Stem Cell Medicine

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Stem Cell Medicine at MCRI

Research excellence in Stem Cell Medicine

The Murdoch Children’s Research Institute is a world leading research institute tackling the toughest issues affecting children’s health. MCRI Stem Cell Medicine is an internationally leading research program established to drive the discoveries of fundamental stem cell biology toward medical outcomes for patients. It is focussed on the delivery of human stem cell-based treatments, including patient disease modelling, drug screening, cell therapies and bioengineered tissues.

Our co-location within the Royal Children’s Hospital, sitting squarely within the Parkville Biomedical Precinct ideally places MCRI to lead research, collaborate closely with clinicians, biomedical scientists, engineers, ethicists and the biotechnology sector, to deliver stem cell medical breakthroughs into the clinic.

At MCRI Stem Cell Medicine, we are proactively building our critical mass of stem cell scientists, strategically investing in cutting-edge technology platforms and state of the art facilities.

Stem Cell Medicine expertise 

Our expertise at the Murdoch Children’s Research Institute lies in our excellence in the generation of stem cells from a patient’s blood or skin cells known as induced pluripotent stem cells (iPSCs) and the subsequent differentiation of those stem cells into specialised tissues we wish to study – for example blood, kidney, brain or heart. This provides the potential for medical application in patient-based disease modelling and the development of treatments at MCRI for:

Key Programs of Stem Cell Medicine Research at MCRI

Program 1: Disease Modelling & Drug Screening using Stem Cells

Disease Modelling & Drug Screening is MCRI Stem Cell Medicine’s flagship research program.

What is disease modelling using stem cells?

Disease modelling takes a patient’s cells, creates stem cells and develops them into tissues and mini organs such as kidney, blood, brain, or heart in the laboratory. By doing this, we create a ‘model’ of their disease – often referred to as a ‘disease in a dish’ – to help us understand what things have gone wrong and predict how the disease will progress in an individual patient. The better understanding we have of a disease, the better we can recognise how to prevent, treat or cure it. 

What is drug screening using stem cells?

By modelling a patient’s ‘disease in a dish’, we’re able to test a range of drugs on their tissues to find the most effective treatments and cures for that patient. MCRI Stem Cell Medicine is currently expanding its Disease Modelling and Drug Screening research capacity by investing in our research talent and state of the art equipment.  This approach capitalises on the ability of stem cells to generate large numbers of normal and diseased human cells in the laboratory on which novel drug compounds can then be applied to determine their toxicity and efficacy. This initiative will take us a step closer to providing effective precision and personalised treatments to patients, provide a new avenue for pharmaceutical companies to discover and test drugs for clinical use, and reduce the need for animal experimentation. 

Program 2: Cellular Therapies & Regenerating Organs using Stem Cells

Using stem cells toward cellular therapies and regenerating organs is the second frontier of research at MCRI Stem Cell Medicine.

What is cellular therapy using stem cells?

Cellular therapies involve the injection or transplantation of human cells from a donor (potentially from the patient itself) to repair and regenerate damaged or malfunctioning tissue.   Some types of cellular therapy are already standard procedure in medical practice, for example, hematopoietic stem cell transplantation (also known as a bone marrow transplant) is an established treatment for a variety of blood cancers and disorders.  Across the globe, including at MCRI Stem Cell Medicine, researchers are working toward delivering the next generation of stem cell cellular therapies.

What is organ regeneration using stem cells?

In some cases, delivering a small amount of cells or tissue into a patient may not be an effective treatment - in cases such as heart or kidney failure - whole organs may need to be replaced.  MCRI Stem Cell Medicine conducts research into approaches for the bioengineering of replacement organs.  The tissue to create these bioengineered organs are grown in the lab from human-derived induced Pluripotent Stem Cells, and stem cell scientists work closely with bioengineers to create 3D organ structures.  The hope is that bioengineered organs may ultimately provide treatments for many conditions, including heart disease, bone disease and kidney disease. 

Working in partnership with the US biotechnology company, Organovo, MCRI Stem Cell Medicine recently set up the Southern Hemisphere’s first 3D bioprinter to support our work in kidney tissue engineering and disease modelling.