New cell therapy raises hope for curing type 1 diabetes – "Never succeeded before"
Av In a first-in-human study at the ATMP center at Uppsala University Hospital in Sweden, the world’s first transplantation of genetically modified, hypoimmune insulin-producing cells from the islets of Langerhans has been used as a treatment. The key aspect is that the transplantation was carried out without immunosuppression. The hope is that, in the long term, this patient group could be cured of type 1 diabetes.
The American company Sana Biotechnology, which developed the hypoimmune technology, saw its stock soar after the first results were presented this week. The company’s shares rose by approximately 200 percent.
For individuals with severe type 1 diabetes, a well-established treatment involving transplantation of islets of Langerhans, a type of cell cluster containing insulin-producing cells, has been available for over 20 years. This treatment involves isolating islets of Langerhans from a deceased donor's pancreas and transplanting them into a diabetes patient with the goal of achieving normal blood sugar control and independence from insulin. However, the transplantation has required immunosuppression in the patient, which often leads to side effects and is not suitable for everyone.
– As with all other organ and cell transplantation, where tissue comes from another individual, it has so far required suppression of the recipient’s immune system to prevent immune rejection of the transplanted cells. The insulin-producing cells we are using in the new study are genetically modified to avoid detection by the immune system and are therefore protected from both rejection and autoimmune attack, explains proffesor Per-Ola Carlsson, study principal investigator, in a press release.
The study is being conducted in collaboration with Sana Biotechnology, based in Seattle, which holds the patent for the hypoimmune technology used in the genetic modification. The company’s founder, Sonja Schrepfer, has long conducted research on animals to determine how cells can survive transplantation without rejection.
– What we have done in Uppsala is a proof of concept to demonstrate that what worked in animals also works in humans, says Per-Ola Carlsson to Life Science Sweden.
The initial results from the treated patient show that the cells evade detection by the immune system and continue to function after transplantation. The study identified no safety concerns.
According to the company, this study is the first to demonstrate survival of an allogeneic transplantation without immunosuppression or an immunoprotective device in a fully immunocompetent individual.
The results are based on data collected four weeks after the cell transplantation. They demonstrated both survival and function of the cells, measured partly through the presence of the biomarker C-peptide in the blood. C-peptide levels also increased during a meal tolerance test (MMTT), indicating that the transplanted cells were stimulated to release insulin in response to a meal.
What conclusions can be drawn from the results after just four weeks?
– In cell transplantation, a rejection reaction typically occurs within the first week, and it is an extremely strong reaction. Therefore, we consider four weeks to be a sufficient time to conclude that it works. We will continue to monitor this transplantation over time, says Per-Ola Carlsson.
How optimistic can one be based on results from just one patient?
– The immune system does not vary significantly in this regard. It has never been successful before to have cells survive for up to a month without using immunosuppressive drugs when transplanting between individuals. So, this is primarily a major immunological breakthrough.
The new data, combined with other research advancements, gives hope that a curative treatment can be offered to type 1 diabetes patients on a larger scale, he believes. However, there are challenges, particularly concerning the manufacturing process of the advanced therapeutic medicine.
Looking ahead, Per-Ola Carlsson suggests moving away from organ donor tissue and focusing instead on producing corresponding genetically modified insulin-producing cells from stem cells. This would allow for the generation of an almost unlimited number of cells for transplantation. Successfully transplanting cells without rejection has significant implications for other diseases as well, he concludes.
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