Medicine is advancing, sometimes faster than you think. In the fight against type 1 diabetes, a new breakthrough could change the situation: researchers are now capable of injecting embryonic stem cells capable of producing missing insulin. An innovation that opens the way to radically different management.
Rapid progress in a long constrained area
It is customary to say that medical research is progressing slowly. However, in diabetology, advances are increasing. The proof: a recent study published in the prestigious review New England Journal of Medicine highlights the promising results of cell therapy developed by the Green Pharmaceuticals laboratory. This targets patients with type 1 diabetes, an autoimmune form of the disease.
Type 1 diabetes, a chronic and demanding pathology
This type of diabetes, formerly called insulin -dependent diabetes (DID), represents between 10 and 15 % of cases in France. It generally occurs in young and not obese patients, before 30 years. In question: the destruction of beta cells of the pancreas, responsible for the production of insulin. Result, chronic hyperglycemia, with a risk of severe complications, such as acidocetosis. The patients must adjust their dose of insulin daily, sometimes time by hour.
It is precisely on these cells that the researchers wanted to act.
Reproduce the function of the pancreas thanks to the stem cells
The objective? Create cells capable of imitating the islets of Langerhans, real insulin factories located in the pancreas, and graft them into the body of patients. An idea that is not new: for the past twenty years, transplants from deceased donors have been practiced with some success.
But this method has its limits.
“”We quickly come up against a glass ceiling, because donors are strokes and their number will always remain limited“Explains Prof. François Pattou, head of the general and endocrine surgery service at the Lille University Hospital, in The Parisian.
And it’s not the only brake.
“”They must also be compatible, especially at the level of the blood group. In addition, there are often two or even three donors for a recipient in order to have a sufficient amount of islets“He adds.
Without forgetting the essential immunosuppressive treatment to avoid rejection. A heavy protocol, which confines this solution to the most serious cases, that is to say patients exposed to severe and frequent hypoglycaemia.
An innovation that changes the situation: the end of the glass ceiling?
Where Vertex Pharmaceuticals innovates is by making pancreatic islets no longer from human donors, but embryonic stem cells, cultivated in the laboratory. These cells, capable of differentiating in many cell types, have been “guided” to become insulin -producing beta cells. Result: a transplant entirely from cell culture.
The laboratory led one clinical trial in 12 patients, using the baptized treatment zimislecel. Three months after the injection, episodes of hypoglycemia have decreased sharply. A year later, ten of the participants no longer needed insulin.
“”A grafting of pancreatic cells derived from allogenic stem cells in type 1 diabetic patients allowed 83 % of patients treated to no longer require insulin treatment one year after injection“, Reports the company in a press release. These results were also presented at the American Diabetes Association Congress in Chicago.
Promising results, but two deaths temper the enthusiasm
Two patients nevertheless died during the test. According to Vertex, their deaths would not be linked to treatment. Therapy would have been “generally well tolerated”.
But some experts question this assertion.
“”One of the patients died of cryptococcal meningitis, which raises questions knowing that immunosuppressive treatment greatly decreases the patient’s immune system“, Underlines Professor Boris Hansel, endocrinologist at Bichat Hospital, for True Medical.
According to him, the efficiency is real.
“”This study shows that this is a technique that works. However, it remains reserved, for the moment, for diabetics most seriously affected by their pathology. We cannot say that this is the treatment of type 1 diabetes that we will apply to all patients“, He tempers.
Heavy treatment, to be reserved for the most serious cases
Why not consider it for all patients?
“”Simply because taking an immunosuppressive treatment remains heavy for a patient, and more restrictive than an insulin pump and a sensor, for patients whose diabetes is well balanced“Explains Professor Hansel.
As often, the decision is based on a balance between profits and risks. This new therapy will therefore only be addressed to the most fragile and unstable patients.
Before a possible generalization, the laboratory must submit authorization requests to regulatory agencies. A process that should not lead before 2026.