Just The Tip Of The Iceberg - Hope For Inherited Childhood Disorders

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9th November 2009, 08:30pm - Views: 912

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November 2009

<EMBARGOED until 7.00 pm Monday 9 November 2009>

Just the tip of the iceberg - Hope for inherited childhood disorders

Top SA medical researcher honoured nationally

Professor John Hopwood, South Australian Scientist of the Year 2008, will this evening follow in the footsteps

of some of Australia’s greatest medical researchers, including immediate past winner Professor Ian Frazer and

Nobel Laureates Professors Barry Marshall and Robin Warren, by being awarded the 2009 CSL Florey Medal

Professor Hopwood today joins a cohort of extraordinary Australian medical researchers who are at the

forefront of breakthroughs globally, and who have dedicated their lives to basic research all the way through

to making a difference to our lives through prevention and treatment. 

This prestigious honour is part of the Australian Institute of Policy and Science’s Tall Poppy Campaign and takes

its name from Australia’s first Nobel Laureate in Medicine, Sir Howard Florey, who made an indelible impact

on world health by developing penicillin. The winner receives a $25,000 prize and a hand sculpted silver medal,

joining the most recent winner, Professor Ian Frazer in 2007.

Hopwood first formed the Lysosomal Diseases Research Unit in 1976 in South Australia. This Unit is the only

group researching lysosomal storage disorders nationally and is the largest multidisciplinary group working on

them world-wide. The Unit is world-renowned for its research capabilities and the translation of research

findings into state-of-the art diagnostic services. 

Lysosomal diseases are genetically inherited and affect about 1 in 5,000 babies, with recent findings suggesting

maybe up to about 1 in 1,000. Symptoms are progressive and impact on many body organs including the

skeleton, heart, lungs and brain with devastating effects. 

Hopwood’s Unit has generated several world firsts, particularly the isolation of genes involved in some of

these disorders and the development of first ever FDA-approved treatments for two disorders which were

marketed world-wide in 2005 and 2006. These outcomes meant improved quality of life for patients and multi-

million dollar royalty returns to South Australia, representing the largest public sector commercialisation

outcome for that state, and possibly Australia. 

Ongoing research commercialisation is well advanced to enable screening and the development of effective

therapies for lysosomal disorders affecting the brain. 

Hopwood says: “we are just tipping the iceberg.”  He is most of all excited about the discoveries that link

lysosomal disease to unexplained cancers, stroke and neurological disorders that concern us all, as well as his

team’s achievements in helping the many children affected.

Professor Peter Rathjen, AIPS Director, Deputy Vice-Chancellor (Research) at the University of Melbourne and

Chair of the 2009 CSL Florey Medal Selection Committee says the bi-annual accolade was created in 1998, the

centenary of Sir Howard Florey’s birth, to celebrate world standing achievements in biomedical science and

human health advancement by Australian researchers.

“Many top ranking scientists were in the running for this year’s medal, a testament to the excellence in

Australian medical research. Professor Hopwood was the winner, reflecting the quality of his long term

dedication from basic research through to clinical outcomes,” he says.  

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“John Hopwood is an Australian medical researcher whom we should celebrate, and who should stand high on

the Australian and world stage for his achievements, alongside other great Australian medical researchers.”

John Hopwood comments: “It is important that biomedical scientists receive recognition for the perseverance

that is required for real outcomes through research. For this I am thankful for this award.  It is important that

we also recognise all the clinicians and scientists involved. But neither they nor I would be where they are

without the patients and families, nor would our work have any meaning without their need. “

CSL, Australia’s leading biopharmaceutical company, became the principal sponsor of the Florey Medal this

year as part of its commitment to recognising and promoting scientific excellence in Australia. 

“Australia has much to celebrate when it comes to achievements in medical research, and we need role

models who can inspire a new generation of innovative scientists.  Professor John Hopwood is one such person

and an extremely deserving winner of the 2009 CSL Florey Medal” Dr Andrew Cuthbertson, Chief Scientific

Officer at CSL said today.

Where & When:

Monday 9 November

Doltone House Loft, Jones Bay Wharf

Pyrmont Point, Sydney

6.30pm for 7.00 pm - 10.00 pm (Awards ceremony and Australian Association of

Medical Research Institutes Annual Dinner)


Professor John Hopwood, and:

Professor Peter Rathjen, Director AIPS/ University of Melbourne

Dr Andrew Cuthbertson, Chief Scientific Officer, CSL Limited

Professor Robert Graham AO, President AAMRI

Hon Mark Butler MP, Parliamentary Secretary for Health

Hon Jodi McKay, NSW Minister for Science and Medical Research

Elektra Spathopoulos, Executive Director AIPS 

Senior representatives of SA Heath, including Dr Tony Sherbon, CEO


For  interviews, to attend the Awards, more information or images contact:

Elektra Spathopoulos, Executive Director AIPS – M:  0422 204 790 or E: director@aips,net.au

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Professor Hopwood is honoured with the 2009 CSL Florey Medal for his life-long work into the diagnosis and

treatment of genetically inherited disorders that affect children with devastating clinical effects leading to

progressive destruction of the brain and other organs. 

Born in Melbourne, and educated at the Swinburne Institute and then Monash University, Hopwood first

formed the Lysosomal Diseases Research Unit in 1976.

From basic science to clinical trials, through to clinical outcomes and commercialisation, Professor Hopwood

has dedicated three decades of research, discovery and personal perseverance in the service of human health

advancement for the sufferers of lysosomal storage disorders (LSD) which affect at least one in 5,000 babies if

not one in 1,000, as recent research suggests. Recently, lysosomes have become implicated in otherwise

unexplained stroke and heart disease, cancers and neurodegenerative disorders. 

Professor Hopwood today heads a group of more than 30 researchers through SA Pathology at the Adelaide's

Women's and Children's Hospital, focusing on the diagnosis and treatment of LSD, and was awarded the South

Australian of the Year (Science) Award in 2008 amongst other accolades. Hopwood and his team have

developed a novel program to enable newborn screening for these disorders. His unit has achieved world-first

treatments for two lysosomal storage diseases that have dramatically improved clinical outcomes for patients

worldwide, numerous patents, as well as FDA-approved drugs and commercial licenses and attracted over $30

million in competitive research funding, to date. 

John Hopwood’s research is world leading. He has displayed continuous and humble dedication and

effectiveness in relating to LSD patients and their families alongside his research team, clinicians, global

colleagues and pharmaceutical companies. John is a man of vision who, through basic research, persistence

and commitment, has accumulated and harnessed scientific knowledge and techniques to achieve his stated

goal of “early diagnosis and effective therapy of lysosomal storage disorders”.  Without his efforts, thousands

worldwide would be without diagnosis, therapy or the hope that either would ever come.


Professor Peter Rathjen - Chair

Deputy Vice-Chancellor (Research) – and AIPS


The University of Melbourne

Professor Warwick Anderson AM

Chief Executive Officer


Professor Suzanne Cory 


Walter Eliza Hall Institute

Professor Geoffrey Donnan


Florey Institute

Steven Dower

Head of Protein Biochemistry

Honorary Professor, Bio21 Institute 

Professor Ian Frazer


Diamantina Institute for Cancer,

Immunology and Metabolic Medicine, QIMR

Professor Peter Schofield

Executive Director and CEO

Prince of Wales Medical Research Institute

Professor Fiona Wood

Winthrop Professor

Burn Injury Research Unit

The University of Western Australia

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