Transplant treatment for diabetes shows promising results

Portrait of Per-Ola Carlsson

Per-Ola Carlsson, Professor of Medical Cell Biology. Photo: Mikael Wallerstedt

A completely new way of treating type 1 diabetes is being tested at Uppsala University Hospital. Patients are being offered transplants of genetically modified insulin-producing cells and the initial results are promising. We put a few questions to Per-Ola Carlsson, Professor of Medical Cell Biology, who is leading the study.

The study started in December and has already attracted a lot of attention. What makes it so special?

“To find a curative treatment for diabetes, two components are needed. Firstly, you have to have a sufficient quantity of insulin-producing cells to be able to treat many individuals with type 1 diabetes. This problem has actually been solved in the last ten years by means of a type of stem cell treatment with insulin-producing cells that is currently undergoing clinical trials.

“The remaining problem has been that as the cells come from another individual, the patients need lifelong immunosuppressive treatment, which can lead to serious side-effects. What we have done is to see whether we can find a possibility and a strategy for transplanting cells between individuals without immunosuppressive drugs.”

You have developed genetically modified insulin-producing cells that are hypoimmune, so that immunosuppressive treatment is not required. For the first time, they have been transplanted into a human. How has it gone?

“We want to see if we can transplant between individuals and get the cells to survive. Though this study is primarily a safety trial, of course we are taking the opportunity to see how the cells are doing.

“At the end of the year, we had just over four weeks of observations and saw that the same quantity of cells remained as when we inserted them. They do not disappear, they have stayed there with their own stable insulin production over the course of these weeks. What we would otherwise have expected in a transplant between individuals is that we would have had a rejection process and would already have lost this cell function in the first few weeks.”

What will be the next stage of your research?

“We will now continue to monitor this over time. This type of study has to carry on for 15 years to show that the treatment is safe. It may not take that long to be able to say that the cells work and that it’s reasonably safe, so we won’t wait all that time before we go ahead with new studies.

“The next step is to apply this genetic modification to stem cells. That would make it possible to produce huge quantities of insulin-producing cells with this genetic modification. We would then have a large number of cells that could be used in clinical trials and could also be made into a pharmaceutical product.”

Annica Hulth

Transplantation treatment for diabetes

  • For people with severe type 1 diabetes, transplantation of islets of Langerhans containing insulin-producing cells has been an established treatment for over 20 years.
  • These cells come from the pancreas of a deceased donor and are transplanted into a patient with the aim of achieving normal blood sugar control and insulin independence.
  • As with all other organ and cell transplants, suppression of the recipient’s immune system has so far been required to prevent immune rejection of the transplanted cells.
  • The insulin-producing cells used in the new study are genetically modified to avoid detection by the immune system, which protects them from both rejection and autoimmune attack.

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