Document Reference: AHQ-WEB-EDU-STEM-001 | Version 1.0 | October 2026
| Important Notice: Educational resource only. Not medical advice or a treatment recommendation. |
Few areas of medicine attract as much attention, or as much confusion, as stem cells. Headlines about cures sit alongside warnings about unproven clinics. This guide explains, in plain language, what stem cells are, the main types scientists work with, which uses are established, and what has changed in stem cell medicine over the past two years.
What Makes a Cell a Stem Cell?
Most cells in the body are specialised: a heart muscle cell beats, a red blood cell carries oxygen. Stem cells differ in two ways. They can renew themselves, dividing to make more stem cells, and they can give rise to specialised cell types. The range of cell types a stem cell can produce is called its “potency”, and it varies considerably between types.
The Main Types of Stem Cells
Embryonic stem cells are derived from very early-stage embryos and are pluripotent, meaning they can become almost any cell type in the body. In Australia, research involving human embryos is tightly regulated under federal legislation and requires a licence.
Adult (tissue-specific) stem cells are found throughout the body, where they maintain and repair particular tissues. The best understood are haematopoietic stem cells in the bone marrow, which produce all blood and immune cells. Another group, mesenchymal stromal cells (MSCs), is found in bone marrow, adipose (fat) tissue and umbilical cord. Many scientists prefer the term “stromal” because MSC preparations are mixed cell populations, and not all of them behave as true stem cells. Much current MSC research focuses on how these cells signal to and modulate the immune system, rather than on whether they become new tissue. Our guide to stromal vascular fraction (SVF) explains how MSCs occur in fat tissue.
Induced pluripotent stem cells (iPSCs) are adult cells, such as skin or blood cells, reprogrammed in the laboratory back into a pluripotent state. Shinya Yamanaka’s team first described the technique in 2006, a discovery recognised with the 2012 Nobel Prize in Physiology or Medicine. Because iPSCs avoid the use of embryos and can be produced in large quantities, they are now the starting material for several of the most closely watched therapies in the world.
What Is Proven Today
For decades, one stem cell therapy has stood well above the rest in evidence. The National Health and Medical Research Council (NHMRC) identifies haematopoietic (blood) stem cell transplantation, which includes bone marrow and cord blood transplants, as the established, proven stem cell treatment. It is a standard treatment for certain blood cancers and serious blood and immune disorders, and has been used for more than fifty years.
What Has Changed Recently
In December 2024, the US Food and Drug Administration approved its first MSC therapy, Ryoncil (remestemcel-L). It is a donor bone marrow-derived MSC product for steroid-refractory acute graft-versus-host disease, a serious complication of blood stem cell transplantation, in children aged two months and older. It was developed by the Australian-founded company Mesoblast. Approval was based primarily on the rate and duration of treatment response 28 days after starting therapy.
In 2026, Japan became the first country to approve medicines made from iPSCs: ReHeart, a cardiac cell sheet for severe heart failure, and Amchepry, a treatment for Parkinson’s disease. According to the manufacturer’s March 2026 announcement, Amchepry received conditional, time-limited approval, and full approval depends on confirming its efficacy and safety in further studies within a set period. The approval rests on a small trial: the Kyoto University Phase I/II study published in Nature in 2025 involved seven patients. No equivalent approval has been granted in Australia.
What Is Still in Clinical Trials
Type 1 diabetes is one of the most closely followed areas. Vertex Pharmaceuticals is testing zimislecel, a donor stem cell-derived islet cell therapy infused into the liver’s portal vein to replace the insulin-producing cells destroyed in type 1 diabetes. Recipients take anti-rejection medication. In the Phase 1–2 results published in the New England Journal of Medicine in 2025, all participants showed engraftment and islet function, and 10 of the 12 who received a full dose no longer needed injected insulin at one year. The authors described the study as small and short-term. The program has since met obstacles: Vertex’s 2025 annual report states that enrolment in the pivotal trial is complete but completion of dosing has been temporarily postponed pending an internal manufacturing analysis. Promising early results are not yet an approved treatment.
How Stem Cell Treatments Are Regulated in Australia
The Therapeutic Goods Administration (TGA) regulates autologous cell products (those taken from and returned to the same person) at three levels: excluded from TGA regulation, regulated with some exemptions, or fully regulated as a medicine or biological. To be excluded, a product must be manufactured and used in a hospital, collected and used under the supervision of the medical or dental practitioner who has clinical care of the patient, and not be advertised to consumers. Exclusion from TGA regulation is not the same as TGA approval of effectiveness. Our article TGA Regulation Explained covers this framework in more detail.
Warning Signs Worth Knowing
Stem Cells Australia, a national research network, lists warning signs including: being asked to pay for an experimental treatment outside a registered clinical trial; one treatment claimed to help many unrelated diseases; testimonials offered as evidence; claims of no risk; and treatments advertised directly to consumers rather than discussed with a doctor. Your GP or treating specialist is the best person to help you weigh up any option.
Frequently Asked Questions
What is the only proven stem cell treatment?
Haematopoietic (blood) stem cell transplantation, including bone marrow and cord blood transplants, is the long-established, proven stem cell treatment, used for certain blood cancers and blood and immune disorders.
What is the difference between embryonic stem cells and iPSCs?
Both are pluripotent. Embryonic stem cells come from early-stage embryos, while induced pluripotent stem cells are made by reprogramming adult cells in the laboratory.
Are any iPSC therapies approved?
Yes, in Japan only. In 2026 Japan granted conditional, time-limited approval to iPSC-derived therapies for Parkinson’s disease and severe heart failure. Further studies are required for full approval.
Who regulates stem cell treatments in Australia?
The Therapeutic Goods Administration (TGA), which applies different levels of regulation depending on how a cell product is manufactured, where it is used and how it is promoted.
The Bottom Line
Stem cell science is moving faster than at any point in its history, but for most conditions stem cell-based treatments remain under investigation. The most reliable signals of progress are peer-reviewed clinical trials and formal decisions by regulators such as the TGA, the US FDA and Japan’s Ministry of Health, Labour and Welfare.
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