Type 1 diabetes is the great scientific challenge and unfortunately, it begins to be the great forgotten, crushed by the enormous epidemiological weight of those suffering from the other face of this chronic disease.
In fact, the only effective therapy to date against type 1 was insulin.In this version, there is a genetic component in its development, which implies first -degree relatives, which makes certain people 'high risk'.
For them there is hope.This is Teplizumab, an immunotherapy capable of delaying the beginning of the disease in this type of patients.
As reported, in the United States (USA) it is already available and it is expected to reach Europe in 2024 according to the Clarivate consultant.This monoclonal antibody is directed to CD3, a molecule present in the T lymphocytes involved in the development of the disease, and slows the destruction of beta cells of insulin producers.
Clinical trials with this drug focused on participants who already experienced a decrease in C peptide levels, which means that insulin production and beta cell function were already affected.
How does it work?
Immunotherapy is a treatment that uses certain parts of a person's immune system to combat diseases, in recent years with more cancer applications but is already showing hopeful results in a broader fan of conditions.In this case, Teplizumab, an anti-CD3 monoclonal antibody, is the first approved therapy that has shown to delay the evolution of type 1 diabetes.
Autoantibodies are substances segregated by lymphocytes, previously manufactured by the immune system, which attack the tissue itself and are usually associated with autoimmune reactions.Autoantibodies related to type 1 diabetes may appear in blood analysis months or years before the appearance of physical symptoms that usually lead to diagnosis.
Type 1 diabetes develops when the T -system T cells erroneously destroy the body's insulin producers of the body.Insulin is necessary to convert glucose into energy.Teplizumab is directed to T cells to reduce the destruction of beta cells.
Thus, the treatment, which acts directly on the immune system, can deactivate the immune cells that attack the beta cells of insulin producers, while increasing the proportion of cells that help moderate the immune response, essentially stabilizing the function ofbeta cells
Although immunotherapy does not avoid the development of diabetes, it approaches a result that opens horizons so far not explored."It is a milestone in the research and treatment of this disease," in the words of Eduard Montanya, a scientific director of the Biomedical Research Center in the Network of Diabetes and Medical Diseases Associated (CIBERDEM) and head of the Diabetes Section of the Diabetes of the Diabetes of the Diabetes of theBellvitge Hospital."Although its initial impact can be limited, for the first time there is a treatment capable of modifying the course of type 1 diabetes."
70% success in the delay of the appearance
The American approval of this therapy is based on a phase II trial published in The New England Journal of Medicine in 76 participants from 8 to 49 years considered high risk of developing type 1 diabetes;All of them with a first degree relative with the disease, with at least two autoantibodies related to diabetes (proteins produced by the immune system) and abnormal glucose tolerance.
The participants were randomly assigned to the treatment group, which received a 14 -day teplizumab cycle, or the group ofControl, which received a placebo.During the essay, 72% of people in the control group developed clinical diabetes, compared to only 43% of the Teplizumab group.
The median time elapsed from the beginning of the study to the appearance of stadium 3 of type 1 diabetes was 50 months for the group that received Teplizumab, compared to 25 months of the group that did not receive the therapy.The appearance of the disease two years later occurred in 70% of the participants.
'' Delaying between one and three years the debut of the disease "is very important in pre -adolescents," says researcher Iria Gómez Touriño to the same mentioned medium, from the Molecular Medicine Research Center and Chronic Diseases (CIMUS) of the University ofSantiago de Compostela."It has been shown that this extra time for the pancreas to grow up to adult size leads to a more benign diagnosis and reduce the progression of long -term disease."
Many factors, including age, could have contributed to Teplizumab's ability to delay clinical disease, since it is known that children and adolescents at risk progress to type 1 diabetes faster than adults.The fastest progression of type 1 diabetes is associated with a highly active immune system, which can explain the impact of the drug -modulating drugs such as Teplizumab.
It also indicates that the drug reduces the risk of diabetic ketoacidosis and long -term complications in people at risk of type 1 diabetes. This is achieved thanks to the active control of the levels of autoantibodies and other markers of this disease.But, above all, the results have important implications for people, especially young people who have relatives with the disease, since they can have a high risk and benefit from early detection and treatment.
inconveniences
On the one hand, the most feared adverse effect is lymphopenia, associated with the response to systemic immunotherapy, and that causes an increase in infections and reaguration of latent infectious processes.For example, hypersensitivity reactions or systemic pictures due to cytokine release syndrome.This picture is presented by a large and fast release in the blood of immune cell cytokines affected by immunotherapy.
Likewise, Teplizumab belongs to a new kind of biological medicines, whose operation in the body is more complex than that of traditional medications of small molecules.Therefore, its use is still experimental and it takes better and broader results over time.
For example, up to 90% of new cases of type 1 diabetes are given in people who do not have affections, so it would also be necessary to identify these people so that the treatment was extensible to much of the population that develops this typeof diabetes.Only 10% could benefit from this therapy.
In addition, another inconvenience of this therapy is that for its administration it requires an intravenous daily infusion for 14 consecutive days.In the trial the infusion was administered for 30 minutes and then the patient stayed two hours of rest to check his response.A prolonged time that will also have to polish so that it does not suppose an impediment in your administration.
Together with monoclonal antibodies, other immunotherapy approaches include cellular therapies, nanoparticles, cytokines and combined therapies.All of them must be tested in the long term to verify their side effects and transfer them to people who have already developed the disease.