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World-first type 1 diabetes drug trial aims to reprogram immune system

World-first type 1 diabetes drug trial aims to reprogram immune system

A world-first human trial of a drug designed to treat the underlying cause of type 1 diabetes has begun in Australia.
Five patients with the condition have already been dosed as part of the trial, including mum-of-two Caecilie Wickstroem Giralde, who was diagnosed last year.
University of Queensland researcher Ranjeny Thomas has spent more than a quarter of a century developing the drug, designed to rebalance the body's immune response in people with type 1 diabetes, which affects more than 130,000 Australians.
Caecilie Wickstrom Giralde, a participant in the drug trial, pictured with her husband Thiago and their children Vincent and Frida.
(
ABC News: Janelle Miles
)
The immune system starts to recognise insulin-producing cells in the pancreas as something it needs to attack and destroy in people with type 1 diabetes — one of dozens of auto-immune diseases in which the body starts to attack itself.
Professor Thomas, who is based at UQ's Frazer Institute, said the experimental drug — dubbed ASITI-201 — was designed to retrain the immune system so it no longer attacks the insulin-producing pancreatic cells, known as beta cells.
The drug, given as an injection under the skin, combines fragments of a protein found in the beta cells of people with type 1 diabetes and vitamin D to calm the immune response.
Professor Ranjeny Thomas has spent more than a quarter of a century developing the experimental drug.
(
ABC News: Will Murray
)
"This therapy is really trying to get to the root cause of the autoimmune disease," Professor Thomas said.
"
We really want to try and stop it in its tracks.
"
She said if effective, the drug would initially be given to patients with type 1 diabetes as soon as possible after diagnosis to preserve remaining pancreatic cells and reduce the amount of insulin needed.
But eventually, if screening programs can be developed to pick up people at risk of developing type 1 diabetes, it may be possible to "prevent the progression of the disease altogether".
"If we can show that this drug is very safe and it does what we expect to calm the immune system, then that is an opportunity for us," Professor Thomas said.
Caecilie Wickstroem Giralde says she will never get used to giving herself insulin injections.
(
ABC News: Janelle Miles
)
'I'm more than happy to give my body for that'
The first in-human trial of 36 participants will test the safety of the drug, but blood tests will also determine the impact of the therapy on a patient's immune system and glucose tolerance.
Three doses of ASITI-201 will be trialled at the Translational Research Institute's clinical research facility based at Brisbane's Princess Alexandra Hospital.
Some patients will also receive a placebo.
The trial is blinded so neither the patients, nor the investigators, are aware of who receives the active drug or the placebo.
Ms Wickstroem Giralde was diagnosed with type 1 diabetes last year.
(
ABC News: Janelle Miles
)
Ms Wickstroem Giralde developed gestational diabetes during pregnancies with Frida, 4, and Vincent, 2, which put her at increased risk of type 1 diabetes.
She was then diagnosed with type 1 diabetes in October last year, which requires regular insulin injections, so volunteered to take part in the trial.
Insulin plays a crucial role in regulating blood glucose levels — the body's primary source of energy — by allowing glucose to move from the bloodstream into cells.
Caecilie Wickstroem Giralde and her four-year-old daughter, Frida.
(
ABC News: Janelle Miles
)
"I think what's special about this trial is that it's so specific," she said.
"If the drug works, it can go and reprogram your immune system and help preserve the beta cells and help you produce your own insulin for longer.
"
If that works, it's kind of magic, isn't it?
"
Having been born in Scandinavia also gave the Denmark-raised 36-year-old a higher chance of developing the condition.
Photo shows
Man in a hat holding up a pumping unit to help control blood sugar levels.
For people who have to inject insulin every day to stay alive, public ignorance about the condition, and its causes, can be intensely frustrating.
Ms Wickstroem Giralde admits to being "needle-phobic" and joined the trial hoping it would reduce her reliance on insulin.
"Multiple daily injections of insulin is so hard," she said.
"I will never get used to giving myself injections. I have to take a deep breath, close my eyes every time I have to do it.
"If I could do anything that could take that away, I would be willing to do that.
"Even if it can't help me, but it can help someone in the future, I'm more than happy to give my body for that."
The 'exhausting' toll of diabetes
The young mum said type 1 diabetes sometimes affected precious time with her children.
The young mum hopes the experimental drug will give her more energy to be fully present during precious family time.
(
ABC News: Janelle Miles
)
"At dinner time, I'll take my insulin, I'll eat, we might muck around a little bit more than what we usually do, and my blood sugar will go low just as I'm about to put the kids to bed," Ms Wickstroem Giralde said.
"Then I'll be like: 'Kids, just go in bed, choose your books'. I'll have to eat some sugar, sit down, make sure my sugar goes back in range so I don't go all shaky and uncomfortable while I'm putting my kids in bed.
"
That's super difficult. I want to be present, I want to be there 100 per cent for my kids. It's a special time.
"
The drug trial's lead investigator Aakansha Zala, an endocrinologist at the Princess Alexandra Hospital, said having type 1 diabetes was "exhausting" for many patients.
Dr Zala said the condition could lead to a "wide array" of complications, including kidney issues, where patients may end up needing dialysis.
Dr Aakansha Zala said people managing type 1 diabetes can often feel exhausted.
(
ABC News: Will Murray
)
"They can get issues with their nerves — where they may not feel things as easily — and sometimes that can affect their ability to drive, so if people have really bad nerve issues, they're not allowed to drive," she said.
"It can also increase the risk of foot infections and amputations. There's an increased risk of heart disease, of strokes. They can get issues with their eyes where they may even potentially lose vision.
"
It can affect many different parts of the body. It's a lifelong condition and affects your day-to-day and minute-by-minute quality of life.
"
To take part in the study, participants need to be at least 18 years old and to have been diagnosed with type 1 diabetes within the past five years.
Ms Wickstroem Giralde hopes the trial will lead to positive health outcomes for people managing type 1 diabetes.
(
ABC News: Janelle Miles
)
Dr Zala said they were
The researchers have some funding available to pay for travel for people living outside of Brisbane who are eligible for the trial.
Future trials, including some with children — who have a much faster progression to insulin dependence — are planned.
Diabetes Australia's director of research Grant Brinkworth, who is not involved in the trial, said the drug being tested could "fundamentally change" the lives of people diagnosed with type 1 diabetes.
Grant Brinkworth said the experimental drug could drastically change the lives of people with type 1 diabetes, if the trial proves successful.
(
Supplied: Diabetes Australia
)
"If successful, this research could reduce or eliminate the need for people living with the condition to endure life-long daily insulin injections and lessen the risk of the health complications associated with living with diabetes," Professor Brinkworth said.
"Living with type 1 diabetes is a life-changing diagnosis and people with the condition make about 250 extra decisions every day to manage their health."
Professor Thomas, who is also a rheumatologist at the Princess Alexandra Hospital, is working on a similar drug for rheumatoid arthritis, another autoimmune disease.

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Prof Harnett says Victoria's Better Health Channel and the US government's National Complementary and Integrative Health website are resources people can look to if they are unsure of using a complementary medicine. When Peter Hill walked into his first pilates class, he had to warm-up for the warm-up. With a feeble lower back and taut hamstrings, the then 63-year-old was hoping the therapy could aid his pain. Six years later, he can dart and dash with little-to-no trouble. "Occasionally I might have a bit of a twinge but I can get out of bed in the morning and do normal stuff without having to warm up," Mr Hill says. "I jog or run once a week and I never have an issue with my hamstrings, and my back is significantly better." 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However due to the rising cost of healthcare, she doubts more people will sign up for premiums. "It would probably be quite smart ... to include those kinds of things," Prof Zhang says. "It would improve the value for private health insurance but in the big scheme of things, I don't think it would have a huge impact." But 83-year-old Colin who attends pilates every week sees the positives of a few more dollars in his pocket. "I might actually go to two classes rather than one," he says. "I really enjoy doing that sort of stretching, that sort of exercise." Prof Harnett says Victoria's Better Health Channel and the US government's National Complementary and Integrative Health website are resources people can look to if they are unsure of using a complementary medicine.

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The opposition matched Labor's promise with spokeswoman Anne Ruston saying the coalition was "strongly committed to strengthening preventative health outcomes ... and these decisions are in line with that focus". Bupa is already on board, telling AAP it will welcome the subsidies. However, Private Healthcare Australia policy director Ben Harris says some firms may only take up the movement-based therapies such as yoga, tai chi and pilates. "The evidence is quite clear that in certain circumstances for certain people, these therapies can make a really big difference and the best thing with healthcare is you need as many options on the table as possible," he says. "It's getting better all the time ... but the broad research is still yet to be done for the community as a whole." Why though are complementary medicines important? 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"It's not an umbrella claim but certainly there is some moderate level of evidence to support effectiveness and ... this can be potentially beneficial to patients," he tells AAP. "The review is not saying yoga can do everything. So, there is a lot more research needed in order to make that more general claim." Medical economist and academic Yuting Zhang expects insurers to include natural therapies in their premiums because of their low-cost compared to private hospital cover. However due to the rising cost of healthcare, she doubts more people will sign up for premiums. "It would probably be quite smart ... to include those kinds of things," Prof Zhang says. "It would improve the value for private health insurance but in the big scheme of things, I don't think it would have a huge impact." But 83-year-old Colin who attends pilates every week sees the positives of a few more dollars in his pocket. "I might actually go to two classes rather than one," he says. "I really enjoy doing that sort of stretching, that sort of exercise." Prof Harnett says Victoria's Better Health Channel and the US government's National Complementary and Integrative Health website are resources people can look to if they are unsure of using a complementary medicine.

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Cheaper complementary health treatments are on the way

When Peter Hill walked into his first pilates class, he had to warm-up for the warm-up. With a feeble lower back and taut hamstrings, the then 63-year-old was hoping the therapy could aid his pain. Six years later, he can dart and dash with little-to-no trouble. "Occasionally I might have a bit of a twinge but I can get out of bed in the morning and do normal stuff without having to warm up," Mr Hill says. "I jog or run once a week and I never have an issue with my hamstrings, and my back is significantly better." The additional good news is that pilates-goers like Mr Hill can soon expect their classes will be cheaper after federal health minister Mark Butler promised during the election campaign that private insurers would be obliged to cover some complementary treatments. A spokesperson for his office says the scheme's rollout is imminent. Complementary medicines are therapies that accompany pharmaceutical treatments with the aim of promoting holistic health. A study found that of the millions of Australians who use them, more than 50 per cent do so the same day they take their pharmaceutical medicine. The therapies include yoga, pilates, tai chi, shiatsu, naturopathy and the Alexander technique, as well as a range of western herbal medicines, and they will be covered by private insurers. A review recommended government subsidise all seven after finding they were moderately effective and safe. This represents a back-track on the 2019 exclusion of 16 natural therapies, which saw insurers lose rebates. "Coverage of natural therapies remains a decision for insurers," according to Mr Butler's office. "Insurers which choose to cover the therapies will work with the natural therapies sector on coverage and to re-establish provider credentialing. "The government will ensure timely re-inclusion of benefits for natural therapies, so this coverage is available as soon as possible." The opposition matched Labor's promise with spokeswoman Anne Ruston saying the coalition was "strongly committed to strengthening preventative health outcomes ... and these decisions are in line with that focus". Bupa is already on board, telling AAP it will welcome the subsidies. However, Private Healthcare Australia policy director Ben Harris says some firms may only take up the movement-based therapies such as yoga, tai chi and pilates. "The evidence is quite clear that in certain circumstances for certain people, these therapies can make a really big difference and the best thing with healthcare is you need as many options on the table as possible," he says. "It's getting better all the time ... but the broad research is still yet to be done for the community as a whole." Why though are complementary medicines important? Well, chronic disease is the leading cause of death worldwide, killing around 41 million people each year, while researchers say natural therapies alongside traditional medicines can help treat chronic symptoms and restore body function. Insurers in Canada, India and the UK are already rebating complementary medicines, while Australia's decision comes nine months after the National Disability Insurance Scheme removed subsidies for therapies like yoga, cuddle therapy and hypnotherapy. Then NDIS head Bill Shorten said these treatments should have never been on the list. But the federal review released in April, said yoga in particular had "some benefit" for people compared to individuals who don't participate. Still, some doctors are sceptical. The Australian Medical Association says people should remain cautious with some of the movement-based treatments. It also warns paients not to stop taking pharmaceutical medicines. "While evidence-based complementary medicine can play a role in patient care under the guidance of a doctor, there is limited evidence on the effectiveness of most complementary medicines," the association says. "People need to be extremely careful if they are sourcing herbal supplements because some are potentially harmful and it's very difficult to be certain about what they contain. "Some have the potential to cause adverse reactions or interact with conventional medicines." Joanna Harnett, an Associate Professor specialising in complementary healthcare, says any medicine people have can cause a reaction but complementary medicines taken orally in conjunction with traditional treatments pose the biggest risk. Natural therapies like shiatsu and western herbal medicines are derived from tradition compared to more research-based pharmaceuticals. But National Integrative Medicine Institute director Dennis Chang says the seven subsidised therapies are scientifically safe. "It's not an umbrella claim but certainly there is some moderate level of evidence to support effectiveness and ... this can be potentially beneficial to patients," he tells AAP. "The review is not saying yoga can do everything. So, there is a lot more research needed in order to make that more general claim." Medical economist and academic Yuting Zhang expects insurers to include natural therapies in their premiums because of their low-cost compared to private hospital cover. However due to the rising cost of healthcare, she doubts more people will sign up for premiums. "It would probably be quite smart ... to include those kinds of things," Prof Zhang says. "It would improve the value for private health insurance but in the big scheme of things, I don't think it would have a huge impact." But 83-year-old Colin who attends pilates every week sees the positives of a few more dollars in his pocket. "I might actually go to two classes rather than one," he says. "I really enjoy doing that sort of stretching, that sort of exercise." Prof Harnett says Victoria's Better Health Channel and the US government's National Complementary and Integrative Health website are resources people can look to if they are unsure of using a complementary medicine.

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