Adapted from Medscape Nov 20 2022 by Miriam E Tucker a freelance journalist from Washington DC.
At last humans have caught up with mice!
Since my son was diagnosed with type one diabetes, some 28 years ago, it has been possible to reverse type one diabetes in mice. At last the huge effort to find a suitable agent to use in humans, and particularly children, has been approved by the FDA.
Thank you everyone who has contributed to this marvellous discovery. The Juvenile Diabetes Research Foundation was set up with this end point in mind and they have been successful in the development of the new drug which they helped fund. The thing is that now such an agent is available, we need to be able to find the people who would most benefit from taking the drug. Thus screening for early type one diabetes is going to become crucial.
The new drug is called Teplizumab-mzwv (Tzield, Provention Bio) and it is a anti-CD 3 monoclonal antibody. It was approved in Nov 17 21 and is the first human disease modifying therapy for impeding the prevention of type one diabetes. It delayed onset by around 2 years and longer in some subjects.
It is given by an intravenous infusion once daily for 14 days and costs around $200,000 dollars for the course of treatment.
It is licensed for children over the age of 8 years. The group it is targeting is those who are asymptomatic but who have raised blood sugar levels and at least two type one diabetes antibodies. Most of those screened are first degree relatives of type ones. The JDRF is offering a screening blood test for $55. But because 85-90% of people who do develop type one diabetes don’t have a first degree relative, screening will need to be developed further.
In Bavaria, Germany, screening of all schoolchildren for type one diabetes has been done, and the organisers said that a major benefit, was that education about the signs of diabetes occurred so that diagnosis occurred before Diabetic Ketoacidosis developed. This is known to cause deaths and wears out the pancreas much faster.
In another study 2 year olds and 6 year olds in the USA and western Europe were screened for islet autoantibodies and this detected almost all of those children who developed type one diabetes by mid adolescence.
Using a genetic risk score at birth has been suggested as more cost effective by Dr William Hagopian of the Pacific Northwest Research Institute in Seattle. 10% of newborns have HLA genes that can identify 80% of those who will get childhood type one diabetes.
My comment: I’m particularly pleased to see that the JDRF has been successful because if my son has offspring they have a one in three chance of developing type one diabetes in childhood. As time goes on, it is to be hoped that the interventions will become cheaper and more effective.