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Winter Edition - June 2019
Winter Edition - June 2019
In this edition:
- Soothe the itching
- Turn your $1 donation into $7 for research this tax time
- For heart disease, stem cells could make a difference
- Snore no more
- Meet the man at the heart of MCRI
- What happens when IVF babies grow up?
- Recognition for an international star
- Adding muscle to the hunt for answers
- Global collaboration for a global problem
- Bring your genes to the party
Soothe the itching
With just one round of treatment, researchers from MCRI have collaborated to knock out two skin diseases in the entire population (26,000+) of Choiseul Province of the Solomon Islands. And their work is now a finalist for the $10 million Macquarie 50th Anniversary Award that recognises research targeting social need.
The two skin diseases – scabies and impetigo – currently infect hundreds of millions of people, mostly in tropical countries. Together, the work of researchers from MCRI, the Kirby Institute of New South Wales, the Solomon Islands Ministry of Health and the London School of Hygeine and Tropical Medicine saw a reduction in cases of almost 90 per cent a year after treatment.
Scabies, a neglected tropical disease, causes extreme itching and leads to bacterial infection of the skin and other organs. It affects an estimated 200 million people worldwide.
Mr Oliver Sokana, from the Solomon Islands Ministry of Health and Medical Services, explains the skin diseases’ impact. “Scabies has the greatest impact on young children and the severe itching can also impact on their attendance and attention in class,” he says. “Both diseases are most common in children aged between five and nine years old.”
MCRI Professor Andrew Steer says the study found that administering two medications (ivermectin and azithromycin) together to a whole population is highly effective at reducing the numbers of people affected by scabies and impetigo.
Prof Steer said significant reductions were also seen in local clinic attendance for skin sores, boils and skin abscesses, the tropical disease yaws, acute respiratory infections and diarrhoeal disease.
Before the study began, the rates of scabies and impetigo for the people of Choiseul Province were 18.7 per cent and 24.8 per cent respectively.
Lead author on the paper, The Kirby Institute’s Dr Lucia Romani says scabies is especially common in rural and remote areas of tropical developing countries, where people share small living and sleeping spaces and access to treatment is limited.
“Our findings show that a simple intervention can have a major impact on a serious health issue that has been too long ignored,” Dr Romani says.
The study was funded by the International Trachoma Initiative; the Murdoch Children’s Research Institute; the Scobie and Claire Mackinnon Trust, Australia; and the Wellcome Trust.
The Solomon Islands result forms the basis of MCRI’s Macquarie Award application for the World Scabies Elimination Program. If successful, the award will deliver $10 million over five years to administer oral ivermectin to treat scabies in large populations.
Read more about the Scabies control program
Turn your $1 donation into $7 for research this tax time
Your donation is vital to seed new and ground-breaking research. When you support MCRI, your dollar goes further for child health research. Last year we turned every $1 of your support into $7 for research through additional competitive funding.
For heart disease, stem cells could make a difference
Nine out of every 10 new drugs developed to stop heart disease fail when they reach the point of being tested in humans.
Billions of dollars and years of research goes down the drain when a new drug fails, but a new development from MCRI’s stem cells researchers is set to revolutionise the process.
A four-year study led by researchers from QIMR Berghofer Medical Research Institute, MCRI and the University of Melbourne, in collaboration with researchers from global biopharmaceutical company AstraZeneca, has used bioengineered human heart muscle to conduct the first ever screening of potential heart regeneration drugs.
The study used thousands of miniature heart muscles grown in the lab, which beat and behave like a human heart, says lead author and head of QIMR Berghofer’s Organoid Research Laboratory, Associate Professor James Hudson.
“These mini heart muscles have similar properties to a functioning human heart and they seem to respond to drugs in a similar way,” A/Prof Hudson says.
AstraZeneca had shared more than 100 compounds with the Australian researchers for them to test in the mini-heart muscles.
Co-lead author Associate Professor Enzo Porrello from MCRI and The University of Melbourne says the group has identified two compounds from the 100 that might help repair hearts.
“Using the mini heart muscles in a dish we were able to eliminate many compounds that didn’t work effectively, or were damaging or toxic, and we ended up finding two potential new drug candidates that may help regenerate damaged heart tissue,” A/Prof Porrello says.
“It’s very early days, and there are years of testing ahead, but our research provides hope of finding therapeutics that could regenerate disease-damaged hearts.”
A/ Prof Hudson says the screening model also has the potential to make drug testing cheaper, quicker, easier and more accurate.
“We can make hundreds of heart muscles in the lab each week and use them to discover treatments that may be beneficial for patients with heart failure, thereby reducing our reliance on using mice and keeping testing costs down,” he says.
The study was funded by QIMR Berghofer, the Murdoch Children’s Research Institute, The University of Melbourne, The University of Sydney, NHMRC, Heart Foundation, Stem Cells Australia and The Royal Children’s Hospital Foundation.
Snore no more
Each year, more than 40,000 people in Australia have their tonsils taken out, making it the most common elective childhood operation in Australia. Now, a trial by researchers at MCRI could significantly reduce this number with a simple nasal spray.
“Surgery is a big deal for any child,” says lead researcher Dr Kristen Perrett from MCRI. “It requires an anaesthetic, can be very painful and there are risks of bleeding. In addition, waiting lists for this surgery in some public hospitals are many months to years.”
Dr Perrett says that, in childhood, the most common treatment for snoring is to surgically remove the adenoids and tonsils. The trial will test whether an anti-inflammatory nasal spray could help relieve the snoring and let those children avoid surgery.
Associate Professor Gillian Nixon, a paediatric respiratory and sleep physician overseeing the trial at Monash Children’s Hospital, says the team is optimistic the nasal spray may be a simple, safe alternative to surgery in many children.
To find out if your child might be eligible for the study, read more at the MIST trial
Meet the man at the heart of MCRI
It was while studying medicine that Dr Michael Cheung became inspired by the idea of working in research.
The prospect of developing new techniques to answer important clinical questions has led him to a long career investigating the health of kids’ hearts all over the world, from Great Ormond Street Hospital in London to Toronto’s Hospital for Sick Children and now, Melbourne.
Michael is Director of Cardiology at The Royal Children’s Hospital and also leads the Heart Group at MCRI. His roles mean he is close to the research, and to the children he is trying to help.
There are many projects on the go, all trying to understand how to best protect the cardiovascular health of children. One of the most exciting projects is looking at the use of a drug to protect the heart during cardio-pulmonary bypass surgery. Other projects include looking at the long-term cardiovascular risk in patients, including children born prematurely, and developing new techniques to assess blood flow in complex circulations.
“My role is quite varied which keeps it interesting with a mix of clinical cardiology, management responsibilities as well as the research. The campus is a great place to work due to the broad extent of expertise and energy amongst the staff.”
What’s left of Michael’s spare time is filled by studying a Masters of Business Administration, providing a taxi service to his kids and also keeping fit.
“I’m hoping that I might be able to take up tennis lessons – again – when the MBA finishes!”
What happens when IVF babies grow up?
The first Australian IVF baby was born in 1980. Since then, more than 200,000 babies have been born in Australia with the help of assisted reproduction technology.
“One in every 25 Australian babies is now born via assisted reproduction technology, which means there is one in every classroom,” says lead author of the study Professor Jane Halliday.
“Given the strong uptake of assisted reproduction technology, studies into the technology’s potential long-term health outcomes are vital.”
Prof Halliday’s trial found that Australians born through IVF conception are just as healthy as people conceived naturally.
“The study showed there is no evidence of increased vascular or cardiometabolic risk such as heart disease and diabetes, growth or respiratory or wellbeing problems in this assisted reproduction technology group, compared with a non-assisted reproduction technology group recruited from the same Victorian population,” Prof. Hallidays says.
The participants in the trial were adults who were among the first Australians to be born through IVF conception. As babies, they were some of the first IVF conception children in the world.
Co-author Associate Professor John McBain, from Melbourne IVF and The Royal Women’s Hospital says that, having worked in the field since before an IVF baby was born, “it is enormously satisfying that our hard work and our patients’ trusting participation in these early days have been rewarded by the positive findings.”
Find out more about all the measures that were taken in the study on MCRI news
Recognition for an international star
Ongoing contributions to kidney research have seen MCRI’s Professor Melissa Little receive the prestigious Alfred Newton Richards Award at the World Congress of Nephrology.
The biennial award recognises Prof Little’s “superb and sustained achievements” in nephrology research over many years, particularly her work in developing mini-kidneys and regenerative treatments for kidney disease.
“I am very proud to receive this esteemed award,” Prof Little says. “I’d like to thank the committee for this recognition and also take the opportunity to pay tribute to the dedicated and passionate researchers who work alongside me at MCRI.”
Prof Little’s research has looked, at the level of the genes and cells, to see how the kidney normally forms.
With this knowledge, she is focusing on generating mini-kidneys from patient stem cells to use in drug screening and disease modelling.
In the long term, she hopes to explore the possibility of growing a new kidney from stem cells to help address the shortage of donor organs as well as transplant complications such as donor organ rejection.
Adding muscle to the hunt for answers
Muscular dystrophies are caused by gene errors passed on to a child by their parents. While the condition varies from person to person in form and severity, MD causes muscle weakness and has a major impact on individuals and their families.
No specific treatments exist for the conditions, but a new research fellowship backed by the Muscular Dystrophy Association of Australia is adding weight to the search for answers.
The inaugural MDA Fellowship has been awarded to MCRI’s Dr Chantal Coles who completed her PhD research on the development of fat and muscle in cows.
Dr Coles is now creating muscle cells out of induced pluripotent human stem cells, with her ultimate aim to develop a 3D mini-organ. Developing a muscle organoid would allow researchers to better study organ development, disease and potential treatments.
“I’m incredibly grateful to be the inaugural MDA Research Fellow and I hope that my research can lead to meaningful outcomes for families in the future,” Dr Coles says.
MDA Executive Director Boris Struk says the fellowship reinforces the organisation’s commitment to research and being part of the solution.
“MDA is passionate about research and the potential it has to influence the outcome for people affected by this devastating muscle destroying disorder,” Mr Struk says.
Head of MCRI’s Office of Research Professor John Bateman says the fellowship builds on MDA’s existing relationship with the institute.
“Hopefully the establishment of a named fellowship marks the next chapter in our shared journey,” Prof Bateman says.
Global collaboration for a global problem
A child having a prolonged epileptic seizure is one of the most frightening situations a parent can imagine.
In hospital emergency departments, prolonged epileptic seizures are the most common neurological emergency seen. These seizures are potentially fatal: up to five per cent of affected children die, and a third suffer long-term complications from brain damage.
The longer the seizure, the greater the chance of long-term complications.
A new Australia-New Zealand study is giving emergency medicine doctors a better way to treat these children.
The study’s senior author, MCRI’s Professor Franz Babl, who is also the inaugural Professor of Paediatric Emergency Medicine at the University of Melbourne, says the study would profoundly improve treatment for children around the world.
Stuart Dalziel, who is Professor of Emergency Medicine and Paediatrics in the Departments of Surgery and Paediatrics at the University of Auckland says the study has provided emergency doctors with clear guidelines for treating children experiencing severe seizures.
“This study has now given us robust evidence to manage children with prolonged seizures without reverting to intubation and intensive care,” says Prof Dalziel, who is also a paediatric emergency medicine specialist at Auckland’s Starship Children’s Hospital.
Bring your genes to the party
In Australia, more than 240,000 people have a stutter.
Researchers at MCRI are now searching for more than 3000 of them to be part of a ground-breaking new study.
By studying their genetic information, researchers want to identify the genes that play a role in stutttering. With that information, they could develop better treatment and even prevent this very common speech disorder.
Participation is free and easy. Volunteers will need to complete a 10-minute online survey and record a short sample of their speech. Those who qualify will be invited to provide a saliva sample so that researchers can analyse their genes.
Co-chief study investigator MCRI Professor Angela Morgan says participants will be contributing to a global effort to unravel the genetics of stuttering, and in the process, may learn more about their own genetic make-up. The study could also lead to new treatment opportunities.
“Many treatments for speech and language disorders focus on symptoms without targeting the underlying cause of the problem,” says Prof Morgan.
“Learning more about the genetic and neurobiological basis of speech and language disorders will help us identify who may be at risk and allow us to develop more targeted treatments."
Prof. Morgan says one per cent of adults globally stutter. With almost seven in 10 of these reporting a family history of the disorder, she says this suggests that genetics could play a role.
The study is international, and MCRI is working with the Walter and Eliza Hall Institute of Medical Research, QIMR Berghofer Medical Research Institute, Griffith University and the University of Melbourne on the Australian arm.
The study is funded by the National Health and Medical Research Council.
To learn more about the study, see www.geneticsofstutteringstudy.org.au
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