Establishment of an efficient and highly automated production of MSC for the treatment of T1D patients with an aim to prevent beta-cell death and restore beta-cell mass.
Type 1 diabetes (T1D) is a chronic disease with a high frequency of long-term complications such as blindness, kidney failure, myocardial infarcts, stroke and premature death. It is caused by a progressive loss of the insulin-producing beta cells located within the islets of Langerhans, which are dispersed in the pancreas. For largely unknown reasons, the incidence of T1D is increasing rapidly in many parts of the world and it has no curative treatment.
Mesenchymal stromals cells (MSC) are non-hematopoietic cells in the bone marrow that produce matrix proteins and contribute to tissue regeneration and repair. In addition, MSC have immunomodulatory capacities and can suppress T cell proliferation while up regulating regulatory T cells. MSC can readily be expanded in vitro and have been shown to reverse diabetes in animal models.
A clinical study evaluating the safety and efficacy of autologous MSC treatment in human T1D was recently concluded by the investigators of iCell Science, which showed that MSC provides a safe and promising alternative to intervene in the disease progression and preserve beta-cell function. In contrast to the patients in the control group, MSC-treated patients showed no loss in C-peptide and even increased their C-peptide levels at one year after diagnosis.
The aim of the project is to improve the production process of MSC, making it more efficient, so that this promising therapy can be made available to larger numbers of T1D patients in the absence of chronic immune suppression. At the end of the project, iCell Science will have established an efficient, cost effective, automated and easily scalable production of MSC for the treatment all subjects diagnosed with T1D in Sweden. iCell Science will also apply for Orphan drug designation of MSC in newly diagnosed T1D patients (incidence about 1 in 10.000 people) in both the EU and US, to further enhance the chances of successful commercialization and market authorization of this cellular product.
The project is carried out in close collaboration with the Centre of Excellence for Diabetes at the Uppsala University Hospital, including Professor Olle Korsgren, Professor Per-Ola Carlsson and Professor Katarina Le Blanc.