The INSPIRE consortium, led by Videregen Ltd, has received approval from the MHRA1 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 £1.9m of funding from Innovate UK.
SSAD is associated with significant airway obstruction leading to high levels of morbidity and a 50% 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.’
Videregen is a clinical-stage regenerative medicine company using its proprietary stem cell-based technology platform to develop a range of personalised, non-immunogenic organ replacement products for orphan indications. Its lead programme is a tissue engineered trachea replacement, and its patented technology and know-how is also being applied to the development of other organ replacement products, including mucosal lining, small bowel and liver replacements. The platform technology, which uses decellularised organ scaffolds seeded with the patient’s own cells to create new organs, has the potential to generate cost-effective and curative therapies for a range of devastating diseases. Founded in 2011, Videregen is an SME based in Liverpool. Its investors include SPARK Impact, London Business Angels and others. For more information please go to www.videregen.com
About the Cell Therapy Catapult
The Cell Therapy Catapult was established in 2012 as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 100 employees focusing on cell and gene therapy technologies, we work with our partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. We offer leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. We aim to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. Regenerative medicine is one of the UK government’s eight great technologies that support UK science strengths and business capabilities. The Cell Therapy Catapult works with Innovate UK. For more information go to ct.catapult.org.uk or visit www.gov.uk/innovate-uk.
About UCL (University College London)
UCL was founded in 1826. We were the first English university established after Oxford and Cambridge, the first to open up university education to those previously excluded from it, and the first to provide systematic teaching of law, architecture and medicine. We are among the world's top universities, as reflected by performance in a range of international rankings and tables. UCL currently has over 35,000 students from 150 countries and over 11,000 staff. Our annual income is more than £1 billion.
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NHS Blood and Transplant (NHSBT) is a joint England and Wales Special Health Authority. Its remit includes the provision of a reliable, efficient supply of blood and associated services to the NHS in England and North Wales. It is also the organ donor organisation for the UK and is responsible for matching and allocating donated organs.
Northwick Park Institute for Medical Research (NPIMR) is a medical research charity, affiliated to UCL. We are dedicated to the acquisition and dissemination, by research, education and publishing, of ethical research based data for the benefit of patients both nationally and internationally. Our expertise is specifically in the invention, development, pre-clinical and safety evaluation of novel tissue scaffolds and their rapid progress to clinical use.
About Royal Free
The Royal Free attracts patients from across the country and beyond to its specialist services in liver, kidney and bone marrow transplantation, haemophilia, surgery for hepatopancreatobiliary (HPB) conditions, clinical neurosciences, renal, HIV, infectious diseases, plastic surgery, immunology, vascular surgery, cardiology, amyloidosis and scleroderma and is a member of the academic health science partnership UCLPartners. For further information, visit www.royalfree.nhs.uk