Type 1 diabetes mellitus (T1DM) is an autoimmune condition characterized by the irreversible destruction of the β cells of the pancreas, which leads to a lifelong dependency on insulin.
Although several treatment strategies have been attempted to target cellular, humoral and innate immunity, very few have had any significant, long lasting, meaningful impact.
Till date the only treatment available to manage Type 1 diabetes is Insulin given as multiple daily doses or through continuous insulin infusion pump.
In November 2022, Teplizumab became the first immunomodulatory agent to be licensed by the US Food and Drug Administration for delaying the onset of T1DM.
Q1) What is Teplizumab ?
A1) Teplizumab, a humanized monoclonal antibody to CD3 on T cells, is approved by the Food and Drug Administration to delay the onset of clinical type 1 diabetes by its action on the insulin producing beta cells of the pancreas.
Q2) What does Teplizumab do?
A2) In the trials conducted, a single course of a 14-day infusion on the progression from stage 2 to stage 3 type 1 diabetes. The teplizumab infusion resulted in a delay of stage 3 type 1 diabetes onset by a median of 24 months.
Q3) Who can it be used in ?
A3) The FDA approval only focused on stage 2 of type 1 diabetes, which requires the identification of individuals with multiple diabetes autoantibodies and dysglycaemia, in high-risk adults upto 45 yrs and children over 8 years
Which means that if the child is diagnosed before the full signs and symptoms have come, then this drug may be tried to prolong the onset of the disease from stage 2 to stage 3.
Stage 1 : Multiple islet autoantibodies, normal blood glucose, pre-symptomatic.
Stage 2 : Multiple islet autoantibodies, abnormal glucose tolerance, usually presymptomatic,
Stage 3 : Blood glucose higher diagnostic thresholds.
Stage 4 : Established T1D (long standing cases)
Q4) Who are the patients that Teplizumab is being currently used in ?
A4) Teplizumab is still being used mostly for research purpose only in high-risk adults upto 45 yrs and children over 8 years. The children need to screened from an early age group to detect antibodies to be able to eligible for use of the drug.
This also means that these tests may need to be repeated several times.
There are challenges regarding identifying the ideal age to start the screening for Type 1 diabetes in children at this point.
Q5) How is the treatment given ?
Q5) In the trial Two 12-day courses of intravenous teplizumab were given to children and adolescents with newly diagnosed type 1 diabetes. In the results it was seen that at the end of 78weeks of treatment, the group treated with teplizumab had higher values of stimulated C-peptide.
Q6) What is the cost of treatment ?
A6) The pricing information on teplizumab was released, at an estimated cost of more than US$193 000 for a single course.
Q7) What are its side effects ?
A7) Adverse events seen in the trial with administration of teplizumab headache, gastrointestinal symptoms, rash, lymphopenia, and mild cytokine release syndrome.
Whether treatment with intravenous teplizumab in patients with newly diagnosed type 1 diabetes can delay the disease occurrence of T1DM, but if it can prevent disease progression is unknown. However it is a step forward in the research in the field of prevention of T1DM diabetes.
