Stem cell medicine in the laboratoryWhat is a stem cell?

Our bodies are made up of billions of cells of many different types that serve many different functions. Each tissue in the body has unique cell characteristics that allows them to function as they do - heart cells make the heart beat, kidney cells filter the blood, and eye cells allow us to see. Each of these specialised cell types originally developed from a stem cell. Stem cells are the building blocks designed for growth and repair. Stem cells have two distinctive characteristics distinguishing them from other cell types in the body: firstly, they have the ability to duplicate and create more of themselves; secondly, they possess the ability to evolve or ‘differentiate’ themselves into specific types of cells such as brain, nerve, kidney etc



A single cell that can replicate itself or

 Pluripotent stem cell
Pluripotent stem cell
Differentiate into many different cell types
arrow pointing right
 different cell types

How do we use stem cells?

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. These cells are known as induced pluripotent stem cells (iPSCs). Our researchers are then able to differentiate the iPSC into specialised tissues we wish to study – for example blood, kidney, brain or heart. These lab-grown tissues are used to understand more about the diseases affecting that tissue, and explore treatment options.

Additionally, through the development of gene editing technology, our researchers can ‘correct’ malfunctioning genes in these iPSC models of disease to see the difference corrected genes may make to their tissues and organs. Read more about how we use these technologies to significantly improve our understanding of what causes a disease, how a disease may progress in patients, and how to treat or cure it.

Current stem cell therapies

The use of stem cells to treat patients is not new. For example, stem cells found in bone marrow and umbilical cord blood have been used to treat diseases of the blood and immune system for over 60 years.

Within Australia, the only proven treatments available involving stem cells are corneal (eye), skin grafting, and blood stem cell transplants for the treatment of some blood, inherited immune and metabolic disorders, cancer and autoimmune diseases. By and large, these represent traditional cell therapies in which cells or tissues are taken from a donor and transplanted into/onto a recipient. There are many other potential treatments, but these are still in the research phase or in clinical trials, and are yet to be proven safe and effective. For those seeking cures, the wait for delivery on these novel treatments can be frustrating, however it is important that they undergo rigorous testing to ensure they are safe.

Medical experts are concerned that there are already clinics and companies offering ‘stem cell treatments’ that have not been validated in registered clinical trials. These unproven ‘stem cell treatments’ are offered across the globe, including Australia, and tend to be expensive, ineffective, and potentially even unsafe. In October 2017, the Therapeutic Goods Administration (TGA) announced that it will be changing Australian regulations for autologous human cell and tissue products. This change is designed to ensure experimental treatments are properly monitored and assessed.

Before embarking on any treatment, it is important you get the facts and discuss all your options with your doctor. Remember the testimonial from a patient is not proof that a treatment works.

To learn more about stem cells, how and why clinical trials are used to evaluate new treatments and the issues you should consider before seeking experimental, unproven stem cell therapies, please visit Stem Cells Australia.