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November 2022

Using stem cells to give sight and controlling rogue blood stem cells – Metcalf Prize winners announced

I am delighted to announce the two brilliant scientists who are the winners of the 2022 Metcalf Prizes for Stem Cell Research, and you can meet them tonight.

Dr Anai Gonzalez-Cordero, a researcher at the Children’s Medical Research Institute, 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 can go rogue, leading to blood cancers. He is a researcher at WEHI and a clinician at the Royal Melbourne Hospital and Peter MacCallum Cancer Centre.

Both will receive a $55,000 prize from the Foundation. We hope that investing in their careers will advance Australian stem cell research and, importantly, improve the prognosis for patients.

Read on to find out more about their research and achievements.

Anai and Ashley are also speaking the Gene therapies and stem cell research: now and in the future public forum tonight at 5.30pm AEDT. There’s still time to register to attend in person in Melbourne or join online via our website.

Also in this newsletter: lab-made blood, using maths to predict the path from cells to humans, and more in our regular round-up of news from around the world.

Kind regards,

Dr Graeme L Blackman AO

Chairman, National Stem Cell Foundation of Australia

In this bulletin:

Using stem cells to give sight

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.

She has already shown that she can grow cultures of healthy photoreceptor cells in a dish in the lab and then use the cells replace the defective cells and restore sight in laboratory models of hereditary blindness. And she has shown that gene therapy can repair diseased human retinal cells grown in the lab as ‘mini-organs’ (or ‘organoids’), providing them with normal light-sensing ability.

Her $55,000 Metcalf Prize will contribute to developing systems to progress both concepts towards clinical trials. She is based at Children’s Medical Research Institute (CMRI) in Western Sydney.

The prize is an initiative of the National Stem Cell Foundation of Australia.

More than 16,000 Australians live with an inherited retinal disease, a broad group of genetic eye conditions. These conditions cause blindness in nearly eight million people worldwide and can occur from birth through to late adulthood. Vision loss occurs due to a ‘spelling mistake’ in the genetic code that causes cells of the eye to malfunction.

Until recently, newly diagnosed patients and their families were told to expect progressive vision loss for which there was no treatment or cure.

Working at University College London, Anai learnt to grow stem cells and direct these to become retinal organoids (laboratory-derived ‘mini-organs), which are small, functional colonies of photoreceptor cells.

Now at CMRI in Australia, she is using this stem cell technology in two ways. 

The first is to determine which retinal cell genes are faulty and replace them with versions that are functional, using gene therapy.  Anai and her team plan to use patient-derived organoids to study how viruses can be employed to smuggle healthy copies of genes into diseased retinal cells to replace the genetic fault.

Read more about Anai and her research.

Controlling rogue blood stem cells

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.

As a researcher at WEHI and a clinician at the Royal Melbourne Hospital and Peter Mac, Ashley will use his $55,000 Metcalf Prize to advance his ideas from the laboratory into treatments for blood and blood cancer diseases.

Blood stem cells can form any cell of the blood system and they self-renew, so they are a source of endless supply. But, blood cancers can also arise from these cells, and are responsible for three per cent of all deaths in Australia. We need to understand why this occurs and use this knowledge to treat disease.

Winning a Metcalf Prize is personal for Ash. While studying for a Bachelor of Medical Science degree at the University of Melbourne, he had the opportunity to work in the late Professor Don Metcalf’s lab and became interested in blood cells and blood disorders.

“Throughout his career Don was always encouraging early career researchers. He was a great mentor and friend to me. So, I’m absolutely blown away by this award. It is very humbling, very emotional for me to receive this award.”

After gaining his BMedSci and his degree in medicine, Ashley did a further 10 years training to become a specialist haematologist. He is currently a consultant in haematology at the Royal Melbourne Hospital and the Peter McCallum Cancer Centre. But in 2008 he also returned to the lab, and a PhD at WEHI leading to postdoctoral work as clinician researcher in the areas of blood stem cells, immunology and leukaemia.

Read more about Ashley and his research.

The Metcalf Prizes for Stem Cell Research are named for the late Professor Donald Metcalf, AC. Over his 50-year career, Don helped transform cancer treatment and transplantation medicine, and paved the way for potential stem cell therapy in the treatment of many other conditions.

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:

CNN: Two teaspoons of lab-made blood could have enormous potential for people with rare blood conditions.

ABC News: Patients receive transfusion of laboratory-grown blood in world-first clinical trial.

EurekAlert: Monoclonal antibodies preserve stem cells in mouse brains, bring promise for future studies.

The Scientist Magazine: Cancer cells in mice may hitch a ride with bone-healing stem cells.

UWA News: Australian scientists develop world-first map showing gene activity changes in diverse human brain cell types from pre-birth to adulthood.

University of Melbourne: Research centre to predict how cells become a person using maths. Scientists discover changes in aging stem cells.

Department of Health and Aged Care: Australia first stem cell research to help adults and children living with joint pain and disfigurement.

Weill Cornell Medicine: Stem cell-based genomic study yields insights on viral infection susceptibility.

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