A small group of patients with a serious form of type 1 diabetes has no longer needed insulin since it has received reprogrammed cells to produce it.
An experimental treatment based on A single infusion of stem cells He may have edited 10 out of 12 patients enrolled in a phase clinical study 1-2 from a serious form of diabetes. A year later, these 10 people they no longer have to take insulinwhile the other 2 must take it, but with lower dosages. The results of the experimentation, announced on Friday 20 June at the Annual Meeting of the American Diabetes Association, were published on New England Journal of Medicine.
The islands of insulin
Insulin, the hormone that allows cells to take the sugar present in the blood and use it as a source of energy, is produced by Langerhans islands or pancreatic islandssmall agglomerations of cells scattered in the pancreas, “as islands in the sea” (hence their name, combined with those of the German pathologist who discovered them in the second half of the nineteenth century). In particular Insulin is secreted by beta cellswhich constitute about 2% of the total mass of the pancreas.
Stem cells in the new treatment, called zimislecel and produced by Boston’s Vertex Pharmaceuticals, they were scheduled for become pancreatic islands and settle not in the pancreas, but in the liver of patients (where cells are usually infused in traditional pancreatic islands transplants). Once on site, they started producing insulin as scheduled. The study still on a very reduced number of people was aimed only at test the safety of therapy.
Hypoglycaemia without warnings
The treatment was designed for patients with type 1 diabetesone autoimmune disease In which out of control and evil antibodies addressed kill the pancreatic beta cells that should produce insulin. The production of insulin is reduced to zero, the body cannot use glucose as a source of energy and sugars accumulate in the blood (hyperglycaemia). Failure to balance between insulin taken with therapy in diabetic people, food consumption and physical activity can also cause hypoglycemic crisisthat is, pathological reductions in blood sugar levels.
The experimentation of Zimislecel turned to a small subgroup of patients with “unconscious hypoglycaemia“, a condition in which There are no symptoms which typically precede hypoglycaemia (blurred vision, hunger, fatigue, weakness, difficulty in reasoning, sweating). The people who suffer from it, about 30% of the total of those who are suffering from type 1 diabetes, do not notice the abrupt reduction of sugars in the blood e They face sudden fainting, convulsions and severe hypoglycaemiapotentially lethal events.
The results of the infusion
Most of the patients who received the infusion needed less and less insulin in the months following therapy, until it reaches the Total suspension after about six months. In the first 90 days the episodes of hypoglycaemia, the most disabling for the quality of life of patients, disappeared. If the continuation of the trials will continue to give convincing results, the pharmaceutical company will ask for the approval of the FDA stem infusions. At the moment the costs of the treatment are not known.
Collateral risks
The therapy was necessarily accompanied by treatments with Immunosuppressor drugs to prevent the immune system from destroying the new cells. These medicines could increase the risk of infections and long -term infections, and it is possible that patients must continue to take them for life. For the moment, they are considered less dangerous than the complications of diabetes, but Studies in the longest period will be needed to be able to establish it with certainty.
