Development of inhalable platelet-based therapy for treatment of acute lung injury

因创伤或感染导致肺损伤的患者可发展为急性呼吸窘迫综合征和肺水肿，死亡率高。富含血小板血浆(PRP)是通过浓缩患者自身血液中的血小板而产生的，在其他需要再生的组织损伤的临床试验中被证明是一种非常有效的治疗方法，例如糖尿病溃疡。该项目将研究一种再生的PRP产品可以通过吸入进入肺部来治疗急性肺损伤的假设。将提供合作生物科学和药物开发方面的培训，以设计一种新的治疗方法，并将其发展为患者的利益。该项目将分阶段进行开发。第一年将提供技术培训和确认关于PRP生产技术和体外模型/生物测试的初步数据，以指导产品开发。第二年将专注于产品特性和开发气雾剂输送系统，以及探索性的体内研究。为期3个月的生物治疗服务部将提供病区绘图方面的培训，以了解这种新疗法的临床需求和对健康的影响。到第三年，产品原型将准备好在临床前疾病模型中确认安全性和有效性。在第4年，将进行一项概念验证研究，以证明优化的吸入PRP在活体肺损伤模型中的有效性。诊所的视线将牢固地嵌入到项目目标设定和学生培训中。这项研究将由生物治疗服务公司领导，并将与实验室调查一起进行。

Patients with lung injury arising from trauma or infection can develop acute respiratory distress syndrome and pulmonary oedema with a high mortality rate. Platelet rich plasma (PRP), produced by concentrating platelets from patients own blood, is a therapy that is proving highly effective in clinical trials for other tissue injuries that require regeneration, e.g. diabetic ulcers. This project will investigate the hypothesis that a regenerative PRP product can be administered to the lungs by inhalation to treat acute lung injury. Training will be provided in collaborative bioscience and pharmaceutical development to design a novel therapy and develop it towards patient benefit. The project will be developed in phases. The first year will provide technical training and confirmation of preliminary data on PRP production techniques and in vitro models/bioassays to guide product development. Year 2 will focus on product characterisation and developing aerosol delivery systems, along with exploratory in vivo studies. A 3-month placement with Biotherapy Services will provide training in Disease Area Mapping to understand the clinical need and health impacts of the novel therapy. By year 3 product prototypes will be ready for confirmatory safety and efficacy in a pre-clinical disease model. In year 4 a proof-of-concept study will be performed to demonstrate the efficacy of the optimised inhaled PRP in an in vivo model of lung injury. The line-of-sight to the clinic will be strongly embedded in project goal setting and student training. This will be led by Biotherapy Services and will run alongside laboratory investigations.