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Providing Australian children and adolescents with equitable access to new and emerging therapies through clinical trials: a call to action

Michelle S Lorentzos, David Metz, Andrew S Moore, Laura K Fawcett, Paula Bray, Lani Attwood, Craig F Munns and Andrew Davidson
Med J Aust || doi: 10.5694/mja2.52191
Published online: 15 January 2024

Opportunities for children to benefit from novel therapies have increased substantially over the past decade. Change is needed to maximise these opportunities, particularly in the paediatric trial environment. Investment and a coordinated national approach are needed to prevent Australian children falling behind their international peers. There is international recognition that collaborative approaches and strategic investment in paediatric clinical trials reap benefits in terms of access to clinical trials and new therapies. This article exemplifies a multi‐state collaboration and presents a united call to action to prioritise paediatric clinical trials, by strategically investing in effective governance and infrastructure, and a shift in culture that embeds clinical trials in the core business of paediatric health care and academic institutions.

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  • 1 Kids Research, Sydney Children's Hospitals Network, Sydney, NSW
  • 2 University of Sydney, Sydney, NSW
  • 3 Monash Children's Clinical Trial Centre, Monash Children's Hospital, Melbourne, VIC
  • 4 Monash University, Melbourne, VIC
  • 5 Child Health Research Centre, University of Queensland, Brisbane, QLD
  • 6 Queensland Children's Hospital, Brisbane, QLD
  • 7 University of New South Wales, Sydney, NSW
  • 8 Sydney Children's Hospitals Network, Sydney, NSW
  • 9 Royal Children's Hospital, Melbourne, VIC
  • 10 Melbourne Children's Trials Centre, Murdoch Children's Research Institute, Melbourne, VIC



Open access:

Open access publishing facilitated by The University of Sydney, as part of the Wiley - The University of Sydney agreement via the Council of Australian University Librarians.
 


Competing interests:

The Murdoch Children's Research Institute and Sydney Children's Hospitals Network have received funding from Roche to aid in building capacity for gene therapy in Australia. All authors’ hospitals and/or institutions are active sites for pharmaceutical company‐sponsored clinical trials and receive enrolment‐based reimbursement on a trial‐by‐trial basis, but do not receive direct funding for clinical trials capacity or capability building unless stated above.

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Rationale and plan for a focus on First Nations urban health research in Australia

Janet Stajic, Adrian Carson and James Ward
Med J Aust || doi: 10.5694/mja2.52181
Published online: 15 January 2024

Urbanisation is a global phenomenon. The World Health Organization reported in 2015 that 55% of the global population lived in cities and is predicting this to increase to 68% by 2050.1 First Nations peoples globally are disproportionately affected by urbanisation, with major drivers being climate change, deforestation and increased pressures created by globalisation. Despite this, there is limited research to address urbanisation and its impact on human health and wellbeing. Similarly, there is an urgent need for a focus on improving health and wellbeing outcomes for urban First Nations peoples in Australia given the rapid urbanisation of First Nations people.

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  • 1 UQ Poche Centre for Indigenous Health, University of Queensland, Brisbane, QLD
  • 2 Institute for Urban Indigenous Health, Brisbane, QLD


Correspondence: j.stajic@uq.edu.au


Open access:

Open access publishing facilitated by The University of Queensland, as part of the Wiley ‐ The University of Queensland agreement via the Council of Australian University Librarians.


Acknowledgements: 

The UQ Poche Centre for Indigenous Health is generously supported by philanthropists Greg Poche AO and Kay Van Norton Poche. We acknowledge Dr Anton Clifford‐Motopi of the UQ Poche Centre for undertaking yarning sessions with members of the Research Alliance for Urban Community‐Controlled Health Services and analysis to inform and develop its research priorities.

Competing interests:

No relevant disclosures.

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The burden of occupational injury attributable to high temperatures in Australia, 2014–19: a retrospective observational study

Blesson M Varghese, Alana Hansen, Nick Mann, Jingwen Liu, Ying Zhang, Tim R Driscoll, Geoffrey G Morgan, Keith Dear, Anthony Capon, Michelle Gourley, Vanessa Prescott, Vergil Dolar and Peng Bi
Med J Aust 2023; 219 (11): . || doi: 10.5694/mja2.52171
Published online: 11 December 2023

Abstract

Objectives: To assess the population health impact of high temperatures on workplace health and safety by estimating the burden of heat‐attributable occupational injury in Australia.

Study design, setting: Retrospective observational study; estimation of burden of occupational injury in Australia attributable to high temperatures during 2014–19, based on Safe Work Australia (work‐related traumatic injury fatalities and workers’ compensation databases) and Australian Institute of Health and Welfare data (Australian Burden of Disease Study and National Hospital Morbidity databases), and a meta‐analysis of climate zone‐specific risk data.

Main outcome measure: Burden of heat‐attributable occupational injuries as disability‐adjusted life years (DALYs), comprising the numbers of years of life lived with disability (YLDs) and years of life lost (YLLs), nationally, by Köppen–Geiger climate zone, and by state and territory.

Results: During 2014–19, an estimated 42 884 years of healthy life were lost to occupational injury, comprising 39 485 YLLs (92.1%) and 3399 YLDs (7.9%), at a rate of 0.80 DALYs per 1000 workers per year. A total of 967 occupational injury‐related DALYs were attributable to heat (2.3% of occupational injury‐related DALYs), comprising 890 YLLs (92%) and 77 YLDs (8%). By climate zone, the heat‐attributable proportion was largest in the tropical Am (12 DALYs; 3.5%) and Aw zones (34 DALYs; 3.5%); by state and territory, the proportion was largest in New South Wales and Queensland (each 2.9%), which also included the largest numbers of heat‐attributable occupational injury‐related DALYs (NSW: 379 DALYs, 39% of national total; Queensland: 308 DALYs; 32%).

Conclusion: An estimated 2.3% of the occupational injury burden in Australia is attributable to high ambient temperatures. To prevent this burden increasing with global warming, adaptive measures and industry‐based policies are needed to safeguard workplace health and safety, particularly in heat‐exposed industries, such as agriculture, transport, and construction.

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  • 1 The University of Adelaide, Adelaide, SA
  • 2 Australian Institute of Health and Welfare, Canberra, ACT
  • 3 The University of Sydney, Sydney, NSW
  • 4 Centre for Rural Health, the University of Sydney, Lismore, NSW
  • 5 Monash Sustainable Development Institute, Monash University, Melbourne, VIC


Correspondence: peng.bi@adelaide.edu.au


Open access:

Open access publishing facilitated by The University of Adelaide, as part of the Wiley ‐ The University of Adelaide agreement via the Council of Australian University Librarians.


Acknowledgements: 

This investigation was funded by the Australian Research Council with a Discovery Project grant (DP200102571) to Peng Bi. We gratefully acknowledge the Australian Institute of Health and Welfare for providing YLD estimates and supplying the disability weights and life tables produced by the Global Burden of Disease study, for providing methodological input, and for their assistance and guidance throughout the project. We acknowledge Safe Work Australia for providing deaths data for calculating YLLs and workers’ compensation claims data for calculating YLDs. We thank Syeda Hira Fatima and Matthew Borg (University of Adelaide) for their assistance with data analysis.

Competing interests:

No relevant disclosures.

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  • 2. Varghese BM, Barnett AG, Hansen AL, et al. Geographical variation in risk of work‐related injuries and illnesses associated with ambient temperatures: a multi‐city case‐crossover study in Australia, 2005–2016. Sci Total Environ 2019; 687: 898‐906.
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Planetary health: a new standard for medical education

Catherine GA Pendrey, Sonia Chanchlani, Laura J Beaton and Diana L Madden
Med J Aust 2023; 219 (11): . || doi: 10.5694/mja2.52158
Published online: 11 December 2023

The Australian Medical Council (AMC) recently released updates to the National Framework for Prevocational (PGY1 and PGY2) Medical Training and the Standards for Assessment and Accreditation of Primary Medical Programs (medical school standards). Together, these cover all medical school programs in Australia and Aotearoa New Zealand and all accredited prevocational training programs in Australia.1,2 In a significant development, the standards recognise the health impacts of climate change, the need for environmentally sustainable health practice,3 and planetary health.4 This welcome and necessary development comes as climate and environmental health indicators deteriorate. All medical education institutions should now integrate planetary health into curricula to ensure the profession is equipped to respond to the unfolding planetary health crisis and realise environmentally sustainable health care.

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  • 1 National Centre for Epidemiology and Population Health, Australian National University, Canberra, ACT
  • 2 Climate and Environmental Medicine Specific Interests Group, Royal Australian College of General Practitioners, Melbourne, VIC
  • 3 Melbourne Medical School, University of Melbourne, Melbourne, VIC
  • 4 Doctors for the Environment Australia, Melbourne, VIC
  • 5 University of Notre Dame Australia, Sydney, NSW
  • 6 Australasian Faculty of Public Health Medicine, Royal Australasian College of Physicians, Sydney, NSW



Open access:

Open access publishing facilitated by Australian National University, as part of the Wiley ‐ Australian National University agreement via the Council of Australian University Librarians.


Acknowledgements: 

We thank all who have contributed to advancing planetary health education and practice in Australia, Aotearoa New Zealand, and globally.

Competing interests:

No relevant disclosures.

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Disruption of gender‐affirming health care, and COVID‐19 illness, testing, and vaccination among trans Australians during the pandemic: a cross‐sectional survey

Sav Zwickl, Tomi Ruggles, Alex FQ Wong, Ariel Ginger, Lachlan M Angus, Kalen Eshin, Teddy Cook and Ada S Cheung
Med J Aust || doi: 10.5694/mja2.52169
Published online: 11 December 2023

Abstract

Objectives: To assess rates of disruption of gender‐affirming health care, of coronavirus disease 2019 (COVID‐19) illness, testing, and vaccination, and of discrimination in health care among Australian trans people during the COVID‐19 pandemic.

Design, setting: Online cross‐sectional survey (1–31 May 2022); respondents were participants recruited by snowball sampling for TRANSform, an Australian longitudinal survey‐based trans health study, 1 May – 30 June 2020.

Participants: People aged 16 years or older, currently living in Australia, and with a gender different to their sex recorded at birth.

Main outcome measures: Proportions of respondents who reported disruptions to gender‐affirming health care, COVID‐19 illness, testing, and vaccination, and positive and negative experiences during health care.

Results: Of 875 people invited, 516 provided valid survey responses (59%). Their median age was 33 years (interquartile range, 26–45 years); 193 identified as women or trans women (37%), 185 as men or trans men (36%), and 138 as non‐binary (27%). Of 448 respondents receiving gender‐affirming hormone therapy, 230 (49%) reported disruptions to treatment during the pandemic; booked gender‐affirming surgery had been cancelled or postponed for 37 of 85 respondents (44%). Trans‐related discrimination during health care was reported by a larger proportion of participants than in a pre‐pandemic survey (56% v 26%). COVID‐19 was reported by 132 respondents (26%), of whom 49 reported health consequences three months or more after the acute illness (37%; estimated Australian rate: 5–10%). Three or more COVID‐19 vaccine doses were reported by 448 participants (87%; Australian adult rate: 70%).

Conclusions: High rates of COVID‐19 vaccination among the trans people we surveyed may reflect the effectiveness of LGBTIQA+ community‐controlled organisation vaccination programs and targeted health promotion. Training health care professionals in inclusive services for trans people could improve access to appropriate health care and reduce discrimination.

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  • 1 The University of Melbourne, Melbourne, VIC
  • 2 Austin Health, Melbourne, VIC
  • 3 La Trobe University, Melbourne, VIC
  • 4 ACON, Sydney, NSW


Correspondence: adac@unimelb.edu.au


Open access:

Open access publishing facilitated by The University of Melbourne, as part of the Wiley ‐ The University of Melbourne agreement via the Council of Australian University Librarians.


Data sharing statement

De‐identified participant data are available upon reasonable request to the corresponding author (adac@unimelb.edu.au), provided that the aim of the request is deemed to be of benefit to the trans and gender‐diverse community and has received Austin Health Human Research Ethics Committee approval (as an amendment).


Acknowledgements: 

We thank the Melbourne Clinical and Translational Sciences (MCATS) research platform staff for the administrative and technical support that facilitated this investigation. Ada Cheung is supported by a National Health and Medical Research Council Investigator Grant (2008956).

Competing interests:

No relevant disclosures.

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  • 2. Bretherton I, Thrower E, Zwickl S, et al. The health and well‐being of transgender Australians: a national community survey. LGBT Health 2021; 8: 42‐49.
  • 3. Zwickl S, Wong AFQ, Dowers E, et al. Factors associated with suicide attempts among Australian transgender adults. BMC Psychiatry 2021; 21: 81.
  • 4. Boyer TL, Youk AO, Blosnich JR, et al. Suicide, homicide, and all‐cause mortality among transgender and cisgender patients in the veterans health administration. LGBT Health 2021; 8: 173‐180.
  • 5. Hughes LD, King WM, Gamarel KE, et al. Differences in all‐cause mortality among transgender and non‐transgender people enrolled in private insurance. Demography 2022; 59: 1023‐1043.
  • 6. Zwickl S, Angus LM, Wong AFQ, et al. The impact of the first three months of the COVID‐19 pandemic on the Australian trans community. Int J Transgend Health 2021; 24: 281‐291.
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  • 9. Balaji JN, Prakash S, Joshi A, Surapaneni KM. A scoping review on COVID‐19 vaccine hesitancy among the lesbian, gay, bisexual, transgender, queer, intersex and asexual (LGBTQIA+) community and factors fostering its refusal. Healthcare (Basel) 2023; 11: 245.
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Invisible wounds of the Israel–Gaza war in Australia

Susan J Rees and Batool Moussa
Med J Aust || doi: 10.5694/mja2.52168
Published online: 20 November 2023

Among the many dire consequences of the Israel–Gaza war that began in October 2023, the impact on the mental health of populations living in multicultural Western countries is significant and should not be overlooked1. The psychosocial reverberations of the conflict are felt in societies throughout the world, embodying unique characteristics of trauma and adding to the complexity of the mental health risk for people living in Western countries. The threat to mental health status is higher for those who have had family members killed, harmed or gone missing, and for those with previous exposure to war, including in Lebanon, Iraq and Syria. The level of stress has been exacerbated by its enduring nature, including systematic oppression, economic hardship, violence, human rights violations and national struggle.2,3

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  • Discipline of Psychiatry and Mental Health, University of New South Wales, Sydney, NSW


Correspondence: s.j.rees@unsw.edu.au


Open access:

Open access publishing facilitated by University of New South Wales, as part of the Wiley ‐ University of New South Wales agreement via the Council of Australian University Librarians.


Competing interests:

No relevant disclosures.

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The Lancet Countdown on health and climate change: Australia a world leader in neglecting its responsibilities

Paul J Beggs and Ying Zhang
Med J Aust || doi: 10.5694/mja2.52152
Published online: 20 November 2023

Climate is integral to good health and wellbeing, but it can also be a forceful driver of death and disease. Many diseases both here in Australia and globally are climate sensitive — the global magnitude of such diseases was estimated to be 39 503 684 deaths (69.9% of total annual deaths) and 1 530 630 442 disability‐adjusted life years in 2019.1,2 Climate change is the biggest health threat facing humanity.3,4

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  • 1 Macquarie University, Sydney, NSW
  • 2 University of Sydney, Sydney, NSW


Correspondence: paul.beggs@mq.edu.au

Competing interests:

No relevant disclosures.

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Cough in Children and Adults: Diagnosis, Assessment and Management (CICADA). Summary of an updated position statement on chronic cough in Australia

Julie M Marchant, Anne B Chang, Emma Kennedy, David King, Jennifer L Perret, Andre Schultz, Maree R Toombs, Lesley Versteegh, Shyamali C Dharmage, Rebecca Dingle, Naomi Fitzerlakey, Johnson George, Anne Holland, Debbie Rigby, Jennifer Mann, Stuart Mazzone, Mearon O'Brien, Kerry‐Ann O'Grady, Helen L Petsky, Jonathan Pham, Sheree MS Smith, Danielle F Wurzel, Anne E Vertigan and Peter Wark
Med J Aust || doi: 10.5694/mja2.52157
Published online: 20 November 2023
Correction(s) for this article: Erratum | Published online: 26 June 2024

Abstract

Introduction: Cough is the most common symptom leading to medical consultation. Chronic cough results in significant health care costs, impairs quality of life, and may indicate the presence of a serious underlying condition. Here, we present a summary of an updated position statement on cough management in the clinical consultation.

Main recommendations: Assessment of children and adults requires a focused history of chronic cough to identify any red flag cough pointers that may indicate an underlying disease. Further assessment with examination should include a chest x‐ray and spirometry (when age > 6 years). Separate paediatric and adult diagnostic management algorithms should be followed. Management of the underlying condition(s) should follow specific disease guidelines, as well as address adverse environmental exposures and patient/carer concerns. First Nations adults and children should be considered a high risk group. The full statement from the Thoracic Society of Australia and New Zealand and Lung Foundation Australia for managing chronic cough is available at https://lungfoundation.com.au/resources/cicada‐full‐position‐statement.

Changes in management as a result of this statement:

  • Algorithms for assessment and diagnosis of adult and paediatric chronic cough are recommended.
  • High quality evidence supports the use of child‐specific chronic cough management algorithms to improve clinical outcomes, but none exist in adults.
  • Red flags that indicate serious underlying conditions requiring investigation or referral should be identified.
  • Early and effective treatment of chronic wet/productive cough in children is critical.
  • Culturally specific strategies for facilitating the management of chronic cough in First Nations populations should be adopted.
  • If the chronic cough does not resolve or is unexplained, the patient should be referred to a respiratory specialist or cough clinic.

 

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  • 1 Australian Centre for Health Services Innovation, Queensland University of Technology, Brisbane, QLD
  • 2 Queensland Children's Hospital, Brisbane, QLD
  • 3 Menzies School of Health Research, Darwin, NT
  • 4 Rural and Remote Health, Flinders University, Darwin, NT
  • 5 University of Queensland, Brisbane, QLD
  • 6 Melbourne School of Population and Global Health, University of Melbourne, Melbourne, VIC
  • 7 Wal‐yan Respiratory Research Centre, Perth, WA
  • 8 Perth Children's Hospital, Perth, WA
  • 9 University of Sydney, Sydney, NSW
  • 10 Lung Foundation Australia, Brisbane, QLD
  • 11 Centre for Medicine Use and Safety, Monash University, Melbourne, VIC
  • 12 Alfred Health, Melbourne, VIC
  • 13 Monash University, Melbourne, VIC
  • 14 Institute for Breathing and Sleep, University of Melbourne, Melbourne, VIC
  • 15 Queensland University of Technology, Brisbane, QLD
  • 16 Austin Health, Melbourne, VIC
  • 17 University of Melbourne, Melbourne, VIC
  • 18 Griffith University, Brisbane, QLD
  • 19 Alfred Health, Melbourne, VIC
  • 20 Western Sydney University, Sydney, NSW
  • 21 Royal Children's Hospital, Melbourne, VIC
  • 22 Hunter Medical Research Institute, University of Newcastle, Newcastle, NSW
  • 23 John Hunter Hospital, Newcastle, NSW


Correspondence: jm.marchant@qut.edu.au


Open access:

Open access publishing facilitated by Queensland University of Technology, as part of the Wiley ‐ Queensland University of Technology agreement via the Council of Australian University Librarians.


Acknowledgements: 

We thank Lung Foundation Australia and the Thoracic Society of Australia and New Zealand for their support in the preparation of these guidelines.

Competing interests:

The authors received no specific funding for this work. Julie Marchant is supported by the Lung Foundation Australia Hope Research Fund Andrew Harrison Fellowship in Bronchiectasis Research 2021 and receives personal fees from being an author of two UpToDate chapters, outside of the submitted work. Anne Chang reports multiple grants from the National Health and Medical Research Council (NHMRC) during the conduct of this work; is an independent data monitoring committee member for an unlicensed vaccine (GSK) and an unlicensed monoclonal antibody (AstraZeneca); is an advisory member on the study design for an unlicensed molecule for chronic cough (Merck); and has received personal fees from being an author of two UpToDate chapters, outside the submitted work. Andre Schultz receives salary support from a Medical Research Future Fund Investigator Grant (APP1193796). Danielle Wurzel has received research grants from the NHMRC and GSK, and honoraria from Merck and MSD. Stuart Mazzone has received honoraria from Merck, NeRRe Therapeutics, Reckitt Benckiser and Bellus Health for consultancy on their antitussive programs, and antitussive‐related grant support from Merck, Bellus Health and Reckitt Benckiser, as well as multiple grants from the NHMRC and the Australian Research Council. Shyamali Dharmage has received multiple grants from the NHMRC and the Australian Research Council, including the NHMRC Investigator Grant (APP1193993) that currently supports her. Additionally, Shyamali Dharmage and Jennifer Perret have received independent investigator‐initiated grants from AstraZeneca and GSK for unrelated research. Jennifer Perret is supported by an NHMRC Early Career Fellowship (APP1159090). Johnson George has received honoraria through consultations for AstraZeneca, GSK and Pfizer which have been paid to his employer, and has held research grants from Boehringer Ingelheim, GSK and Pfizer through investigator‐initiated research schemes. All other authors have no conflicts of interest to declare in relation to this work.

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The Future Healthy Countdown 2030: holding us to account for children's and young people's health and wellbeing

Sandro Demaio, Sharon R Goldfeld and Susan Maury
Med J Aust 2023; 219 (10): . || doi: 10.5694/mja2.52141
Published online: 20 November 2023

Too many Australian children and young people are faring poorly across key measures of health and wellbeing, and too often these reflect systemic inequities that could be mitigated or eliminated through policy change. Despite Australia's abundant resources and wealth, we are failing a key test of societal progress: that our children have opportunities to live as well or better than their parents.

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  • 1 Victorian Health Promotion Foundation, Melbourne, VIC
  • 2 University of Melbourne, Melbourne, VIC
  • 3 Centre for Community Child Health, Royal Children's Hospital, Melbourne, VIC


Correspondence: smaury@vichealth.vic.gov.au

Acknowledgements: 

The Victorian Health Promotion Foundation (VicHealth) has played a convening role in scoping and commissioning the articles contained in the MJA supplement.

Competing interests:

Sandro Demaio and Susan Maury are VicHealth employees.

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100 years on: the first use of insulin in Australia

Sophie Templer
Med J Aust 2023; 219 (10): . || doi: 10.5694/mja2.52137
Published online: 20 November 2023

A century ago, the only accepted treatment for diabetes in Australia and worldwide was the so‐called “starvation diet” of severely restricted carbohydrate intake. However, near‐starvation only prolonged the natural course of disease, and profound hunger and progressive emaciation would almost inevitably give way to coma and eventually death. The successful isolation of a therapeutic pancreatic extract in the Canadian summer of 1921 by Frederick Banting, Charles Best, James Collip and John Macleod followed numerous less successful attempts by other researchers over preceding decades. The extract, which they named “insulin” (from the Latin insula, meaning island), would become, in the words of medical historian Michael Bliss, “the elixir of life for millions of human beings around the world”.1


  • Bankstown–Lidcombe Hospital, Sydney, NSW



Competing interests:

No relevant disclosures.

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