this has not translated into the development of new types of therapeutics for what is actually the most common neurodegenerative disease in man. A significant portion of our focus has been on understanding the pathways that control the genesis of auditory and vestibular hair cells in an effort to apply this in patients. We will review some of the key discoveries in the patterning of the neuro-sensory epithelium of the inner ear and what factors go into taking that information and developing a hair cell regeneration drug that is testable and human subjects. These initial studies promise to expand our ability to develop novel treatments that require delivery of standard and molecular therapeutics into the inner ear.
---
Early phase trials of novel hearing therapies; challenges and opportunities.
Anne GM Schilder, Shakeel Saeed, Joanne Palmer, Helen Blackshaw, ThanosBibas, Dimitris Kikidis, Stephan Wolpert, on behalf of the REGAIN consortium
Introduction: With biotech companies moving rapidly from discovery to the development of drug, gene and cell therapies for sensorineural hearing loss, and their investors keen to see return on their investments, clinical trials have become the decisive moment to success. This creates new opportunities and challenges to clinicians and researchers in the hearing field, most of whom are new to this type of research. In this presentation, I will share some of the dilemma’s our international team faced while preparing for the REGAIN trials.
Methodology: Supported by a €5.8 million EU Horizon 2020 Research and Innovation Programme award, REGAIN will test the safety and efficacy of a gamma-secretase inhibitor (GSI) in patients with adult onset hearing loss.
Results: Preclinical work (candidate selection, GMP production, formulation, pharmacokinetics, safety and toxicology) and regulatory submissions will be completed by May 2017. The phase 1 open label multi-ascending dose safety study and phase 2 open label randomised efficacy study of two doses taken forward from the safety study, in adult patients with mild to moderate sensorineural hearing loss, will be rolled out from July 2017 in the UK, Germany and Greece.
Conclusion: Novel therapies targeting the underlying biological causes of hearing loss in a safe way may meet a need for millions of patients. Challenges in designing and delivering proof of concept include selection of patients and outcome measures, governance and regulatory requirements and costs. With patient safety and benefit at the heart of these new and exciting developments, it is vital that translational expertise is sought and shared and essential quality criteria are agreed upon by hearing researchers.
The REGAIN consortium includes 7 dedicated partners, coordinated by Audion Therapeutics and involving evidENT at the UCL Ear Institute, the Eberhard Karls University of Tübingen, National and Kapodistrian University of Athens, Eli Lilly, Nordic Biosciences and ttopstart.
This project receives funding from the EU Horizon 2020 Research and Innovation Programme (No 634893).
Page 1 sur 3