Stem cell–derived islets produce insulin in non-human primates.
The startup company Semma Therapeutics is developing a stem cell therapy for diabetes. The approach is based on research by Semma scientific founder and Harvard professor Doug Melton. Melton used stem cells to create insulin-producing pancreatic beta cells, which were successfully protected from an immune response when inserted into mice in bulk.
The company recently announced () positive results for its preclinical studies. Its stem cell–derived islets produced insulin in non-human primates whose immune systems had been flattened to prevent a rejection — and thus performed as anticipated. In a separate study, the engineered islets were contained in a specially designed package and administered to two pigs. The islets successfully produced insulin without having to give the pigs an immunosuppressant. They also functioned in response to blood glucose levels, causing the production of insulin on an as-needed basis. In addition, they work without causing fibrosis or cell suffocation.
Semma is now preparing to launch two small phase II clinical trials in 2020, to see if the same results obtained in non-human primates and pigs can be achieved in humans with and without an immunosuppressant, respectively. The company will first target type 1 diabetes patients that are difficult to treat and have no other options. They will then focus on the broader type 1 population and eventually particular subgroups of type 2 diabetes patients.
One of the challenges ahead is the development of a scalable manufacturing process to produce the stem cell–based islet at large scale. The company also hopes to identify a cell design that will eliminate the need for the specially designed packaging that protects the islets from immune system attack.