Teplizumab Therapy to Delay the Onset of Type 1 Diabetes.

Type 1 diabetes mellitus (T1DM) is an autoimmune disease that results in the destruction of insulin-producing pancreatic beta cells. The incidence and prevalence of T1DM are increasing, making this one of the most common diseases of childhood. The disease is associated with significant morbidity and mortality with patients experiencing reduced quality of life and decreased life expectancy compared with the general population. Patients become dependent on exogenous insulin which has been the primary treatment since its first clinical use over 100 years ago. Although there have been advancements in glucose monitoring technology and insulin delivery devices, most patients fail to meet glycemic targets. Research has therefore focused on different treatment options to delay or prevent disease progression. Monoclonal antibodies have previously been utilized to suppress the immune response following an organ transplant and were subsequently studied for their ability to treat autoimmune diseases. Teplizumab, a monoclonal antibody (manufactured by Provention Bio and marketed as Tzield), was recently approved by the Food and Drug Administration as the first preventative treatment for T1DM. The approval came after a 3-decade history of research and development. This article provides an overview of the discovery and mechanism of action of teplizumab, as well as the clinical trials that led to its approval.