View of a Smarter Australia in 2030 - veski

To succeed in 2030 Australia will need to overcome the barriers and issues threatening the future of its industries, scientific discoveries, global reputation and its connected communities, according to a symposium to mark the tenth anniversary of Not for Profit, veski.

Founding veski board member, Australian IVF pioneer and now President of the California Institute for Regenerative Medicine, Alan Trounson told the symposium that there is currently a disconnect between science discoveries and community utilization of their potential.

veski – which was funded with a science, knowledge and innovation endowment by the Victorian Government in 2004 – was set up to identify globally competitive individuals and leading researchers and bring them to Victoria for the benefit of the Australian economy.

Here’s an extract from Alan Trounson’s presentation:

Australian Science and Medicine is undoubtedly first rate – we have great medical research institutions, plenty of young scientists and a great record in the international science and medical community.

Nobel Laureates feature regularly from Brian Schmidt in physics (2011) to a raft of Laureates in physiology/medicine including Elizabeth Blackburn (2009) for chromosome protection by telomeres and the enzyme telomerase that extends the multiplication of cells; Barry Marshall and J Robin Warren (2005) for discovery of the role of Helicobacter pylori in gastric ulcer disease; Peter Doherty (1996) for cell mediated immune defense; Bernard Katz (1970) for humoral transmitters in nerve terminals; John Eccles (1963) for ionic mechanisms of nerve cell membranes; Macfarlane Burnet (1960) for acquired immune tolerance; Howard Florey (1945) for penicillin.

Many other great medical discoveries have arisen recently from Australian labs – including Ian Fraser’s HPV vaccine for preventing cervical cancer; Don Metcalf’s colony stimulating factors that have impacted medicine broadly; Carl Wood’s work on human IVF; among many others.

One might have cause to be optimistic about the next 20 years! Maybe even expect Australia to have a Minister of Science driving high tech developments and a strong biotechnology industry employing scientists and medical graduates maturing in universities and research centers.

This might rival the quantum benefits of the mining boom and particular sectors of the food industry that appear to rise and fall pretty regularly.

However, it doesn’t take much searching to demonstrate the disconnect between science discoveries and community utilization of their potential discoveries.

What is hampering our smartness in converting high quality science discoveries to community benefit?

Is Australian science research sustainable without the ability to expand beyond the publically funded institutional base of universities, medical centers and CSIRO?

Where is our Biotech Industry? Who spans the Valley of Death?

There is a distinct scarcity of industry connecting with the Australian medical research community and a lack of venture investment in the biosciences.

However, a company such as Mesoblast can arise in the Australian environment to be a world leader in stem cell medicine (>$2 billion mc) in a relatively short time and with a strong Australian investor base.

In California, I developed academic research teams partnered with companies, or utilizing commercial consulting together with independent and in house guidance, to successfully navigate the translation pathway to clinical trials.

Beginning in 2010 from a pure discovery base our Disease Teams are now entering registered clinical trials – expect 10 by the end of 2014.

They are now connected to industry and are expected to fare extremely well. Areas such as the destruction of malignant cancer stem cells, cure of genetic diseases such as β thalassemia, sickle cell disease, HIV AIDS, blindness (macular degeneration and retinitis pigmentosa), juvenile diabetes (type I), treatment for heart muscle damage from myocardial infarct (heart attack), spinal cord injury and deadly motor neuron disease.

A Big Idea for 2030 – Immunology and Stem Cells – Defeating Cancer

In 2030 we can expect that research has delivered us a way to defeat cancer by engineering the T cell receptor (TCR) to recognize cancer specific antigens – this chimeric antigen receptor (CAR) technology can be introduced into patient’s T cells (our scavenging killer thymic instructed cells that aggressively target foreign infection or abnormal or non-self cells) or through their blood or pluripotent stem cells.

The TCR is expressed only in T cells and is activated to destroy malignant cancer cells expressing any abnormal antigen of the patient’s tumor.

We can select these cells for expansion or genetically engineer them to be extremely potent killers of our cancers. Now we have an internal surveillance system capable of removing any cancer stem cell that develops – this can last a lifetime if introduced through the stem cells in the body or be made patient specific in large numbers by converting the patient’s own T cells to iPS cells – expanding them and redirecting them into activated T cells for tumor killing purposes and using these as cell therapy.

The beauty of this system is that it can be modified and tailored to work in concert with an active immune system – not against it. We should also be able to reawaken the immune system for recognizing and tolerating autoimmunity – the thymus can be regenerated by cell therapy or with small molecules (e.g. targeting the thymic epithelium transcription factor Forkhead box N1 – FoxN1)(Blackburn 2014). Wow, very Melbourne eh! Foreign or allogeneic cells and tissues will be tolerated and,  potentially universal donor tissues will be available that are immune compatible for use by patients.

This requires we apply the power of tools discovered in immunology together with developments in stem cell biology and ensure we translate that for patient benefit.

What do we need?

It is time that academies of science, funding agencies and government looked at such models to refocus the limited public resources available in a more strategic way.

We need to really encourage the very smart academics to become interested in applying their discoveries for the community benefit – universities have to change how they recognize and reward scientists who enter this pathway – not penalize them.

It isn’t about number of publications or grants from NH&MRC – these are not the measure for making the innovative contributions needed. They probably handicap the conversion to a more effective system that the community will recognize and endorse.

The current federal grant system is broken in the USA (see Alberts etal PNAS 2014), and would not be any better here. Money is trickling down an unsustainable pipeline that fails to encourage developments of use to the community.

The system must be fixed despite the inertia that exists even among the majority of scientists. We will have no viable research environment soon if we continue to look away from the problems that are evident and are turning away the smart young scientists.

We must quickly revise the direction we are taking.

We need a viable and robust biotechnology industry with investors who are confident in the returns on investment. Australia is an attractive place for basic research but it needs to become competent in translation of these discoveries. Preclinical demonstration of safety and sound proof of concept for the candidate product with the accompanying regulatory requirements for pharmokinetic data, processing and manufacture of the therapeutic candidate, development and validation of a potency assay etc. are areas of biotech experience that should be available for the raft of opportunities that exist here for translation.

Typically patent lawyers drafting patents should have experience in prosecution of IP. I think we need to link with US biotech industry to move this capacity into places in Australia where sufficient clinical and scientific power exists. The experience may be illuminating and rewarding.

Get with it and Get Global

The Australian research community must be globally linked to the other big engines of progress. This hasn't been a feature of research in Australia. Indeed it is rare for university researchers in the same city to collaborate.

We cannot be competitive by working in isolation. Niche discoveries aside, we are less represented in the major journals with time, less recognized and less respected.

The Asian tiger research communities (China, Japan, Korea, Singapore) are moving strongly to challenge North American dominance and Europe is awake to the merits of collaborating – using vehicles such as EU granting.

I encouraged Australians to collaborate with California in stem cells – they have been extremely competitive – were awarded more collaborative grants than any other country besides Germany.

These high quality scientists are currently starving for funding here in Australia. We need to re-evaluate the merit of becoming global partners in medical research and sharing the benefits with our collaborators that will flow from this work.

Communicate with the Whole Community – Inform them of the Extraordinary Opportunities of Wealth and Health Creation

Time is of the essence here. The so-called Australian “cleaver country” will not be recognized as that in the next decade or two without major change.

We must change the way we fund research, we must consider how to reward scientists properly. We must expand the opportunities for expressing their smart ideas.

We need to power up industry linkages to, and partnerships with, academia and medicine. We need to globalize our collaborations. We need to make science and medicine an industry to compete with resource-based commodities for wealth and prosperity of the whole Australian community.

Most of all we need to begin a dialogue with the Australian community about the benefits of medical research for our healthy ageing.

The example of returns over 23 years of $1400 for every $1 invested in the original grants for sequencing the human genome – and the likely large economic benefit from investing in medical research in stem cells, pathogenic vaccines, cancer drugs etc. needs to resonate in the halls of government – not a just be a whisper from scientists returning to Australia with the aid of VESKI, or in my case, after managing $3 billion of Californian bond funds to drive a revolution in regenerative medicine.

It might take 20 years to reward so well this kind of investment here but we need the support and changes to the way we arrange this support, for Australians to be living really smart and hand healthy in 2030.

About the author: In the mid-1990’s Dr. Trounson led a team to discover human embryonic stem cells and directed their differentiation into nerve and other tissue cells (published 2000). Following his extensive career in this area he was a Director of the Monash Centre for Early Human Development from 1985-2002 and founding Deputy Director/Director of the Institute for Reproductive Biology from 1990-2002.

In 2003 he founded the first Australian Biotechnology Centre of Excellence – Australian Stem Cell Centre and was the founding CEO. Alan then went on to establish the Monash Immunology and Stem Cell Laboratories in 2004.

In January 2008 he was appointed President of the California Institute for Regenerative Medicine, responsible for the management of the $3 billion fund for stem cell research in California (CIRM).