The Food and Drug Administration, the federal agency that supervises the safety of medicines in the USA, has approved the first drug capable of postponing the onset of symptoms of the type 1 diabetesthe “juvenile” form of the disease.
The teplizumabwhich should be available in the US later this year, doesn’t cure diabetes but slows its progression, giving patients destined to develop it some extra freedom before routine glucose monitoring and insulin management becomes a necessity.
An autoimmune form. Type 1 diabetes affects about 10% of patients with diabetes e usually arises in children, adolescents and young adults under the age of 40. It initially manifests itself with a dramatic weight loss accompanied by fatigue, an unquenchable thirst and a constant need to urinate, and results from an autoimmune attack on the pancreas, the organ responsible for the production of insulin.
The “gasoline” of the cells of the human body is glucose and the hormone insulin it is the key to getting glucose into cells. In patients with type 1 diabetes, the immune system mistakenly targets the pancreatic beta cells responsible for secreting it. The pancreas then stops producing insulin and the body is no longer able to regulate blood sugar.
How does it work. Teplizumab (trade name Tzield) is a monoclonal antibody, i.e. a modified antibody obtained in the laboratory, which binds to a molecule present on the surface of T cells, the white blood cells responsible for self-sabotaging beta cells in patients with type 1 diabetes. The drug typically targeted active the T cells: by hooking it, the drug avoids their autoimmune response against the pancreas for a while.
How long does the effect last? Even though the drug has been under investigation for decades, the FDA’s approval came after that a study on 76 patients with type 1 diabetes it demonstrated its safety and efficacy. The volunteers were in the presymptomatic stage of the disease but showed some the alarm bellsi.e. the presence of autoantibodies and abnormal glucose metabolism.
Half of the patients received the drug and half a placebo, and all were followed up for 51 months. In the first group, 44% of patients developed diabetes symptoms against 72% in the second group. In people treated with the drug who developed diabetes during this time, the onset of symptomatic disease occurred on average a couple of years later than in the others.
Not for all. Difficult to imagine for now a large-scale distribution. The treatment should be given by intravenous infusion, i.e. drip, once a day for 14 days. According to New York Times the drug, produced by Provention Bio, would cost 13,850 dollars per vial (about 13,500 euros) or 193,900 (about 189,000 euros) for a complete treatment: a figure that could be covered by insurance or health systems but still considerable.
Who is it aimed at? The main difficulty will be to recruit patients who can benefit from it, before they develop the symptoms of diabetes. Although disease onset is usually sudden and often occurs in adolescence, autoantibodies that indicate pancreatic attack are often present as early as 5 or 6 years of age.
However, early screening for diabetes is not yet widespread, and testing only those who have close family members with the same disease would still miss 85% of diagnoses. Type 1 diabetes patient organizations in the US would like to see juvenile diabetes screening as part of routine care in children.
What’s its purpose? One might wonder why intervene, if in any case the drug cannot prevent the development of diabetes: according to the authors of the study that led to the approval, which appeared in 2019 on the prestigious New England Journal of Medicinethe drug is used to gain time to prepare, to grow a little more and to know the disease, but also to get a few more years without the glycemic swings that can lead to even serious complications.
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