Year 1

We have built a highly experienced, multidisciplinary clinical, managerial, and scientific team to deliver a new stem cell based treatment for children with advanced airway disorders. There are no satisfactory treatments for severe collapse of the windpipe in children. Conventional surgery leads many children requiring repeated hospital admissions and can have serious complications, whilst the use of internal supports (stents) can cause infections, bleeding and coughing. We have performed one adult and one child airway replacement operations using implants based on the patient’s own stem cells. The windpipe scaffolds were derived from transplant donors, with all the donor cells removed but all the good biological properties retained. At three and one half and two years respectively, both patients are doing very well: the adult is working and the child at school. From the lessons learned by these ‘first-in-human’ experiences, we have been able to propose research work leading to, and including, the world’s first clinical trial of an organ replacement based on stem cells, here the windpipe in needy children. With our first-in-human experiences, we believe this project has a very high chance of successful delivery of a landmark clinical trial in California within the four-year period of funding requested.