# Pre-Clinical Testing of the Safety and Efficacy of Treatments for Voice Disorders

> **NIH NIH R01** · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · 2020 · $383,351

## Abstract

Project Summary
The lifetime prevalence of voice disorders in the adult United States population is 30% with point prevalence
rates of 6.6% to 7.5%. These disorders are debilitating and can lead to significant socioemotional
consequences, loss of income, and long-term disability. Point prevalence and census estimates suggest that
nearly 20-23 million adults may experience dysphonia annually, with the cost of treatment and lost wages
approaching $13 billion dollars. These annual direct costs are comparable to those associated with chronic
obstructive pulmonary disease, asthma, diabetes, and allergic rhinitis. Among otolaryngology evaluations for
dysphonia, nearly 43% of short-term disability claims are associated with phonotraumatic lesions. Thus,
improving the care of patients with voice disorders remains a significant public health need. As a necessary
and essential first step to address this need, our research program has established a systematic effort in
discovery and pre-clinical testing in the area of voice disorders. Our efforts to date have provided critical
new insights into the cellular and molecular pathophysiology of phonotrauma and novel approaches that can
support studies of gene/protein, structure, and function in pre-clinical models. In this five-year proposal, we
concentrate our efforts on the safety and efficacy of treatments for acute phonotrauma. We focus on a class of
steroid hormones extensively used in humans, despite a limited understanding of mechanisms and indications
for use, safety, and demonstration of efficacy. Beyond the anti-inflammatory actions, emerging evidence
supports a role for glucocorticoids in the maintenance of the epithelial barrier. As a result, glucocorticoids have
evolved as a first line therapy for many inflammatory diseases and disorders involving impaired epithelial
barrier function. Unfortunately, despite widespread clinical use, the specific effects of glucocorticoids on the
vocal fold epithelial barrier remain unknown. The need for treatment efficacy research in voice disorders has
been acknowledged as a high priority area in the NIDCD strategic plan. The development of phase I/II/III
clinical testing in humans strongly depends on pre-clinical studies to provide information regarding
mechanisms underlying therapeutic safety and efficacy. Federal regulations require demonstration of safety
and efficacy prior to the approval of large-scale human trials. Pre-clinical testing is thus necessary not only to
improve care, but also to reduce the burden of voice impairment on patients and society. To address this need,
our research program is well-positioned to launch a systematic effort on the efficacy of treatments for voicedisorders. In the current proposal, we will test the pharmacodynamics of glucocorticoids using in vitro and in
vivo preclinical models in order to determine the mechanism of action, desired activity, and drug safety profile
of glucocorticoids.

## Key facts

- **NIH application ID:** 9959388
- **Project number:** 5R01DC015405-06
- **Recipient organization:** UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- **Principal Investigator:** Bernard Rousseau
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $383,351
- **Award type:** 5
- **Project period:** 2016-07-15 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9959388

## Citation

> US National Institutes of Health, RePORTER application 9959388, Pre-Clinical Testing of the Safety and Efficacy of Treatments for Voice Disorders (5R01DC015405-06). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9959388. Licensed CC0.

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