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June 2020

Hijacking viruses to save sight; 2 x $50,000 prizes for rising stars in stem cell research; new talent on the board

Please join the Foundation in supporting the development of a new treatment to save the sight of thousands of Australians born with one of the many types of inherited retinal diseases.

We are getting behind Melbourne clinician-scientist Dr Tom Edwards’ efforts to bring to human trials a new stem cell enabled gene therapy for these conditions. But we need your help. Read on for more information.

Another important investment of the Foundation is the Metcalf Prizes for Stem Cell Research, rewarding exceptional mid-career scientists who are conducting excellent and sustained work in the discipline.

There are two $50,000 prizes, one each to be awarded to a successful male and female researcher. Nominations for the 2020 prizes are now open and will close on Friday, 31 July. If you know any strong candidates, encourage them to apply. Read on for details.

Also in this newsletter, we welcome a new director: Professor Megan Munsie. Megan is an expert in the ethical, legal and social implications of stem cell research. She is a great communicator and educator, a compassionate voice for patient welfare, and a defender of sound science. We’re delighted to have her join the team. Read more about Megan below.

Finally, our regular round-up of stem cell news includes stories on their use as pharmaceutical factories, 3D printing for cartilage repair, calls for caution with unproven treatments in multiple countries, and Australian research projects collectively receiving a $5.9 million research injection.

 
Kind regards,
 
Dr Graeme L Blackman AO
Chairman, National Stem Cell Foundation of Australia


In this bulletin:


Hijacking viruses to save sight

Your chance to support research on a gene therapy for inherited eye diseases

Dr Tom Edwards wants to use the infectious power of viruses to develop cures for blinding eye disease using gene therapy.

More than 16,000 Australians live with an inherited retinal disease (IRD). One type, retinitis pigmentosa, is the leading cause of blindness in the working-age population. Currently, there are no cures or treatments.

IRDs affect adults and children alike. For most young people, vision loss is due to a ‘spelling mistake’ in the genetic code that causes cells to malfunction.

With your support, the National Stem Cell Foundation of Australia wants to help fund Tom’s research into new gene therapies for these conditions.

The Foundation aims to provide $100,000 for the project, by matching, dollar for dollar, every public donation of $500 or more, capped at $50,000. You can contribute via our website – just specify ‘EYE’ when prompted.

Tom is a surgeon at the Centre for Eye Research Australia (CERA) in Melbourne, dividing his time between the lab and seeing and treating patients.

He says that one of the hardest parts of being a clinician-researcher is giving patients, especially children, the life-changing news that they have an inherited disease that will inevitably take away their eyesight. He leads a laboratory group focused on developing treatments that may stop this vision loss from progressing.

“Thanks to the exciting progress being made in this field, for the first time we’re able to tell patients there’s hope,” he says.

Gene therapies work by correcting the disease-causing spelling mistakes in a person’s genetic code. Modified viruses are the delivery vehicles.

“When you get a common cold, that’s an example of a virus delivering its trouble-making genetic material into your body,” Tom explains.

“We are borrowing this ability to deliver a genetic payload of DNA to dysfunctional retinal cells.”

Tom and his colleagues reengineer a naturally harmless virus, removing most of its DNA and replacing it with a corrected copy of the patient’s original misspelt retinal gene.

“You’re essentially hijacking the virus to deliver useful genetic material,” he says. “In its simplest form, you’re delivering a functioning copy of the gene that is not working.”

To do his research, Tom needs eye cells with faulty genes. That’s where stem cells come in.

“The retina is the light-detecting layer inside the eye,” he explains. “It’s less than half a millimetre thick and extremely delicate. You can’t just go in and biopsy that tissue because you would damage the eye.”

Skin biopsies, in contrast, are far easier and less invasive, so his colleagues at CERA and the University of Melbourne take skin cells from patients, turn them into stem cells and then into new retinal cells.

“Induced pluripotent stem cell-derived eye cells are an immensely helpful model that can help us to understand how a gene mutation causes disease, and allow us to test different therapies,” said Tom.

Tom hopes the funding support from the Foundation and its donors will help bring new treatments for IRDs to clinical trial.

“All the surgical techniques required to deliver this treatment have already been developed,” he said.

“We’re just applying them to the task of delivering a precise volume of virus underneath the retina.

“It’s a huge honour to have my project selected and I’m immensely grateful for the Foundation’s support. We now feel a sense of duty to deliver results. Thankfully, I’m fortunate to have a great team at CERA and outstanding collaborators working hard to deliver for our patients.”

 


Do you know a rising star in stem cell research?

Applications for $50,000 Metcalf Prizes now open

The 2020 Metcalf Prizes for Stem Cell Research will see two up-and-coming leaders in the field awarded $50,000 each to turbocharge their work. If you know a promising researcher, encourage them to apply.

Potential winners could be working in medicine or agriculture, government or academia.

Applications are open to scientists who completed research-based PhDs or MDs between August 2010 and August 2015 – although allowances will be made for career breaks, such as maternity leave periods.

Winners will be chosen for their scientific excellence, leadership ability and the potential to have a continuing influence on stem cell research in Australia.

Past Metcalf Prizes have honoured:

  • Brisbane mammary biologist Felicity Davis,
  • Brisbane bioengineer James Hudson,
  • Newcastle leukaemia researcher Heather Lee,
  • Melbourne heart development researcher Enzo Porrello,
  • Melbourne haematologist Mark Dawson,
  • Brisbane computational biologist Jessica Mar,
  • Sydney heart clinician and researcher James Chong,
  • Melbourne immunologist Tracy Heng,
  • Melbourne bioinformatician Christine Wells,
  • Perth geneticist Ryan Lister,
  • Tasmanian neural stem cell researcher Kaylene Young, and
  • Monash University reprogramming legend Jose Polo.

You can read more about these alumni and their research on our website.

The Metcalf Prizes for Stem Cell Research honour the exceptional contribution made by the late Professor Donald Metcalf. 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.

The prizes support the Foundation’s mission to promote the study and use of stem cells in the prevention or control of disease in human beings, and to enhance public education around the subject.

Applications close Friday, 31 July. We encourage last year’s unsuccessful applicants to nominate again this year, if still eligible.

To apply online, and for a full list of criteria and conditions, head to our website: www.stemcellfoundation.net.au/metcalf_prizes.

If you have any questions about eligibility or the application process, please contact Tanya Ha at Science in Public: tanya@scienceinpublic.com.au.

 


Stem cells in society expert Megan Munsie joins Foundation board

We’re delighted to welcome Professor Megan Munsie as a new director of the Foundation. She also joins the Metcalf Prizes jury.

Megan is Deputy Director of the University of Melbourne’s Centre for Stem Cell Systems in the School of Biomedical Sciences, and a strong voice in the Australian media for patient welfare and sound science.

She is an internationally recognised biologist who has made a significant contribution to policy development and community engagement in stem cell science and regenerative medicine.

Megan serves on advisory committees to national and international scientific organisations, and is the chair of the ethics committee for the International Society for Stem Cell Research (ISSCR) and of the policy, ethics and translation committee of the Australasian Society for Stem Cell Research.

She was awarded the ISSCR’s 2018 Public Service Award in recognition of her contribution to outreach and policy advocacy in stem cell science.

Professor Munsie was appointed to the National Stem Cell Foundation of Australia board on 25 May 2020 and also serves as a member of the science and ethics committee.

Here is a selection of media interviews she has done to help sort science from snake oil:

 


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:

International Society for Stem Cell Research: ISSCR urges FDA and EMA to enforce regulation of clinics offering unproven and unapproved stem cell-based interventions

Yahoo! News: 5-year-old Thai boy with COVID-19 saves sister’s life with his stem cells

The Irish Times: Stem cell therapy: why we need to be suspicious about cure-all claims

Office of Minister for Health Greg Hunt: Treatment for incurable diseases receives $5.9 million research injection

Scientific American: Inside the stem-cell pharmaceutical factory

EurekAlert: Microneedling therapeutic stem cells into damaged tissues. Paper

Study Finds: Scientists grow mini human livers from stem cells, successfully transplant them in rats. Paper

University of Queensland: Stopping the clones may help win skin cancer battle. Paper

Science Alert: There's been a dangerous rise in people selling stem cell 'treatments' for coronavirus. Paper

Phys.org: A nanostructure that stimulates growth of stem cells for Parkinson's disease treatment

Nine News (Melbourne): BioPen: 3D printing with stem cells for cartilage repair

Nature: Revealed: two men in China were first to receive pioneering stem-cell treatment for heart-disease

Technology News: Can stem cells heal a brain injury? A new biomarker may predict the answer.

Los Angeles Times: Column: Don’t be taken in by stem cell firms offering unsubstantiated therapies for COVID-19


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