Late last year, JDRF hosted its second Type One Summit event in Melbourne, bringing together some of Australia’s most notable names in type 1 diabetes research to share their insights with our community. The feature session of the day saw all Summit guests come together for a keynote presentations and a clinical trials Q&A, starring an expert panel. We’re pleased to make a video of the morning sessions available for the JDRF community nationwide, along with a summary of the clinical trials session below.
Type One Summit – Keynote and morning session
Watch our video to hear from the following speakers:
Mike Wilson, JDRF Australia CEO and Managing Director
Rebecca Davies AO, JDRF Australia Board Member and mum to a daughter with type 1 diabetes
Alana Hearne, Abbott guest speaker on T1D technology
Clinical trials panel session, convened by Professor Tom Kay
If watching a video isn’t convenient for you, read the written account of the clinical trials Q&A session below.
Demystifying clinical trials – The experts tell all
Professor Tom Kay, Director St Vincent’s Institute of Medical Research and Aikenhead Centre at St. Vincent’s in Melbourne, and session chair, took to the stage to introduce the panel. He set the scene by stating some of the world’s best minds in type 1 diabetes (T1D) research are in the room, and that’s why we can say Australia punches above its weight in the T1D research space.
Professor Kay is an endocrinologist by background, and has worked in T1D research for 30 years. He enjoyed working in area of diabetes as a junior doctor, and had lots of terrific mentors and role models who encouraged his enduring interest in the space.
The main message he put to the audience is that although we hear a lot of good things about the benefits of research there are also the frustrations. Research takes longer than you think, but it’s important to recognise that there’s been an enormous change.
“I can look back to the start of my working life when I was a resident medical doctor in a hospital and there’s been enormous change in treatment and understanding of how T1D comes about. That change can only happen through research; that’s the only way it happens… including research that happens right here in Australia. That can only happen if people participate.”
“That can only happen if we’re not about curing mouse diabetes. We’re about curing humans.”
Professor Kay went on to explain that this really means that the community needs to be involved.
“There will be enormous changes again in the next working lifetime and these will benefit you and your children.”
Professor Kay then introduced the panel:
Associate Professor John Wentworth is based at Royal Melbourne Hospital and Walter and Eliza Hall Institute. His main area of interest is immune therapies which turn off the immune attack that causes T1D. He’s involved with a number of human trials, including TrialNet and ENDIA. His research is focused on people who are just diagnosed and people who will go on to develop to get T1D by virtue of having antibodies.
Catriona Sims is a project portfolio manager at University of Melbourne’s St Vincent’s Hospital diabetes research technology group. She’s also the research coordinator for the adult hybrid closed loop study across seven Australian sites. Catriona is also involved in several other studies investigating hypo unawareness and exercise.
Shannon Ryan has had T1D for 32 years. His wife Ilona, and little girls Audra and Eden who are enrolled in the ENDIA study, also join the panel. Ilona’s background as a nurse means she understands the importance of clinical research. She was keen for her girls to get involved because her husband and brother-in-law have T1D.
Belinda Moore, is a research nurse for ENDIA. Belinda works with the awesome families enrolled in the study, including doing home visits. She also is involved in the TrialNet study.
Louise Ginnivan has had T1D since the 1970s and was the one and only in the family until her grandchild was diagnosed at 18 months. Louise is also a diabetes educator and has participated in clinical trials since the ‘80s including closed loop studies. All three of Louise’s children were in clinical trials in ‘80s to investigate a T1D genetic link. She puts the reason she’s so fit and healthy down to her involvement in various trials over the years. Louise also has two grandchildren enrolled in the ENDIA study.
Professor Kay then called on each of the panel members to explain a bit more about their areas of research.
A/Prof. Wentworth explained the purpose of immune therapy trials is about hitting T1D at the source. His research goes to the heart of the problem by looking at why the immune system attacks the insulin-producing beta cells. The vision is to find safe and effective ways of turning the attack off. However, research is very slow at evolving immune therapies. A/Prof. Wentworth’s mission is informed by other studies, including ENDIA, which investigate what kicks the disease off in some people but not in others. A/Prof. Wentworth states researchers need to do this as international community, as we need such large numbers of participants. That’s why Australian researchers are joining forces with number of centres around the world.
Belinda stated the goal of ENDIA is to look into environmental and genetic triggers and why we’re seeing children at risk developing autoantibodies. ENDIA looks at what is in every child’s world that’s stopping or not stopping them from developing T1D. Is it viral or gut microbiome or something to do with genes? The study includes tracking of autoantibodies. These are markers in the bloodstream that show the immune attack of T1D is occurring. If someone shows the autoantibodies for T1D, they might develop the disease at some point in the future. Showing two autoantibodies means there’s around an 80% chance of this. ENDIA has enrolled 1,200 babies from all around the country who have immediate family members with T1D. The process starts during pregnancy, where the mum gives samples until birth, where each family then collects genetic and environmental samples from the babies.
“Wees, poos, saliva, skin swabs mouth swabs, colostrum, and blood if the parents are happy”, said Belinda on what samples ENDIA collects.
Out of all the babies and children in ENDIA, eight have developed T1D and are no longer in the study. Some are able to go on to be involved in TrialNet.
ENDIA babies and children have a tenfold increased risk of developing T1D compared to the general population, but it’s still a low risk overall.
Catriona’s research goal is to investigate the next generation of the technology that’s currently available in Australia. Her team is looking at a hybrid closed loop device that’s not commercially available, and investigating the impact of this on factors including blood glucose levels, biomarkers, sleep quality and impact on equipment on psychological state. The aim is to understand how this device improves quality of life. The three-year study involves 120 people across Australia. Participants on injections and pumps are randomised to a control group (staying on their current treatment regime) or the hybrid closed loop technology for six months. This is a very important study to see the impact of the closed loop system and whether the algorithm needs more work.
From a parent’s perspective, Ilona explained the reason the family became involved in ENDIA is because she and Shannon are passionate about doing their bit to contribute.
“It’s a simple process; no hassle. The girls love seeing the ENDIA nurses. If you’re a little bit concerned, the nurses are fantastic – so don’t stress.”
Louise, who has taken part in many clinical trials was asked if it is scary using a device that’s not on the market. She said not in the environment she was in. She spent the first week of the hybrid closed loop trial in a hotel, enjoying the week off household duties.
“There was a few disturbed nights, where they were taking samples all night. But I have the incentive of grandchildren and that’s why I continue to be involved.”
Louise said the device she trialled looked after her basal insulin in a beautiful way.
“It was amazing how it copes with meals. All week, I had only one hypo which was mild and it was during a two-hour walk. It’s wonderful seeing what is coming and our lives will be much easier to manage.”
On the benefits of clinical trials, Professor Kay listed close contact with researchers in the field, the opportunity to see what’s coming in the future and the benefits for an individual’s own T1D. He gave the example that it would be extremely unlikely that any kids in the ENDIA study would end up with diabetic ketoacidosis, as we would be shown the disease was developing a long time before they become unwell.
He balanced this by saying that researchers are very conscious of the risks of being involved as a clinical trial participant.
“We’re put through the hoops by ethics committees to make sure things are safe. But people need to be prepared to try things that aren’t yet validated.”
Professor Kay continued that the benefits really outweigh the concerns. He explains that part of reason is to help others and future people, in addition to yourself. There’s a sense of altruism in helping the community.
Catriona took a question from the audience around the device the hybrid closed loop study is investigating. She said it won’t be the exact same device as what goes into market but the difference between it and current devices is that background infusion is controlled by an algorithm. The device delivers ‘microboluses’ of insulin as the basal rate. These are small amounts of insulin which the device determines based on the wearer’s history and the predictions of what they will do. The device is not completely automated and can be used out of closed loop mode. Wearers will still need to consider and calculate carb amounts. Catriona explains it’s called a hybrid system because people are still required to use bolus for meals and exercise. Closed loop studies are finding good results overnight, as these hours are not influenced by food and exercise. Researchers want to try and replicate this success in the day, as the algorithms get better.
The panel took another question on devices – is there any thought of pumps having insulin and glucose onboard? A/Prof. Wentworth answered that there are pumps in development that can give glucogon, but nothing is in mainstream use yet.
The audience asked a question around trials for long-acting insulin that lasts for weeks or months. A/Prof. Wentworth said that there are research teams looking into this e.g. patch pumps but that these are in very early stages of development. He explained that the challenge in T1D treatment is mimicking what the body’s own systems can do, which are incredible.
“You can see this when the beta cells are not there anymore. The amount of insulin we need goes up and down a lot during the day naturally. Giving one shot of long-acting insulin won’t be able to mimic the body’s own systems and there would need to be something sophisticated on top of that to manage that. That’s why we’re so interested in biological solutions which try to prevent or reverse T1D entirely. It’s so difficult for technology to mimic what the body’s own systems do. We ideally want to have the body’s own systems take over, and do that again.”
An audience member commented that his family is discussing TrialNet involvement. He asked the panel: “Are you better off knowing? What things can we do about the triggers? How can we slow the process down today? Does this increase anxiety in kids or family?”
A/Prof. Wentworth gave context for the wider audience that TrialNet is currently the only T1D antibody screening program available and the blood test is free of charge. If antibodies are found, individuals can go into a monitoring program and possibly even intervention studies. He acknowledged that not everyone wants to know that they will get T1D someday in the future. There are pros and cons to knowing you have, or your child has, the autoantibodies. Studies have looked at how people have reacted to knowing they will develop a disease in future. There is shown to be an initial anxiety and with monitoring and extra information and support, the anxiety levels drop to below that of general population. What does this mean for insurance? This is a very grey area. If your insurer asks if you have background medical history, you may need to disclose this. There’s not yet a clear answer, especially as people with type 1 diabetes generally have the same life expectancy as the general population now.
Professor Kay took a question on what else people at risk of T1D can do apart from becoming involved trials? A/Prof. Wentworth answered that we currently don’t know of anything that works from lifestyle perspective in terms of preventing T1D. That’s why these population studies are so important.
“The advice I give people is to be alert about symptoms and lead a healthy lifestyle generally.”
So, what are some other exciting prevention developments on the horizon? A/Prof. Wentworth explains that prevention studies using existing drugs for rheumatoid arthritis and lupus are underway. These drugs are chosen because they’re exquisitely safe, and the really key thing is that things are safe – predominately for paediatrics. Two insulin prevention trials have recently finished and didn’t show any benefits. Another immune therapy trial will publish results this year. This trial caused a few early side effects, but it will be interesting to see whether it has delayed T1D in the population in which it was tested.
The final question was, “is there going to be better communication between hospitals on what we can do?” A/Prof. Wentworth answered honestly that clinical trial referrals are not top priority for clinicians, so researchers can struggle to get the word out. One thing the community can do is to sign up for JDRF’s T1D Game Changer program, to be told about new and recruiting clinical trials. Alternatively, you can check out JDRF’s T1DCRN website, as well as the Australian Clinical Trials Registry.
The session finished with a few final words:
“Get involved as I’ve only benefited ever,” said Louise.
“All these trials are good, it’s about getting ahead of the game. Back in ‘86 when I was diagnosed things were pretty horrific, so anything that gets ahead of the game is good. Heaven forbid, if my girls had any issues they wouldn’t have to go through what I did,” added Shannon.
Catriona said “I’m very very thankful to anyone who does participate… The benefit is extraordinary. At the end of the day if we can’t have you involved we can’t advance and get the data we need which is vital to progressing forward.”
A/Prof. Wentworth gave his perspective, “We’re all in this together. We’re very grateful for help and we’re doing this as a team”.