Genetically modified islet cells are now being tested as a treatment for type 1 diabetes
For the first time, patients with type 1 diabetes will be offered a transplant using genetically modified, hypoimmune insulin-producing cells from the islets of Langerhans. The cells have been modified so that they cannot be detected by the immune system. The treatment is part of a clinical study at Uppsala University Hospital and has previously proved very successful in preclinical studies. The hope is that patients with type 1 diabetes can eventually be cured of the disease. The Uppsala University Hospital writes this in a press release.
Per-Ola Carlsson
"This is a potentially curative cell therapy that is now being tested on patients for the first time ever. The insulin-producing cells being transplanted have been genetically modified to avoid detection by the immune system. This could mean that the patient does not need lifelong treatment with immunosuppressant drugs, which have been required for islet transplantation to date and which often have side effects and are not suitable for everyone. The long-term hope is that all patients with type 1 diabetes can be cured of the disease by replacing their destroyed insulin-producing cells with new ones," says Per-Ola Carlsson, senior physician and professor of diabetes at Uppsala University /Uppsala University Hospital.
The study, a so-called first-in-human safety study, will last for one year. It is carried out within the framework of the new centre for advanced drug therapies, ATMP, which was inaugurated at Uppsala University Hospital in September 2023 and primarily aims to ensure that more patients benefit from the development and benefit from new treatments.
The study will evaluate the treatment in up to two patients aged 30-45 years with elevated blood sugar levels despite standard treatment. The patients will have the cells transplanted into their forearm. The patients will then be followed for one year in the study, but a long-term follow-up is also planned. The aim of the study is to evaluate the safety and whether the insulin-producing cells escape "attack" from the immune system, survive and generate measurable stabilisation of blood sugar levels. If patients regain their own insulin production, the treatment can be a cure.
Treatment of type 1 diabetes has progressed greatly in recent years, for example with insulin pumps. For people with severe type 1 diabetes and unpredictable, sharp variations in blood sugar, island transplantation with so-called Langerhans islands has been available as an alternative treatment for more than 20 years. Since its inception in 2001, 165 islet transplants have been performed at Uppsala University Hospital on about 60 patients.
"As with previous islet transplants, in this case insulin-producing cells, known as Langerhans' islets, are taken from the pancreas of a deceased donor. The islet isolation takes place at the Rudbeck Laboratory here at Uppsala University Hospital. What is new is that three genomic changes are made in the cells before they are given to the patients to avoid rejection, that the cells are made hypoimmune, so to speak, which means that the patients have an opportunity to avoid immunosuppressive treatment. Already after 2-4 weeks we can determine whether the cells have survived and not been rejected," explains Per-Ola Carlsson.