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Expert opinion

Teplizumab is just the beginning

We hear from Dr Rachel Besser, Consultant and Research Lead at Oxfordshire Children’s Diabetes Service and researcher at the University of Oxford, on how teplizumab being approved in the US benefits children in the UK.

Dr Rachel Besser

Dr Besser is leading a study investigating how to introduce screening for type 1 diabetes in children into the NHS.

Teplizumab is a massive leap forward

I was diagnosed with type 1 diabetes when I was nine. That’s when I decided I was going to be a paediatrician and look after children with diabetes. It is very exciting that we now have teplizumab, a disease-modifying drug with the potential to push diabetes into adulthood and give children back their childhood. No parent wants their child to have type 1, so anything that delays diabetes onset is a good thing.


Better outcomes with age

One benefit to delaying the onset of type 1 is that it allows time for your pancreas to grow and develop more. The bigger your pancreas is, the more insulin-producing cells you have, so the longer it takes for the immune system to destroy them. This means that people diagnosed at an older age can sometimes keep making small amounts of insulin. Any amount of insulin that the body can still make can help with type 1 diabetes management by providing a buffer against severe hypoglycemia and long-term complications of diabetes.

We need screening for type 1

If the MHRA, who license new drugs in the UK, approves teplizumab, we need to know who to give it to. We don’t currently have a process of identifying who would benefit from teplizumab. That’s where screening comes in.

We are running a feasibility study called T1 Early to find out if we can screen children for type 1 diabetes when they have their pre-school vaccinations at age three or four. We are testing children’s blood for immune markers which are a sign that the pancreas’ insulin-producing cells are being attacked. A child with two or more forms of these immune markers is very likely to develop type 1 in childhood, and certainly in their lifetime.

Teplizumab is a benefit for screening

Screening children is a balance of risk and harm. There can be a long period between being identified as high risk of type 1 and needing insulin. While we can educate people about type 1 before their diagnosis, we are wary of the anxiety and medical monitoring it may bring. But now there’s a treatment that we can use to delay type 1, it reduces that anxiety and offers an extra benefit to screening.

The NHS needs to be ready

But it doesn’t end with screening. The NHS, rather than a research programme, will need to deliver teplizumab if it is licensed. So, we need to be ready with the infrastructure in place for when we can identify a child is at high risk and know how to follow them up and support them. Early identification of type 1 will also have the benefit of stopping children becoming seriously unwell or dying due to a late or missed diagnosis. In paediatric diabetes we are currently only funded to look after children who take insulin. There’s nothing in place for children who don’t yet have type 1. We need to teach healthcare professionals this new specialism of pre-clinical type 1 diabetes.

Integrating screening into the NHS

I have a grant from the NIHR (the research arm of the NHS) and Novo Nordisk Research Foundation to determine how we integrate screening and follow-up into clinical care. I’m looking at its cost effectiveness as well as willingness in the general population to screen. I am setting up an NHS healthcare professional group to advise how to deliver screening and teplizumab. We are also interviewing parents in the T1 Early screening programme and getting feedback from people who turned it down, to better understand their experience. This project has already started, and we expect our first results in December.

I’m happy to be part of the story

People with diabetes have been waiting a long time for a therapy to delay type 1. The approval of teplizumab is the beginning of a journey. It paves the way for multiple drugs to emerge that can help delay and ultimately prevent type 1. We’ve had a hundred years of insulin. It’s time for a hundred years of immune therapies.

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