Self-regulation of autologous cell therapies

A first step for a virtually unregulated industry

Autologous cell therapies are being used privately by increasing numbers of clinicians to treat a number of disorders including rheumatological conditions, sports injuries, strokes, Parkinson disease and multiple sclerosis.1 Sources of cells include stromal vascular fractions obtained during liposuction which contain mesenchymal stem cells,2 as well as platelet rich plasma obtained from peripheral blood.3

In Australia, prospective authorisation of autologous cell therapies by the Therapeutic Goods Administration (TGA) is often not required. The current regulations contain exceptionally broad exemptions permitting clinicians to treat their patients with autologous cells without restrictions either on the extent of manipulation (excluding genetic manipulation) or the intended final use of the cells. The TGA's expectation is that the Australian Health Practitioner Regulation Agency (AHPRA) would regulate these clinical practices. In contrast, the United States' Food and Drug Administration (FDA) requires minimum standards for manufacturing autologous cells, and more than minimal manipulation of cells requires both manufacturing and product approvals.

Some Australian companies have become concerned about the lack of oversight of the safety, efficacy and ethics of cell-based therapies and related advertising claims. To better understand the current situation in Australia, the New South Wales Stem Cell Network conducted its 17th Workshop in October 2012 on this subject.4 In the workshop, those using autologous cell therapies expressed a desire for more structured industry self-regulation than occurs at present. A steering committee to achieve this goal was established, which includes us and representatives from nine entities practising autologous cell therapies: Cell Innovations, Hunter Regenerative Medicine, Lakeside Sports Centre, Macquarie Stem Cells, Magellan Stem Cells, New Zealand Stem Cell Clinic, Regeneus, South Sydney Sports Medicine Centre and Stem Cell Solutions.

Six teleconferences were held, after which it was agreed a written code of conduct would be created. The code, currently being written, is to be modelled on that produced by Medicines Australia, using a template provided by the Australian Competition and Consumer Commission. Four goals were agreed t practise evidence-based medicine; ensure fully informed consent is obtained; manufacture injected autologous products using internationally accepted standards; and follow the advertising standards set for medical practitioners by AHPRA.

Most forms of regulation are external to an industry, and are often by governmental agencies. Medicines Australia, however, regulates aspects of the pharmaceutical industry through a code of conduct. The organisation imposes large financial penalties and publicly exposes phar maceutical companies breaching the code. A second example is fertility clinics, with fertility specialists banding together to decide the rules of conduct for in-vitro fertilisation.

However, examples abound of limited regulation not providing necessary checks and balances in medicine. For example, in France, the regulatory agency AFSSAPS (Agence française de sécurité sanitaire des produits de santé) was found to have responded too slowly to allegations of cardiotoxicity and patient deaths from the antidiabetes drug Mediator (benfluorex). In 2012, the French Government replaced AFSSAPS with a new agency with a stronger mandate to protect patients' interests.5

Whether self-regulation will protect any patients receiving autologous cell therapies remains to be seen. Nevertheless, some form of regulation is needed urgently, and neither the TGA nor the AHPRA seem empowered or ready to engage actively in the matter. That provides an opportunity for clinicians and scientists to step in. It is true that the area is a commercially lucrative one, but then so are the Australian pharmaceutical industry and fertility clinics for which self-regulation appears to be working. However, one difference is that autologous cell therapies, though in high demand, are mostly unproven. This is a concern, but self-governance could be an interim solution, provided the code of conduct pursues the goal of evidence-based medicine and seeks input from regulatory and other bodies as well as potential consumers.

In the short time we have been working with members of the autologous cell therapy consortium, we have been heartened by the movement so far towards accepting and practising the goals of the code of conduct. Self-regulation of autologous cell therapies is a first step in an industry which would otherwise have virtually no regulation.