A group of researchers successfully reversed a 25-year-old patient’s type 1 diabetes through a transplant of her own cells reprogrammed to start producing insulin without the need for medication. This is a scientific milestone published in the journal cell and collected by Nature.
Behind this success, currently limited to a single person, lies a sophisticated technique. Deng Hongkui, a cell biologist at Peking University, and his team took cells from three people with type 1 diabetes and returned them to a pluripotent state, from which they can be transformed into any type cell of the body.
This reprogramming technique is not new: it was first developed by Shinya Yamanaka of Kyoto University (Japan) almost two decades ago. However, the researchers modified the technique slightly and “instead of introducing proteins that trigger gene expression, as Yamanaka had done, they exposed the cells to small molecules, with greater control over the process,” according to the article. Nature.
One year of production
They then used these chemically induced stem cells to generate groups of 3D islets which they injected, in June 2023, into the patient’s abdominal muscles, a location from which they could monitor him with MRI. After two and a half months, the woman produced enough insulin to last and production was maintained for a year. During this time, he did not experience any spikes or decreases in his glucose levels.
“With due caution, this progress should be applauded. It’s quite complex and sophisticated and we’ll have to see how well it can be replicated, but it starts somewhere. You have to be very careful when throwing the bells into the air. Imagine that they have three more patients and that they do not work,” assesses Lluis Montoliu, geneticist and vice-director of the National Center for Biotechnology (CNB-CSIC).
With all due caution, this progress should be applauded. It’s quite complex and sophisticated and we’ll have to see how well it can be replicated, but it starts somewhere.
Lluís Montoliu, vice-director of the National Center for Biotechnology (CNB-CSIC)
This step is important for two reasons, according to Montoliu. The first is that cells produce insulin “in a regulated manner, that is, they act when they should act.” “They located the cells in a place exposed to the blood circulation, in a sort of abdominal sheath so that if there is a sugar spike, they detect this increase and produce insulin. They do what a pancreas would do but inside a cavity in the abdomen,” he explains.
The second reason is linked to safety: the process was developed without the stem cells being involved in the development of a tumor, the most feared side effect of these experiments. The researchers admit, however, that such procedures are “difficult to scale and commercialize.”
A growing pathology
Type 1 diabetes, or diabetes sugaris a chronic autoimmune pathology in which the immune system (defense system against pathogens) attacks and destroys the cells of the pancreas responsible for producing insulin. This released hormone helps sugar enter cells and can be used as an energy source. Without it, glucose builds up in the blood. To make up for this deficit, patients must obtain this hormone through injections or delivery devices (insulin pumps). It affects between 5 and 10% of people with this pathology (the majority are type 2) and its incidence has doubled among European minors over the last 30 years.
Beyond revolutionary drugs, such as Ozempic, several pioneering trials are underway with stem cells to treat a pathology that affects 500 million people worldwide, whose prevalence is increasing and which represents a challenge for health systems from all over the world.
Preliminary results from a trial conducted by Vertex Pharmaceuticals of Boston, Massachusetts, were presented in June. “A dozen participants with type 1 diabetes received islets derived from donated embryonic stem cells by injection into the liver. All were treated with immunosuppressants. Three months after the transplant, all participants started producing insulin when there was glucose in their blood. Some had become insulin independent,” he says. Nature.
