Pre-clinical development of a hematopoietic stem cell therapy product

点击翻译按钮获取中文摘要

Many patients with blood disorders, such as cancers, can usually be treated by receiving a bone marrow transplant from another person. Usually this donor has to be a blood relation or have a compatible tissue type to ensure the transplant is not rejected. However, finding such a match is not always possible. For some years now, parents have been donating the blood found in the umbilical cord of their new-born babies. Just as with bone marrow, this 'cord blood' contains stem cells which are capable of reconstituting the entire blood and immune system of a transplant recipient. Furthermore, complete matching is not required, so cord blood cells can be used to treat patients that can't find exact matches, lowering the risk of tissue rejection. Unfortunately, the number of cells in each cord blood unit is limited and too small to treat most adults, therefore methods to expand the number of these cells in the laboratory are highly desirable. Plasticell has developed and patented formulations which, in preliminary _in vitro_ studies, allow the stem cells in the cord blood units to be multiplied by around 500-fold without affecting their properties. The company has further developed manufacturing methods in which these cells can be expanded ready for clinical use. The resulting cells have been tested _in vitro_ in the laboratory and in preliminary _in vivo_ transplantation using immune-deficient mouse models. Nevertheless, to ensure the cells are useful to patients we must carry out more extensive _in vivo_ analyses. In this project, we will test the cells ability to reconstitute the entire blood system when injected into immune-deficient mice and also aim to develop innovative solutions to ensure the stem cell product we are developing can be made and delivered to the patients that need it in a format that is safe, effective, user-friendly and affordable.