Confronting unproven stem cell treatments in Australia: The new regulation

Stem cell research holds tremendous promise for the treatment of a wide variety of illnesses and conditions, from spinal cord injury to autism. However, much more work is necessary to translate stem cell research into safe and effective therapies. Many clinics are bypassing this crucial stage, advertising stem-cell based procedures on the internet despite little evidence for their safety or efficacy (see BioNews 908). Some patients, especially the acutely ill and desperate, are prepared to pay a high price for these stem-cell-based treatments.

The range of illnesses for which there are proven treatments based on stem cells is negligible. At present, the only established safe and effective stem-cell treatment is haematopoietic stem cell transplantation (HPC) transplantation; for decades, doctors have been using stem cells from bone marrow and umbilical cord blood to treat disorders of the blood and immune system such as leukaemia.

But other stem cell therapies are merely experimental. Just like a new drug, stem cell therapies must be assessed and meet stringent standards before obtaining the formal approval from regulatory bodies to be used to treat people. Thus, medical research into stem cells is still at infancy stages and more research is needed to create safe and effective treatments.

The grave concern is that stem cell therapies are being sold around the world, including Australia, before they have been proven safe and effective (see BioNews 921). There is direct advertising to consumers via the internet. There have been disturbing reports of baseless claims of cures; charlatans and adverse medical events. The potential risks include infection, allergic reactions, development of cancer, rejection of the cells by the patient's immune system and other complications that can be fatal. Also there is no evidence that these treatments will work. They may cause financial hardship as they are expensive and the procedures may involve repeat treatments. There may even be the cost of emergency medical care if something goes wrong. Undergoing unproven stem cell treatment may interfere with proven therapies and it can also disqualify the patient from participation in clinical trials.

Previously, these treatments are available mostly in developing countries with weak laws or lax enforcement such as China and India. However, these days, Australia too has some stem-cell businesses, growing to more than 60 such enterprises since 2011. Australia had among the world's highest concentration of stem-cell clinics, with websites advertising medical procedures and even anti-ageing therapies. Other developed countries that also engage in the direct-to-consumer marketing of stem-cell treatments are Ireland, Singapore, Germany, Italy, Japan, and the United States. Desperate people are at risk of becoming patients without the safeguards of a controlled clinical trial.

Australia's Therapeutic Goods Administration (TGA), which is responsible for ensuring that therapeutic goods are of an acceptable standard, introduced an exemption for certain types of biologicals including autologous therapies (cells and tissues that are removed from and applied to the same person, i.e., the donor and the recipient are the same person) in 2011. These cells may be treated, processed or purified after removal. As the cells come from the same person, there is a much lower chance of rejection of the cells by the patient's immune system. The exception was introduced because they were seen as an extension of medical practice and it was intended to exclude straightforward procedures from undue regulatory interference. Under the Therapeutic Goods (Excluded Goods) Order No1 of 2011, 'stem cell treatments may not be subject to regulation by TGA if they are human cells that are collected, processed and returned to the same patient, in a single course of treatment while under the clinical care and supervision of a registered medical practitioner'. However, these unproven stem cell-based therapies may be unsafe.

Recognising the potentially severe risks to human health and welfare posed by these stem cell-based therapies, TGA responded to this serious problem by conducting two consultations (one in 2015 and the other in 2016) with various stakeholders to seek public input to determine an appropriate regulatory framework. Four options were proposed for the future regulation of autologous human cell and tissue products. Option one maintains the status quo allowing providers to continue marketing unproven autologous stem cell directly to consumers. Options two, three and four prohibit the direct-to-consumer marketing of autologous stem cell but they differ according to the degree of manipulation that is permitted before regulatory exemption no longer applies.

Last month, TGA announced that changes to the law on autologous stem-cell therapies will be implemented in 2018. A transition period will permit providers some time to adjust to the new regulations. Some of the products will be subject to regulation as biologicals by the TGA with the level of control being determined by the risk posed to patient safety. This long-awaited and welcomed crucial step will place Australia into closer alignment with other jurisdictions such as the European Union and the US. When the new regulation is fully implemented, it is likely to be a successful effort to push back against unscrupulous doctors. While this is undoubtedly a progress, much would depend on the practical reality of the enforcement measures. It remains to be seen.