Internationally, stem cell science has developed incredible
momentum with the promise of arevolution in medical ther-
apies.This hasbeen spurred on in 2007 with the discovery by
Japanese scientistShinya Yamanaka (who wonthe NobelPrize
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). Inthe laboratory,iPScells 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-derivediPScells,
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 theirst to derivehumanembryonic stem cells,
and our discoveries on neural, skin, breast, blood and kidney stem
cells have kept us at the forefront of the ield.
It’s against this background that the Australian Academy of
Scienc 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 (http://tinyurl.com/hx397ts),
identiied a number of vulnerabilities for the sector as Australia
strives to keep up with international competition and to secure
health and economic beneits from the stem cell revolution.
Science funding in Australia has been very lat 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 eicacy. 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 proit, with no obligation to meet normal
standards for the manufacture and delivery of therapeutics or
to report indings that contribute to scientiic knowledge. Their
business models offer a single therapy for a range of disease
states, from autism to cancer, and usethe internet, social media
and unveriiable patient anecdotes to recruit new patients. This
pseudoscience is a growing problem in Australia under permis-
sive 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
clinicaleicacy.Thismodel 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 bene-
its of the stem cell revolution and allowed stem cell tourism to
lourish. We must maintain our excellent international proile
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 lagship centres of excellence that amalgamate inter-
disciplinary 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 accept-
able boundaries of ethical practice for the provision and commer-
cialisation 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 Innova-
tion 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.MAY 2016|| 39Stem Cell Industry Must Tread a Fine Line
The emerging stem cell industry needs to be able to fast-track therapies into clinical trials
without clearing the way for clinics to offerunproven therapies to vulnerable patients.conSCIENCE Richard Harvey, Martin Pera & Megan MunsieIt would be unthinkable if Australia
missed out on the benefits of the
stem cell revolution and allowed
stem cell tourism to flourish.