Breakthrough jab could provide 'functional cure' deadly diabetes
by CIARAN FOREMAN, REPORTER · Mail OnlineA single jab hailed as a 'potential cure' for type 1 diabetes will be trialled this year – raising hopes it could finally end the need for multiple daily insulin injections.
The one-off treatment is designed to help the body control blood sugar on its own – potentially for life.
In a world-first study, patients will be given a gene therapy that turns muscle into a long-term insulin producer, with effects that could last for years – or even decades.
Dr Partha Kar, NHS England's national speciality adviser for diabetes, said the approach is 'really exciting' and has the potential to be a 'functional cure' – adding that it could 'help a lot of people if it works'.
Adults with type 1 diabetes – an incurable condition affecting around 464,000 people in the UK – currently rely on daily insulin injections or pumps to stay alive. The disease occurs when the body's immune system destroys insulin-producing cells in the pancreas, leaving patients unable to regulate blood sugar.
The new treatment, known as KRIYA-839, takes a radically different approach.
Rather than replacing insulin through injections or devices, it aims to turn the patient's own muscle into a long-term insulin factory.
Scientists hope that after a single injection into the thigh, muscle cells will begin producing insulin and other blood sugar–regulating proteins – removing or dramatically reducing the need for daily treatment.
Crucially, researchers say the therapy is not gene editing and does not alter a person's DNA. Instead, it delivers genetic instructions into muscle cells, allowing them to produce insulin in a controlled way over time.
Early animal studies have shown promising results, with the treatment continuing to work for up to four years without the need for ongoing immune suppression.
Now, for the first time, it will be tested in people.
The trial – unveiled at this year's International Conference on Advanced Technologies and Treatments for Diabetes – will enrol adults with poorly controlled blood sugar who are already using automated insulin delivery systems. This will allow scientists to closely track how much insulin the therapy produces and how effectively it stabilises glucose levels.
Participants will receive injections in both thighs during a single outpatient appointment lasting up to an hour. The treatment is expected to take two to three months to reach full effect.
There is also a short phase of 'immune modulation', where the immune system is temporarily dampened to help the therapy successfully enter cells – a step researchers say is key to making the treatment work.
If successful, the effects could last for years – or even a lifetime.
Jeremy Pettus, an endocrinologist and associate professor of medicine at the University of California, said the field is entering a new phase.
'In the type 1 community, we're used to (hearing) this will happen in 10 to 15 years and maybe will come one day,' he said, as first reported by Medscape.
'It's very exciting to stand here and say that this is actually something that's in the works and happening now.'
Dr Kar said the potential impact could be transformative – even if the therapy does not completely eliminate the need for insulin.
'If you're saying, 'hey, listen, we can reproduce 75 per cent of your need of insulin', then you probably would be like, 'wow, that's a big thing',' he said.
He added that even a partial effect could mean coming off high doses of insulin or reducing reliance on pumps and continuous monitoring systems.
However, he cautioned that key questions remain – particularly around how much insulin the therapy will produce, and how long the effects will last.
'If it works, it could help a lot of people,' he said. 'I see it as positive… I certainly would be keeping a very close eye on this.'
Other experts have also urged caution.
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Tadej Battelino, head of endocrinology at UCH-UMC Ljubljana, said the term 'cure' should be used carefully at this stage.
'I tend to be cautious, so I really can't give promises,' he said. 'Does this have a potential? Very much so.'
He added that if the therapy can keep blood sugar in a healthy range most of the time – particularly when combined with existing technology – it could effectively function as a cure in practical terms.
'I'm not saying it's a cure, but a functional cure, for sure.'
The initial trial will run for one year, with future studies expected to expand to a wider group of patients, including those managing their condition with daily injections.
If the results are positive, the therapy could mark a turning point in the treatment of type 1 diabetes – shifting it from a condition managed day-to-day to one controlled by a single intervention.
For patients used to a lifetime of injections, monitoring and constant vigilance, that prospect alone is enough to generate real excitement.