Australasian Science: Australia's authority on science since 1938

Stem Cell Loophole Must Be Closed

By Richard Harvey, Martin Pera and Megan Munsie

Unproven stem cell treatments are being offered in Australia without regulatory oversight.

The use of stem cells to treat currently intractable conditions has the capacity to revolutionise medicine. However, despite more than a decade of intensive research on a number of stem cell systems, the only proven and approved stem cell therapy available for Australian patients is the transplantation of stem cells from bone marrow to treat diseases of the blood and immune system. Other stem cell applications are only just starting to be evaluated in clinical trials.

Despite this there has already been a proliferation of commercial clinics offering unproven stem cell therapies for a wide range of conditions. These treatments are not cheap – often with an asking price around $10,000 per treatment.

Bizarrely, single preparations of “autologous” (patient-derived) stem cells are being injected to treat diseases as diverse as osteoarthritis and autism, injuries including spinal fracture, cosmetic applications and ageing. Such practices rely on a business model that exploits the hope and hype surrounding stem cells, the expectations of desperately ill and frustrated patients seeking the latest therapies, and the trust normally afforded medical professionals.

There is real potential to harm patients because the behaviour of stem cells is unknown and unpredictable in many circumstances, particularly where the cells have been manipulated outside of the body. Practitioners involved in selling unproven treatments flaunt a number of medical ethical standards, and often misrepresent the nature of cells harvested, the benefits of therapies, and even their own expertise.

Due to a loophole in our regulations, Australia is set to become a destination for stem cell tourism. In 2011 the Therapeutic Goods Administration (TGA), which is responsible for ensuring that therapeutic goods in Australia are of an acceptable standard, introduced an exemption from undue regulatory interference for routine autologous therapies such as cardiac bypass grafts.

This exemption inadvertently created a loophole for opportunistic clinics, because the exemption also effectively allows clinicians to perform any type of unproven cell therapy as long as cells are obtained from the patient and used under the supervision of a medical practitioner for a single indication.

There are currently no standards for quality control of the manufacturing process or delivery of the cell preparation, and there is no obligation for practitioners to attest to the safety or efficacy of therapies. There is also no requirement to follow-up on the well-being of patients, or to report adverse events or findings in the scientific literature. The normal process of proving the safety of new cellular therapies through step-wise clinical trials has been completely bypassed.

The opinion of international experts and societies is clear: that provision of unproven autologous therapies to large numbers of individuals for profit is unethical. Local stem cell,patient and medical professional bodies have also spoken out, including the National Stem Cell Foundation of Australia, Motor Neuron Disease Australia, the Australian Rheumatology Association and The Royal Australasian College of Physicians. Even other government agencies such as the National Health and Medical Research Council recognise the dichotomy between the TGA loophole and their duty to promote and support evidence-based medicine.

In January the TGA signalled that it would consider tightening the regulatory framework for autologous stem cell therapies, and sought community feedback on possible solutions. A variety of options were flagged. Under all but the status quo option, the TGA exemption would only apply if autologous cells are minimally manipulated and used in applications where their physiological function remains identical to their role in the tissue from which they are harvested.

Expert scientific bodies including the Australian Academy of Science and Stem Cells Australia have called for the highest level of regulation, whereby cells would need to be registered with the TGA as therapeutic goods, with the risk and benefits assessed for evidence of efficacy, safety and quality control during manufacturing. There would also be an obligation to report adverse findings. None of these requirements would hinder the exploration of novel therapies through the normal channels of clinical trials.

This move towards robust community debate and education provides an opportunity for Australia to arrest the most egregious exploitation of the current regulatory loophole without compromising patient access to novel therapies. Ultimately, evidence and a duty of care to patients must underpin responsible translation of stem cell science and regenerative medicine into accepted clinical practice.

There is too much at stake to ignore due diligence and appropriate regulatory oversight. We eagerly await the TGA’s next step.

Richard Harvey (VCCRI, UNSW), Martin Pera (UoM, Florey & WEHI) and Megan Munsie (UoM) are members of Stem Cells Australia, an ARC-funded Special Research Initiative.