DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hearing loss, autosomal dominant 78 — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHearing loss, autosomal dominant 78 maps to a 1-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for hearing loss, autosomal dominant 78 is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
solute carrier family 12 member 2 (SLC12A2) — SLC12A2 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet butylaminodrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9C0H · 2.5 Å · ligand 3-(butylamino)-4-phenoxy-5-sulfamoylbenzoic acid (82U). Experimental structure, not a prediction.
What the evidence adds up to
No drug is tested or proposed for hearing loss, autosomal dominant 78 in any of these abstracts. The 2021 review notes that no federally approved drugs exist to prevent or treat hearing loss caused by aminoglycoside antibiotics or cisplatin, and states that further research is urgently needed. The 2010 study on DFNA9 — a different autosomal dominant hearing loss caused by COCH mutations — shows that mutant cochlin protein forms stable dimers, can stabilise wild-type cochlin in dimer conformation, and eventually induces wild-type cochlin to form stable, reduction-resistant oligomers. Mutant cochlin is cytotoxic in vitro and in vivo. This provides a molecular mechanism for the dominant inheritance pattern but does not test any intervention.
The 2025 review on gene therapy for hereditary hearing loss describes three strategies — gene replacement, gene suppression, and gene editing — and notes that clinical trial results using gene therapy for hereditary hearing loss have recently been approved. It summarises successful preclinical trials but gives no specific numbers for any drug or therapy. The 2005 and 2014 reviews confirm that half of congenital hearing loss has a genetic basis, that treatment devices do not correct the underlying pathology, and that genetic approaches have identified functionally relevant molecules in the inner ear. The 2025 collection of 31 papers includes work on basic mechanisms, protection methods, and gene therapy, but no concrete efficacy data for any drug in any form of hearing loss.
What is still missing: a drug candidate tested in DFNA78 specifically; any clinical trial data for any compound in autosomal dominant hearing loss; funding for preclinical drug screening or repurposing studies; and patient stratification by genotype to enable targeted trials.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
American Journal of Audiology · 2021 · 74 citations · open access
Mechanisms of Aminoglycoside- and Cisplatin-Induced Ototoxicity
AbstractPurpose This review article summarizes our current understanding of the mechanisms underlying acquired hearing loss from hospital-prescribed medications that affects as many as 1 million people each year in Western Europe and North America. Yet, there are currently no federally approved drugs to prevent or treat the debilitating and permanent hearing loss caused by the life-saving platinum-based anticancer drugs or the bactericidal aminoglycoside antibiotics. Hearing loss has long-term impacts on quality-of-life measures, especially in young children and older adults. This review article also highlights some of the current knowledge gaps regarding iatrogenic causes of hearing loss. Conclusion Further research is urgently needed to further refine clinical practice and better ameliorate iatrogenic drug-induced hearing loss.
Journal of Biological Chemistry · 2010 · 41 citations · open access
Role of Protein Misfolding in DFNA9 Hearing Loss
AbstractMutations in the COCH (coagulation factor C homology) gene have been attributed to DFNA9 (deafness, autosomal-dominant 9), an autosomal-dominant non-syndromic hearing loss disorder. However, the mechanisms responsible for DFNA9 hearing loss remain unknown. Here, we demonstrate that mutant cochlin, the protein product of the COCH gene, forms a stable dimer that is sensitive to reducing agent. In contrast, wild-type (WT) cochlin may form only dimers transiently. Interestingly, the presence of mutant cochlin can stabilize WT cochlin in dimer conformation, providing a possible mechanism for the dominant nature of DFNA9 mutations. Furthermore, the expression of mutant cochlin eventually induces WT cochlin to form stable oligomers that are resistant to reducing agent. Finally, we show that mutant cochlin is cytotoxic in vitro and in vivo. Our study suggests a possible molecular mechanism underlying DFNA9 hearing loss and provides an in vitro model that may be used to explore protein-misfolding diseases in general.
The Journal of Laryngology & Otology · 1999 · 20 citations
Metronidazole ototoxicity - report of two cases
AbstractTwo cases of bilateral moderate to severe sensorineural hearing loss due to oral administration of metronidazole are reported. There has been only one case report of deafness following metronidazole therapy in the world literature. The hearing loss recovered gradually in a period of four to six weeks following withdrawal of drug and oral steroid therapy. The possible mechanism of ototoxicity is discussed. Awareness by the treating physician of ototoxicity due to any drug is stressed.
Sensory Neuroscience · 2025 · 7 citations · open access
Update on Gene Therapy in the Treatment of Hereditary Hearing Loss
AbstractABSTRACT Gene therapy is a promising therapeutic approach for genetic disorders, involving genetic modification to repair or reconstruct faulty genetic material. It is particularly relevant to hereditary hearing loss (HHL), a common monogenic condition that can lead to congenital deafness. The recent approval of clinical trial results using gene therapy for HHL underscores the growing interest in this field. To further advance inner ear gene therapy and its application in genetic diseases, it is crucial to review the progress of gene therapy for HHL. This review focuses on the three major gene therapy strategies—gene replacement, gene suppression, and gene editing—highlighting their application across different monogenic disorders and successful preclinical trials in HHL. We summarize the primary gene therapy strategies used in recent years, discuss recent achievements in preclinical studies, and explore potential advancements in this field.
British Journal of Hospital Medicine · 2005 · 4 citations
The genetics of hearing loss
AbstractHearing impairment is the most common sensory deficit with half of the causes of hearing loss having a genetic basis. There is a range of treatment devices but these do not correct the underlying pathology. Advances in molecular biology have greatly enhanced our understanding of the pathophysiology of genetic hearing loss, including potential treatments.
Hereditary hearing loss in humans: the importance of genetic approaches for clinical medicine and basic science
AbstractHereditary hearing loss is one of the most common monogenic diseases in humans and, depending on the severity of symptoms and age of onset, dysfunction of one of the main sensory systems can cause significant problems for the affected individual and his/her social environment. The diagnostic workup of hearing impairment is complicated by a pronounced phenotypic variability and extensive genetic heterogeneity. Nevertheless, many forms of monogenic hearing impairment have been elucidated in recent years by genetic approaches. In addition to improved counselling and medical management of patients and families, these research findings have contributed significantly to the identification of functionally relevant molecules of the inner ear and have thus helped us to better understand the molecular physiology of hearing and the pathophysiology of hearing impairment.
Advanced Science · 2025 · 1 citations · open access
Hearing Loss: From Basic to Clinical Science
AbstractHearing loss (HL) affects over 1.5 billion people globally, with genetic factors accounting for ≈50% of congenital cases. Therefore, HL has become a global health issue, driving extensive research from basic science to clinical applications. This Special Collection includes a total of 31 papers, among which 9 are review papers, 21 are research article papers, 1 is a perspective paper, that highlight the basic mechanisms and possible protection methods of HL, the application of gene therapy for treating HL, and the clinical study and application in HL.
Greater South Information System · 2023 · 0 citations · open access
Potential Mechanisms of Hearing Loss Due to Impaired Potassium Circulation in the Organ of Corti
AbstractHearing loss (HL) is a common condition that significantly affects an individual's quality of life. Impaired potassium circulation in the organ of Corti (OC), including the movement of potassium into hair cells (HCs) and from hair cells to supporting cells (SCs), can contribute to hearing loss. This chapter aims to provide a better understanding of cochlear potassium ion homeostasis and its dysfunction in this context. Sensorineural hearing loss (SNHL) is caused by damage to the inner ear or the auditory nerve. Various factors contribute to it, including aging, exposure to loud noise, genetics, medications, and infections. In all of them, some level of potassium circulation alteration is present. Potassium plays a crucial role in hearing function as it is the moving charge that depolarizes hair cells in response to sound perception. It generates the endocochlear potential (EP) which provides the driving force for potassium movement. Disruptions in potassium circulation due to molecular alterations in ion channels and transporters can lead to hair cells dysfunction and cell death. Moreover, drugs that affect potassium circulation can also cause hearing loss. Understanding the molecular and tissue changes resulting from potassium circulation deficits is essential for developing targeted treatments and preventive measures for potassium-related hearing disorders.
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
