The US Food and Drugs Administration (FDA) has approved the world’s first ever immunotherapy for type 1 diabetes, teplizumab, also called Tzield. This is the biggest treatment breakthrough for the condition since the discovery of insulin 100 years ago.
In a clinical trial, teplizumab delayed type 1 diabetes by an average of three years in people who were at high risk of developing the condition. It can now be prescribed in the United States outside of a research setting to people who don't yet have type 1 diabetes but are at high risk of developing type 1 diabetes in the future.
After weighing up the evidence, the FDA decided teplizumab was safe and effective at holding off the development of type 1 diabetes and delaying a diagnosis. It's been approved for use in people aged eight and older.
And this matters – each day without type 1 diabetes counts. A delay in diagnosis means people spend more years free from worry about injections, hypos, carb counting and the relentlessness of living with type 1 diabetes. It means people spend more years with their blood sugars in a healthy range, which could protect them 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, perhaps avoiding childhood diabetes altogether.
Teplizumab is under review in the UK but it hasn’t yet been approved yet. However, the U.S. approval paves the way for this life-changing treatment to be made available to people at high risk of type 1 diabetes in the UK too.
Chris Askew OBE, Chief Executive at Diabetes UK, said:
“Today’s landmark approval of teplizumab in the U.S. is the start of a seismic shift in how type 1 diabetes is treated. For 100 years, people living with type 1 diabetes have relied on insulin to treat the condition, and today’s decision means that for the first time, the root cause of the condition – an immune system attack – can be tackled, and type 1 diabetes potentially delayed for up to three years.
“The licensing of teplizumab in the UK must now be accelerated, and we’re working with the NHS, other diabetes charities and key stakeholders to ensure that people in the UK can benefit from this life-changing treatment as soon as possible. Preventative treatments for type 1 diabetes can only be effective when combined with screening programmes to identify those at risk, and the launch of the ELSA screening trial this week is perfectly timed to help unleash the benefits of teplizumab here in the UK.
“Diabetes UK is funding immunotherapy research to help people at all stages of type 1 diabetes, and we hope this monumental breakthrough will open the door for increased research investment, to develop further effective immunotherapies to treat the condition. Today is a significant moment in the history of type 1 diabetes, and one that will shape the future – propelling us closer to the day where type 1 diabetes can be prevented or cured altogether.”
Professor Colin Dayan, co-lead of the Type 1 Diabetes Immunotherapy Consortium, said:
“This is a very important announcement ushering in a new era for type 1 diabetes. For the first time in 100 years new treatments can move away from focussing on improvements in insulin therapy and glucose control, to prolonging the period when insulin is not needed. And the possibility of making insulin treatment for type 1 diabetes in children a thing of the past, is in sight.”
What about the UK?
Teplizumab has only been approved by the FDA so far, who decide what should happen in the U.S. For a new drug to be licensed in the UK, the UK's Medicines and Healthcare products Regulatory Agency (MHRA) would need to approve it as safe and effective.
The company who own teplizumab has submitted an application to the MHRA too. The MHRA has awarded teplizumab an ‘Innovation Passport’ under the ‘Innovative Licensing and Access Pathway’. This pathway is designed to help speed up access to promising new medicines in the UK. Teplizumab was one of the first drugs to be given this special status. It means the MHRA will accelerate its assessment of teplizumab and that other important organisations, like the NHS and the National Institute for Clinical Excellence (NICE), will be involved in the process from the start.
The MHRA’s assessment is at an earlier stage and we’ll update you when we learn more about possible timelines. But the FDA’s approval is a hugely encouraging step. And we’ll continue to work with key players to help make sure teplizumab becomes available in the NHS too.
We’re also funding research into other immunotherapy treatments and the Type 1 Diabetes Grand Challenge, in partnership with JDRF and the Steve Morgan Foundation. The partnership is investing £50 million into the most promising type 1 diabetes treatments to speed up breakthroughs and lead the race towards a cure for the condition.
We hope this work, alongside the enormous milestone of teplizumab becoming the world’s first licensed immunotherapy, will open the floodgates to more investment, more treatments, and – most importantly – more people living with or at risk of type 1 diabetes benefitting from immunotherapies at different stages of the condition.
What is teplizumab?
In type 1 diabetes, the body’s immune system attacks the part of the pancreas that produces insulin, so you can’t produce insulin anymore. Immunotherapies are new treatments that reprogramme the immune system to disrupt its attack on the pancreas, dealing with the root cause of type 1 diabetes for the first time.
Teplizumab is a type of immunotherapy drug called a monoclonal antibody. Antibodies are proteins made by our immune system. They’re the ‘search’ part of the immune system's search-and-destroy task force. They find harmful invaders, like bacteria or viruses, and mark them for destruction.
Scientists can also made antibodies in the lab to create treatments that introduce new antibodies to the immune system. With teplizumab, scientists made an antibody that’s programmed to hunt down and weaken the killer immune cells responsible for destroying insulin-making beta cells in type 1 diabetes. This helps to protect some beta cells from the immune attack, and people can keep on making enough of their own insulin for longer.
Scientists first tested teplizumab in a landmark trial, funded by JDRF and the National Institutes of Health. The trial involved 76 people aged between eight and 49 years who were at high risk of type 1 diabetes. Those given teplizumab were less likely to be diagnosed with type 1 diabetes than a comparison group who were given a dummy (placebo) drug. Those who did develop type 1 diabetes during the study were, on average, diagnosed three years later than people who had received the placebo. This was the first clinical trial to show that it’s possible to intervene and hold off the development of type 1 diabetes in people at high risk.
Finding people at risk of type 1 diabetes
Scientists have previously discovered that signs of the immune system’s attack diabetes can be detected long before someone develops symptoms of type 1 and receives a diagnosis. With a simple blood test, it’s possible to spot early signs that the immune system is planning an attack and identify those who have a high risk of developing the condition in the coming months, years or decades. Knowing who is at risk opens up a window to use immunotherapy to disrupt the immune attack before it progresses too far.
Excitingly, earlier this week we announced the launch of a UK-first trial we’re funding with JDRF. The ELSA (EarLy Surveillance for Autoimmune diabetes) study is screening 20,000 children, aged three to 13 years, to test them for their risk of developing type 1 diabetes in the future. You can find out how your child could take part and sign up today.
ELSA will answer important questions about how a type 1 diabetes screening programme for children in the UK would best work, and lay the groundwork for routine, widespread screening. A screening programme will be a critical to harness the benefits of teplizumab, and other immunotherapies, by giving us a system to identify those at high risk of type 1 who could benefit from preventative treatments.
Could teplizumab help people already living with type 1 diabetes?
Based on the research conducted so far, the FDA has approved teplizumab for use in the U.S. in people who have a high risk of getting type 1 diabetes. But teplizumab and other immunotherapies are also being tested right now in people who are newly diagnosed with type 1 diabetes to see if they can help to protect their remaining beta cells. The more beta cells we can protect, the more insulin you can produce on your own.
In the future, immunotherapies might form part of a cure for people who have been living with type 1 diabetes for a longer time. Our 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 replacement therapy to protect new cells from another immune system attack.
How you can get involved
This breakthrough couldn’t have happened without the people who took part in trials. You could play an important role in advancing immunotherapy research.
- If you’re a parent of a child aged three to 13 years, you can sign up to ELSA and get them screened to find out your child’s risk of developing type 1 diabetes in the future. Or if you’re aged 45 years and under and have a close relative with type 1 diabetes, you could get screened through INNODIA.
- If you’re newly diagnosed with type 1 diabetes, you could be eligible to take part in an immunotherapy trial within 100 days of your diagnosis. Sign up to the type 1 diabetes immunotherapy consortium or ADDRESS-2 to find out about opportunities.
- If you live with type 1 diabetes, find out more about opportunities to take part in research.
