February 2017

Australian of the Year puts spotlight on stem cell science

Welcome to the National Stem Cell Foundation of Australia’s bulletin on stem cell medicine and research in Australia.
The new Australian of the Year is a stem cell scientist and a stem cell transplant recipient.
Emeritus Professor Alan Mackay-Sim’s story is drawing the nation’s attention to stem cell science and the potential of regenerative medicine. More on his Australian of the Year recognition below.
This represents both challenges and opportunities for scientists, regulators and patient advocates alike.
For the science sector, it’s an opportunity to spark the interest of bright young Australians considering science careers and to raise awareness of what Australia is achieving in this field.
However, discussion of the link between his research and a trial stem cell treatment in Poland that allowed a quadriplegic firefighter to walk again runs the risk of raising the hopes and expectations of people suffering from spinal cord injuries and a host of other conditions.
It may also provide a new marketing hook for clinics offering dubious and expensive stem cell therapies to vulnerable patients.
For the Foundation and our friends in the sector, this renews our determination to invest in the science and provide the information needed for people to make informed choices regarding their health.
In this bulletin, we have included an article on the need to balance the hype and hope of stem cells, written by Professor Melissa Little who has just been announced as the new program leader for Stem Cells Australia. We’re also delighted to see that 2015 Metcalf Prize winner Professor Christine Wells will serve as her deputy.
This month marks four years since the Foundation’s official launch where we announced our first major investment—bringing Dr Kathryn Davidson back from America to apply her stem cell techniques to eye disease research. We catch up with Kathryn below.
Kind regards,
Dr Graeme L Blackman AO
Chairman, National Stem Cell Foundation of Australia

In this bulletin:

Australian of the Year announcement shines a spotlight on stem cell science


Nasal stem cell researcher and patient Alan Mackay-Sim awarded 2017 Australian of the Year

Queensland biomolecular scientist and nasal stem cell expert Emeritus Professor Alan Mackay-Sim has been named the 2017 Australian of the Year, recognition he hopes will raise awareness of stem cell research, and of scientific research in general.

“I’m hoping 2017 will be a wonderful opportunity to talk about the importance of research on spinal cord and rare brain diseases; about the therapeutic futures of stem cells and cell transplantation, which were undreamed of 20 years ago,” he said, accepting his award at Parliament house.
Professor Mackay-Sim is an authority on the human sense of smell and the biology of nasal cells, including adult olfactory stem cells, which can give rise to many cell types, including nerve cells. Taking these stem cells and turning them into nerve cells and other cell types in the lab allows him and his colleagues to study the biological basis of disease such as schizophrenia and Parkinson’s disease.
Alan led the world’s first clinical trial using olfactory stem cells in spinal cord injury, with the aim of testing the safety of the procedure. This work was a precursor to the world’s first successful restoration of mobility in a quadriplegic man in 2014.
Professor Mackay-Sim is also a beneficiary of stem cell science, having had a stem cell transplant to treat multiple myeloma, a cancer of the blood’s plasma cells.
For more information, visit www.australianoftheyear.org.au.
Media coverage of Australian of the Year
ABC news: Australian of the Year winner: Emeritus Professor Alan Mackay-Sim
SBS news: Stem cell researcher Alan Mackay-Sim named 2017 Australian of the Year
Huffington Post: Meet the New Australian Of The Year: Professor Alan Mackay-Sim

The future of stem cells: tackling hype versus hope

 Melissa Little, new head of Stem Cells Australia, reflects on what the Australian of the Year news means for society in this article for The Conversation. 
For many people suffering from disabling conditions, such as Parkinson’s disease, spinal injury and paralysis, multiple sclerosis, macular degeneration, heart disease, renal failure and even cancer, announcements in the press around breakthroughs in stem cell research undoubtedly bring hope.
The challenge remains how to accurately communicate what is genuinely possible in terms of therapies and what we scientists hope might be possible but do not yet have strong evidence for.
Keeping the balance between hope and hype is a difficult one, particularly when there are vulnerable and suffering people relying on the hope medical research offers. As Australian of the Year, Emeritus Professor Alan Mackay-Sim, stated in his acceptance speech, there are now many clinical trials being performed in Australia and around the globe, to determine whether the delivery of certain types of cells, including some grown from stem cells, into the spinal column can allow patients with spinal cord injury to regain function.
For these individuals, even a small gain of function—such as being able to sense the touch of a loved one or regain bowel or bladder control—is a major advance. However, as yet there is no stem cell silver bullet…

Read the full story online.

From skin cells to eye cells, and on to cell therapies: catching up with Kathryn Davidson

The Foundation’s first funded researcher has:
  • helped establish a tissue bank for researching a suite of eye diseases,
  • been part of a group of top young stem cell researchers making recommendations for the sector’s future, and
  • is now part of a team paving the way for living cells as treatments.
And she’s just won an Endeavour Fellowship from the Australian Government.

Turning skin cells into eye cells via stem cells

Foundation chairman Graeme Blackman welcomes Kathryn Davidson to Australia at the Foundation's launch in 2013.

Dr Kathryn Davidson knows how to take mature skin cells and turn them into stem cells, then eye cells, using the cell ‘reprogramming’ technique developed by Nobel Prize-winning scientist Shinya Yamanaka.
She’s the young American stem cell expert the Foundation funded to return to Melbourne to take up a post at the Centre for Eye Research Australia (CERA).
Kathryn uses tissue samples from patients with eye diseases so that these conditions can be studied and treatments improved. Obtaining eye cells ultimately from a skin biopsy is far more convenient and comfortable than collecting samples from the eye itself.
“We turn those skin cells into stem cells and from those stem cells we can generate all the different cell types within the retina. We can then study how those cells behave and how they’re different from those of non-diseased patients,” says Kathryn.
Many causes of vision loss are complex and involved a genetic predisposition. As such, cells containing specific genetic risks of patients with eye diseases are invaluable for research.
Kathryn’s work at CERA largely focused on age-related macular degeneration—Australia’s leading cause of blindness. But her reprogramming skills have also allowed her and her colleagues to build a ‘tissue bank’ of stem cells representing the genetics of a wide range of eye diseases, including glaucoma, macular telangiectasia and Leber’s hereditary optic neuropathy.
Kathryn says the Foundation funding came at a crucial time for both her and CERA, and was a wonderful way to return to the city where she did her PhD and started her research career.
“The most important thing was it helped provide support for me for two years at CERA, allowing us to jump-start the research while seeking federal funding for the project, which we later achieved through the NHMRC program.
“The Foundation has also supported me to attend several conferences, to present my research and learn what’s new in the field. These meetings served as excellent networking opportunities and facilitated new collaborations with other Australian scientists.”
Informing the future of stem cell science in Australia
 Kathryn’s career has continued to grow from strength to strength.
Kathryn participated in the 2015 Theo Murphy High Flyers Think Tank ‘The Stem Cell Revolution’, hosted by the Australian Academy of Science. Each year, this initiative brings together the brightest early and mid-career researchers in a chosen field to explore the strengths, knowledge gaps and challenges of their field and, make recommendations for its future.
Kathryn was one of a core team of eight participants who wrote the initiative’s report, written to inform policy makers and stakeholders, and was one of the experts on hand to brief the media when the report was launched.
Read the report at the Australian Academy of Science website.
Moving to Monash to pave the way for cell therapies
 Kathryn is now part of Nagy Group at the Australian Regenerative Medicine Institute, a satellite lab of Canadian stem cell scientist Professor Andras Nagy. They’re working to engineer ‘designer’ stem cells so that these living cells can be used as treatments.
Kathryn explains that there’s a streamlined process for how chemical drugs are developed, tested and approved. However, cell therapies are a new frontier, with new challenges for researchers and regulators.
“In the future we hope to deliver cells in order to target a number of conditions that aren’t treatable with conventional drug therapies. But there are several hurdles to overcome.”
The challenges Kathryn is working to address include:
  • ensuring grafted cells that don’t share the patient’s genetics are tolerated, rather than rejected,
  • enhancing safety functions in cell therapies, so that if some cells produce tumours they can be eliminated without killing the entire graft,
  • engineering cells to enhance their therapeutic function by combining gene therapy with cell therapy, and
  • conducting research with non-human primate stem cells, which are more relevant for predicting how human tissues will behave, compared with mouse models—an important part of pre-clinical studies to identify the therapies most likely to translate successfully into human patients.
The ultimate aim is to understand, engineer and bring these functions together into a stem cell that can serve as a universal cell therapy.
“It’s a pretty big idea, but hopefully in five to ten years we and others will have made enough progress to push cell therapies from the research lab into the clinic.”
This year, Kathryn will head to Canada for six months to learn patented genome editing techniques at the Lunenfeld-Tanenbaum Research Institute, having won an Endeavour Research Fellowship through the Australia Awards program. Then she will return to Melbourne to teach the techniques to her Australian colleagues.
Kathryn’s story is just one example of how funds raised by the Foundation are put to work. People who want to support the Foundation by making a donation can do so securely online via our website: stemcellfoundation.net.au/support-us/donate

Stem cell news from around the world

Between newsletters, we share stem cell news on social media: Here are a few of the stories we’ve shared recently.
Sydney Morning Herald: Rapid repair by basal stem cells: Lung cancer origin breakthrough made by Australian scientists at Walter and Eliza Hall Institute
The Conversation: Shark study reveals taste buds were key to evolution of teeth
New Scientist: Cellular recovery: How self-help could aid the damaged brain
Science Alert: Scientists say it's time we discussed creating humans from stem cells
The Guardian: Researchers use stem cells to regenerate outer layer of heart (Nature paper)
New York Times: The stem cell revolution is coming—slowly: interview with Nobel Laureate Shinya Yamanaka
Stem Cells Australia: Monash researchers start company to reprogram human cells
Knoepfler lab blog: Clinics can’t retract stem cell treatments gone bad
Australia Unlimited: An Australian Neuroscientist is using yeast and stem cells to develop new therapies for Alzheimer’s disease

About the Foundation

The National Stem Cell Foundation of Australia is an ATO-registered tax-deductible Health Promotion Charity dedicated to promoting the study and responsible use of stem cells to reduce the burden of disease.
The Foundation’s activities include:
  • supporting research that pursues cures for as-yet-untreatable diseases
  • building a community of people with a shared interest in stem cell science
  • providing the Australian public with objective, reliable information on both the potential and risks of stem cell medicine.
We are working to build a community of people with a stake in stem cell science and to promote collaboration between scientists locally and internationally.
Please feel free to contact the Foundation’s Executive Officer Julia Mason via jmason@stemcellfoundation.net.au.

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We aim to:

  • Promote the study and use of stem cells

  • Prevent or control diseases or illness

  • Enhance public education about stem cells