Behind the headlines: Delaying Type 1 diabetes

You may have seen the headlines today about a Type 1 diabetes research breakthrough. It’s a really exciting moment – scientists have shown that a new drug can delay a diagnosis of Type 1 diabetes in some people. But you probably have some questions, so we’ve delved a little deeper into the study for you.

What were the results and why were they such a big deal? 

In the trial, people at high risk of Type 1 diabetes (more on this later) were given an immunotherapy drug, called Teplizumab, or a placebo (dummy) drug. The researchers wanted to know if Teplizumab could slow down the development of Type 1 diabetes.

They found that 72% of people in the placebo group were diagnosed with Type 1 diabetes during the study, compared to 43% of participants who were treated with the drug. The median time for people in the placebo group to develop clinical Type 1 diabetes was just over 24 months, while the median time in those taking Teplizumab was 48 months.

(The median is a way of calculating the average. You line up all of your results – in this case, the time it took for each participant to develop Type 1 diabetes – and take the number that falls in the middle of that list.)

These results are an incredible step forward. For the first time we’ve seen that it’s possible to intervene in the progression of Type 1 diabetes in people at risk. And this matters – each day without Type 1 diabetes counts. A two-year delay in diagnosis could benefit blood glucose control, providing protection against long-term complications. And it means someone could get a diagnosis when they’re older, and might be better equipped to manage their condition.

Whilst this is all really promising, we should bear in mind that the number of people involved in the study was relatively small (76 people). And the researchers did see short-term side-effects in some people who took the drug: a rash and low white blood cell counts, which can increase your risk of infections.

And is this the first time we’re seeing results like this?

It’s the first time we’ve seen a median delay of two years. The drug has already been tested in a previous trial, with people recently diagnosed with Type 1. The researchers found that it could protect insulin-producing beta cells and help participants to continue making some of their own insulin for longer. So the TrialNet scientists decided to move earlier in development of Type 1, to those at high risk.

How did they find people at high risk of Type 1? What does that mean?

The trial participants were found as part of TrialNet’s Pathway to Prevention. This screens relatives of people with Type 1 to find people in the early stages of the condition, before a diagnosis.

The TrialNet team worked out that the immune attack behind Type 1 diabetes begins long before someone is diagnosed, and has three different stages (highlighted in the picture below). TrialNet’s latest study involved people who were in stage 2. Scientists wanted to delay them reaching stage 3: a diagnosis.

Stages of Type 1
Image courtesy of TrialNet

Stage 1 is considered to be the start of Type 1 diabetes. At this point, you have normal blood glucose levels but have autoantibodies in your blood, which tell us that the immune system has started attacking the insulin-producing beta cells in your pancreas. Autoantibodies can appear years – sometimes decades – before a diagnosis. At stage 1, people test positive for at least two autoantibodies (there are five different autoantibodies that we can test for at the moment).

In stage 2, your blood glucose levels become higher than normal, as more beta cells are being destroyed. But you don’t yet have any symptoms and you don’t need to take insulin. At this stage, your likelihood of developing Type 1 diabetes at some point in the future is almost 100%.

Lastly, at stage 3 the immune system has killed off so many beta cells that you’re no longer able to produce enough of your own insulin. Blood glucose levels become dangerously high and the symptoms of Type 1 diabetes appear. This is known as the clinical onset of Type 1 diabetes and is usually the point you’d get diagnosed.

We can also spot people who are at risk of Type 1 even earlier by looking at your genes. For example, we’re funding research to develop a ‘risk calculator’ that uses information from genes linked to Type 1 diabetes to help find babies who are at higher risk of developing the condition in the future.

What’s Teplizumab and how does it work?

Teplizumab is a type of immunotherapy drug called a monoclonal antibody. A company called Provention Bio makes it. It works by interfering with the immune system’s destruction of beta cells.

‘Monoclonal’ means that they are all copies of one type of antibody, and they are made in the laboratory. Antibodies recognise and bind to specific molecules in the body. In the case of Teplizumab, it recognises a molecule found on T cells: a type of immune cell involved in the immune attack.

In the earlier studies of Teplizumab involving people recently diagnosed with Type 1 diabetes, scientists found the drug could exhaust the T cells, so that they became less effective at attacking the insulin-producing cells in the pancreas. This meant more beta cells could survive for longer.

You can find out more about T cells, and the other immune cells involved in Type 1 diabetes, on our immunotherapy spotlight page.

This all sounds amazing – when will it be made available?

We don’t know yet. For any new treatment to be licensed, it needs to be approved by regulatory authorities who weigh up the evidence and decide if a drug is safe and effective.

Although the results so far are really encouraging, we still need more research to fully understand the effects of Teplizumab. The TrialNet team plan to follow up the participants who were treated with the drug, to learn more about its impact on the progression of Type 1. They’re also hoping to conduct additional studies to look for ways to extend the possible benefits of Teplizumab.

If we get any additional information on the licencing of Teplizumab, we’ll make sure to update you.

I’ve already got Type 1 diabetes – is there any way these results could help me?

The researchers will be following up those who developed Type 1 diabetes during the trial, to find out if the Teplizumab could still provide some benefits. For example, could it make blood glucose levels easier to manage?

But importantly, now that we know that we can intervene in the immune attack, it opens up new avenues of investigation and provides hope for other immunotherapy research.

For people who already have Type 1, immunotherapies could form part of a cure in the future. Scientists are looking for ways to replace or regenerate the beta cells that have already been destroyed. Once we can do this, immunotherapies could potentially be used alongside the beta cell therapy to protect new cells from another immune system attack.

Did Diabetes UK fund this work?

No, the National Institutes of Health (NIH) and NIH’s Institute of Diabetes and Digestive and Kidney Diseases did, with additional support from our friends at JDRF.

We’re funding over £6 million of immunotherapy research like this, including the Type 1 diabetes Immunotherapy Consortium. We just announced that we’re investing more in this network of scientists and research centres. They’re bringing scientists together to run faster, more efficient clinical trials than ever before, with the aim of finally reaching a licensed immunotherapy for people with Type 1 diabetes.

This all sounds pretty exciting. How can I get involved?

This breakthrough couldn’t have happened without the people who took part in the trial. Recruitment for this trial has closed, but there are other opportunities to get involved in research.

Research also can’t happen without funding. It’s only with your help that we can push ahead. Make a donation, adopt one of our research projects or fundraise for us and help us reach the next breakthrough.

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