The INSPIRE consortium, led by Videregen Ltd, a clinical-stage regenerative medicine company, has received approval from the MHRA to start a UK clinical trial with its tissue engineered replacement trachea. The trial, which is expected to start in the first half of 2016, will focus on severe structural airway disease (SSAD), a potentially life-threatening condition believed to affect 19,000 people in Europe and the US.

Along with Videregen as commercial partner, the consortium includes the Cell Therapy Catapult, University College London (UCL), NHS Blood and Transplant in Speke and the Royal Free London NHS Foundation Trust.

The phase I trial, which will recruit four patients with SSAD, is to assess the safety and initial efficacy of the product. While replacement trachea technologies have been tested previously in compassionate use cases, this is the first formal clinical trial of a tissue engineered autologous stem cell trachea replacement. The INSPIRE project started in 2014, and has GBP 1.9 million of funding from Innovate UK.

SSAD is associated with significant airway obstruction leading to high levels of morbidity and a 50 per cent mortality rate if not treated successfully. Current treatment involves surgery which has a high incidence of failure and lack of efficacy. In contrast, this tracheal replacement approach, which involves repopulating an acellular trachea 'scaffold' with the patient's own stem cells, is designed to be a one-off curative treatment without the need for a lifetime of anti-rejection drugs thereafter.

The INSPIRE clinical trial will be led by professors Martin Birchall and Mark Lowdell at UCL with the Cell Therapy Catapult as clinical trial sponsor and a provider of regulatory expertise. Videregen’s proprietary decellularisation technology, originally developed at Northwick Park Institute for Medical Research, is used by NHS Blood and Transplant to manufacture the trachea scaffold. The Centre for Cell, Gene and Tissue Therapy at the Royal Free London NHS Foundation Trust has developed the stem cell seeding processes so that the final product can be manufactured in its state-of-the-art facilities.

Speaking for the consortium, Dr Steve Bloor, CEO of Videregen, said, “Approval from the UK MHRA for this clinical trial is an important milestone for Videregen and the INSPIRE team, moving this transformative approach to airways disease one stage closer to market. The consortium approach, bringing together expertise from the Cell Therapy Catapult, clinician scientists at UCL, NHS Blood and Transplant and the Royal Free London NHS Foundation Trust, has enabled us to make good progress with this exciting project. Innovate UK's funding was an important first step towards the trial, which is critical in transforming trachea replacement from occasional compassionate use to a treatment that could benefit thousands of patients. It also helps to expand the technology into other indications, and the consortium is grateful for Innovate UK's continued support.”