Program for Translational Diabetes
The Program for Translational Diabetes Research is a translational research program at DanStem funded by the Novo Nordisk Foundation with the vision to initiate new strategies for bringing advanced stem cell-derived therapy to a broad population of type 1 diabetes (T1D) patients. Currently led by Professor Henrik Semb, the program aims to conceive a plan for scalable engineering of human pluripotent stem cell (hPSC)-derived pancreatic islet of Langerhans-like cellular grafts for future T1D cell therapy trials.
There is currently no cure for T1D, and with age, the frequency of complications such as kidney failure and blindness due to vascular complications dramatically worsens patients’ quality and length of life. Furthermore, patients with severe T1D who respond poorly to available treatment, are at risk to die prematurely (“brittle diabetes”). Importantly, when these patients receive donor islet transplants their uncontrolled glucose fluctuations dramatically improve, providing encouraging proof-of-concept data that cell-replacement therapy can improve the quality of life and lower the risk of premature death in patients with severe forms of T1D.
The overall goal of the Program for Translational Diabetes Research is to establish a safe, scalable and cost-effective stem cell therapy for T1D patients. This goal signifies a key step in the long-term objective to develop an academic program that, in collaboration with leading basic and clinical researchers, regulatory and ethical consultants, and cell manufacturing, hospital and industrial partners, aims to provide a stem cell-based therapy for a large number of T1D patients.
Manufacturing of cells for future clinical trials requires state-of-the-art GMP facilities that can provide appropriate experience and access to the facility. An adequate facility is not available in Denmark, and only the UK and Germany have GMP facilities with sufficient requirements for this work. As a result of this situation, DanStem recently established a partnership with the Helmholtz Zentrum München (HMGU), Munich, Germany, to strengthen translational diabetes research in Copenhagen. In this partnership, HMGU will finance the GMP-adaptation of our current differentiation protocol of hPSCs into glucose-responsive beta cells for future clinical trials.
To fortify the partnership, and thereby move more quickly towards the clinic, the Program in Translational Diabetes Research aims to establish a stem cell-based cell therapy program at University of Copenhagen covering all relevant steps from bench to patient.
The specific objectives of the Program in Translational Diabetes Research are to:
- Develop a transferable platform for manufacturing of cells for clinical supply
- Optimize the functionality and stability of the hPSC-derived cellular graft (advanced therapy medicinal product (ATMP))
- Develop safe expandable manufacturing of the graft and innovative strategies to improve ATMP quality control
- Harmonize EU regulation and ethics with ATMP approval for hPSC-based therapy in T1D
- Carry out the 1st European stem cell-based clinical trial in T1D
- Disseminate results, solutions, and technology transfer to stakeholders
- Collaborate with industry to develop a new business model for marketing of ATMPs in T1D, (see: DanStem spinoff company Pancryos)
- Initiate new strategies for bringing this treatment to a broader population of T1D patients by addressing immunological barriers