RGM.20.010 – Beta cell replacement therapy for type 1 diabetes

Route: Regenerative medicine: game changer moving to broad areas of application

Cluster question: 100 How can we use cells, stem cells, and biomaterials to engineer and regenerate tissues and organs?

Type 1 diabetes is a lifelong autoimmune disease which specifically destroys the insulin producing beta-cells residing in the pancreatic islets and often occurs at a very young age. Standard insulin therapy is a symptomatic treatment for patients to maintain normal blood glucose levels, but inevitably leads to long term complication such as cardiovascular disease, kidney damage and blindness. A better treatment would be to provide patients with a sufficient amount of new beta cells to become insulin independent again. Clinical islet transplantation today has been shown to be a good alternative for insulin therapy, but unfortunately it is not a very efficient method. Most of the allogeneic donor islets are lost within two weeks after transplantation. In order to reach full insulin independence, patients have to undergo multiple transplantations with cells from different donor pancreata to become fully insulin independent. Risks associated with the accompanying immunosuppressive therapy allow only very few severely affected patients access to this treatment. In the past 5 years we developed two important strategies to make beta-cell replacement therapy widely available for type 1 diabetes patients namely: a tailor-made biomaterial-based delivery device for beta cells, and development of stem cell derived beta cells. The delivery device enables one to transplant islets and beta-cells in an optimal location while maximizing their survival and blood glucose maintenance function, while the development of IPSC derived beta-cells will provide us with an unlimited source of beta cells, which can be used for clinical transplantation. We are already able to produce delivery devices with different dimensions for research purposes, which we will test relatively soon in a first phase clinical trial. We aim to further optimize the stem cell derived beta cells and would like to develop a next generation delivery device for future clinical application in combination with these cells.

Keywords

bioengineering, clinical islet transplantation, stem cell derived beta cells, type 1 diabetes

Other organisations

Hubrecht Laboratory, Leiden Universitair Medisch Centrum (LUMC)

Submitter

Organisation Maastricht University (UM)
Name Dr. Aart van Apeldoorn
E-mail a.vanapeldoorn@maastrichtuniversity.nl
Website https://merlninstitute.com/merln/aart-van-apeldoorn