Objectives 3

The third scientific objective of The ONE Study is to learn more about the comparative characteristics of suppressive cell types and to assess their tolerogenic capacity and potential for clinical translation.

Expert scientists in immunotherapy across Europe, supported by motivated biotech industry, will be generating end-stage cell products that can be compared at different levels. For example, a complete molecular profiling (e.g. microarrays) of each of the purified cell types is planned, which is expected to uncover overlapping mechanisms of their activities, and hopefully unveil new possible mechanisms of action. Moreover, humanised mouse models will be used to investigate the functional mechanisms of cell products. The key to this testing is that it will be done at central facilities, reducing the chance for bias and centre-to-centre variation. The database generated for the different cell types will be an invaluable resource for advancing the field of haematopoietic cell therapy. This aim could only be accomplished with an EU consortium of this nature. All academic partners will contribute to this objective.

With the information gained from the clinical trial and from laboratory analyses of the immunoregulatory cell types, we expect to be in a strong position to begin development of a second-generation of improved cell products. Therefore, the basic science development of currently less advanced haematopoietic cell therapies will be promoted, particularly those likely to require more than three years for licensing as a cell product.