Transforming diabetes care through stem cell-based therapies
Type 1 diabetes (T1D) imposes a lifelong clinical and social burden on patients and their families. It also represents a significant and growing burden on healthcare systems, accounting for approximately 10 % of total healthcare expenditure. “The average annual direct care costs for T1D patients are estimated to be around EUR 8 000 per patient,” notes ISLET(opens in new window) project coordinator Henrik Semb from Helmholtz Munich(opens in new window) in Germany. “This excludes hospitalisation and the use of high-end technologies such as continuous glucose monitors and insulin pumps.”
Durable cell therapy treatments
The EU-funded ISLET project set out to find ways of improving the standard of T1D care. The opportunity identified was the development of a durable cell therapy treatment. Cell therapies represent a promising pathway towards replacing damaged beta cells in people living with T1D, removing the need for patients to manage their own insulin levels. “We wanted to establish a programme for the engineering and testing of stem cell-derived insulin-producing beta cell products, for future treatment of T1D patients,” explains Semb. “For this, we needed to develop a first stem cell-derived product, ready to enter preclinical and clinical trials.” To achieve its aims, the project brought together expertise in stem cell and developmental biology, beta cell biology, and clinical pancreatic islet transplantation. It also utilised omics technologies (a combination of techniques used to holistically analyse biological systems) and adopted high-quality manufacturing practices. Another key element of the project was the inclusion of voices from the diabetes community – the International Diabetes Federation Europe(opens in new window) was a project participant.
First-generation stem cell-derived products
Through pooling this expertise, the ISLET project was able to develop a first-generation stem cell-derived beta cell product for the future treatment of T1D patients. These lab-engineered islet-like clusters, containing beta and alpha cells from stem cells, are designed to reproduce the function of islets in a person’s body. Islets are the clusters of cells found within the pancreas that mainly contain insulin-producing beta cells and glucagon-producing alpha cells. The ISLET project delivered proof of concept showing that this newly developed stem cell-based product efficiently and safely restores normal glucose concentrations in diabetic animal models. “We reached our goal of performing all the development work necessary to be able to continue with non-clinical and clinical studies,” says Semb. Additional key findings include new leads on how omics can help researchers to assess the health status of cells during manufacturing. The ISLET project also expanded fundamental knowledge of human pancreatic development. The goal now is to translate these mechanistic insights into tangible product concepts with improved safety and functionality.
Combination of multidisciplinary expertise
Semb believes that the combination of multidisciplinary expertise was a key strength of the project. “This integrated approach has helped to reinforce Europe’s competitive position in regenerative medicine,” he adds. Moving forward, the first-generation stem cell product pioneered in ISLET will be further evaluated in preclinical animal studies to assess safety and functionality. These studies will help to pave the way for future clinical studies in patients with T1D. “This work has already been initiated and will be carried out at Lund University and Skåne University Hospital (SUS) in Lund/Malmö, Sweden,” notes Semb.
