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Aerosolized hyaluronic acid as a treatment for emphysema

雾化透明质酸治疗肺气肿

基本信息

批准号：
6404780
负责人：
JEROME O CANTOR
金额：
$ 9.94万
依托单位：
EXHALE THERAPEUTICS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2001
资助国家：
美国
起止时间：
2001-09-10 至 2003-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/6404780
关键词：
aerosols alpha 1 antitrypsin deficiency chronic obstructive pulmonary disease drug design /synthesis /production drug screening /evaluation emphysema hyaluronate inhalation drug administration laboratory mouse smoking tobacco abuse

项目摘要

DESCRIPTION (provided by applicant): Chronic obstructive pulmonary disease afflicts 12-14 million people in the US and in an advanced form, emphysema, disables 2 million and ultimately leads to death. No approved, existing treatment can arrest emphysema's progressive destruction of the lung. Exhale Therapeutics, Inc intends to develop and commercialize aerosolized hyaluronic acid (HA) into the first effective therapeutic product that can preserve lung function. The long-term objectives of the proposed project (Phases I) are: (1) to show that aerosolized low-molecular weight HA inhaled daily protects against lung elastic fiber injury and subsequent destruction of alveoli; and (2) to elucidate the protective mechanism. The specific aim of Phase I is to demonstrate that in mice exposed regularly to tobacco smoke in a chamber that simulates cigarette smoking, daily treatment with HA administered as an aerosol significantly reduces the severity of emphysematous damage to their lungs. Two groups of mice will smoke, the first will receive daily treatment with HA dissolved in distilled water, and the second will receive nebulized water only. A comparison of the mean linear intercept of lung sections of the two groups will provide a direct quantitative measure of the protective effect of HA. Other semi-quantitative measures will provide supporting evidence. PROPOSED COMMERCIAL APPLICATION: Hyaluronic acid (HA) has the potential to become an effective therapeutic agent that can protect lung function against the chronic destruction of alveoli in emphysema. Since no treatment for pulmonary emphysema currently exists, proof of safety and efficacy in humans will permit rapid commercialization. The proposed research in an animal model will accelerate the formulation of a therapeutic product for study and use in humans.

描述（由申请人提供）：慢性阻塞性肺疾病在美国有1200万到1400万人患有肺气肿，使200万人失去能力最终导致死亡无批准，现有治疗可以阻止肺气肿对肺部的逐渐破坏。呼气 Therapeutics公司打算开发和商业化雾化透明质酸酸（HA）成为第一个有效的治疗产品，可以保护肺功能建议项目（第一期）的长远目标是：（1）表明每日吸入雾化低分子量HA可预防肺弹性纤维损伤和随后的肺泡破坏;和（2）阐明保护机制。第一阶段的具体目标是在一个实验室中，模拟吸烟，每日以气雾剂形式给予HA治疗显著降低肺气肿对肺部的损害程度。两一组小鼠吸烟，第一组小鼠每天接受HA治疗，第一个将溶解在蒸馏水中，第二个将仅接收雾化水。两组肺切片平均直线截距的比较将提供HA保护作用的直接定量测量。其他半定量指标将提供支持证据。拟定商业应用：透明质酸（HA）有可能成为一种有效的治疗剂，保护肺功能，防止肺气肿时肺泡的慢性破坏。由于没有目前存在肺气肿的治疗方法，人类将允许快速商业化。拟议的动物模型研究将加速用于人类研究和使用的治疗产品的配制。