World-leading clinical trials at Northern Sydney Local Health District
Northern Sydney Local Health District TOMORROW’S MEDICINE TODAY World-leading clinical trials at Northern Sydney Local Health District
Edition 1
Northern Sydney Local Health District is home to world-leading investigators and scientists conducting clinical trials across a wide range of specialties, including oncology, cardiology, geriatrics, clinical genetics, intensive care and many other fields. Each year, hundreds of patients join our trials, driving advancements in new treatments, devices and medications. Incorporating clinical trials into clinical care enhances the long-term health outcomes of our patients.
A new era
We are delighted that Northern Sydney Local Health District is ushering in a transformative new era in research through clinical trials. Clinical trials are the bedrock of medical innovation, providing our patients with access to the latest treatments and therapies. They pave the way for tomorrow’s medicines and are crucial in advancing medical science.
For decades, our district has been home to world-leading clinical investigators conducting research that has changed medical practice, not only locally but also globally. Having witnessed firsthand the extraordinary impact clinical trials can have on patient outcomes, we have long seen their essential role in advancing medical science. Through these trials, we have seen lives transformed and patients receive cutting-edge care that significantly improves their health and quality of life. Our new vision for the district is to offer every patient the opportunity to participate in a clinical trial. By integrating clinical trials into our standard patient care protocols, we can ensure that all patients benefit from the latest medical innovations. This approach will enhance current patient care and set the stage for ongoing medical breakthroughs. A key factor in achieving this vision is robust philanthropic support. Generous contributions from individuals, foundations, and corporations are vital to funding these trials. By investing in clinical trials, donors directly contribute to medical discoveries and improved patient outcomes. We urge everyone in our community to consider how they can support and advocate for this crucial initiative. Together, we can create a healthcare environment where every patient has access to the most advanced treatments and where continuous innovation is the norm.
This publication, Tomorrow’s medicine today , highlights our incredible clinical trial work and celebrates the researchers and clinicians whose passion drives this innovative research. Scientific discovery is not easy and requires the dedication of both professionals and patients. We both want to thank all involved in advancing medical science and patient care. Your support in researching or participating in clinical trials will make a significant difference to the lives of people in our community and the future of healthcare. This is an exciting chapter for our district as we continue to prioritise and elevate our research initiatives. Adjunct Professor Anthony M. Schembri AM Chief Executive NSLHD Nadia Levin Chair of the NSLHD Research, Innovation and Technology Committee
Nadia Levin and Adjunct Professor Anthony M. Schembri AM
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NSLHD clinical trials snapshot
In 2024, there were 400 clinical investigators and staff working on 350 clinical trials across the district.
Our clinical investigators are recognised in the world’s top 2% of scientists in the prestigious Stanford/ Elsevier rankings.
Our research is published in the top scientific journals in the world, including Nature, The Lancet, JAMA, The BMJ and The New England Journal of Medicine.
We collaborate with clinical investigators and researchers
at many institutions at the University of Sydney, Macquarie University, University of Technology Sydney, NSW Health, Sydney Health Partners, Sydney North Healthcare Network, and others.
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Tomorrow’s medicine today
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Royal North Shore Hospital’s data-driven approach to radiation oncology
Fifteen years ago, when Professor Tom Eade began working at Royal North Shore Hospital (RNSH) as a radiation oncologist, he was eager to set up a solid system to collect patient data for research.
He’d been inspired by the approach to cancer research he’d seen during his fellowship in the United States. “There was a lot of interest in collecting clinical trial data and evidence-based practice,” he says. Today, the department boasts a sophisticated system that helps doctors and many other highly-skilled staff run a range of pioneering studies, which help improve cancer treatment and patient outcomes. A focused approach to collecting patient data means doctors and staff can run real-time audits of the approximately 1,000 patients treated at the centre each year. “We use the data to help understand where we need to do better for our patients, which educates us in future trial design, and which studies we should recruit to.” Aside from running data-driven studies, Tom says the department also has a long history of being strong in the technical aspects of cancer treatments due to ongoing basic scientific research. “We’re getting better imaging of the cancer, and we’ve got better technology for delivering and planning the radiation.” Impact of radiation treatment The data collected at RNSH encompasses not just cancer outcomes but also the side effects of radiation treatment, an area that is of particular interest to the radiation therapy community.
“It’s quite hard for most clinicians to report because if you cure a patient, they don’t tend to turn up after five years and obviously, if you haven’t cured them, then it’s sometimes hard to work out what was the cancer and what was the radiation treatment,” says Tom. But the department is in a unique position because they have well-established communication with patients, which in some cases stretches over 15 years, so they can keep asking questions about side effects. “Having our infrastructure in place also allows us to have a really good feeling about our patient cohort – the risk group, the stage of their cancer, and what types [of cancer].” Trials unique to NSLHD With a strong database, the department can more easily enrol patients in multicenter studies and run their own in-house research. Over the past few years, Tom and the team have been able to design and complete randomised phase III clinical trials that are specific to the patient cohort in the district. Recent successes include completing a 500-patient prostate cancer study comparing treatment durations, and a breast cancer study focused on shorter fractionation periods for radiation therapy.
The department is also researching palliative radiation.
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Tomorrow’s medicine today
“People often talk about our work in a curative setting – about how we use radiation to cure cancer. Radiation also has very good benefits for patients who have incurable cancer and have symptoms and pain.” Tom says there is very little strong clinical trial data on how to do palliative radiation well, but the department has nearly completed one study and is halfway through another. Patient feedback He says a lot of their recent work and clinical trials also includes patient-reported outcomes. “We’ve really moved towards having studies which use a lot of patient reports about how they think things went, rather than historically where the clinician would grade outcomes and say whether it’s good or bad,” he says, adding that they have also begun asking carers whether they think treatments are beneficial or difficult for patients in their care. Tom has seen huge changes in radiation oncology in the last 20 years, often connected to technological shifts. He says RNSH’s focus on data-driven studies, combined with robust research in the basic sciences, aims to push the boundaries of radiation oncology in a structured, evidence-based manner.
Professor Tom Eade 7
Groundbreaking work in geriatric pharmacology
For the past 20 years, Professor Sarah Hilmer AM has dedicated her career to optimising medicine use for older people and has achieved recognition as a national and world leader in geriatric pharmacology.
Eager to put the DBI into clinical practice, Sarah and her colleagues developed a calculator that could measure a patient’s drug burden. They produced a way to integrate it into a hospital’s electronic medical record so that staff could use it when treating frail, elderly patients. In 2021 — at the height of the pandemic — Sarah ran a successful pilot study implementing the calculator at RNSH. “We managed to show that we could really improve prescribing,” she says. For the past 18 months, she and her team have run a clinical trial in three hospitals in Northern Sydney and three on the Central Coast to see if this package of tools can help clinicians to identify patients who are not functioning optimally because of the medication they have been taking and to minimise medication- related harm. The tools are now available for clinical care across Northern Sydney and Central Coast health districts and are being implemented at other NSW health districts. Sarah is eager to see the tool as part of routine hospital care. Nurses regularly have a “huddle” to discuss patient issues, which can include falls or delirium. Her team has been encouraging nurses to look at the Drug Burden Index during a huddle, and if it is high, arrange for a medication review. “It might be that the medication is causing the falls or confusion,” she says.
Not only has the Royal North Shore Hospital (RNSH) clinical pharmacologist and geriatrician set about improving medication practices for older people, but she has long advocated for ‘age-friendly’ clinical trials. One of Sarah’s notable contributions has been the development of the Drug Burden Index (DBI), a risk assessment tool that measures an older person’s exposure to medications that slow them down physically and mentally. “Over the past 20 years or so, we have been validating the tool in populations around the world to show that the higher your Drug Burden Index, the worse your physical function and your cognition is, and the more likely you are to fall or wind up in a nursing home,” she says.
Professor Sarah Hilmer
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Tomorrow’s medicine today
Sarah has long advocated for ‘age-friendly’ clinical trials that make it easier for frail, older people to be included. While the average older person takes up to eight prescription
medications, globally, they are often underrepresented in clinical trials.
“We have a situation where we test drugs in healthy, older people or in middle aged people, and then use them in frail, older people with a lot of different complex problems. We wind up with all sorts of interactions and unexpected effects.” “I think it’s really important that if we’re going to do clinical trials, we need to make sure that they’re inclusive of the people who are going to actually wind up using the drugs in clinical practice,” she says. Her advocacy in this space extends beyond the NSLHD. The U.S. Food and Drug Administration recently designed a roadmap for drug evaluation for older adults, and Sarah was the only Australian on the project, representing the Geriatric Committee of International Union of Basic and Clinical Pharmacologists, which she chairs. Sarah’s work through development and implementation of the DBI and her advocacy for age-friendly trials has significantly improved the quality of life for older adults. “We need to ensure our ageing population receives the best possible evidence-based care,” she says.
This means they have more than one medical problem and what is known as a geriatric syndrome, which include falls, delirium or polypharmacy.
These days, ‘geriatric’ describes patients over 65 who have complex medical problems.
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Pioneering kidney disease research
Dr Brendon Neuen is Director of the Kidney Trials Unit at Royal North Shore Hospital who is driving clinical trials to improve patient outcomes.
His research focuses on identifying new treatments for patients with kidney disease, tackling the dual risk of kidney failure and cardiovascular disease. He collaborates with medical institutes and universities, as well as with the pharmaceutical industry to conduct large international randomised clinical trials. Trained as a nephrologist in Sydney, Brendon further honed his clinical trials expertise with a Masters, PhD and postdoctoral fellowship at the University of Oxford, UNSW Sydney, and Brigham and Women’s Hospital, Harvard Medical School. What fuels his passion for trials? “Clinical trials are the best way to improve the health of millions of people worldwide by better understanding what works and what doesn’t in healthcare settings,” he says. “I really enjoy the challenge of being a clinician and triallist. You have to have in-depth understanding of the science and how to conduct a clinical trial. But you also need to be excellent operationally in terms of logistics and often working with hundreds of sites across 20 or 30 countries. Finally, you must be able to lead large groups of people and bring them with you over the course of a trial which can takes several years.” “The reward is if you can do that, you can make a huge impact on patient care because what changes guidelines and what changes people’s treatments are clinical trials.”
Since 2022, Brendon established and has led SMART-C, a global consortium pooling all the available clinical trial data for sodium-glucose cotransporter-2 (SGLT2) inhibitors, a drug used for treating type 2 diabetes, heart failure and chronic kidney disease. The consortium includes data on over 90,000 patients enrolled in 13 global clinical trials. This public-private partnership involves more than 12 institutions including Harvard, Yale, Stanford, Oxford and Sydney Universities, and others in Europe, as well as major pharmaceutical companies. While the drug class was known to have cardiovascular benefits, Brendon’s work has definitively shown that the SGLT2 inhibitors reduce kidney disease progression, even in people without diabetes, and that the drug class has particularly prominent benefits in preventing heart failure and sudden cardiac death.
Dr Brendon Neuen
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Royal North Shore Hospital’s nephrology department is spearheading 20 clinical trials with over 50 patients enrolled in 2024. But he stresses there is much more to do to improve outcomes for people with kidney disease. He is involved in the leadership of several international trials testing new medicines to reduce the risk of kidney failure in people with non-diabetic kidney disease and prevent cardiovascular complications in people receiving dialysis. Brendon’s dedication to clinical trials and his work with SGLT2 inhibitors exemplify the profound impact that rigorous scientific research can have on global health. “The research allowed us to better understand the effects of SGLT2 inhibitors on a range of important outcomes for different types of patients,” he says. Brendon is proud the work has had a global impact. “It has, in part, helped support the inclusion of SGLT2 inhibitors on the World Health Organisation List of Essential Medicines,” he says. The drug is now available in almost every country for less than a cup of coffee, and widely recommended in almost every major clinical practice guideline for patients with type 2 diabetes, kidney disease or heart failure.
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Clinical trials are tomorrow’s medicine
Endocrinologist Professor Rory Clifton-Bligh has been involved in clinical trials at Royal North Shore Hospital for over 20 years, focusing on conditions such as osteoporosis, thyroid cancer, and diabetes. Patients in the endocrinology clinic have sometimes accessed medication 10 years ahead of other Australians. Many of these drugs have gone on to be listed on the Pharmaceutical Benefits Scheme. “The trials have all been quite transformational in their areas,” he says.
Addressing rare diseases Unfortunately, patients with rare diseases have often faced limited treatment options. However, over the past 10 to 15 years, Rory has seen a significant increase in the number of clinical trials for rare diseases, including some involving his department. The shift has flowed on from the Orphan Drug Act , a US law passed in 1983 that incentivised drug development for rare diseases, making it easier for the US Food and Drug Administration (FDA) to grant approvals, as well as encouraging greater investment. participants for several trials spanning rare bone, thyroid cancer, cholesterol, and lipid diseases. “It’s a fairly diverse portfolio of clinical trials in the rare disease space,” he says. Recruiting subjects can be challenging but multicentre study centres around the world help. Rare diseases are defined differently in parts of the world. In Australia, it is considered rare if five in 10,000 people have the condition while in the US, it’s rare if it affects fewer than 200,000 people. Paradoxically, rare diseases are not rare. Rory points out that rare diseases are more common than people think. Importance of trials He emphasises the broader importance of clinical trials for providing access to new therapeutics and enhancing clinical practice. “What we call best practice is yesterday’s medicine. Clinical trials are tomorrow’s medicine.” Current trials and recruitment challenges Rory and his team are currently enrolling
Professor Rory Clifton-Bligh
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A life-saving rare disease trial
Stuart Robinson was on a holiday in late 2011 with a bad cold. When he got home, he felt a lump in his neck and thought it was swollen lymph nodes connected to his cold. Later, the lump was still there. Doctors in his hometown of Adelaide initially thought he had a small cell cancer of unknown origin, and he underwent chemotherapy and radiation. When the treatment didn’t work, a more in-depth pathological investigation found he had medullary thyroid carcinoma (MTC), a rare form of thyroid cancer. MTC makes up six per cent of all thyroid cases. It is estimated there are only around 60 to 100 people in Australia with the disease. With limited treatment options, Stuart’s wife Jenny began researching, and in a social media group for sufferers in the US, discovered there was a clinical trial for a drug which was heading to Royal North Shore Hospital. The cancer produces a chemical in the blood called calcitonin. By measuring calcitonin, it is possible to tell how active the cancer is. By the time Stuart joined the trial in May 2018, his calcitonin levels were at 7000 pg/mL, and the cancer was spreading. The trial involved taking a pill twice a day that contained a molecule targeting the mutated cells causing the cancer. Within three weeks, Stuart’s calcitonin levels plummeted from 7000 pg/mL to 40 pg/mL. The drug had effectively deactivated the mutant cells, offering Stuart a new lease on life.
“I often say I was unlucky to get cancer, but I was unbelievably lucky this trial came to Australia in 2018 just when mine was starting to take off,” he says. Being on a trial meant he was closely monitored throughout the process. While it has ended, he is now part of an ongoing monitoring program, travelling to Sydney every three months to see Dr Rory Clifton-Bligh, Dr Bruce Robinson and Clinical Trials Coordinator Rhonda Siddall. “They’re very generous with their time and I feel like I’m their only patient,” he says. Stuart says there are side effects such as dry mouth and upset stomach, but he has stuck with it. “We still lead an active life, and it is 12 years now since I first felt the lump in my neck. I’m very happy.”
Stuart Robinson
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Innovation at the Kolling’s core
In the heart of the Kolling Institute, a world-renowned research centre, a raft of clinical trials is driving the future of healthcare.
“Clinical trials are intrinsic to research,” says Professor James Elliott, Academic Director of the Institute. “They help us test something so that we can better inform care for patients with various conditions who come to our hospitals for treatment.” The Kolling Institute has a strong track record in conducting impactful clinical trials. Notably, it has been involved in running multiple trials for those with heart and kidney disease, musculoskeletal conditions and chronic pain. Their cancer trials have made significant contributions across various tumour streams, including pancreatic, haematological, breast, and bowel cancers.
James emphasises that basic science, discovery, and scientific innovation are embedded in the Kolling’s over 100-year history. “Often, it is those innovations from our basic science laboratories that ultimately inform the mechanisms by which trials are developed.” James adds that understanding the ways diseases work allows researchers to target trials more precisely, leading to better patient outcomes. However, the need for trials sometimes outpaces scientific understanding. “We can’t always wait for a full understanding of a disease,” James notes. “In some cases, we must move forward with trials to push the needle on patient care, even as we continue to explore the underlying mechanisms.” A key advantage for the Institute is its proximity to one of Australia’s largest tertiary hospitals, Royal North Shore Hospital. This connection provides access to a vast patient population, which is crucial for conducting large-scale clinical trials. “We serve over a million patients,” says James, stressing the importance of their “trial as therapy” culture. “We want patients to expect that they may be invited to participate in a clinical trial or research study as part of their care.” The Kolling Institute is a world leader in researching osteoarthritis, musculoskeletal conditions, cancer, neuroscience and pain, and cardiac, cardiovascular, and renal disorders.
Professor James Elliott
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A new era in cancer research is dawning at Northern Sydney Local Health District (NSLHD), with the opening of an international cancer clinical trials centre thanks to a generous $20 million donation from Greg Poche AO and the late Kay Van Norton Poche AO. Launch of global cancer clinical trials centre
The NORTH Sydney Trials and Research Van Norton Poche Centre, or NORTH S.T.A.R VNP centre, is an innovative partnership between NSLHD and New York’s Memorial Sloan Kettering Cancer Centre (MSK) and aims to revolutionise cancer treatment and research. Professor Bruce Robinson AC, Chair of Research at NSLHD, welcomed the donation, stating: “The generosity of this gift is extraordinary and will further increase the ability of local cancer patients to participate in clinical trials with a world-class cancer centre in New York.” NORTH S.T.A.R VNP will attract the next generation of doctors, scientists, and researchers to Australia through international fellowships and substantial funding dedicated to discovering cures for cancer. Set to operate out of Royal North Shore and North Shore Private hospitals at the Sydney North Health Precinct, the centre will be accessible to all Australians. Partnerships with regional NSW hospitals have already been established, ensuring wide-reaching impact. Initially, four clinical trials will be available for eligible patients living in areas including Sydney, Coffs Harbour, Port Macquarie, Armidale, Bathurst, Orange, Dubbo, and Gosford, with plans to expand enrolments nationwide. A key component of the centre’s vision is the creation of a national database to enhance collaboration among hospitals, doctors, and allied health professionals. This initiative aims to guarantee that every patient, regardless of location or treating physician, can participate in clinical trials, with a particular focus on rural and regional residents and First Nations people.
The generous $20 million donation marked the largest single donation ever made to the NORTH Foundation. Kay Van Norton and Gregory Poche AO made the landmark donation to the NORTH Foundation, the official fundraising partner of NSLHD, in December 2023. At the time, Kay expressed their desire to make a difference: “We hope this gift will attract more donors to show their support to help eradicate cancer as a major cause of death.” “It was through groundbreaking cancer trials from the US that Kay was able to extend her life by many years and she was determined that all Australians should have access to the same opportunities she had,” said Professor Robinson.
Australians can build upon this legacy and help to create a future without cancer by donating at: northfoundation.org.au/northstar
Greg Poche AO and the late Kay Van Norton Poche AO
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Advancing genetic disease research
Associate Professor Mimi Berman is head of Royal North Shore Hospital’s busy Clinical Genetics Department, where a dedicated team of genetic doctors, nurses and counsellors look after people diagnosed or suspected of having an inherited condition.
The department also specialises in treating neurofibromatosis, a complex and unpredictable disease that causes tumours to form on any nerve in the body. “It’s a very debilitating condition,” says Mimi, mentioning symptoms that range from pain and disfigurement caused by the thousands of tumours to learning and thinking difficulties. The tumours can also sometimes become cancerous. Mimi and her team are running several International clinical trials investigating the disease, which affects one in 3,000 people. One trial sees adult patients taking drugs known as MEK inhibitors to block tumour growth. “It is the current drug of hope,” she says. Another trial they are about to recruit for is the Strength Study, which investigates muscle weakness and fatigue in children aged 8-12 years with the disease. “It’s a big issue,” says Mimi. “It affects their ability to participate in sport and their ability to participate in educational activities as they become fatigued towards the tail end of the day.”
There are two major types of the disease: neurofibromatosis type 1 (NF1) and Schwannomatosis. NF1 is diagnosed at birth or during early childhood. Schwannomatosis is a related disorder and is more frequently diagnosed in adults aged 30 or older.
Associate Professor Mimi Berman (centre in blue) and some of the clinical genetics department team.
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Imaging is another area of research. One study uses 360-degree imaging to record changes in the thousands of tumours throughout a sufferer’s body. Another pilot study is conducting whole-body MRIs for young people, which looks at how much disease is in the body. “We want to detect tumours early and identify those with a higher risk of turning malignant,” she says. Around 80 per cent of people with neurofibromatosis will get through their life without their tumours becoming cancerous but many more suffer from progressive disfigurement. To provide support, the clinic also offers laser treatment and excision for the small lumps which can develop on the skin. The national clinic is also involved in world-leading research, including a collaboration with John Hopkins University in the US to develop ways to measure treatment outcomes. Mimi is overseeing other neurofibromatosis trials and remains optimistic about the path forward. “Despite the challenges, we’re committed to advancing treatments and support for our patients. There is so much opportunity here to improve the lives of people living with neurofibromatosis.”
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Trailblazing intensive care unit trials
For over three decades, critical care physician Dr Simon Finfer AO has led groundbreaking clinical trials with thousands of participants, shaping global patient care.
His journey began in 2004 with the publication of the first intensive care unit (ICU) mega trial in the world, involving 7,000 patients. This landmark study compared outcomes in patients received fluid resuscitation with either an albumin or saline solution. The study was spurred by an earlier Cochrane review suggesting increased mortality risk associated with albumin use in critically ill patients. “Our study was very influential in not only proving that albumin was safe to use in critically ill patients, but it also changed the landscape of people's thinking about what could be done in terms of proving or disproving the effectiveness and safety of treatments for critically ill patients,” he says. Since then, the Senior Staff Specialist at Royal North Shore Hospital has spearheaded numerous influential trials. In some studies — like in the case of a 6,100-patient ‘NICE-SUGAR study’ which involved controlling blood glucose with large doses of insulin — the results swiftly altered clinical guidelines worldwide. Conducting clinical trials with ICU patients is challenging due to issues around consent and the need to start treatment within minutes or hours. “The patients are usually profoundly unwell, they may be on ventilators, they may be sedated to tolerate such treatments, and you can't seek consent from them,” says Simon. Instead, consent is sought from family members or next of kin, a process which can be stressful for both staff and families and must be handled with care and sensitivity.
But the benefits are wide-reaching. “If we don't include critically ill people in trials, we don't have any other way to improve treatments,” he says, adding that the results will help patients around the world. “It is very gratifying because there is a lot of effort that goes into these trials.” The ‘NICE-SUGAR study’, for example, took eight years of planning, raising funding, setting up the processes, recruiting patients and analysing data. Simon fell into research as a young ICU clinician in the late 1980s and early 1990s, when he could see there was a lack of evidence for some interventions. While he has retired from clinical practice — he’s spent 42 years being on call nights and weekends — he continues to research, focusing on ongoing studies into sepsis. He's never tallied the exact number of trials he has conducted over the years but estimates it to be around 25 to 30, involving nearly 100,000 patients. “The goal has been to try to produce really robust, high-quality evidence to guide clinicians' behaviours in terms of how they treat patients, and therefore to standardise and improve treatment leading to more patients surviving and with a better quality of life.”
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The NICE-SUGAR trial ran in the ICUs of 42 hospitals , including Royal North Shore. This large, international, randomised controlled trial found that intensive glucose control increased mortality among adults in the ICU.
Dr Simon Finfer
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Pioneering partnerships Chair of Research Professor Bruce Robinson
For decades across NSLHD, we have conducted clinical trials in disciplines such as cancer, geriatrics, surgery, cardiovascular, diabetes, bone disease, and rheumatology.
By collaborating with patients, our clinicians and researchers have made life-changing discoveries, as well as stopping treatments that were found to be of little or no benefit to our patients. Our long-term goal has always been to involve more patients in clinical trials because of their extraordinary benefits. This vision is now becoming a reality as we enter a new chapter where clinical trials are the standard of care across the district.
We aim for every hospital in our district to run clinical trials, advancing science and providing care that patients might not otherwise receive. The advantages for patients joining clinical trials are immense. They can gain access to drugs that are not yet available through the Pharmaceutical Benefits Scheme, often before they are listed. These drugs can be prohibitively expensive, but on a trial, the treatment cost is completely covered by the pharmaceutical or device company. Additionally, the close monitoring during trials ensures any issues are swiftly addressed, making it a very safe way to receive care. To advance medical science requires partnership. Doctors and health-care professionals need to discuss trials with patients. The community must understand that participating in trials provides real benefits. Many drugs in clinical trials have already proven safe in phase 1 studies. Phase two trials test effectiveness, followed by larger phase III trials to confirm results. Most trials in our hospitals are in these advanced phases. By working together, we can transform clinical trials into a cornerstone of healthcare, benefiting patients today and paving the way for tomorrow’s medical breakthroughs. I look forward to making this new chapter a reality.
Professor Bruce Robinson
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Arthritis focus group shapes future research
At a large table, six individuals, each battling arthritis in various forms— be it in their knees, hips, toes, or feet—are gathered.
For the past five years, the OACR group has organised two to three focus groups annually, engaging individuals living with osteoarthritis. They are offered lunch and a gift card for their time. World-leading clinician researcher and head of OACR Professor David Hunter says consumers play a vital role in shaping medical research. “They’re key in making studies better, boosting recruitment and retention, ensuring ethical research, spreading findings, and pushing for implementation.” NSLHD is home to world-class clinical investigators and scientists. Our teams regularly run clinical trials and focus groups with consumers across all medical disciplines.
Over the next four hours, the Kolling Institute’s Osteoarthritis Clinical Research (OACR) team will unveil a clinical trial in need of volunteers and delve into multiple projects on which they are eagerly seeking feedback. Research Fellow and physiotherapist Dr Vicky Doung probes the group: Can a digital osteoarthritis coach app inspire better arthritis management? The My Joint Pain website fact sheets also come under scrutiny. What’s the verdict? Clinical Trials Program Lead Dr Karen Bracken asks if the advertisements for recruiting patients to a new clinical trial are clear. As the focus group banters, offers ideas, and delivers the occasional joke, researchers diligently take notes. Glenda Gartrell has joined various clinical trials, and this is her second time in a focus group. “I’ve been living with osteoarthritis for ages. Hearing others’ experiences is enlightening, and it reinforces my approach to managing my condition,” she says.
Knee osteoarthritis affects more than 650 million people worldwide.
To join an osteoarthritis clinical trial, go to: https://www.osteoarthritisresearch.com.au/
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Frontline work with older people shapes research
Professor Sue Kurrle and her team at the Rehabilitation and Aged Care Network at Hornsby Ku-ring-gai Hospital have been running clinical trials for 25 years, looking at dementia, frailty, falls and various other areas.
“We do trials as it keeps you cutting edge and they are really important for our patients,” she says. As a geriatrician on the frontline, Sue is well- positioned to identify the critical areas that need attention. “You want to do the research that helps answers some of the questions that your clinical practice throws up,” she says. Falls research Sue says one of the most impactful trials she participated in was during her PhD research on hip protectors for older people. Her study found that the underwear with built in plastic “shields” stopped people from breaking or fracturing their hips when they fell.
“I got involved in hip protectors, because we were looking after so many hip fracture patients in our rehab ward here at Hornsby,” she says. “Ditto with dementia and with frailty. Everything we’ve done in research has been driven by what you see in clinical practice.” Dementia With an ageing population in the area, Sue and the team have had a long interest in dementia. Since 1999, they have run 56 trials for dementia drugs, some of which are part of global studies. Although most haven’t been especially successful, Sue says a recent tablet is promising, with the results soon to be published.
Professor Sue Kurrle (left) and the Rehabilitation and the Aged Care team
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“The biggest message is that exercise is much more effective than medication in slowing the decline once you have dementia, or in delaying its onset. It’s one of the 12 modifiable risk factors ,” she explains. In 2012, the NHMRC awarded her $25 million for the Cognitive Decline Partnership Centre, which funded 32 projects across the country which looked at care for people with dementia. “We developed the clinical practice guidelines for management of dementia, which were the first ones ever done in Australia,” she says. Frailty The frailty intervention trial (2011 to 2013) involved 241 frail Hornsby residents. One group received exercise and nutrition interventions, while other received normal care. The experimental group saw numerous benefits. “We turned frailty around,” she says, adding that the research is now being implemented in hospitals throughout Northern Sydney, with another frailty study soon to be published. Clinical trials can last up to five years, and older patients often bring a relative along. Sue and her colleagues, including clinical trials research coordinator Roseanne Hogarth and clinical nurse specialist Bronwyn Cook have developed close relationships, knowing their families, pets, and even sharing coffee outings. “They are like family,” says Sue. “The advantage of working in a smaller hospital is we really get to know our patients.”
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Embedding clinical trials in patient care Executive Director of Research Associate Professor Naomi Hammond I have been deeply involved in intensive care research and clinical trials for the past 20 years, witnessing the extraordinary impact they can have on patient care and outcomes.
My background is in nursing, and I have seen how some treatments and healthcare practices that were commonplace early in my career have either been abandoned due to causing harm or replaced due to discoveries made in clinical trials. It is crucial that we continue to question and improve how we conduct clinical practice. The clinical trials I have been involved with have had both local and international impact. It is incredible to know that research conducted at Royal North Shore Hospital has improved mortality rates and patient outcomes around the world.
As the inaugural Executive Director of Research at NSLHD, one of the key elements of my role will be refocusing our efforts to support and promote research, with a particular emphasis on expanding our clinical trials, which is a priority for the district. I am excited to lead our district to embed research and clinical trials within our clinical care. It will be an era where participation in clinical trials or research studies is a standard option for everyone entering our health service. This approach will ultimately enhance patient outcomes and benefit the broader community and future generations. I want to thank all the clinical trials staff who have been engaged in running trials, which can take years of work and require enormous dedication. I also thank our patients, their families, and the community at large for walking with us and participating in the trials we offer. Together, we can make significant strides in medical research and clinical practice. I look forward to the journey ahead and the advancements we will achieve together.
Associate Professor Naomi Hammond
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Nine researchers have been announced as winners of scholarships to study Australia’s first Graduate Certificate of Clinical Trials Operations in a special ceremony. Celebrating clinical trials scholarship winners
The NORTH Foundation awarded scholarships to the nine Northern Sydney Local Health District clinical trials staff members to undertake the new course at Macquarie University. Yue Zhao, a scholarship recipient and senior Clinical Research Coordinator in endocrinology at Royal North Shore Hospital, said there was a training gap in the market when it came to clinical trials. While she has received on-the-job training, she said she was eager to learn more. “I want to be able to extend myself and have some proper training.” Erin Li, a clinical trials coordinator in cardiology, said the scholarship would give her the opportunity to learn more about regulations and the whole clinical trial process in general. “It will benefit the hospital and the patients.” Dr Teresa Lee Chang, a lymphoedema physiotherapist, has worked as a clinical trial investigator, but is looking forward to learning how to manage a trial.
“I normally outsource the management aspects to other people, but I want to know what goes on to have a fully sponsored or investigator-led trial,” she said. NSLHD Chief Executive Anthony Schembri welcomed the opportunity for staff and said clinical trials were about “bringing tomorrow’s medicine today.” “Clinical trials are about ensuring that we embed the very best treatments in ordinary care for our patients, and that we strive for the very latest in medical and healthcare treatments,” he said. Professor Patrick McNeil, Deputy Vice- Chancellor (Medicine and Health) at Macquarie University, said clinical trials were a win-win for patients and researchers. “Most importantly, they allow our patients to access devices that are cutting-edge and not yet approved,” he said. The NORTH Foundation provided 80% of the funding for the new scholarships, while Macquarie University contributed 20%.
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Transforming care for vulval health
When Professor Gayle Fischer OAM started as a dermatologist, she made a promise to herself to sort out as many unanswered questions about vulval disease as she could before she retired. Thirty years later, she has realised her life plan.
Gayle laments the stigma and shame that people feel when they have a skin condition affecting their genitals. “Any condition on the skin can involve genital skin but when it’s the genital area – somehow it becomes much more of a problem to treat.” She is proud of her 10-year-long lichen schelorus research, which investigated the long-term outcomes of 507 women using a corticosteroid cream. This showed that regular preventative treatment could change the course of the disease and result in good clinical outcomes. It was published in the prestigious JAMA Dermatology journal 2015 and led to the creation of new Australasian guidelines on how to treat the condition. Gayle says research can be a challenging space, particularly when it comes to discoveries. “It’s hard to change people’s minds. People tend to resist change, but eventually if you are persistent, they come to embrace it,” she says. After decades of
The head of the Dermatology Department and principal paediatric dermatologist at Royal North Shore Hospital is a global authority in vulvovaginal diseases. She co-authored ‘ The Vulva: A Practical Handbook for Clinicians ,’ a seminal textbook in the field. But significantly, she is renowned for her expertise in lichen sclerosus—a distinctive skin condition on the vulva known for scarring and potential cancer risks. Her groundbreaking decade-long clinical trials into the disease have significantly impacted the field. Gayle’s interest in vulval skin problems began, when as a young consultant, she set up a practice in Penrith as there were so few dermatologists in the area. Local GPs were soon sending her patients with vulval problems. “I think they had tweaked they were skin-related and not gynaecological,” she says. Patients were presenting with vulval psoriasis and eczema, as well as lichen sclerosus, the condition for which she was to become a renowned expert. She fell into research when Professor Bryan Spurrett, an obstetrician and gynaecologist at Nepean Hospital, called and asked her to collaborate He’d heard through the grapevine that she was successfully treating people using basic principles of dermatologic treatment. Together, they studied what vulval conditions led women to consult a doctor and set out to disprove the concept of HPV (human papillomavirus) vulvitis, which at the time was a devastating diagnosis for many women. “They were led to believe they had an incurable sexually transmitted condition,” she says. The pair’s work eventually demonstrated that HPV vulvitis was no more than a myth.
groundbreaking research and compassionate care, Gayle continues to run several clinical trials.
Patients with dermatological conditions suffer a reduced quality of life.
They often suffer from low self-esteem, low confidence and embarrassment. Their
conditions can impact them at school and in the workplace.
Professor Gayle Fischer
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Empowering patients on clinical trials
In her 25-year career in clinical trials, Linda Pallot, a registered nurse, has worked on trials in neurology, gene therapy and vascular surgery. What has struck her repeatedly has been the benefits they bring. Beyond the innovation, she has always enjoyed seeing what positive outcomes can occur when patients consent to participate in a clinical trial. “I love that trials provide new treatment options. They have the potential to improve health when no other treatments are available, and participating in a clinical trial can help patients learn how to better manage their health and hopefully improve it,” she says. Linda is currently a Senior Clinical Trial Coordinator in Vascular Surgery at Royal North Shore Hospital. She has observed that young participants in trials take a keen interest in their new treatments. “I like that we are educating them about their health.” Older participants, who are less familiar with clinical trials, are equally eager to participate, especially when it provides them with specialised care and more prompt treatment. Throughout her career, which has included stints at other hospitals, Linda has witnessed Multiple Sclerosis (MS) treatments reaching
It can take around 10 years to demonstrate a treatment being tested in a clinical trial is safe and effective. “In my clinical trial career, if a treatment does make it to market, it’s incredibly exciting to think that you have been a small part of a process that could help so many people around the world,” she says. The department currently runs trials involving new devices and conducts research into improving surgical practices. Clinical trials provide a variety of health benefits. Each participant’s journey is unique, offering access to innovative treatments, expert medical attention, and careful monitoring by dedicated research teams. “What more could you possibly want when no other treatment is available?” Linda encourages anyone presented with a research opportunity to embrace it and discover its potential.
the market thanks to clinical trials. She has also managed treatments for gene therapies for haemophilia and thalassemia, which continue today.
Debbie Knagge, Linda Pallot and Clare Mundell
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HISTORICAL VIEWPOINT Shaping the future of diabetes treatment through trials at RNSH By Catherine Storey OAM – Honorary Archivist RNSH
In 1923 the Institute of Pathological Research opened on the grounds of RNSH in a small workman’s cottage, “Oakleigh”. Dr William Wilson Ingram, a young Scottish physician/pathologist was appointed the first Director.
On the other side of the world in Toronto, Canada, researchers Banting, Best, Macleod and Collip had discovered insulin in 1921. The team first used the untrialled substance on a young boy dying of diabetes mellitus in January 1922 with miraculous results.
When news first reached Australia, the editor of the Medical Journal of Australia cautioned against accepting the findings without further research. He felt that there had not been sufficient experimental clinical data to warrant its widespread acceptance. Where were the trials? However, by October 1923, Banting and Macleod received the Nobel Prize in Medicine for their discovery.
By mid-1923, CSL received the licence to manufacture insulin in Australia and began local production. However, the use of the product was initially restricted. When the Director General of Health announced that only certain hospitals (those who could perform a blood sugar level and had a recognised metabolic specialist to oversee its use) would receive a supply of insulin, RNSH was not on the list. Dr Ingram then employed two local medical graduates, Dr Elsie Dalyell and Dr Beatrix Durie, as research assistants to make his own insulin! When insulin became more freely available, the young female graduates continued further experiments into the management of diabetes.
Dr Ingram established one of the first diabetic clinics in Australia at RNSH.
28 Tomorrow’s medicine today
Perhaps one of the earliest clinical trials performed at RNSH was to disprove the use of prickly pear as a treatment for diabetes, a popular misconception. In the Medical Journal of Australia in 1930, Dr Ingram published the results of his carefully conceived and conducted clinical trials. In one experiment he used the extract of prickly pear given to a normal rabbit to see whether there was any change in the Glucose Tolerance Test. He failed to show any active ingredient in prickly pear. Then, with a series of diabetic patients, using an oral extract he showed no demonstrable effect on the blood sugar levels. So, it is likely that the first clinical trial at RNSH was to show that prickly pear was not a useful adjunct in the treatment of diabetes mellitus. Dr Ingram continued his research into diabetes and in 1933, he published a monograph titled “ The Diagnosis and Management of Diabetes ,” which proved to be extremely popular.
Oakleigh was one of the cottages acquired in 1920 to house the Institute of Pathological Research. It was later demolished to make way for the new nurses’ home, Vindin House. [ courtesy of Heritage and Archive Collection RNSH ]
William (Bill) Wilson Ingram during his first expedition with BANZARE to Antarctica 1929-1930 [ courtesy of Heritage and Archive Collection RNSH ]
The pathology lab in Oakleigh in 1925
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