Can stem cells save sight? 20 years since Nobel discovery
Discover how stem cells could boost our understanding of eye diseases and potentially treat vision loss. Hear about the latest research and ask your own questions at a free online event, Future Medicine: Can stem cells save sight? on 21 May. More below.
It’s 20 years since the announcement that Japanese scientists had found a way to take specialised adult cells, such as skin or blood cells that usually cannot change, back in time and return them to a stem-cell-like state. This led to a Nobel Prize in 2012. What has this discovery led to? Find out below.
In June, we will open the 2026 Metcalf Prizes for applications. We caught up with lung researcher Dr Rhiannon Werder from Murdoch Children’s Research Institute (MCRI) in Melbourne to see how her career has progressed since winning a 2024 Prize. Read on for details. Spoiler: she has won an Al & Val Rosenstrauss Fellowship and other grant funding.
Plus:
- MCRI’s Katie Ayers has secured funding to uncover the genetic links between reproductive differences and the increased risk of cancer. We supported Katie’s earlier research into how sex development occurs in the womb and the role of genetics in variations.
- researchers in Mexico are calling for better regulation of its stem cell medical tourism industry
- the Australian-Danish reNEW collaboration has announced a spin-out company to deliver stem cell-derived heart tissue into clinical trials as a treatment for heart failure
- and more in our regular round-up of stem cell news from around the world.
Kind regards,
Dr Graeme L Blackman AO
Chairman, National Stem Cell Foundation of Australia
In this bulletin:
- Eye diseases and damage: what do stem cells have to offer?
- 20 years of turning adult cells back into stem cells
- Helping people with lung disease breathe easier
- Stem cell news from around the world
Eye diseases and damage: what do stem cells have to offer?
Hear the latest science and bring your questions to a free online event at 7:00pm AEST Thursday 21 May 2026.
Find out how stem cells are helping us understand and potentially treat causes of blindness and low vision including inherited eye diseases such as retinitis pigmentosa, Stargardt disease, Usher syndrome, and acquired conditions, such as corneal damage.
Three of Australia’s top stem cell researchers and eye clinicians will discuss Future Medicine: Can stem cells save sight? Come along to this free webinar with your questions for the panel.
Register to attend via Humanitix: https://events.humanitix.com/future-medicine-can-stem-cells-save-sight.
More than 453,000 Australians are blind or vision impaired, which affects their ability to read, work, drive, take part in hobbies, and other activities. Prescription glasses and contact lenses help many, but some eye injuries and diseases are currently beyond repair or cure.
About 19,000 Australians have an inherited retinal disease, caused by genetics. The lifetime cost of living with an inherited eye disease in Australia is about $5.2 million per person. Corneal blindness affects all ages and burdens over 2 million people worldwide.
The good news is that scientists and clinicians working with stem cells are:
- developing lab-grown or patient-derived healthy eye stem cells for transplantation to treat corneal blindness.
- running clinical trials for gene therapies targeting inherited eye diseases, which could potentially stop vision loss from progressing or even restore some sight.
- using a patient’s own cells to better understand their eye disease, test treatments, and potentially develop new cell sight-saving therapies.
Find out more from our panel of researchers and clinicians:
- Clinician-scientist and eye surgeon Dr Tom Edwards, Centre for Eye Research Australia
- Cell biologist Associate Professor Anai Gonzalez-Cordero, Children’s Medical Research Institute
- Eye surgeon Professor Stephanie Watson OAM, University of Sydney, Sydney Eye Hospital, Sydney Children's Hospital, and Prince of Wales Hospital.
The webinar is presented by the National Stem Cell Foundation of Australia and hosted by Tanya Ha, Director of Engagement at Science in Public.
Read more about the event and panellists online.
20 years of turning adult cells back into stem cells
Anniversary of Nobel Prize winning paper
Stem cell research 20 years ago was primarily focused on two sources: adult stem cells found in developed tissues such as bone marrow, fat, skin, and embryonic stem cells derived from the inner cell mass of three- to five-day-old embryos. Embryonic stem cells can self-renew for long periods of time and are ‘pluripotent’, meaning they can give rise to most cell types in the body.
The understanding that embryonic stem cells could become any cell type – termed ‘pluripotency’ – brought with it the exciting potential to develop new treatments or replacement tissue. However, their origin raised ethical concerns for many people.
Then, in 2006, Japanese physician and researcher Shinya Yamanaka and fellow scientist Kazutoshi Takahashi reported a breakthrough in a landmark paper published in Cell. They showed that mature adult mouse cells could be reprogrammed into so-called ‘induced pluripotent stem cells’ (iPSCs), which like embryonic stem cells can give rise to most cell types in the body.
Yamanaka won the 2012 Nobel Prize in Physiology or Medicine jointly with British developmental biologist Sir John B. Gurdon, “…two scientists who discovered that mature, specialised cells can be reprogrammed to become immature cells capable of developing into all tissues of the body. Their findings have revolutionised our understanding of how cells and organisms develop.”
Today, because of this work, cells from your body – usually skin or blood cells – can be reprogrammed and coaxed into becoming various cell types such as heart, kidney, muscle, eye, or even brain cells. It is a much easier way of obtaining cells with your genetic fingerprint than potentially more invasive and painful tissue biopsies.
That means that scientists can, for example, investigate an individual’s kidney disease, assembling 3D models made entirely from the patient’s cells that mimic structure and function of the kidney in the lab, and work out a personal treatment.
Scientists can also see how different diseases develop at a cellular level, identify the genes and processed involved, and identify new or better ways to treat the condition.
It also offers the possibility of generating patient-derived cells and tissues for transplantation, which can help reduce the risk of immune rejection.
Gene editing also raises the possibility of correcting disease-causing mutations in a patient’s cells, then generating healthy replacement cells genetically changed for therapy. In some cases, this approach has already reached the clinic. For example, gene-edited blood stem cells are now approved for sickle cell anaemia in some countries. This approach is also under development to treat other genetic conditions such as Duchenne muscular dystrophy, cystic fibrosis, and some inherited eye diseases.
Adult cells from healthy donors are also being reprogrammed back into stem cells both to study normal tissue and organ development, and to provide cells for therapy. Two treatments, one for heart failure and one for Parkinson’s disease, received conditional approvals from Japan’s health ministry, coinciding with the 20th anniversary of Yamanaka and Takahashi’s landmark 2006 paper.
The Foundation has invested in projects and scientists doing this kind of work. Examples include:
-
Giving cystic fibrosis patients longer, better lives: Gerard Kaiko, Hunter Medical Research Institute and the University of Newcastle - A brain bank of schizophrenia to fast-track diagnosis and better treatments: Maria Di Biase, The University of Melbourne
- Turning blood cells into muscle cells to test treatments for childhood muscular dystrophy: Dr Peter Houweling, Murdoch Children’s Research Institute.
We have been able to support these researchers thanks to the generosity of our donors. Please make a tax-deductible donation to the Foundation so that we can continue this work: www.stemcellfoundation.net.au/donate.
Read Nobel Laureate Prof Shinya Yamanaka’s reflections on this historical milestone in Cell Stem Cell: Two decades of induced pluripotent stem cell research: From discovery to diverse applications.
Helping people with lung disease breathe easier
Catching up with 2024 Metcalf Prize winner Rhiannon Werder
Dr Rhiannon Werder is growing ‘mini-lungs in a dish’, which mimic the complexity and function of lungs in living people.
The Group Leader at Murdoch Children’s Research Institute (MCRI) has already used stem cell-derived lung cells to create models of human genetic diseases, such as cystic fibrosis, and acquired lung diseases and infections, including the common cold.
Now she plans to grow three-dimensional models of human lung tissue, or ‘mini-lungs’, in a dish to better investigate respiratory infections and drive new treatment discoveries. She is also using stem cell-derived models to investigate chronic obstructive pulmonary disease (COPD). This progressive, incurable lung disease, often comprising chronic bronchitis and emphysema, makes breathing difficult.
In November 2024 she received one of our two annual $60,000 Metcalf Prizes. We caught up with Rhiannon to see how her career has progressed.
How has your work advanced since you won a Metcalf Prize?
My work has advanced in developing new protocols to generate a broader range of lung cell types for incorporation into our miniature lung tissue models. This has allowed us to build more physiologically relevant systems. Our first publication in this space has just been accepted!
This progress has translated into several major milestones, including receiving the Rebecca Cooper Foundation Al & Val Rosenstrauss Fellowship and an MRFF Chronic Respiratory Conditions grant focused on shared mechanisms of exacerbations in preschool wheeze and COPD.
The Metcalf funding was pivotal in helping us generate the preliminary data needed to secure these grants. In addition, the recognition from the prize provided a valuable boost to my profile at a key stage, helping to build collaborations and momentum for my research program.
What first drew you into stem cell research?
I was initially drawn to stem cell research by a curiosity about fundamental biology and a desire to better understand how complex cell types and tissues form. At the same time, I was motivated by the limitations of existing models for studying human disease. Stem cell systems provided a way to study disease processes directly in human cells, which felt both scientifically exciting and highly meaningful.
What’s the biggest difference you want your research to make and why?
The biggest difference I want my research to make is to enable the development of new treatments for respiratory diseases. Progress in this area has been relatively slow compared to other fields, and as a result, patients continue to live with significant symptoms and limited therapeutic options. Driving more effective, targeted interventions is what motivates my work and is the impact I am most committed to achieving.
Stem cell news from around the world
Between newsletters, we share stem cell news on social media:
Here are a few stories we’ve shared recently:
The Australian: How controversial cell therapy is making a comeback to fight incurable diseases [paywall]
reNEW: Spin-out company targets treatments for severe heart disease
MCRI: Denmark’s Queen Mary and King Frederick tour MCRI
Science: Stem cell therapies ‘come of age’ with two conditional approvals in Japan
Nature: Inside Mexico’s stem-cell industry
MCRI: Associate Professor Katie Ayers awarded Rebecca Cooper Fellowship for research exploring the genetic variations responsible for differences of sex development
Dr Maria Di Biase has created a ‘brain bank’ of schizophrenia: blobs of brain cells from 100 patients, growing in the lab. She’s using these brain organoids to develop urgently needed new approaches to diagnosis and treatment.
Waking up brain stem cells at the right time could one day improve cognition and fight neurodegenerative diseases and cancer, according to Dr Lachlan Harris, a researcher at QIMR Berghofer in Brisbane.
The brain remains one of the greatest biological mysteries. Often referred to as the body’s ‘control centre’, this complex organ of nervous tissue makes us uniquely who we are: living, breathing, thinking, feeling human beings.
Up until a few decades ago it was widely believed that the brain had neither stem cells nor the ability to regenerate itself.
Neuroscientist Professor Kaylene Young – one of two recipients of the Foundation’s inaugural Metcalf Prizes – assisted with the establishment of the Queensland Brain Institute in 2003.
Monash University researcher Associate Professor Atul Malhotra is leading a clinical trial to explore feasibility and safety of using donated umbilical cord blood-derived cells to help repair brain injury associated with preterm births.
This year, 24 researchers [pictured with ASSCR 2025 organising committee members] received grants to attend the meeting on the Gold Coast in Queensland, and present their work.
Unacceptably so, according to Monash University researcher, Associate Professor Atul Malhotra, Senior Consultant Neonatologist and Head of Monash Children’s Hospital’s Early Neurodevelopment Clinic.
Dr Felicity Davis won a 2019 Metcalf Prize for her research into 
Dr Rhiannon Werder is growing ‘mini-lungs in a dish’ that mimic the complexity and function of lungs in living people.
Dr William Roman is growing human muscles on a chip. He’s using them to understand how the skeletal muscle cell, the largest cell in a human body, connects with neurons and tendons to create working muscles.
Stem cell scientists have turned their research into works of art in a competition and exhibition to celebrate Global Stem Cell Awareness Day 2024 (October 9).
Two past Metcalf Prize winners – cardiologist and researcher 
University of Technology Sydney biomedical engineer Dr Jiao Jiao Li plans to use stem cells as biofactories to make drugs to reduce inflammation and encourage repair in painful osteoarthritic joints.
Rare but extremely debilitating gut disorders, like paediatric achalasia and Hirschsprung’s disease, can affect the fundamental ability to swallow and digest food and pass stools. Children with these conditions endure a lifetime of potentially life-threatening symptoms that severely impact their daily lives, require repeat hospitalisation, surgery and ongoing care.
Endometriosis, a painful condition caused by the growth of endometrial-like tissue outside the uterus, affects one in nine reproductive age women, half of all infertile women, and more than half of women and teenaged girls with pelvic pain.
Each year we award two $60,000 prizes to support the field’s rising stars and set them up for future success. And it’s working!
Now is the time to apply for grants and submit abstracts.
Human sex characteristics develop in the womb. About one in 100 babies are born with variations, some of which are linked to health problems such as increased cancer risk, heart disease, osteoporosis, intellectual disability, or infertility.
Little Lilly Lloyd-Morgan, aged 8, lives with a debilitating condition which stops her swallowing food. But there is hope on the horizon thanks to a potential stem cell therapy being developed by Dr Lincon Stamp and Dr Marlene Hao at the University of Melbourne.
Lincon and Marlene are developing a therapy that involves making healthy nerve cells from stem cells. These cells would be transplanted into patients like Lilly to replace the damaged or lost nerve cells and restore function.
Grow heart cells together in a petri dish and they know what to do – they start beating. Now they’re being used to find drugs to heal the heart. Eye cells are being grown to test gene therapies for blindness. Human brain cells growing together make connections and can even learn to play Pong! They are also providing a valuable tool to reveal how the brain ages.
Dr Jiao Jiao Li plans to use stem cells as biofactories to make drugs to reduce inflammation and encourage repair in painful osteoarthritic joints.
People diagnosed with late-stage stomach cancer have a less than 10 per cent chance of surviving more than 5 years.
“Australians will pay a lot to relieve the pain of arthritis—sometimes opting for unproven therapies using stem cells.”
Extremely premature babies’ own stem cells could hold the key to a new treatment for brain injury associated with preterm birth.
Dr Anai Gonzalez-Cordero’s research aims to restore sight in people with inherited retinal diseases, by repairing or replacing damaged photoreceptor (light-sensing) cells in the eye.
Dr Ashley Ng is revealing how blood stem cells are controlled, and how they sometime go rogue, leading to blood cancers. He has discovered how a protein known as ‘ERG’ underpins healthy development of blood cells, and how it also plays a role in Down syndrome-associated leukaemia and a range of other blood cancers.
Melbourne researcher Dr Peter Houweling is growing mini-muscles in the lab, in order to study muscular dystrophy in children, without the need for invasive and painful tissue biopsies.
Developmental biologist Dr Sarah Withey is using mini liver ‘organoids’ made from patients’ stem cells to test a potential treatment for children with a rare, genetic, progressive, life-limiting disease – Ataxia Telangiectasia (A-T).
Cardiac stem cell researcher Professor Enzo Porrello, a 2018 Metcalf Prize-winner, will be Director of reNEWS’s Melbourne node.
With your support, the Foundation wants to help fund Gerard’s research and bring this life-changing treatment to clinical trials.
Associate Professor Siok Tey is researching treatments that will improve the survival and quality of life for her patients with leukaemia or other blood cancers.
Dr Pengyi Yang plans to transform stem cell research.
Melbourne university student and long-distance runner Billy Morton, 22, was first diagnosed in his early teens with a rare genetic disease that is causing his eyesight to deteriorate. At the time there was no prospect of a treatment, but progress in gene therapy research is providing new hope.
Billy, with the rare genetic disease choroideremia, is one of 190 million people worldwide with diseases causing the death of light-sensing photoreceptor cells in the eye.
Cardiovascular disease is the leading cause of death for all Australians. Every year more than 57,000 Australians suffer a heart attack. Many develop heart failure. 
Each year, the Foundation selects up to four promising Australian stem cell research projects that need a funding boost to help them progress new therapies to human clinical trials.
Past
ISCT 2021 took place last week.
Back in Melbourne, Graeme visited Professor Tom Edwards in his lab at the Centre for Eye Research Australia where he is developing 
The Foundation is backing Professor Mark Shackleton’s stem cell research aimed at preventing melanoma and treating vitiligo – two diseases affecting the melanin pigment-producing cells, called melanocytes.
“In essence, there’s two main diseases which arise from melanocytes: one involves having too many melanocytes, which is melanoma, and the other results from not having enough melanocytes, which is vitiligo,” Mark says.
Proteins which control the growth of cells in embryos could teach us how to stop the uncontrolled growth of cells that is the hallmark of cancer, thanks to work by molecular biologist Dr Melanie Eckersley-Maslin.
Clinical haematologist Associate Professor Steven Lane wants to lift the survival rates of his leukaemia patients. He thinks the key could lie in the genetic fingerprints of the blood cancer stem cells that proliferate the disease.
2016 Metcalf Prize winner James Chong kicked off 2020 with a major journal publication on New Year’s Day. His study, published in Science Translational Medicine, showed a new protein therapy could encourage 
US researchers created programmable living machines – dubbed xenobots – by assembling cells from African clawed frogs into robots that can move around on short stumpy legs. These living robots could deliver small amounts of material, such as medicines or useful reagents. 
“Australia’s research workforce will be severely impacted by the pandemic and the effects are likely to be felt for an extended period.”
One such is Professor Ryan Lister (pictured with WA Science Minister Dave Kelly) who, in late September, was named joint 
Frozen cord blood became a vital back-up source of blood-forming stem cells during COVID-19 travel restrictions.
Preclinical results for the MCDS drugs have been encouraging, but if we are to progress the drug to the next phase, and identify other drug candidates, we need to know exactly how it works. 
Associate Professor Mike Doran from Queensland University of Technology is exploring the potential of special cells taken from placentas to repair non-union bone fractures.
We join the International Society for Stem Cell Research (ISSCR) and International Society for Cell & Gene Therapy (ISCT) in raising concern about the marketing of unproven stem cell therapies to treat people infected with Coronavirus disease (COVID-19).
ISCT 2020 will be accessible remotely and on-demand, including live and recorded sessions that are certified for accreditation programs, in addition to networking events, poster halls, and one-on-one partnering opportunities. 
The ISSCR Annual Meeting brings together scientists, clinicians, business leaders, ethicists, and educators from more than 65 countries.
Stem Cells Australia (SCA), a $24 million-dollar Australian Research Council Special Research Initiative, has formally come to an end. 
SCA will continue as an educational website, administered by the University of Melbourne, providing teaching resources for schools, and reliable information for people making choices about their own health care.
Cardiologist and researcher Associate Professor James Chong has already used human stem cells to repair the damaged hearts of other large primates — a world first. 
Professor Emeritus James A. Angus AO is a biomedical pharmacologist with a strong scientific and leadership background. He was Dean of the Faculty of Medicine, Dentistry and Health Sciences at the University of Melbourne from 2003 to 2013.
The Foundation took the opportunity to transition to a different management model. Graeme Mehegan has taken over the day-to-day running in the newly created role of General Manager. He has served the Foundation as Company Secretary and CFO since its inception in 2012.
On 28 November 2018, He Jiankui claimed to a packed conference room at the Second International Summit on Human Genome Editing in Hong Kong to have edited the genomes of two twin girls, Lulu and Nana, who were born in China.
2017 Metcalf Prize winning computational biologist Jessica Mar is analysing stem cells to discover the changes that influence ageing. Who will win the 2018 Prizes?
Your bone marrow makes one million new mature blood cells every second. Your life depends on it!
Cardiac disease affects at least 1.4 million Australians and almost 350,000 will have a heart attack at some point in their life. One in every two people with severe heart failure will die within one year of diagnosis. Stem cells may have the potential to treat heart failure.
These are just some of the things a live audience learnt from six Australian stem cell scientists at the ‘Stem Cell Research—Now and in the Future’ forum—the public event held in the lead up to the ISSCR 2018 Annual Meeting in Melbourne.
The audience heard about the current state of play with research and treatments for eye, skin, heart, kidney and blood disorders. They also had the opportunity to ask the panel their own questions; from what inspired the panellists to become stem cell scientists (with one freaky sci-fi related answer), to the treatment options here and overseas for MS.
On Wednesday 26 June, Melissa received the National Health and Medical Research Council Elizabeth Blackburn Fellowship – Biomedical. The Fellowship is named after 2009 Nobel Laureate Elizabeth Blackburn who discovered how chromosomes are protected by telomeres. Melissa was one of 20 of Australia’s finest health and medical researchers honoured at the NHMRC’s annual Research Excellence Awards in Canberra.
