Development of a nebulised cell based therapy for acute respiratory distress syndrome

Acute lung injury and acute respiratory distress syndrome (ARDS) constitute a spectrum of severe acute respiratory failure. Mortality rates over 60% have been reported and ARDS is the leading cause of death in the critically ill. ARDS develops most commonly in the context of severe sepsis, particularly infection with gram-negative bacteria such as Escherichia Coli. Currently there is no effective treatment for ARDS and little evidence to support any specific intervention. Treatment remains supportive with mechanical ventilation in intensive care units, a mainstay in ARDS, providing only symptomatic care which can lead to further lung injury and multi-organ failure. Therefore, there is an urgent need to develop novel strategies to treat ARDS.

Mesenchymal stem cells (MSC) have demonstrated anti-inflammatory, anti-bacterial and pro-healing effects in preclinical models of ARDS and have confirmed safety in early clinical trials. We have recently established that MSC derived products such as the MSC secretome are equally functional in preclinical models. However, unlike cells, these products are nebulisable, opening up direct therapeutic delivery to the lung. Delivery to the lung has a number of advantages as systemic side effects are minimised and the therapeutic is delivered to the injured site with minimal therapeutic loss. We plan to use nebuliser technology to deliver these MSC derived products to the lung and assess their effectiveness in a preclinical model of ARDS. We will investigate dosage regimes, assess the therapeutic window of effect and therefore determine the most efficient strategy to deliver the therapeutic to the lung epithelium. If these evaluations are successful we will proceed to planning a phase I/II clinical trials and seek further funding to progress this novel therapeutic towards clinic.

Award Date
30 June 2017
Award Value
€364,687
Principal Investigator
Dr Daniel O'Toole
Host Institution
National University of Ireland, Galway
Scheme
Investigator Led Projects