Thanks to an experimental Teplizubam treatment, 21-year-old Mikayla Olsten has not developed type 1 diabetes (T1D), a disease to which she is predisposed and has delayed its onset.
The drug has also shown a “road map” for the development of drugs for other autoimmune diseases, according to an article in the journal Nature.
The operation of Teplizubam consists of an autoimmune type therapy that blocks T cells, the “attack dogs” of the immune system, causing them to stop destroying the cells that produce insulin.
Mikayla received the two-week treatment in July 2016 as part of a clinical study conducted between 2011 and 2018 to determine the efficacy of the therapy.
The study found that those who received the drug took an average of five years to develop the disease, three years longer than those who received the placebo.
So far, Mikayla has been 6 and a half years without type 1 diabetes, others have been more than a decade. In November, the drug was approved by the US health authorities to be administered to patients 8 years of age and older who meet the established criteria.
It is the first medicine that has been proven to delay the onset of an autoimmune disease.
The challenge, however, is identifying the people who might benefit from these therapies.
“Optimal screening strategies have yet to be established and the presymptomatic signs of diseases are not yet well defined for each condition,” Nature abounds.
Also, the publication noted, because Teplizumab offers only a temporary delay of T1D, researchers need to find better approaches.
Its predecessor was OKT3, which, although it had beneficial effects for the prevention of organ rejection in liver transplants, also triggered a life-threatening complication at high doses, cytokine release syndrome.
Another challenge is the cost, which is close to $194,000.
Despite the difficulties, according to autoimmune disease specialists, the drug’s pathway holds promise for application to other conditions, especially in the area of disease prevention and delay.
Paul Emery, a rheumatologist at the University of Leeks in the United Kingdom, explained that type 1 diabetes is the model for the prevention of autoimmunity.
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