Treatment Options for Diabetes Using Stem Cells

Type 1 diabetes mellitus is an autoimmune disease in which the immune system of the patient attacks the insulin-producing beta-cells in the pancreas as part of an inflammatory response. The resulting destruction of the beta cells leads to a harmful insulin deficiency. Up until now, type 1 diabetes has chiefly been treated by artificially administering the deficient hormone in the form of insulin preparations. However, this type of treatment does not, strictly speaking, provide a 'cure'. In fact, the missing endogenous substance has to be administered to the patient until the end of his or her life. Stem cells, on the other hand, are a potential resource for producing functioning beta cells which can also be used to treat the more widespread type 2 diabetes. Stem cells of varying origins which have the potential to differentiate into beta cells are suitable for this kind of treatment. 

The following studies are considered to be milestones in the development of stem cell-based diabetes treatments: In 2007, a team of scientists led by Julio Voltarelli succeeded in developing a procedure that temporarily reverses the diabetes in type 1 diabetes patients (although it does not permanently cure it). Stem cells were taken from the bone marrow of 15 volunteers aged between 14 and 31 who had recently been diagnosed with type 1 diabetes. Henceforth, all of their immune cells were destroyed by medication and their own stem cells were then re-administered intravenously. 12 out of the 15 patients had recovered so much within a few days that they no longer needed insulin injections. This effect lasted on average 18 months; with one patient the effect even persisted for 35 months. In a follow-up study carried out two years later, it was possible to reproduce these effects; however, neither study was a randomised controlled trial.

In 2008, researchers led by Evert Kroon were able to treat mice with human embryonic stem cells whose stem cells had been chemically destroyed. In the study, pancreatic tissue was derived from human embryonic stem cells and this was then implanted into the test mice. After around one month, cells formed from the human tissue which then synthesised insulin depending on blood sugar levels. After three months, the majority of the mice even produced enough insulin to compensate for the artificially induced diabetes. To what extent the findings of this study and those of similar experiments on animal models can be applied to humans is something that needs to be clarified further in follow-up studies.

Voltarelli, J. C./ Couri, C. E./ Stracieri, A. B./ Oliveira, M. C./ Moraes, D. A./ Pieroni, F. / Coutinho, M. / Malmegrim, K. C./ Foss-Freitas, M. C./ Somoes, B. P./ Foss, M. C./ Squiers, E. / Burt, R. K. (2007): Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetesmellitus. In: JAMA 297 (14), 1568–1576. doi: 10.1001/jama.297.14.1568 Online Version 

Kroon, E. / Martinson, L. A. / Kadoya, K. / Bang, A. G./ Kelly, O. G. / Eliazer, S. / Young, H. / Richardson, M. / Smart, N. G ./ Cunnigham, J. / Augulick, A. D. / D'Amour, K. D. / Carpenter, M. K. / Baetge, E. E. (2008): Pancreatic endoderm derived from human embryonic stem cells generates glucose-responsive insulin-secreting cells in vivo. In: Nature Biotechnology, Published online: 20 February, 2008. doi: 10.1038/nbt1393 Online Version 

Couri, C. E. / Oliveira, M. C. / Stracieri, A. B./ Moraes, D. A. / Pieroni, F. / Barros, G. M./ Madeira, M. I. A. / Malmegrim, K. C. / Foss-Freitas, M. C. / Somoes, B. P. / Martinez, E. Z. / Foss, M. C. / Burt, R. K. / Voltarelli, J. (2009): C-peptide levels and insulin independence following autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. In: JAMA 301(15), 1573–1579. doi: 10.1001/jama.2009.470 Online Version 

Since these initial success stories, further experiments and several clinical trials have been carried out (see the module on Pluripotent Stem Cells as a Treatment for Diabetes). Largely due to the subpar efficiency of producing large volumes of beta cells, the incomplete reproducibility of the achieved outcomes and the ethical debate surrounding the derivation of human embryonic stem cells, current research is chiefly targeting the production of functioning beta cells on the basis of adult and induced pluripotent stem cells.

An overview of the current status of research and clinical trials using a variety of stem cell types with regard to developing treatments for diabetes can be found here: 

Chen, S. / Du, K. / Zou, C. (2020): Current progress in stem cell therapy for type 1 diabetes mellitus. In: Stem Cell Res Ther 11(1), 275. doi: 10.1186/s13287-020-01793-6 Online Version 

Bluestone, J. A. / Desai, T. / Hebrok, M. / Roy, S. / Sneddon, J. B. / Stock, P. / Tang, Q. (2018): Stem Cell Therapies for Treating Diabetes: Progress and Remaining Challenges. In: Cell Stem Cell 22(6), 810–823. doi: 10.1016/j.stem.2018.05.016 Online Version

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