Hopeful new NHS treatment offers more time before type 1 diabetes symptoms begin
A landmark treatment that can postpone the start of type 1 diabetes symptoms is becoming available through the NHS in England and Wales, bringing welcome hope to children, young people and families.
Teplizumab, an immunotherapy drug, can delay the point at which people develop symptoms and need lifelong insulin treatment by around three years. The decision has been warmly welcomed by the NHS medicines body, charities and families affected by the condition.
Helen Knight, director of medicines evaluation at the National Institute for Health and Care Excellence, called the recommendation "genuinely exciting".
"For the first time, we have a treatment that can give people diagnosed at an early stage precious extra time before they need to manage the full demands of the condition."
Charities say the development marks a major step forward after many years of research and campaigning.
Karen Addington, chief executive of Breakthrough T1D, said: "If it were your child or someone you love, you would want to do everything possible to give them more years without the daily burden of managing this relentless condition."
"We now have a treatment that can help make that possible".
A welcome breakthrough for families
Type 1 diabetes happens when the immune system mistakenly attacks insulin-producing cells in the pancreas. It is different from type 2 diabetes, which is often, though not always, linked with being overweight.
People living with type 1 diabetes need to regularly check their blood glucose levels and take insulin through injections or a pump, because their bodies can no longer make enough of the hormone naturally.
The condition can appear at any age, although it is most often diagnosed in the early teenage years.
For families, the arrival of a treatment that can delay the full onset of the disease is being seen as a meaningful gift of time.
Theo Sebastian-Jenkins, now eight, was diagnosed with type 1 diabetes when he was four. His parents took him to A&E after noticing he was unusually tired, constantly thirsty and losing weight.
At the time, Theo was too young to receive teplizumab, which can be given from the age of eight. He is now doing well, but his family still has to manage his condition carefully every day.
His mother, Vicky, said: "It's something which you can never switch off from."
"When you make breakfast, you've got to weigh everything, counting how many carbohydrates are in it, so that you know the right amount of insulin to give."
If blood sugar levels fall too low, it can become a medical emergency. If they stay too high for too long, they can damage blood vessels and nerves.
Theo’s father, Ben, said a medicine that could postpone the start of symptoms would be life-changing for many families.
"It would be huge for any family to have those three years of childhood back without the worry and the things he's had to deal with," he said.
How the treatment works
Teplizumab is given before symptoms develop. Patients first need a blood test showing that their immune system has begun attacking the pancreas.
The treatment is delivered in hospital through an infusion, or drip into a vein, once a day for 14 consecutive days.
Routine NHS screening for type 1 diabetes is not yet available in the UK, although diabetes charities are calling for it. At present, people are most likely to learn they are at risk through blood tests carried out for other medical reasons, or because type 1 diabetes runs in their family.
However, most people diagnosed with the condition do not have a close family history, suggesting environmental factors may also help trigger it.
Italy is currently the only country with a national screening programme for children and young people.
“The biggest gift” is time
One of the first patients in the country to receive teplizumab was Dima Boichak, from Newbury in Berkshire.
Dima was nine when he discovered he was highly likely to develop type 1 diabetes after joining a UK-wide research study funded by diabetes charities. His family took part partly because Dima’s cousin had already been diagnosed and was using insulin.
His mother, Elena, said: “If we hadn't done it, Dima might have been diagnosed much later, possibly at a stage where he'd need urgent intervention, like my niece."
Before the drug was formally recommended for NHS use, Dima received it on compassionate grounds. The course involved going to hospital every day for two weeks.
Elena described the process as "hard work", but said it was worth it for the chance to give her son more time before insulin treatment becomes necessary.
"The biggest gift teplizumab gives us is time - just time being a normal kid, living a normal life," she said.
"That's wonderful for him, and for me, as his mother."
Who could benefit
The published price of teplizumab is about £150,000 per treatment course, although the NHS has agreed a confidential discount with Sanofi, the drugmaker.
NICE, which advises on treatments for the NHS in England and Wales, estimates that around 1,100 adults and children could be eligible in the first year. Once initial demand has been met, about 820 people a year are expected to be offered the treatment.
The guidance applies in England and Wales. In Northern Ireland, NICE guidance does not apply automatically. In Scotland, the Scottish Medicines Consortium makes decisions on which medicines should be provided by the NHS. The SMC said it expects to issue advice on teplizumab in early 2027.
More progress may be ahead
Breakthrough T1D, which helped fund earlier research that contributed to the development of teplizumab, said other immunotherapy treatments for type 1 diabetes are also being developed.
Looking ahead, researchers hope newly diagnosed patients may one day receive personalised combinations of treatments that could prevent them from becoming dependent on insulin therapy.
