OTOSTEM - Human stem cell applications for the treatment of hearing loss

Hearing impairment is the most frequent human sensory deficit and is mainly caused by the irreversible loss of neurosensory cells in the cochlea. The lack of human otic (‘relating to the ear’) cell models represents a significant roadblock that has hampered development of either drug-based or cell-based therapies for the treatment of hearing loss. OTOSTEM aims to generate human otic progenitors and differentiated otic cells from different human stem cell sources via guidance protocols that reprogram stem cells toward inner ear cell types. Use of principles of early germ layer formation and otic induction will improve the efficacy of otic progenitor cell generation and purification.

OTOSTEM will develop novel bioassays for drug screens to identify means to decrease potential tumorigenicity after cell transplantation by such purified otic cells. New hit compounds identified from screening efforts will then be tested and validated further in established organ culture models. The relevant candidate compounds defined by these studies will be further developed as lead drug candidates in noise and ototoxic drug –induced in vivo models, with clear otoregeneration as a key objective.

The scope of such stem cell technology development requires a collaborative team effort, with 9 groups from Tübingen to Palo Alto, including two SMEs, that have substantial combined experience in human ES/iPS cell work, inner ear stem cell biology, high-throughput assay development, and in translating research findings into both the clinical and the biotechnology realm.

Because an established translational route from bench to bedside exists in the consortium for the commercial development of human otic stem cell derived technology, medical applications aimed at the restoration of hearing function are anticipated.

SCIPROM has accompanied OTOSTEM since the project preparation phase, and now acts as the project management and dissemination partner.

The project began on 1 Nov 2013 and will run for 48 months. For more information, please visit http://www.otostem.org.