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May 2020

Stem cells and mending bones; COVID-19; and more

The Foundation is investing $50,000 in research to develop a new treatment for hard-to-fix fractures, matching a $50,000 donation from the public.

We’re also matching public donations to bring a new diabetes treatment and a heart attack treatment to clinical trials, and have selected a further three stem cell therapy research projects to partner with. Read on to see how you can join us to make your donated dollars go further.

We’re concerned to see yet another rise in the marketing of unproven stem cell treatments, now being hyped to treat COVID-19, the disease caused by the new coronavirus. We join the international stem cell research community in sounding the warning to beware of misleading information. More below.

Stem cell science continues, with two major international research conferences happening online in the coming weeks. Read on for details.

And our support for evidence-based regenerative medicine continues, despite COVID-19 disrupting society.

Next month we will invite stem cell researchers to nominate for this year’s $50,000 Metcalf Prizes.

The two 2018 Metcalf Prize winners are investigating cancer survival and mass-producing mini-hearts. In this newsletter, we catch up with leukaemia researcher Heather Lee and heart researcher Enzo Porrello, to see what they’re achieving. More below.

Finally, enjoy our regular round-up of links to stories we’ve shared via social media, including news of reputable trials involving stem cells for COVID-19.

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

In this bulletin:

Eye, skin, and bone treatment research selected for fundraising boost

Treatment targeting better broken bone-mending shows promise

A new regenerative medicine treatment to help mend broken bones is the first of four projects chosen to receive donations through the Foundation’s 2020 Matched Funding Program.

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.

While we’ve reached the cap for matching funding to this project, we’re happy to pass on new donations to Mike and his colleagues. You can contribute via our website – just specify ‘BONE’ when prompted. We are also still seeking donations to match for Bernie Tuch’s diabetes project and James Chong’s heart project.

“Everyone knows someone who has broken a bone,” explains Mike. “In most cases, bone tissue will repair on its own very efficiently.

“However, in some patients, fractures do not heal, and non-unions form.  This outcome is more common in populations of older people, smokers, or those in the early stages of diabetes.  Non-unions also form in healthy individuals, and it is not always clear why the repair process stalled.”

Mike describes the normal healing of damaged bone and other tissues as an elegant cascade of events, with each stage triggering the next.

“However, some underlying pathologies or events can interrupt this cascade, and the bone ends don’t properly knit together,” he explains.

Mike thinks a treatment where placenta-derived stem cells – more conservatively called placenta-derived stromal cells – could be implanted into the fracture site to promote tissue repair.  

Mesenchymal stem cells are thought to repair tissue either by directly contributing to the cells that make up the repair tissue, or by secreting factors that upregulate natural tissue repair processes.

“The important thing about placenta-derived stromal cells is that they secrete a lot of growth factors that encourage tissue regeneration,” says Mike. “It is unlikely that placenta stromal cells will make a long-term contribution to new bone cells.  Instead, the very large number of placental stromal cells used in the therapy will secrete factors that will ‘kick start’ stalled healing cascades and upregulate natural bone repair processes.”

Mike says it takes about 100 million cells to make a cubic centimetre of tissue. In this instance, these cells act like small biofactories at the site of the injury, producing the factors needed for healing exactly where they’re needed.

“And we’re getting them from placental tissue, which is normally discarded. The placenta is a wonderful resource! We can get 1000 doses of this treatment from a single donated placenta.”

Once restrictions related to the COVID-19 pandemic ease, Mike will be ready to start safety trials of the treatment. He will be joined by his colleagues at the Queensland University of Technology at the Translational Research Institute, working in collaboration with the Departments of Orthopaedic Surgery at the Royal Brisbane and Woman’s Hospital and at the Princess Alexandra Hospital.

We’re thrilled to share the news that this project has already reached the $100,000 target, thanks to a $50,000 donation from Inner Wheel Australia, the local arm of the international women’s community group formed by a group of wives of Rotary International club members in 1924.

The group has a 20-year history of sponsoring cord blood research, choosing a medical research field aligned with their membership, which is largely made up of mothers and wives. Sponsoring clinical trials involving placental cells was therefore a natural fit.

Mike’s first research grant, many years ago, was from Inner Wheel Australia for a cord blood project he was working on.

“I’m very grateful for the support from the National Stem Cell Foundation of Australia and Inner Wheel Australia,” he says. “Especially at this time. Once we can get through this COVID-19 period, it will be a huge time for cell therapies. They are the next big thing in medicine.”

The other selected projects

We will share more information about the three other projects, outlined briefly below, in our next newsletter.

  • Dr Tom Edwards from the Centre for Eye Research Australia (CERA) and the University of Melbourne is working on gene therapy for inherited retinal diseases.
  • Professor John Bateman from Murdoch Children's Research Institute is researching genetic disorders of bone and cartilage.
  • Professor Mark Shackleton is from the Cancer Development and Treatment Group Laboratory at the Alfred Hospital and Monash University seeks to develop new treatments for the skin pigment disorder vitiligo, and for melanoma, a deadly type of skin cancer.

 

Warning about unproven stem cell treatments for COVID-19

An important message from the Centre for Stem Cell Systems, University of Melbourne, shared with permission:

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).

While stem cell research shows promise for numerous diseases and conditions, there currently are no approved stem cell-based approaches for the prevention and treatment of COVID-19 infection.

Despite the unmet need at this challenging time, we need to continue to insist that new therapies are appropriately tested and authorised prior to their use.

Clinics claiming that expensive stem cell products can protect people or cause miraculous improvements for those suffering from COVID-19 should be viewed with caution.

Legitimate clinical trials are underway to evaluate a range of interventions, from drugs to vaccines and cellular therapies. It remains too early to know if these will work or are safe. Experimental interventions should only be explored under expert medical care and as part of recognised clinical investigation.

Read full statement online.

From ethical organoids to accelerating cell and gene therapies: annual international scientific meetings go online

Scientists in Australia and around the world will be able to participate in two major conferences without the costs of international travel.

The International Society for Cell and Gene Therapy 2020 Annual Meeting (ISCT 2020) and the International Society for Stem Cell Research 2020 Annual Meeting (ISSCR 2020) have both adapted their programs to take place online. This virtual offering will allow the communities to share knowledge, collaborate and network within the constraints imposed by coronavirus pandemic.

ISCT 2020: ‘Accelerating Cell and Gene Therapy Adoption: From Proof of Concept to Standard of Care’ – May 28 – 29

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 program features:

  • ‘The Force Awakens’ – an opening plenary showcase of COVID-19 therapies in development, with live Q&A
  • A look at the history, present and future of gene engineering
  • Emerging issues in unproven therapies
  • Addressing the practical challenges of industrialising CAR T-cell therapies.

More information and to register: www.isct2020.com.

ISSCR 2020: ‘The Future Starts Here’ – 23 – 27 June

The ISSCR Annual Meeting brings together scientists, clinicians, business leaders, ethicists, and educators from more than 65 countries.

This year’s program features:

  • A keynote address from Sekar Kathiresan, CEO of Verve Therapeutics, a biotechnology company focused on treatments for heart disease
  • Four core themes of tissue stem cells and regeneration, cellular identity, modelling development and disease, and clinical applications
  • Sessions on the progress of cell therapies and tissue engineering to the clinic
  • Ethical considerations for brain organoid research
  • A science advocacy and communication seminar
  • A Women in Science panel discussion.

More information and to register: www.isscr.org/meetings-events/annual-meetings/isscr-2020-virtual.

 

Understanding the cancer cells that get away: Newcastle leukaemia researcher Heather Lee

Dr Heather Lee of the University of Newcastle won a Metcalf Prize for her work analysing individual cancer cells to understand how some survive therapy. Her research ultimately aims to prevent relapse and lift survival rates for leukaemia. We caught up with her recently and asked what’s she’s been up to since her win.

What have you been up to since you won a Metcalf Prize?

“My scientific highlights have been developing a new experimental technique with PhD student, Kooper Hunt, and developing closer collaborations with haematologist Dr Anoop Enjeti. I’ve also contributed to research manuscripts from UNSW and the University of Melbourne.

“A major achievement has been the award of an NHMRC Ideas Grant with my colleague, Dr Carls Riveros. We propose to study how acute myeloid leukaemia cells can change over time, to see if epigenetics can drive evolution of this disease.

“On the home front, I have welcomed a second daughter into our family. Felicity Rose was born in May last year and is a very happy and healthy baby.”

Read full story online.

 

Making masses of mini-hearts: Melbourne cardiac development researcher Enzo Porrello

2018 Metcalf Prize for his work exploring newborn heart development to develop heart attack drugs and engineer ‘artificial hearts’ from patient stem cells.

He was delighted to see his regular collaborator and friend Associate Professor James Hudson win a Metcalf in 2019. We sat down with him for a chat.

What have you been up to since you won a Metcalf Prize?

“It has been an incredibly busy year since I won the Metcalf Prize in 2018. A major highlight for my team was our publication in Cell Stem Cell, which described the first ever large-scale drug screen in human stem cell-derived cardiac organoids. This work led to the identification of several drugs that can promote regeneration of human heart muscle cells, which may have therapeutic potential for patients with heart failure.

“This publication was the culmination of several years of work and resulted from a collaboration with Astra Zeneca that started in 2014, so it was a real highlight to finally publish this work in the world’s top-ranked stem cell journal.”

Read full story online.

 

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:

Science Daily: Lipid metabolism controls brain development. Paper

7 News: Coronavirus Australia: Stem cell treatment for virus patients to be trialled in Sydney

ABC Radio National: COVID-19 update with Norman Swan (includes reporting on stem cell trial for COVID-19)

The New Daily: Australia joins international bid to use stem cells against COVID-19

EurekAlert: Study shows stem cells constitute alternative approach for treating corneal scarring. Paper

Washington University: Diabetes reversed in mice with genetically edited stem cells derived from patients. Paper

US News: Transplanted skin stem cells help blind mice see light. Paper

European Pharmaceutical Review: Stem cells in the clinic: how are they regulated?

Cosmos magazine: Cancer gene could help repair hearts

Science Daily: Parkinson's disease may start before birth

The Niche: Rudy Giuliani now plugging unproven stem cells for COVID19: is Trump next?

Tech Explorist: Scientists reprogrammed cells from a 114-year-old woman

EurekAlert: Blood stem cells boost immunity by keeping a record of previous infections

ABC Triple J Hack: Graham is waiting on lifesaving stem cell treatment. Coronavirus is stopping delivery

SciTechDaily: Stanford scientists discovered the invisible pattern that growing neurons follow to form a brain

Future Medicine: Portrayal of umbilical cord blood research in the North American popular press: promise or hype?