It may sound like science fiction or an attempt to inject novelty into the overworked vampire genre, but the production of artificial blood, on a scale that would rival existing transfusion sources, is being countenanced by researchers eager to exploit stem cell technology. In particular, researchers at the University of Edinburgh and the Scottish National Blood Transfusion Service (SNBTS) are looking forward to assess how well people accept blood transfusions derived from induced pluripotent stem cells. Clinical trials assessing the safety of the approach may commence by the end of 2016.
A team of scientists led by SNBTS director Marc Turner, M.D., Ph.D. is heading the project, which reflects the combined efforts of the BloodPharma consortium, which has benefited from a Strategic Award, provided by the Wellcome Trust, in the amount of £5 million (approximately $8.4 million).
The research funded by the award involves multiplying and converting induced pluripotent stem cells (iPSCs) into red blood cells for use in humans, with the aim of making the process scalable for manufacture on a commercial scale. In hopes of refining the process, one of the BloodPharma consortium members, the Cell Therapy Catapult, intends to develop the nonclinical and clinical studies, and to provide guidance on data requirements for market access optimization.
The clinical trial will begin conservatively, with three patients who are in need of regular transfusions because they have thalassemia, a disorder of the red blood cells. These patients will receive about 5 mL of blood initially, and the researchers will determine whether the laboratory-made blood cells survive and behave normally once they are in the patients' bodies.
“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,” explained Dr. Turner. “These developments will also provide information of value to other researchers working on the development of cellular therapies.”
Dr. Turner added that while the researchers are optimistic about the clinical applications of their approach, it may take at least 20 years before artificial blood is widely used. Accordingly, the news that artificial blood is entering early clinical trials should not be taken as a signal that blood donations are any less necessary than ever.
While the use of induced pluripotent stem cells rather than embryonic stem cells offers a key advantage—the ability to develop cells of particular cell types—researchers also acknowledge that many development challenges remain.
Dr. Turner and colleagues, in an article published October 2013 in Cell Stem Cell, wrote the following: "While it is possible that clinical GMP-grade autologous iPSC lines could be derived on an individual basis, it seems unlikely that these will be used as a source for large numbers of patients in the near future, given the time and cost required to produce clinical GMP cell lines and to differentiate these into cells and tissues of clinical utility. It is likely, therefore, that a bank of allogeneic clinical GMP cell lines will be required to allow the field to develop over the next few years, raising the issues of how such a bank, or network of banks, could be established and, in particular, how immune incompatibility can best be managed."