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Stem Cell Industry Must Tread a Fine Line

By Richard Harvey, Martin Pera and Megan Munsie

The emerging stem cell industry needs to be able to fast-track therapies into clinical trials without clearing the way for clinics to offer unproven therapies to vulnerable patients.

Internationally, stem cell science has developed incredible momentum with the promise of a revolution in medical therapies. This has been spurred on in 2007 with the discovery by Japanese scientist Shinya Yamanaka (who won the Nobel Prize in 2012) that the blood or skin cells of any person can be converted easily to a powerful form of stem cells called induced pluripotent stem cells (iPS). In the laboratory, iPS cells can be expanded and differentiated into any cell type in the body, offering possibilities for using them to treat a range of diseases. We can now correct genetic defects in patient-derived iPS cells, which will lead to “personalised” therapies.

Australia has a strong legacy of excellence in stem cell research, including fundamental discoveries that have led to the routine use of bone marrow transplantation for blood disorders and cancers, and developments that made in vitro fertilisation safe for hundreds of thousands of couples worldwide each year. We were among the first to derive human embryonic stem cells, and our discoveries on neural, skin, breast, blood and kidney stem cells have kept us at the forefront of the field.

It’s against this background that the Australian Academy of Science last year convened a think tank for over 60 of Australia’s brightest early and mid-career researchers to imagine the future of stem cell science and therapies over the next decades, and to take stock of our capability in this emerging sector. The Think Tank’s report, released in March (, identified a number of vulnerabilities for the sector as Australia strives to keep up with international competition and to secure health and economic benefits from the stem cell revolution.

Science funding in Australia has been very flat and there is a current capacity crisis. Although stem cell science has been well supported, Australia is now well behind the rest of the world in moving basic science discoveries into clinical trials. There is a worry that we will slip further behind if funding for stem cell research and translation is not restructured.

New therapies take time to emerge, leading to tension between public expectations for access to new experimental therapies and the ability of medical science to deliver on their safety and efficacy. The normal route is through clinical trials with randomised patients receiving a control treatment. These are time-consuming and expensive, and come with crippling administrative burdens.

However, some clinics are offering unproven therapies to vulnerable patients for profit, with no obligation to meet normal standards for the manufacture and delivery of therapeutics or to report findings that contribute to scientific knowledge. Their business models offer a single therapy for a range of disease states, from autism to cancer, and use the internet, social media and unverifiable patient anecdotes to recruit new patients. This pseudoscience is a growing problem in Australia under permissive legislation that thankfully is under review.

Japan has taken the bold step of streamlining the clinical trials registration process by relaxing requirements for high level evidence of safety and allowing the market to determine clinical efficacy. This model is driven by economic imperatives and is a worrisome departure from the evidence-based medicine to which we must aspire.

It would be unthinkable if Australia missed out on the benefits of the stem cell revolution and allowed stem cell tourism to flourish. We must maintain our excellent international profile in fundamental stem cell science and critical human capacity. Immediate action is needed to inject optimism into the Australian science culture to stop our best minds from going overseas or leaving science altogether.

Universities, medical research institutes and hospitals must champion flagship centres of excellence that amalgamate interdisciplinary discovery science with translational research and clinical trials capacity, and have the capacity to train a new generation of researchers who work seamlessly across these spaces. We must develop new funding models that engage industry as well as government funding bodies.

New ways to fund clinical trials are also needed so that patients have improved access in Australia and don’t seek out bogus clinics. Government must also legislate to establish acceptable boundaries of ethical practice for the provision and commercialisation of stem cell therapies, and must arm its regulators with the teeth to effectively oversee this.

The report of the Theo Murphy Think Tank is timely. There is a lot of work ahead to maintain our legacy in stem cell science and convince government and the public that the promise of stem cell science is a value proposition.

We look forward to what the emerging National Innovation and Science Agenda will deliver in the way of much-needed relief and a long-term vision for this sector.

Richard Harvey, Martin Pera and Megan Munsie are Chief Investigator, Program Leader and Associate Investigator, respectively, for Stem Cells Australia.