Stem cell treatments and regulation - a quick guide for consumers

Our bodies are made up of about 200 different types of cells, such as muscle cells, skin cells and nerve cells. Each cell type performs a specialised function. Stem cells are cells that are not specialised. Their role is to replace specialised cells that have been lost through injury, diseases, or the normal course of events.

Medical research into stem cells is still at the very early stages. Our understanding of the risks associated with stem cell treatment is limited. Further research is required to create safe and effective treatments.

In Australia, the only proven safe and effective stem cell treatment is haematopoietic stem cell transplantation (using stem cells from umbilical cord blood or bone marrow) for the treatment of disorders of the blood and immune system such as leukaemia.

Currently, no other stem cell treatment has been demonstrated to be safe and effective. However, some clinics located both in Australia and overseas offer unproven stem cell treatments.

Any unproven stem cell treatment may be unsafe, posing serious risks to your health. The risks include infection, allergic reactions or the development of cancer; complications that can be fatal.

The only established stem cell treatment approved in Australia is haematopoietic stem cell (HPC) transplantation (using stem cells from umbilical cord blood or bone marrow), which is standard practice for the treatment of disorders of the blood and immune system such as leukaemia. The use of fresh bone marrow-derived HPC is standard practice and is exempt from oversight by TGA. Cord-blood derived HPC is usually stored frozen prior to use, so involves some processing, and is approved by TGA.

An unproven stem cell treatment may also pose serious, potentially fatal, risks to your health including infection, allergic reactions, rejection of the cells by your immune system and the development of cancer.

Unproven stem cell treatments may cause financial hardship, including treatment and ongoing follow-up costs, as well as the cost of emergency medical care in the event that something goes wrong. If you undergo treatment outside Australia, there are additional costs associated with travel.

Importantly, undergoing an unproven stem cell treatment may interfere with proven and potentially beneficial therapies recommended by your general practitioner or specialist. It can also disqualify you from participation in a registered clinical trial.

In Australia, the Therapeutic Goods Administration (TGA) is responsible for the regulation of products for therapeutic use including human cells and tissues (termed 'biologicals'). This includes human stem cell treatments.

However, the TGA does not regulate medical practice. Some products that would otherwise be considered biologicals are excluded from TGA regulation (through the Therapeutic Goods (Excluded Goods) Determination 2018). Under this provision stem cell treatments may not be subject to regulation by TGA if they meet all of the following criteria:

1. collected from a patient who is under the clinical care of a medical or dental practitioner registered under a law of a State or an internal Territory
2. manufactured by that medical or dental practitioner, or by a person or persons under the professional supervision of that medical or dental practitioner in a hospital (except storage and testing), for that patient who must be a patient of that hospital
3. not advertised to consumers

Where one or more criteria are not met, including advertising to consumers, regulation by TGA will apply.

Cord blood can be stored in Australia. There are approved facilities for storing cord blood stem cells (collected from a newborn's umbilical cord). There are currently no TGA approved facilities for the storage of other stem cells in Australia.

Stem cells derived from sources such as dental pulp are subject to the same regulatory requirements as all other human cell and tissue products for therapeutic use.

However, the use of these stem cells to produce a product that could be of therapeutic use is only in the research and development stage. It is possible that in coming years these stem cells will be used in products for clinical trials, but products suitable for full marketing approval by the TGA are still some time away.

Autologous cells are those that are removed from, and applied to, the same person, so the donor and recipient are the same.

Autologous cells are those that are removed from and applied to the same person. These cells may be treated, processed or purified after removal. As the cells come from the same person, there is a lower chance of reaction and rejection of the cells by the patient's immune system.

However, an unproven autologous stem cell treatment may be unsafe, posing serious risks to your health. The risks include infection and growth of specialised tissue in the wrong place (ectopic tissue formation).

Unproven stem cell treatments may also involve significant financial costs, including treatment and follow-up costs, as well as the cost of emergency medical care in the event that something goes wrong. Undergoing an unproven stem cell treatment may interfere with proven and potentially beneficial therapies or treatment plans recommended by your general practitioner or specialist. It can also disqualify you from participation in a registered clinical trial.

The collection and use of cord blood stem cells for a specific patient is known as directed donation. On rare occasions, a hospital will collect and store a baby's cord blood for a family member who has a medical condition that can be treated with cord blood transplantation. This requires a treating doctor's approval.

The TGA ensures that where specific therapeutic claims are made about a product or therapy then these are supported and the product is safe. For natural therapies that do not have oversight by TGA there is no guarantee of safety or effective use.