Annex: Visit to UCL and Centre for Cell Gene and Tissue Therapeutics, Royal Free Hospital
1)On Tuesday 29 November 2016, the Chair and Carol Monaghan visited the UCL Institute of Ophthalmology. The UCL Institute of Ophthalmology is working in partnership with Moorfields Eye Hospital.
2)Following a tour of the stem cell laboratories, they had the opportunity to speak to Professor Pete Coffey and Professor Robin Ali about their MRC-funded research in gene and stem cell therapy.
3)Robin Ali is Professor of Human Molecular Genetics at UCL Institute of Ophthalmology. The main focus of Professor Ali’s research is the development of gene and cell therapy for the treatment of retinal disorders. As chief investigator, he established the world’s first clinical trial of gene therapy for retinopathy. The results from this trial reporting an improvement in vision along with results from two other trials, established proof-of-concept for gene therapy for inherited retinal degeneration. His group has also provided the first proof-of-concept for effective transplantation of photoreceptors that has provided the basis for ES cell-derived photoreceptor transplantation, now a major programme in his laboratory.
4)Professor Coffey is director of the London Project to Cure Blindness and Professor of Cellular Therapy and Visual Sciences at the Institute of Ophthalmology, University College of London (UCL) and is an MRC funded researcher.
5)In September 2015, a pioneering trial of a new treatment derived from stem cells for people with ‘wet’ age-related macular degeneration (AMD) commenced at Moorfields Eye Hospital following a successful operation on a patient. This first operation was a major milestone in the London Project to Cure Blindness, which was established 10 years ago with the aim of curing vision loss in patients with wet AMD, and is the result of a partnership between the hospital, the UCL Institute of Ophthalmology, and the National Institute for Health Research (NIHR). Pfizer Inc. joined the partnership in 2009 with the goal of helping to turn the original idea into a potential therapy.
6)Professor Coffey’s team are transforming cells taken from skin biopsies into stem cells. These stem cells will be converted into eye cells that will be transplanted back into patients’ eyes to preserve their sight. In the recent clinical trial for the treatment of dry age-related macular degeneration, these cells have been grown on a membrane which is then inserted into the eye as a patch of cells.
7)On Tuesday 13 December 2016, the Chair visited the Centre for Cell Gene and Tissue Therapeutics (CCGTT) at the Royal Free Hospital in London. A tour of the CCGTT manufacturing suites and process development laboratories was provided by Professor Martin Birchall and Professor Mark Lowdell. The CCGTT is a manufacturing and development facility for Advanced Therapeutic Medicinal Products regulated by both the Human Tissue Authority and the MHRA. The cell manufacturing labs allow scientists at the hospital to create or modify cells which can be used to treat a range of conditions, including lung cancer, haemophilia and macular degeneration, using state of the art platform technologies. The suite is utilised by both academic and industrial partners, bridging the gap between basic research and manufactured medicinal products.
8)In 2008, MRC-funded researchers at University College London carried out the first transplant of a human trachea (wind pipe) reconstructed using stem cells. By 2013, the group were ready to build on this success by developing the first clinical trials of a stem cell-derived larynx transplant in a project known as “RegenVOX”. The RegenVOX procedure involves preparing a reconstructed larynx made from the patient’s own stem cells and a donor larynx. The team removes the cells from the donor larynx, leaving behind a scaffold onto which the patient’s stem cells are grafted. This means that the new larynx will not be rejected by the immune system so patients do not need immunosuppressant medication.
Note: See also paras 95–96 of Sixth Report of Session 2017–19
9)On both visits, the challenges for securing funding for ‘translational’ research were raised. Such programmes require significant and sustained funding. Funding for research is available from the MRC and research charities, and Innovate UK supported developmental stages and industry-led work. Company and venture capital funding can be more difficult to secure than from other biomedical drug development activities due to uncertainties over the business model and challenges in product development. A major challenge is sustaining funding support for the critical GMP facilities needed to underpin product development and testing, which is expensive. Ideally, long term support needed to be made available to underpin facilities with costs recovered through individual projects, potentially with commercial partners.
28 April 2017