Group 2 - Clinical translation

Chair: Dr James Chong

Whilst Australia has a rich history in the development of stem cell-based clinical therapies, a meaningful future role for Australia in the clinical translation of emerging therapies requires decisive action now. In North America and Europe many regulated clinical trials of experimental stem cell therapies have been initiated. In contrast, comparatively few trials are being initiated in Australia. In addition, regulatory ‘loop holes’ have opened the door for unregulated delivery of unproven stem cell treatments for commercial gain, involving many diseases in several organ systems.

Australia’s relatively small population and biotechnology sector particularly disadvantage our participation in costly clinical translation in stem cell fields. It is vital that novel solutions are identified to address the hurdles impeding Australia’s engagement in this potentially transformative medical field.

Questions to get you thinking:

1. Managing the ‘valley of death’: Translating stem cell therapies from the research ‘bench’ to the clinical bedside involves high cost and great effort both at the preclinical and the early-phase clinical trials stages. What novel strategies, designed to particularly suit the Australian stem cell environment, might give the most promising therapies the best chance of success?

2. Cost of clinical trials: Clinical trials—particularly late-stage efficacy studies—are extremely expensive. Stem cell therapies pose a very high risk investment and are more reliant on government funding rather than industry financing. Is the standard model of clinical trials viable for Australian clinical translation of stem cell therapies?

3. Infrastructure for preclinical and clinical stem cell research: In comparison to other regions around the world, Australia has not fostered the creation of specific infrastructure for successful preclinical and clinical translation of stem cell therapies. How can Australia best take advantage of currently available infrastructure and secure funds to build on this? What role should collaborative projects with other countries play within Australian programs?

4. Careers: With traditional grant funding at record lows, researchers in clinical translation are leaving research for more secure positions including those in provision of non-research industry services. With a specific focus on clinical translation of stem cell therapies, how can Australia best ensure that researchers are supported and career paths protected?

5. Safety versus efficacy of therapies: There are an increasing number of clinicians willing to charge patients for unproven treatments using purported ‘stem cells’. Justifications given for such activities include:

• the need for clinical exposure of novel and innovative therapies so that safety and efficacy can be tested
• the high expense and long time-frame of conventional clinical trials
• the unmet needs of patients, many of whom are desperate for treatment of their (often incurable) conditions.

The current relatively unregulated environment means that there are few incentives for those working outside the proven framework of clinical trials to prove safety, disclose protocols, engage with peer-review or ethics committees and to report findings (including those that are adverse). This has led in some cases to a lack of transparency and even false advertising. How can Australia create a regulatory environment that ensures patient safety and enables the scientific community to learn from any clinical administration of stem cells whilst remaining sensitive to a patient’s right to new and innovative therapies?

References and reading material

ISSCR Guidelines for the clinical translation of stem cells. 2008. http://www.isscr.org/home/publications/ClinTransGuide (update mid-2015; will be circulated)

Hey SP and Kimellman J. (2013) ‘Ethics, error and initial trials of efficacy’. Science Translational Medicine. 5(184):1-3 http://stm.sciencemag.org/content/5/184/184fs16.full

Main H, Munsie M and O’Connor MD. (2014) ‘Managing the potential and pitfalls during clinical translation of emerging stem cell therapies’. Clinical and Translational Medicine 3:10. http://www.clintransmed.com/content/3/1/10

Tabar V and Studer L. (2014) ‘Pluripotent stem cells in regenerative medicine: challenges and recent progress’. Nature Reviews Genetics, 15(2):82-92. http://www.nature.com/nrg/journal/v15/n2/full/nrg3563.html