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What are stem cells?

What are stem cells?

Stem cells are stirring up great excitement in medical research. What are they and why are scientists so intrigued by them?

Stem cells have been part of us since birth. They are in our hair, skin, intestines, bone marrow, brain, joints, and muscles. As we grow through childhood and adolescence, stem cells generate new tissue and when we have stopped growing, they replace damaged and aged tissue. It is this ability to repair damaged tissue that makes stem cells so unique, and their potential application in modern medicine so far-reaching.

What makes stem cells different?

What makes stem cells different?


What makes stem cells different?

Stem cells are different from other cells in the body in three main ways:

  • Stem cells are unspecialised. They have not developed into cells that perform a specific function. 

  • Stem cells can differentiate. This means they can divide and produce cells that have the potential to become other more specific cell types, tissues or organs. These new cells and tissues are used to repair or replace damaged or diseased cells in the body. Once cells have differentiated, they have less capacity to form multiple different cell types, and become ‘committed’ to becoming a particular cell type. Skin stem cells, for example, give rise to new skin cells when needed; to assist regeneration after damage and as part of the normal ageing process.

  • Stem cells are capable of self-renewal. Stem cells are able to divide and produce copies of themselves, which leads to self-renewal. Once a cell has become specialised (has differentiated) to a particular tissue or organ, it has a very limited capacity to self-renew (produce new stem cells) but instead produces only cells relevant to that organ.

Types of stem cells

Stem cells are with us right through to adulthood as part of the process to repair ageing or damaged tissues. 

There are four types of stem cells:

  • Adult Stem Cells (ASC) are found in most organs in the body and also in infants and children.  Sometimes they are called non-embryonic stem cells.
  • Induced Pluripotent Stem cells (iPSC) are genetically modified mature adult cells. This means they are more like embryonic stem cells; a discovery which has eased concerns about using Embryonic Stem Cells.  iPSC act like pluripotent stem cells, which can change into any cell type. Using iPSC eliminates the need for embryonic stem cells in research.
  • Embryonic Stem Cells (ESC) are found in early embryos. Their most important characteristic is their ability to turn into all the tissues of the body, called “pluripotency”.
  • Cord blood stem cells and amniotic fluid stem cells Cord blood stem cells are harvested from the umbilical cord after childbirth. They can be frozen in cell banks for use in the future.  These cells have been successfully used to treat children with blood cancers such as leukemia and certain genetic blood disorders.

Stem cells have also been found in amniotic fluid. This is the fluid that surrounds a developing baby inside the mother’s womb. However, more research is needed to help understand the potential uses of amniotic fluid stem cells.

What do stem cells do?

What do stem cells do?

Most of the 300 trillion cells that make up the human body are fully specialised for particular functions in organs such as the heart or the brain, or in tissues like muscle, fat and bone. Others play a supply or defensive role in the blood or immune system. 

In fact, each cell type has a specific lifespan and function, which is dependent on the activity that cell is required to perform. Some cells are replaced, others live for the duration of a person’s life. For example, blood cells only live for up to a few months, and are replaced at a rate of several billion each day, whereas brain cells may last a lifetime.

Why is stem cell research important?

Why is stem cell research important?

Over the next few decades, as we learn more about stem cells, we will see them become part of many modern medical treatments, giving hope to many facing currently untreatable and sometimes life-threatening illnesses. 

Key areas of research for future therapies include:

  • stroke and traumatic brain injury
  • blindness
  • deafness
  • baldness
  • spinal cord injury
  • lung and breast cancers
  • chronic lung disease in premature babies
  • acute heart damage
  • motor neurone disease
  • liver cirrhosis
  • diabetes
  • uterine disorders
  • prostate disease
  • muscular dystrophy
  • osteoarthritis and rheumatoid arthritis
  • genetic bone disease
  • multiple sclerosis and other neurological disorders

How does stem cell research work?

How does stem cell research work?

1. Treating illnesses
Stem cells found in bone marrow have been in use for 40 years to replace cells destroyed by high dose chemotherapy in a variety of cancers and blood diseases. As stem cells can create new tissues, they promise new treatments for conditions like spinal cord injury, where they may replace nerve cells destroyed in accidents, restoring the ability to walk and move the arms. Another example is strokes, where brain stem cells are important in restoring function after loss of brain tissue. They are also in advanced clinical trials for degenerative conditions like arthritis, macular degeneration, which causes blindness in an increasing number of people, and diabetes. There is early evidence that scientific knowledge gained from studying stem cells could provide a completely new approach to treating cancers.

2. Working out how important illnesses are caused
Stem cells can be altered to behave like abnormal cells found in illnesses. Cancer is a prime example. Scientists can use actual human cells to study conditions that could previously only be approached using animals. New techniques such as Induced Pluripotent Stem cells (iPSC) enable scientists to study diseases in cells derived from the patient’s own faulty cells.

3. Understanding fundamental functions of living cells
Stem cells kept under the right conditions are immortal - they can be maintained forever. They therefore provide a unique medium for observing in depth the way living cells develop, work and repair themselves. Understanding the basic biology of stem cells is opening the doors to a vast field of new and vital research. This research drives the search for new cures for diseases that up to now have been difficult or impossible to treat effectively.

Clinical Trials

Clinical Trials

The path to bring a promising cure from a laboratory to a medical treatment we can access in a clinic or hospital takes several years and is in several stages:

  • Basic research
  • Preclinical research
  • Clinical research and clinical trials
  • Approval for use by regulators and government authorities

Further information about this process can be found in the resource:

How Science Becomes Medicine

The development of all medical treatments requires clinical trials. This stage of medical research must be undertaken to ensure the treatment is safe for use on humans.

  • Disease prevention — using drugs, vitamins, foods to reduce risk
  • Treatments — new drugs or combinations of drugs; new ways of giving treatment, new types of treatment
  • Diagnosing disease — new tests or scans
  • Controlling symptoms — new drugs or complementary therapies.

Trials aim to find out if a new experimental drug or procedure

  • Is safe
  • Has side effects
  • Works better than the currently used treatment
  • Helps you feel better.

New treatments must be thoroughly tested. A new drug, for example is investigated first in the laboratory. If it looks promising, it is carefully studied in people. There are three different types of clinical trials. These are called Phase 1, 2 and 3.

Phase 1 is the earliest trials in the life of a new drug or treatment. They are usually small trials, recruiting up to 30 patients.

About 70% of new treatments tested at Phase 1 make it to Phase 2 trials. Phase 2 trials are often larger than Phase 1. There may be up to 50 people taking part. If the results of Phase 2 trials show that a new treatment may be as good or better than existing treatment, it then moves to Phase 3.

Phase 3 trials compare new treatments with the best currently available treatment (the standard treatment). Phase 3 trials are usually much larger than Phase 1 or 2. This is because differences in success rates may be small, so many results are required to show the difference. Sometimes Phase 3 trials involve thousands of patients in many different hospitals and even different countries. Phase 3 trials are usually randomised. This means the researchers put the people taking part into two groups at random. One group gets the new treatment and the other the standard treatment.

Clinical trials are essential to the development of new interventions. For example, without clinical trials, we cannot properly determine whether new medicines developed in the laboratory or by using animal models are effective or safe, or whether a diagnostic test works properly in a clinical setting. This is because computer simulation and animal testing can only tell us so much about how a new treatment might work and are no substitute for testing in a living human body.

Clinical trials also permit testing and monitoring of the effect of an intervention on a large number of people to ensure that any improvement as a result of the intervention occurs for many people and is not just a random effect for a one person.

Most modern medical interventions are a direct result of clinical research. New interventions for most diseases and conditions — including cancer, heart disease, high blood pressure and asthma — have been developed through clinical research. Clinical trials often lead to new interventions becoming available that help people to live longer and to have less pain or disability.

Clinical trials can also help to improve health care services by raising standards of treatment. Doctors and hospital staff involved in clinical trials are continually trained to provide best practice patient care. Australian clinical trials are recognised internationally for including very high quality patient care.

You can access further information from the International Society for Stem Cell Research's public education resource About Stem Cells which has a section on clinical trials.

The Australian Government also has resources for learning about clinical trials in the Australian context: www.australianclinicaltrials.gov.au. This website

  • enables you to search for clinical trials in Australia,
  • to understand how to enroll in a clinical trial
  • the questions you should consider.

Stem Cell Research in Australia

Stem Cell Research in Australia

Australia’s scientists have been at the forefront of stem cell research since stem cells were first identified. Right now, Australian scientists hold senior positions on international scientific committees and conference organisations.

However we have a challenge: our top scientists find it increasingly difficult to secure funding for their projects whether this be through Commonwealth Government funding and the National Health and Medical Research Council (NHMRC), state government sources, or their own institutions.

Together, we must work to grow the potential of stem cell research in this country and protect the future success of Australian stem cell research. We can do this by ensuring the best and brightest researchers and the most promising projects have the funds they need to thrive.

All too often, Australian scientists are lured away to other countries because they can’t get the financial support needed to continue their research. Moreover, many potentially ground breaking research projects fail to ever achieve the results researchers know could be possible – due to lack of funding.

We must not let this continue to happen. Join us in funding the next phase of stem cell research in this country and help Australian researchers create the type of future we all know is possible.

Donate now

FAQs

The National Stem cell Foundation of Australia is a philanthropic foundation supporting stem cell research and its potential applications in the treatment of illnesses for which current treatments are unsatisfactory. We do not treat patients, do not have any direct affiliation with treatment centres, and cannot give personal medical advice or recommendations.

Although there have been major advances in the study of stem cell biology, and in our understanding of how stem cells can be manipulated to produce different cell types for potential treatment applications, at present most stem cell treatment remains experimental, with benefits and safety still to be clearly established.

Yes, and in fact it was Australian stem cell scientiss who were successful in the use of blood forming stems in bone marrow transplantation, which has saved many lives over the past 50 years. This treatment is known as a bone marrow transplant, and you may heard of procedure being used to treatment some forms of leaukaemia.
Besides bone marrow transplants, stem cells found in the skin and cornea of the eye have also been used successfully in skin grafts and corneal grafts. In all these instances the stem cells are already committed to producing the cells of the tissues which is damaged – ie they are doing the repair job that they are already programmed to perform.

The Canadian Stem Cell Network website https://stemcellnetwork.ca/stem-cells/disease-regional-profiles/ has good information relating to disease specific progress in stem cell treatment research, as does the European Stem Cell website where you can search for progress in a specific disease area:  https://www.eurostemcell.org/what-diseases-and-conditions-can-be-treated-stem-cells

Your treating specialist is the person who will be most up to date on the standard treatments available for your condition and on the progress of exciting new therapies. They will also be able to assist in assessing the suitability of a clinical trial that you may be interested in, and in assessing your eligibility to participate. We strongly advise you to visit your GP and not seek medical advice from the internet.

The Australian and New Zealand Clinical Trials registry at www.ANZCTR.org.au lists current trials open within Australia and NZ. The US trials register at www.clinicaltrials.gov lists studies that are taking place in the US and many other countries. Eligibility criteria and contact details for the Study coordinator are listed for each study.

Many registered studies are well designed trials, approved by ethics committees, registered with the NIH and carried out in academic institutions, without charge to the patient. Unfortunately the US site now also lists ‘trials’ carried out by private clinics that may not meet appropriate scientific or ethical standards. There are unfortunately now many 'Treatment Centres' and ‘Regenerative Medicine Clinics’ around the world, including in Australia, that advertise unproven and unregulated stem cell treatment (usually using autologous patient cells obtained by liposuction, see below) for a large number of diseases. Sadly these trials are carried out without evidence of safety or efficacy of this treatment. and usually at significant cost to the patient (not reimbursable by Medicare).

The ‘Australian Stem Cell Handbook’ available free on our website, has a chapter on ‘Unproven Therapies’ and lists some of the questions you should ask in trying to establish the credentials of any centre from which you might consider seeking treatment. The booklet also provides further information on stem cells and how they may ultimately be used. You may find stem cell treatment centres outside Australia advertised on the internet. It is common for such centres to also promote unsafe stem cell treatments. Information about “stem cell tourism” can be found in the Foundation’s handbook, as well as from
The European Stem Cell website at eurostemcell.org has good information relating to disease-specific progress in stem cell treatment research. Your treating specialist is the person who will be most up to date on the standard treatments available for your condition and on the progress of exciting new therapies. They will also be able to assist in assessing the suitability of a clinical trial that you may be interested in, and in assessing your eligibility to participate

Human embryo research and the use of human eggs for research and training in assisted reproductive techniques (ART), is tightly regulated in Australia, requiring licensing of the researchers by the National Health and Medical Research Council (the NHMRC) for each research project.
The members of the Science and Ethics Committee of the NSCFA are not experts in this complex legal/ethical field. We did however seek advice from a senior consultant in the field and the reply received is copied below, explaining why altruistic donation of eggs or embryos for research may not always be possible – there are many ART clinics but not all will be associated with embryo research projects (and much research involving donated eggs would involve creation of embryos, so falls under these provisions).

In Australia, human embryo research is licensed and regulated by the NHMRC and their website has an Embryo Research Licensing section that lists current and past licensed projects. Essentially, all of the projects are carried out either within an ART unit or at least with their involvement so the licence holders will be the ART clinics.

There is tight control of the numbers of embryos that can be used in each project so, in general terms, there is no shortage of donated embryos for the projects i.e. clinics will be able to complete their projects using embryos donated from their own patients. This is why there has been really no need to transfer embryos between clinics. In fact, in many cases, patients within embryo research licensed clinics who wish to have their embryos used for research are told that it may not be possible to guarantee their inclusion in the research.

If you have attended a specialist within a Assisted Reproduction Clinic then this would be the most appropriate person with whom to discuss this further.

Australia is well served with 3 public Cord Blood Banks (CBB), one each in Melbourne, Sydney and Brisbane, collecting from a number of different maternity hospitals. These banks form the AusCord network and have been in operation for 30 years and are licensed by the Australian regulatory agency, the TGA as well as being internationally accredited by a body setting international standards for cord blood banking and release, called FACT (the Foundation for Accreditation of Cellular Therapies). 

There is no charge for the collection or storage of cord blood in the public banks but there are strict eligibility criteria and collection can only occur in one of the licensed obstetric units associated with the Banks, and if baby’s delivery occurs during the times that the collection staff are on duty.

This cord blood is not stored specifically for use by a member of your family. Cord bloods from Public Banks are made available for patients with suitable matching, in Australia and internationally, who need a marrow transplant but do not have a suitably matched family or adult unrelated donor. To date over a thousand cord blood units have been released to patients in need from the Auscord Banks.

Information about cord blood donation, including the AusCord Booklet, is available on the Stem Cell Donors Australia website.

For further information about altruistic cord blood donation and the hospitals at which cord blood collection is possible, the Banks may be contacted as follows:

  • BMDI CBB located at the Royal Children’s Hospital Melbourne: 03 8345 3385 or 03 8345 3384
  • Brisbane CBB at the Mater Hospital: 07 3163 1614 
  • Sydney CBB at the Sydney Children’s Hospital Randwick: 02 9382 0371.

In addition to the public banks there is a private cord blood bank in Australia: CellCare. They arrange collection of an infant’s cord blood at the obstetric hospital, process and store the cord blood for potential use by the infant donor, or another close family member, at some time in the future. There are costs associated with private cord blood collection and private banking. Contact details information about the services provided and the schedule of charges, may be found on the CellCare’s website.

After more than twenty years of research into stem cells, advancing our understanding how stem cells function and can be manipulated, scientists have opened a wide range of opportunities to diagnose and treat illnesses and tissue injuries that, up to now, have resisted treatment. Severe injuries to the spinal cord and Parkinson's disease in the brain are two that have received a lot of public attention. And the use of stem cells and their derivatives to carry or be therapeutic agents for treating serious diseases like cancer is growing exponentially. The Foundation aims to accelerate the rate of development of effective treatments based on this research.

Our web site contains up to date information about stem cell science and links to major sites in our field.


Both the European Stem Cell Foundation and the International Society for Stem Cell Research's A Closer Look at Stem Cells websites contain good information relating to progress in stem cell research and clinical application.

All three sites include sections on the ethical issues relating to stem cell research and the dangerous illnesses that are likely to respond to treatments based on stem cell science.

In surveys of public opinion, more than 80% of Australians support research into stem cells so the word "controversial" is not really accurate. Originally, controversy arose around the use of embryonic stem cells. Hardly any research in Australia now involves such cells. Most of it is on adult stem cells or their derivatives, such as induced pluripotent stem cells (iPSC). When you go to the relevant web sites, you will be able to find these things out for yourself.

The pressing ethical issue currently is the growth of private commercial clinics claiming to provide stem cell treatments for a wide range of diseases. Their treatments are largely untested in proper clinical trials, very expensive, and the companies prey on the fears of people desperate for help for their illnesses or those of relatives or friends. One might argue that there ought to be more controversy about this.

‘Therapeutic cloning’ refers to the formation of embryonic stem cells by Somatic Cell Nuclear Transfer (SCNT). In this process, the nucleus of an animal/human gamete (egg) is replaced with one from a mature cell (e.g. a skin cell) from another animal/individual, leading to the formation of a pluripotent cell with the genetic characteristics of the donor of the nuclear material. This pluripotent cell has the ability to divide and form an embryo. Although it is theoretically possible for an embryo formed through SCNT to develop into a ‘clone’ of the nuclear donor this could only occur if the embryo were to be implanted into a uterus. This is strictly prohibited in Australia, Europe and the USA and has never occurred. It is important to note also that SCNT is an extremely difficult process with a low success rate. The use of pluripotent cells derived from embryos formed by SCNT has very largely been superseded by the discovery in 2006 that it was possible to reprogram ‘adult’ somatic cells to revert to a pluripotent embryonic like cell – these cells are termed induced pluripotent cells and show great promise for future therapeutic and research applications without the ethical concerns raised by the use of embryonic stem cells whether derived from ’excess’ fertilised embryos created by IVF for fertility treatment, or by SCNT.


The ethical considerations around the use of embryonic SC in research and treatment of disease as well as further information about the different types of stem cells, are well laid out in several FACT sheets to be found on the this link to the European Stem Cell Foundation. Secondary teachers and students may also find information on ethical issues in this subject sequence for VCE Biology here.

As stem cells can be induced to grow into new tissues, they have great potential to treat illnesses where tissue has been completely lost, for instance after injury to the spinal cord or nerves, death of heart muscle from a heart attack, liver failure from destruction of cells by viruses or toxins or destruction of functioning lung tissue by disease. Formal clinical trials of adult stem cells are in progress in several countries to assess whether such treatments are effective and safe.

The ability to “manufacture” stem cells from adult donors and patients themselves (see section above on iPSC) has reduced the need to use embryonic stem cells, and opened up a wide range of new possibilities in immunological treatments for cancer, production of vaccines and new therapeutic proteins and diagnostic tests.

In addition, stem cells and the tissues derived from them are an increasingly useful tool for researchers, providing living cells and tissues for understanding diseases and testing new treatments.

Current Stem Cell Treatments

Stem cell therapy using tissue stem cells has been in routine use since the 1970s! The most well-established and widely used stem cell treatment is the transplantation of blood stem cells to treat diseases and conditions of the blood and immune system, or to restore the blood system after treatments for specific cancers, for example, leukaemia. Bone marrow transplants are able to replace a patient’s diseased blood system for life, thanks to the properties of blood stem cells.

The late Professor Donald Metcalf, AC, who transformed cancer treatment and transplantation medicine paved the way for stem cell therapy in the treatment of leukaemia and many other conditions. Don Metcalf’s research on blood cell formation led directly to the development of bone marrow transplantation. This treatment is used worldwide every day, and has saved many thousands of lives. Each year the NSCFA celebrates Professor Metcalf’s achievement with The annual Metcalf Prize. This prize of $50,000 is awarded to the brightest mid-career female and male Australian stem cell researcher.

Stem cells are seen as a key tool in the future of medicine, for their abilities to regenerate damaged tissue, replace missing tissue and repair the effects of disease or injury. Experimental stem cell therapy is being developed for many common diseases, including blindness, stroke, arthritis, multiple sclerosis and heart attacks.

Important research is being undertaken by reputable hospital, biotechnology and university groups in Australia and around the world. However, it is still early days, and some private non-academic organisations and individual practitioners are offering stem cell treatments that have not been shown to be effective and have not been proven to be safe. If you’d like to help Australian researchers progress stem cell treatments, donate to the NSCFA because that’s exactly the work we support.

Currently, the range of diseases for which there are proven treatments using stem cells is quite small and the only established stem cell therapies are those of the blood system involving transplants of blood stem cells (usually from bone marrow but with cord blood also being developed as an alternative).

We recognise that patients and families who are facing severe illness or injury may consider trying untested treatments, especially when there seem to be few other options. People in this situation may be at risk of harm, through lack of information or misinformation provided by less reputable practitioners who are not recognised specialists or experts in the diseases they are proposing to treat.

We recommend that you are very cautious before you agree to try an untested treatment, particularly if it costs a lot of money. Your regular hospital doctors, specialists, and your general practitioner, should be able to offer accurate advice on which treatments have been proven to work, and which are unproven, unlikely to help, and may even cause harm. If it sounds to be too good to be true, be wary. The best protection for patients and families in this situation is to be fully informed.

Fortunately several highly respected resources are available via the internet to help you.

Several booklets can be downloaded to be read later or printed. If you cannot download a document or do not have access to a printer please send us an email: [email protected]. Please note we do not provide advice on stem cell treatments, clinics, or any medical advice whatsoever.

  • The National Stem Cell Foundation of Australia has produced a handbook to help you: What you need to know about stem cell therapies. The handbook aims to help patients critically analyse stem cell treatments before considering taking part in them. As with all medical treatments, there are risks involved. This handbook does not seek to advise or evaluate the treatments, or an individual’s reasons for travelling for treatment. Instead it aims to provide the patient with as much information as possible prior to considering any therapy. This handbook summarises the pros and cons of medical travel, potential risks and benefits, and helps patients evaluate their options when considering travelling overseas for unproven treatments.
  • The International Society for Stem Cell Research (ISSCR) has prepared a Patient Handbook on Stem Cell Therapies (pdf) that addresses frequently asked questions about clinical therapies using stem cells.
  • Access this international website on stem cells, research, and how stem cells become medicine. The website is aimed at the non-medical general public: www.aboutstemcells.org.
  • Check the section on the ISSCR website for their checklist of What to Ask when talking to someone promoting a stem cell treatment of any type.

Australia is home to many leading stem cell scientists and research institutes. These frequently hold seminars to discuss recent stem cell discoveries, research in Australia, and the promotion of non-medically proven stem cells treatments via the internet and social media. Below is a list of resources from Australia and international sources.

Educational resources