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March 2022

Testing treatments for heart defects and cystic fibrosis; grants of $100K for projects approaching the clinic

Extra pumping power for hearts with congenital defects is just one of the outcomes being pursued by a new international collaborative regenerative medicine centre.

The Australian arm will be led by Melbourne cardiac stem cell researcher and Metcalf Prize alumnus Enzo Porrello. Read on for details.

The Foundation has raised funds to support Newcastle researcher Gerard Kaiko’s efforts to bring a life-extending treatment for cystic fibrosis to clinical trials. But we need more - can you help? More below.

Gerard’s project is one of four selected for our 2021 Matched Funding Program.

We’re now looking for the next four projects. If you know a stem cell scientist whose research projects are nearing clinical trials, encourage them to be in the running for the 2022 funding round’s four $100,000 grants. Details below.

Finally, some of our directors have been in the news. Read on to find out more about the experts who lead the Foundation.

Kind regards,

Dr Graeme L Blackman AO

Chairman, National Stem Cell Foundation of Australia

In this bulletin:

Engineered heart tissue and other treatments the target of a new half-billion-dollar international research effort

Australian scientists lead new international research centre

An international collaboration of three research institutes will develop new drugs and therapies using human stem cells to treat heart, respiratory and kidney disease, diabetes, cancer, and other conditions.

The Novo Nordisk Foundation Center for Stem Cell Medicine, or ‘reNEW’, is a €300 million (A$468 million) collaboration between Murdoch Children’s Research Institute (MCRI) in Australia, the University of Copenhagen in Denmark, and Leiden University Medical Center in The Netherlands.

Pioneering Australian scientist Professor Melissa Little has been appointed CEO.

Professor Little, Chief Scientist at MCRI, is known for creating functioning ‘mini-kidneys’ from stem cells, used to study diseases and test treatments.

“Stem cell research has come so far,” says Melissa. “Right now we are producing beating heart tissue that may be able to treat children with congenital heart disease. And we’re really only scratching the surface.”

Cardiac stem cell researcher Professor Enzo Porrello, a 2018 Metcalf Prize-winner, will be Director of reNEWS’s Melbourne node.

He will oversee the centre’s research at MCRI and continue his own work developing patches of heart tissue that can contract to provide extra blood-pumping power to people with heart conditions.

“I’m really excited about the development of these engineered heart patches,” he says. “Over the next few years, we want to move that work forward to a point where we’ve established it’s safe and effective in large animal trials. Then we can begin to think about taking that into the clinic for human trials.”

Enzo says the MCRI’s inclusion as one of reNEW’s three institutes is a recognition of the strengths in stem cell research in Australia.

“This is the first time in its history that the Novo Nordisk Foundation has invested in an international centre of this nature. All of their previous centres are Danish,” he explains.

Under the collaboration, University of Melbourne Professor Megan Munsie will also be joining MCRI part-time to lead reNEW’s social, ethical and regulatory research theme. Megan is an expert in stem cell ethics, legislation and the global regulatory environment, and is one of our Directors.

Watch a video about MCRI’s involvement in reNEW.

Read more about reNEW on MCRI’s website.

The National Stem Cell Foundation of Australia would like support more mid-career scientists like Enzo Porrello, which is why we award two $55,000 Metcalf Prizes to promising local researchers each year. Thanks to the support of our donors, we will open the 2022 Prizes for nominations mid-year.

Read more about the Metcalf Prizes.

Giving cystic fibrosis patients longer, better lives

Join the effort to bring a new life-changing treatment to the clinic

Dr Gerard Kaiko has developed a new molecule that could help cystic fibrosis patients live longer and with a better quality of life.

With your support, the Foundation wants to help fund Gerard’s research and bring this life-changing treatment to clinical trials.

A baby is born with cystic fibrosis every four days on average in Australia. The inherited genetic disease causes severe damage to the lungs, digestive system and other organs.

When cystic fibrosis was first recognised 80 years ago, most babies with the condition died before their first birthday.

Life expectancy is about 38 years – less than half that of the general population – and Gerard wants to extend it further.

“Existing drugs don’t restore enough of the function of the defective gene to give patients the same life expectancy as the general population,” Gerard says.

“There’s no reason why, with the advent of targeted therapies, like those we’re working on, that we can’t make greater strides.”

Gerard and his colleagues at Hunter Medical Research Institute and the University of Newcastle were selected for the Foundation’s 2021 Matched Funding Program for their project using personalised medicine for the treatment of cystic fibrosis.

Under the Program, the Foundation will match, dollar-for-dollar, donations made by other supporters to provide a total of $100,000.

A generous donation from Whiteley Corporation, an Australian manufacturing company specialising in cleaning and infection control and prevention products, has brought the funds raised to $70,000.

We’re looking for donors to cover another $15,000, which, matched by the Foundation, will meet the funding target.

The project uses stem cells in two ways. The drug molecule is made using living stem cell ‘biofactories’; it can’t be made synthetically through chemical reactions in a traditional manufacturing lab.

“Stem cells allow us to make the large quantities we will need for treatments in the clinic.”

The project also uses stem cell-derived tissues from individual patients to test how different people might respond to the drug.

Gerard says that personalised treatment is particularly important in cystic fibrosis. Although only one gene is involved, there are about 2000 different mutations. Not everyone has the same prognosis or the same symptoms.

A biopsy from a patient’s airways or colon provides cells that are grown into ‘mini-lung’ or ‘mini-gut’ tissues that allow Gerard’s team to test the stem cell ‘biofactory’ drug candidate in living cells with the patient’s unique genetics.

“With this disease, you see some really sick kids. You can also see patients who don’t go downhill until later in life. Being able to study how treatments work in a wide range of patient-derived tissues from the actual patients in the clinic with different mutations will mean we can get to clinical trials much faster.”

If you want to make a donation to support Gerard’s research, contact Graeme Mehegan via [email protected].

$100K funding opportunity for stem cell scientists

Applications closing soon

The National Stem Cell Foundation of Australia invites applications for up to four $100,000 research grants as part of its 2022 Matched Funding Program.

Under the Program, the Foundation will match any donation it receives from an approved donor (or donors) up to a maximum of $50,000 with up to $50,000 dollars of its own funds, to potentially provide a total of $100,000 for a successful research project.

Ideally, the lead researcher (or their host institution) would find and introduce the donor to the Foundation, however if this does not occur the Foundation will try to assist by using its resources to source an appropriate donor (through its newsletters, website and social media).

To be eligible the research project must be:

  • Using stem cell technology
  • Performed predominantly in Australia
  • At the late pre-clinical trial stage in readiness for clinical trials OR ready to conduct a clinical trial.

Researchers interested in applying for a grant under the program should complete an Expression of Interest (found on our website) and forward it to us as directed.

Applications will close on Tuesday 8 March 2022. For more information, an overview of past funding recipients, and to download the Expression of Interest form, visit the Foundation’s website.

The Foundation hopes to support a diversity of projects in stem cell research. Applications from gender and culturally diverse backgrounds are encouraged.

Foundation directors in the news

Independent oversight from experts

From left to right: Governor of Victoria the Honourable Linda Dessau AC and Professor James Angus AO; Professor Caroline Gargett; Zali Steggall OAM, MP and Professor Megan Munsie.

The Foundation’s work is overseen by a voluntary board of independent directors, with expertise in governance, finance, scientific leadership, stem cell and medical research, and ethics. Recent news from our directors:

  • Biomedical pharmacologist and research sector leader Professor James Angus AO was sworn in as the 15th Lieutenant-Governor of Victoria on 12 November 2021. James comes to the role with 45 years of experience across academia and medical research.
  • Professor Caroline Gargett from Hudson Institute of Medical Research is the key international member of the team which was awarded the $US1 million Magee Prize for their work on identifying vaginal stem cells to use with new biomaterials to repair tissue loss in women with compromised vaginal structure and function.
  • In addition to her role at reNEW, Professor Megan Munsie from The University of Melbourne received a High Commendation in the Advocacy Award category at Research Australia’s 18th Health and Medical Research Awards for her work on the public education and ethical considerations for stem cell medicine. Read an AusBiotech story about her achievement.

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:

Technology Networks: Newly discovered stem cell resembles cells in early human embryo. Paper.

RMIT: Sonic advance: How sound waves could help regrow bones.

Department of Health: New PBS listing to help fight chronic graft versus host disease.

BBC Health: Sickle cell: ‘The revolutionary gene-editing treatment that gave me new life’.

Al Jazeera: Woman cured of HIV after stem cell transplant.

Wired: A twist on stem cell transplants could help blood cancer patients. Paper.

NPR: Watch these robotic fish swim to the beat of human heart cells. Paper.

Medical News: Individual dosing of preparatory drug makes stem cell transplants safer for children. Paper.

New Atlas: Stem cell rejuvenation helps immunotherapy fight cancer tirelessly. Paper.

Business Insider Australia: Researchers are getting better at regenerating lab animals’ limbs. They might regrow human body parts in your lifetime. Paper.

The Niche: Fact-checking stem cell therapy for hair loss.

ABC: Calls for stem cell donation to change in Australia to reduce reliance on overseas donors.

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