Diabetes Moonshot
-A proof-of-concept therapy for type 1 diabetes-
The RegMed XB Diabetes Moonshot focuses on making a combination product of insulin-producing cells protected by a biomaterial implant for patients with type-1 diabetes.
Type-1 diabetes is a chronic autoimmune disease that cannot be cured. Globally almost 50 million patients suffer from this disease while in the Netherlands up to 120.000 patients are diagnosed. In 2017, total expenditure on diabetes care (both type 1 and type 2) in the Netherlands amounted to almost 1.6 billion euros (1.8% of total healthcare expenditure in the Netherlands). In patients with this form of diabetes, their own immune system destroys the insulin-producing beta cells in the pancreas. The hormone insulin is vital because it allows the cells in the body to absorb glucose, a form of sugar, to function properly. Moreover, it regulates the glucose concentration in the blood so that there are no major fluctuations.
What does it mean for patients?
Because the body produces too little/no insulin, patients have to measure blood sugar several times a day. Using this information together with lifestyle factors (meals, exercise) they can then calculate and inject the required dose of insulin. However, mimicking the natural insulin dosage is impossible and therefore large fluctuations in glucose concentration take place. Prolonged high blood glucose levels greatly increase the risk of serious complications such as blindness, damage to heart and blood vessels, nerve damage, and kidney failure. Too low blood glucose levels can, at worst, lead to drowsiness, confusion, and even coma. As a result, the overall quality of life deteriorates. Not only physically but also mentally this can be very difficult for patients.
For more information about Diabetes or how the interests of these patients are represented visit the Diabetes Fonds website.
What do we do in the RegMed XB Moonshot?
Within this Moonshot, the goal is to make a beta cell replacement implant that can be placed in humans and in which the insulin-producing beta cells are located. This implant must respond to the patient's blood glucose levels while protecting insulin-producing cells from the body's immune responses.
Status
Over the past few years, it has been possible to establish an infrastructure and apply protocols in both Leiden and Utrecht, making it possible to develop in vitro insulin-producing cells from stem cells. A number of cells have also been modified so that their function can be easily monitored. The main challenge here is to make the final steps in this transformation to insulin-producing cells more efficient and faster.
In the future research will focus on their precise function, lifespan, and safety. As far as the biomaterials are concerned, an open protective structure has been selected and has already been extensively tested. This is now being further validated by us as well as external partners before the first clinical trials will take place. The biggest challenges here lie in converting production to a level that is robust and safe enough to guarantee quality and longevity every time. Moreover, the construction is currently being extensively tested in various experiments to show that the function of the cells is good enough and the biomaterial is well tolerated by the body.
