First volunteers to receive blood cultured from stem cells in 2016
Red blood cells cultured in a laboratory will be trialled in human volunteers for the first time within the next three years, as part of a long-term research programme funded by the Wellcome Trust.
The £5 million Strategic Award was granted to a consortium led by the Scottish National Blood Transfusion Service (SNBTS), and will follow on from previous research that proved that red blood cells could be generated from stem cells.
The consortium includes the University of Glasgow, the University of Edinburgh, Loughborough University, NHS Blood and Transplant, the Irish Blood Transfusion Service, Roslin Cells Ltd and the Cell Therapy Catapult, in collaboration with Bristol University and the University of Cambridge.
The consortium will be using pluripotent stem cells, which are able to form any other cell in the body. The team will guide these cells in the lab to multiply and become fresh red blood cells for use in humans, with the hope of making the process scalable for manufacture on a commercial scale. The team hopes to start the first-in-man trial by late 2016.
Blood transfusions play a critical role in current clinical practice, with over 90m red blood cell transfusions taking place each year worldwide. Transfusions are currently made possible by blood donation programmes, but supplies are insufficient in many countries globally. Blood donations also bring a range of challenges with them, including the risk of transmitting infections, the potential for incompatibility with the recipient's immune system and the possibility of iron overload. The use of cultured red blood cells in transfusions could avoid these risks and provide fresh, younger cells that may have a clinical advantage by surviving longer and performing better.
Professor Marc Turner, Principal Investigator, said: "Producing a cellular therapy which is of the scale, quality and safety required for human clinical trials is a very significant challenge, but if we can achieve success with this first-in-man clinical study it will be an important step forward to enable populations all over the world to benefit from blood transfusions. These developments will also provide information of value to other researchers working on the development of cellular therapies."
Dr Ted Bianco, Director of Technology Transfer at the Wellcome Trust, said: "Harnessing the power of stem cell biology to contribute to healthcare is one of the most exciting opportunities we can expect to see reach fruition in the coming years. But one should not underestimate the challenge of translating the science into routine procedures for the clinic. Nowhere is this more apparent than in the challenge Professor Turner and colleagues have set out to address, which is to replace the human blood donor as the source of supply for life-saving transfusions, knowing that each unit of blood contains no less than a trillion red cells."
Before clinical trials can begin the cultured cells will have to be manufactured to a very high standard and be approved by UK regulatory authorities.
About the Scottish National Blood Transfusion Service
The Scottish National Blood Transfusion Service is part of NHS National Services Scotland (NSS). Accountable to the Scottish government, NSS works at the very heart of the health service, providing national support services and expert advice to NHS Scotland. It also plays an active and crucial role in the delivery of effective healthcare to patients and the public.
About the consortium
The consortium has been working on the development of red blood cells for transfusion from pluripotent stem cells for around four years. The original programme demonstrated that it is possible to generate enucleated red cells at a sufficient scale to allow initial studies in human volunteers. The purpose of the current programme is to manufacture red cells that meet sufficient standards of quality and safety to allow clinical studies to proceed with regulatory approval. The development of a complex cellular therapy such as this requires the combined talents of many different professionals from across the fields of biology, manufacturing and clinical translation, as well as the support of universities, blood services and commercial organisations, so as to jointly solve the fundamental problems in reaching this difficult goal.
About the Cell Therapy Catapult
The Cell Therapy Catapult is a centre of translational excellence for cell therapy and regenerative medicine. Its vision is for the UK to be a global leader in the development, delivery and commercialisation of cell therapy, making it a location for business start-up and growth. Based in London at Guy's Hospital, the centre takes products into early clinical trials, providing clinical, technical, manufacturing and regulatory expertise and access to the NHS. There is a focus on collaboration and lowering barriers to investment and funding, and operations have grown rapidly since inception. UK Trade & Investment has valued the global regenerative medicine industry at just over £500 million, and estimates that it will be generating revenues of over £5 billion by 2021.
About NHS Blood and Transplant
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.
About Roslin Cells
Roslin Cells is a leader in the development and production of stem cells for research and the production of cell therapies. The company was established at the world-renowned Roslin Institute in 2006 and is now based at the Edinburgh BioQuarter where, in addition to its iPSC core facility, it operates state-of-the-art clean rooms for the production of new cell therapies. This enables the company to provide research and clinical grade iPS cell services to prominent industry, academic and clinical researchers who are breaking new ground in cellular models for research, drug discovery and also the next generation of cells to be manufactured for cellular therapies.
About the Wellcome Trust
The Wellcome Trust is a global charitable foundation dedicated to achieving extraordinary improvements in human and animal health. It supports the brightest minds in biomedical research and the medical humanities. The Trust’s breadth of support includes public engagement, education and the application of research to improve health. It is independent of both political and commercial interests.