Incredible life-changing medical breakthroughs Qld scientists made in 2023
From an aspirin-inspired boost for cancer patients, to possible preventions for childhood asthma and irreversible blindness, Queensland scientists continued to lead the way in medical advances in 2023. Here are their biggest breakthroughs.
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Queensland scientists lead the way in scientific and medical breakthroughs with 2024 likely to be another standout year for the researchers at QIMR Berghofer.
Last year they made extraordinary advances in the understanding, diagnosis and treatment of serious diseases such as leukaemia, heart disease, bowel cancer, obsessive-compulsive disorder, COVID-19, multiple sclerosis, and ovarian cancer.
Researchers’ studies and papers have been published in a number of prestigious publications, including Nature Genetics, Nature Communications, Nature Cancer, Nature Mental Health, Cell Reports, BMJ and the Journal of the National Cancer Institute.
These papers investigate everything from glaucoma genetics to maternal diet and childhood asthma risk, to the genetics of binge eating disorder, vascularisation of heart organoids and the benefits of vitamin D for heart health.
Mid-year, the globally recognised Nature Index ranked QIMR Berghofer in the top 40 not-for-profit science institutions worldwide, one of only two Australian institutes to make the top 40.
These are some of the life-changing projects.
FEBRUARY 2023: SIMPLE ASPIRIN’S POSSIBLE CANCER BOOST
The year began with a bang when new QIMR Berghofer research found that low-dose aspirin may improve ovarian cancer survival.
The study followed more than 900 Australian women newly-diagnosed with ovarian cancer, and asked them how often they used nonsteroidal anti-inflammatory drugs (NSAIDs) such as aspirin.
Lead researcher Dr Azam Majidi said the women who reported taking NSAIDs at least four days a week in the 12 months after diagnosis, lived longer on average than occasional or non-users.
“Our findings suggest that frequent NSAID use might improve survival for women with ovarian cancer, regardless of whether they start taking the drugs before or after diagnosis,” Dr Majidi said.
“We found the difference would translate to an average of an extra 2.5 months’ survival in the five years post-diagnosis. While this might not sound like a lot, it is significant for ovarian cancer. The disease is often diagnosed at an advanced stage when the prognosis is poor, and treatment options are limited.”
MARCH: “POO TRANSPLANT” TRIAL GIVES HOPE TO CANCER PATIENTS
The immune-boosting power of a healthy gut microbiome was harnessed in an Australian-first “poo transplant” trial aimed at saving the lives of severely ill blood cancer patients and meeting an urgent need for new treatments.
Faecal microbiota transplant (FMT) is emerging as an exciting potential treatment for a range of conditions. Trillions of gut microbes from the stool of a healthy donor are transferred into a patient to replenish their depleted gut microbiome and improve their immune response to fight disease.
In March, FMT was trialled in Australia in blood cancer survivors who had developed severe Graft-versus-Host-Disease (GVHD), which was caused by their lifesaving bone marrow transplant when donor immune cells attack the recipient’s organs and tissues.
QIMR Berghofer Researcher and RBWH Haematology and Bone Marrow Transplantation Specialist Dr Andrea Henden said new treatments are urgently needed.
“A bone-marrow transplant can be a double-edged sword. It saves the lives of people with aggressive blood cancers but can then take their lives by causing GVHD which is heartbreaking,” Dr Henden said.
“GVHD is particularly hard to treat in the gut with patients often hospitalised for long periods of time where they are dependent on hospital care and intensive immune suppressing medications.
“Conventional immune-suppressing steroid medication fails to work in half of all GVHD patients.
“FMT is a really exciting new approach that could save lives. If we can restore a healthy gut
microbiome in these patients we can potentially influence immune function in the gut and treat the GVHD in a safer way that preserves the patient’s immunity,” Dr Henden said.
APRIL: NEW CLUES ON POTENTIAL CAUSE OF OCD
QIMR Berghofer researchers moved closer to solving what causes obsessive-compulsive disorder, after discovering changes in how distinct brain regions communicate. This important finding could guide the development of more targeted and effective treatments for this debilitating condition impacting thousands of Australians.
In their new study, the researchers confirmed that the disorder likely emerges from a complex
imbalance affecting particular signalling pathways deep within the brain.
Senior researcher, Associate Professor Luca Cocchi says the information will be instrumental to QIMR Berghofer’s efforts to develop innovative brain stimulation treatments for OCD – a condition with limited treatment options and no known cure.
“This study offers some important clues as to how we might research and develop treatments that better target OCD and its symptoms,” A/Prof Cocchi said.
The QIMR Berghofer researchers used the findings to inform clinical trials of a range of non-invasive and invasive brain stimulation therapies for OCD during the year.
APRIL: TINY HEARTS BREAKTHROUGH
An Australian research team led by QIMR Berghofer succeeded in introducing a vascular system into tiny living and beating model human heart muscles, an achievement which it’s hoped will accelerate progress towards the ultimate goal of repairing damage from heart disease.
The new vascularised tiny heart muscles, or organoids, more closely mimic the human heart and will allow much more accurate testing of new drugs to treat disease and inflammation, and take scientists a step closer to the holy grail of repairing heart tissue.
Lead researcher Professor James Hudson, who heads QIMR Berghofer’s Cardiac Bioengineering Research Group, said vascularising the tiny hearts is a game changer for their work.
“Each organoid is only about the size of a chia seed, measuring just 1.5 millimetres across, but inside are 50,000 cells representing the different cell types that make up the heart,” Professor Hudson said.
Organoids are grown from human pluripotent stem cells which can be generated using
“reprogramming” of skin or blood cells. Until now, the model hearts included a range of cell types including the cells that hold the tissue together and the cells that make them beat, but researchers had not been able to add the critical vascular cells.
“Incorporating the vascular cells for the first time in our mini heart muscles is very significant because we found they had a key role in the biology of the tissues. Vascular cells made the organoids function better and beat strongly. This has really opened up our ability to better understand the heart and accurately model disease,” Professor Hudson said.
MAY 2023: MAJOR DEVELOPMENT FOR CANCER-KILLING ‘MASTERSWITCH’ DISCOVERY
QIMR Berghofer’s ‘Masterswitch’ technology that unleashes the immune system against two deadly types of cancer moved closer to the clinic in May with the Institute’s biotech spin-out Fovero Therapeutics receiving prestigious CUREator funding.
The ‘Masterswitch’ is an antibody that has produced remarkable preclinical results against triple- negative breast cancer and the most common form of bowel cancer, Micro Satellite Stable (MSS) bowel cancer. Treatment options and outcomes for patients with these diseases are currently very poor.
QIMR Berghofer’s Group Leader of Molecular Immunology Associate Professor Michelle Wykes’s discovery turns on a key type of immune cell called dendritic cells which in turn activate the body’s critical T cells to recognise and attack cancer.
“Cancer cells are very good at hiding from the immune system but our ‘Masterswitch’ antibodies make the cancers visible again, so the dendritic cells can go back to work and ‘organise’ the T cells to kill the cancer,” A/Prof Wykes said.
“We’re seeing palpable tumours that completely disappear and melt away. In our preclinical lab models, 80 per cent of both the triple negative breast cancers and bowel cancers were cleared and hadn’t grown back after ten months. We’re seeing similar results from our tests on samples takenfrom patients with bowel cancer,” she said.
JUNE: DRUG COULD TREAT LONG Covid AND PREVENT RE-INFECTION
Mid-year, QIMR Berghofer announced the development of a new drug which is set to transform the treatment of COVID-19 by potentially protecting against infection by any SARS-CoV-2 variant and reversing the persistent inflammation that is a major driver of debilitating long Covid.
Epigeneticist and co-lead author, Professor Sudha Rao who heads QIMR Berghofer’s Gene Regulation & Translational Medicine Group, said the peptide-based drug, NACE2i, was tested repeatedly by independent laboratories using a variety of preclinical models.
“The results of this second major study are really exciting. It shows our drug, NACE2i, stops the virus replicating and protects against reinfection,” Professor Rao said.
“We believe it could be a highly promising adjuvant to boost the effectiveness of existing vaccines providing long-lasting protection against any variant of the virus that tries to enter the cells.
“The other major discovery is that we uncovered the pathway that the virus uses to induce the
persistent inflammation which causes organ damage found in long Covid.
“This study shows our drug prevents that inflammation and even repairs damaged lung tissue in pre- clinical models. It is both a prevention and a treatment.”
JUNE: BENEFITS OF VITAMIN D TO PREVENT HEART ATTACKS
A major QIMR Berghofer clinical trial, the D-Health Trial, found vitamin D supplements may reduce the risk of cardiovascular events such as heart attacks in people aged over 60.
The researchers emphasised that the findings are not conclusive, but that they warrant further
investigation, particularly for patients who take heart disease medications such as those used to treat high blood pressure or high cholesterol.
Lead author and epidemiologist Professor Rachel Neale, who heads QIMR Berghofer’s Cancer
Aetiology & Prevention Group, said the trial was the second-largest of its kind to date to investigate whether vitamin D could reduce the risk of cardiovascular events.
“Our trial found vitamin D supplementation may reduce the risk of major cardiovascular events, and the protective effect could be more marked in those taking statins and other heart disease drugs. It does suggest that further research into this is needed,” she said.
JULY: GENETIC DISCOVERY COULD HELP PREVENT IRREVERSIBLE BLINDNESS
International research led by QIMR Berghofer found hundreds of new genes linked to a person’s risk of developing glaucoma, including key genetic targets that could, for the first time, pave the way for treatments that prevent the retinal damage that causes blindness.
The research significantly advances our understanding of the genetics of glaucoma, building on a previous 2021 study to identify another 185 previously unknown genes linked to glaucoma risk, bringing the total number to 312 genes.
Glaucoma is the leading cause of irreversible blindness globally affecting more than 75 million people around the world, including 300,000 Australians. Around 50 per cent of all glaucoma cases aren’t diagnosed until permanent optic nerve damage has already occurred, so early diagnosis and treatment is vital.
Lead researcher and internationally-recognised genetic epidemiologist Professor Stuart
MacGregor who heads QIMR Berghofer’s Statistical Genetics Laboratory, said the discoveries could rapidly accelerate a new approach to treatment.
“Existing treatments focus only on lowering eye pressure. The dream has always been to find a way to make the retina itself stronger so it can withstand the build-up of pressure and prevent the damage that causes permanent blindness,” Prof Stuart MacGregor said.
“Our findings are really exciting because for the first time we’ve discovered the set of genes that could be targeted to induce this ‘neuro-protection’ in the retinal cells.
“We’ve also identified existing drugs that could be used on those genetic targets. This could rapidly advance effective treatment to finally prevent retina and optic nerve damage.”
The findings helped guide the development a groundbreaking genetic test to predict a person’s risk of developing glaucoma, which is currently in a large-scale clinical trial.
JULY: MATERNAL DIET COULD REDUCE CHILDHOOD ASTHMA RISK
In July, new research from QIMR Berghofer showed a high-fibre diet in breastfeeding mothers could potentially protect infants from serious respiratory conditions such as asthma.
In a preclinical study, researchers demonstrated that a diet rich in fibre helped reduce the risk of babies developing severe lower respiratory infections (sLRIs), which can predispose the infant to chronic lung diseases.
Senior researcher, Associate Professor Simon Phipps said the study’s finding was significant because it suggested diet affected the health of a mother’s breastmilk, which was key to triggering a baby’s immune resistance to sLRIs.
“Poor maternal diet increases the incidence of severe lower respiratory infections, such as
pneumonia and bronchiolitis, in an infant. These infections are a major cause of infant morbidity worldwide, and increase childhood asthma risk,” said A/Prof Phipps.
“In preclinical models, we found that dietary fibre affects the composition of the mother’s milk microbiota, which in turn affects the development of the infant’s gut microbiota.
“When a breastfeeding mother eats a high-fibre diet, her healthy milk microbiota kicks off a process that promotes the development of an important population of immune cells. We have identified that the microbes talk to the cells that line the gut, and these cells then produce a growth factor that supports the immune cells in the bone marrow. These immune cells then protect the infant against severe lower respiratory infections,” explained A/Prof Phipps.
AUGUST: VACCINE BREAKTHROUGH OFFERS HOPE AGAINST MS
A cutting-edge vaccine candidate developed by researchers at QIMR Berghofer could potentiallyprotect against a common virus thought to be a leading cause of multiple sclerosis (MS) and various cancers.
Epstein-Barr virus (EBV) is carried by about 95 per cent of the population, with most of us unaware it lies dormant in our bodies. However, in some people the virus can lead to severe illness.
QIMR Berghofer’s vaccine candidate potentially offers a breakthrough approach that combines two powerful arms of the immune system to target EBV, and hopefully prevent associated conditions like MS, Hodgkin’s lymphoma and nasopharyngeal cancer.
In promising early findings, the vaccine has achieved potential and durable immune protection
against EBV in preclinical models.
“For decades, QIMR Berghofer has been researching the role of EBV in causing multiple diseases including cancers. It is a really proud moment for us to see all of this work coming together, with this vaccine now heading towards the next important stages of development,” said lead author, Dr Vijayendra Dasari.
OCTOBER: POTENTIAL NEW ARSENAL TO TARGET LEUKAEMIA AND OTHER CANCERS
This year a QIMR Berghofer-led team of international scientists had an exciting ‘eureka’ moment when they potentially unlocked an entirely new approach to targeting acute myeloid leukaemia.
Their findings, published in the journal Nature Cancer, are highly significant not only for advancing the treatment of blood cancers but potentially other types of cancer too.
The research team led by Dr Claudia Bruedigam and Professor Steven Lane made their unexpected discovery while investigating a new class of drug, imetelstat, against leukaemia cancer cells in the laboratory.
They found the drug triggers a certain type of cell death in leukaemia samples, with the study also offering insight into the biological process involved.
“This is very exciting because it essentially means we potentially have a new option to kill blood cancer cells and with this, a new explanation for how the drug works,” says Professor Lane.
“This could transform the way we think about treating patients with blood cancers, especially those who have run out of options.”
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