A patient with diabetes has received cells that produce “invisible” insulin to the immune system: therefore, the use of immunosuppressors do not require.
A 42 -year -old man with type 1 diabetes is the first person in the world to have received a transplant of pancreatic cells for the production of genetically modified insulin so as to be unassailable from the immune system. Even if the cells received do not secrete, alone, insulin sufficient to “cure” the disease completely, the patient will not need to take immunosuppressive drugs for the rest of life, as must be done today who has undergone transplants of this type.
The case described on New England Journal of Medicine It represents a big step forward in the future treatment of type 1 diabetes and other autoimmune disorders, because immunosuppressants, which remove the risk of rejection, however increase that of infections and cancer.
Cells to be replaced
Type 1 diabetes is an autoimmune disease that involves the destruction by the immune system, the beta cells of the pancreas, in charge of producing insulin (the hormone that allows cells to take the sugar present in the blood and use it as a source of energy).
Pancreatic islands transplant is a procedure that allows you to provide the patient with new functioning beta cells with: however, these are quickly attacked by the immune system of an organism with type 1 diabetes. This kind of interventions therefore always accompanies the need to depend on that moment on on an immunosuppressive therapy.
Great hopes also derive from the infusion of stem cells reprogrammed to transform into pancreas cells and produce insulin, whose first experiments, on a dozen patients with type 1 diabetes, have given good results. However, these therapies are also necessarily accompanied by treatments with immunosuppressive drugs to prevent the immune system from destroying the new cells. The goal to which we tend to be a little around the world is therefore to study forms of transplantation for patients with diabetes who do not require immunosuppressive therapy.
Two invisibility cloaks
The scientists led by Per-Ale Carlsson of the University of Uppsala in Sweden injected 80 million pancreatic cells of a deceased donor modified with the genetic editing Crispr in the forearm of the patient to have two “superpowers”: they were difficult to recognize by the immune system thanks to the deactivation of two genes that control the expression of certain proteins; And in some cases they showed higher levels of a gene that discourages the attacks of t killer lymphocytes and macrophages, two types of immune cells.
Three months after the infusion, the transplanted cells were still alive and active in the production of insulin, and the blood tests did not detect any measurable immune activation or production of antibodies in response to the new arrivals.
Before transplantation, man no longer produced insulin naturally and had to integrate it with injections several times a day. After the transplant, the hormone concentration began to increase slightly after meals: the new beta cells were producing it in response to the increase in glucose.
The next steps
However, transplanted cells are sufficient to provide only 7% of the insulin levels necessary for a healthy metabolism, and the patient will have to continue with daily injections. It will however be monitored for a year and will receive new infusions, if the procedure continues to be considered safe. And only reached all the answers on safety can you think of extending the treatment to other patients with type 1 diabetes.
Meanwhile, transplants of stem cells are able to generate a greater number of pancreatic cells responsible for the production of the hormone. Even if it is too early to consider this approach a cure for diabetes, the premises are very promising.
