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A first-of-its-kind gene therapy trial for type 1 diabetics is set to begin this year, raising hopes that a single jab could transform treatment for millions worldwide.<\/p>\n
Adults with the incurable condition will be given a one-time dose of an injection which scientists hope will help the body control blood sugar levels without the need for daily insulin.<\/p>\n
Early animal model tests showed the jab – called KRIYA-839 – worked for up to four years without suppressing the immune system.<\/p>\n
Unveiled at this year’s International Conference on Advanced Technologies and Treatments for Diabetes<\/a>, the treatment will now be tested in humans for the first time – and could potentially last a lifetime.<\/p>\n ‘In the type 1 community, we’re used to (hearing) this will happen in 10 to 15 years and maybe will come one day,’\u00a0announced Jeremy Pettus, an endocrinologist and associate professor of medicine at the University of\u00a0California<\/a>, as first reported by\u00a0Medscape<\/a>.<\/p>\n ‘It’s very exciting to stand here and say that this is actually something that’s in the works and happening now.’<\/p>\n It is hoped that the therapy will work by making thigh muscles – where the injection is given – produce insulin and help regulate blood sugar.<\/p>\n Dr Partha Kar, NHS England’s national speciality adviser for diabetes told the Daily Mail that the development is ‘really exciting’ and has the potential to be a ‘functional cure’ for the condition.\u00a0<\/p>\n A first-of-its-kind gene therapy trial for type 1 diabetics is set to begin this year, raising hopes that a single jab could transform treatment for millions worldwide<\/p>\n<\/div>\n Prof Pettus stressed that the method ‘isn’t gene editing or changing genetic makeup’. Instead it is said to deliver insulin-related genes into muscle cells, where they will stay and keep producing proteins long-term.\u00a0<\/p>\n He added:\u00a0‘This is a proven technology. Now the manufacturing has improved such that we can move away from the (rare) cases to more common diseases like diabetes.’<\/p>\n Around 464,000 people in the UK live with type 1 diabetes, a condition which means the body cannot produce insulin – a hormone needed to convert sugar into energy.<\/p>\n Patients currently rely on daily insulin injections or pumps to manage their blood sugar.<\/p>\n The new therapy could significantly reduce that burden for type 1 diabetics across the world, with Prof Pettus suggesting it could ‘potentially be for the masses.’\u00a0<\/p>\n Dr Kar said the injection has the potential to ‘help a lot of people if it works’ and that ‘it’s got a potential for being a functional cure’ – even if ‘it’s not a complete one’.<\/p>\n He added: ‘(I see it as really exciting). We’re talking about a lifetime of coming off high doses of insulin or having an artificial system around you because at the end of the day, there’s technology and everything.\u00a0<\/p>\n Dr Kar believes the two ‘big unknowns’ going into the trials are how much insulin the injection will produce, and how long the effects will last.<\/p>\n But, he said: ‘If you’re saying, ‘hey, listen, we can reproduce 75 per cent of your need of insulin’, and then you probably would be like, ‘wow, that’s a big thing’.<\/p>\n ‘I see it as positive. I think I’d be excited if me or if my children had type 1 diabetes. I certainly would be keeping a very close eye on this.’<\/p>\n The treatment is said to involve a short period of ‘immune modulation,’ meaning the immune system is temporarily dampened to help the therapy enter cells effectively.\u00a0<\/p>\n The injection may be given in both legs during a single outpatient visit lasting up to an hour and could take two to three months to reach full effect.\u00a0<\/span><\/p>\n ‘And then, if all goes well, this could last for the entire life of the patient,’ Prof Pettus added.<\/span><\/p>\n The initial trial will run for one year and enrol adults with higher blood sugar levels who already use automated insulin delivery systems, allowing researchers to\u00a0monitor changes.<\/span><\/p>\n If successful, future trials could include those who use daily injections.\u00a0<\/span>\u00a0<\/p>\n Tadej Battelino, head of endocrinology at UCH-UMC Ljubljana, told Medscape that he wouldn’t use the word ‘cure’ just yet, adding: ‘I tend to be cautious, so I really can’t give promises. Does this have a potential? Very much so.’<\/p>\n Battelino added that if it can keep blood sugar in a healthy range most of the time, perhaps in combination with technology, then ‘I’m not saying it’s a cure, but a functional cure, for sure.’<\/p>\n<\/div>\n