Bio21 Director's Message - Funding Freedom to Fail...& Discover - 14 December 2020

This year the Nobel Prize for Medicine and Physiology went to Harvey J Walter, Michael Houghton and Charles M Rice for their "discovery of the Hepatitis C virus".

The Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer Doudna “for the development of a method for genome editing”.

The lectures took place and were broadcast last week on the 7th December 2020 via the website and Facebook, where you can still find them.

It is interesting to note that when they started out Emmanuelle Charpentier and Jennifer Doudna did not know they were going to discover a powerful new molecular tool that would go on to be considered ‘the greatest benefit to humankind.’

A microbiologist, Emmanuelle was studying a defence mechanism in bacteria. She was conducting fundamental discovery research on her organism of choice, the gram-negative bacterium Streptococcus pyrogenes.

Although it has long been possible to ‘cut’ and ‘paste’ genes in bacteria and other organisms, the process was slow and difficult. Emmanuelle explains in her lecture:

“This started in the 70s with different types of enzymes and technologies that would allow you to recombine DNA, to sequence DNA, amplify DNA, target genes. The zinc-finger and TALEN nucleases that were discovered over the last 20 years, allowing the same as what CRISPR Cas9 does, except that CRISPR Cas9 brings a level of programmability and a level of simplicity and versatility that is quite unique. So, we are all very happy because we can now study certain organisms that were difficult to study prior to CRISPR Cas9.”

Famously it was Louis Pasteur who said that: ‘Chance favours the prepared mind.’

Often when we dig into the stories of scientific discovery, we find that it was a chance observation; something strange, that could not at first be easily explained, that led to something truly new being discovered.

In the case of Emmanuelle Charpentier and Jennifer Doudna, it was stumbling upon the ancient mechanism by which bacteria defend themselves against viruses.

For the Hepatitis C discovery, it was the observation that there was an agent in blood causing hepatitis, that was not being picked up by the existing testing methods, that triggered the hunt for the virus.

To have the opportunity to make discoveries that win Nobel Prizes or to make an impact on the lives of people, scientists need long term, consistent funding, that provides the opportunity to take a risk, explore, fail and stumble upon something new and exciting.

At a time when medical research has received enormous exposure and recognition by the Australian public, we are facing a huge exodus of young researchers, who fail to receive funding to continue their research careers. This is a tragedy for individuals and for science.

According the ASMR’s Fact Sheet, the full time health and medical research workforce declined by 20% between 2012 and 2017.

Read Bio21’s Dezerae Cox’s insightful piece in Research Professional News that shows that gains in gender diversity are slow and that emerging researchers continue to struggle:

Investigator Grants fund outstanding researchers from a limited pool of resources, but the reality is that success rates so low – just over 13 per cent in 2019 and 2020 – are unsustainable for long-term retention of Australian-trained scientists,” writes Dezerae.

Last week the Australian Society of Medical Research initiated a Twitter campaign to highlight the lack of federal funding for medical research.

I became involved through my own Twitter account and was even challenged to name high impact medical discoveries from basic research funding at Bio21.

At Bio21, we do have numerous high impact scientific discoveries that we can be proud of and I sent out the following tweets:

Director, Bio21 Institute @Bio21Director·Dec 9
The @Bio21Institute is one of Australia's largest life science institutes with >800 people distributed over 4 buildings, including @CSL's global research hub. We have made a number of high impact medical discoveries that would not have been possible without basic research funding

Director, Bio21 Institute @Bio21Director·Dec 9 Replying to @Bio21Director @DwanPrice and 6 others
Paul Donnelly developed SARTATE @Bio21Institute to find & destroy neuroendocrine tumours #menigioma & #neuroblastoma in kids. Clarity Pharmaceuticals has been granted Investigational New Drug status for SARTATE by the @US_FDA & a Phase 1-2a trial has been approved.

Dr Dwan Price Glowing star Retweeted Director, Bio21 Institute @Bio21Director·Dec 9 Replying to @Bio21Director @DwanPrice and 7 others
A potential treatment for Motor Neurone Disease #MND and #Parkinsons Disease, that was developed in the @Bio21Institute, has progressed to a Phase 2/3 Trial in MND patients led by Collaborative Medicinal Development #AdvanceAustraliaCare #auspol2020

Dr Dwan Price Glowing star @DwanPrice·Dec 9
Fibrosis can develop during chronic kidney and heart disease, pulmonary fibrosis and even arthritis. #medicalresearch from @Bio21Institute developed critical drugs that targeted these disease complications. We need more of this, that's why we need: #AdvanceAustraliaCare @TheASMR1 Quote Tweet

Director, Bio21 Institute @Bio21Director· Dec 9
Replying to @DwanPrice @anthonyjhannan and 2 others

Professor Spencer Williams @sjwill99 co-founded Fibrotech which developed a family of anti-fibrotic drugs at @Bio21Institute @ChemistryUoM @scimelb for treating #diabetes complications #AdvanceAustraliaCare #auspol2020

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For instance, the discovery of Cu-ATSM, synthesised by Paul Donnelly’s lab, as a potential treatment for Motor Neurone Disease and Parkinson’s Disease was made at Bio21. At the time, Kevin Barnham was the fortunate recipient of a NHMRC program grant.

He says:

At this time, Tony White and I were part of a NHMRC program grant team, and this flexible funding allowed us to take a few risks and try things that we would not otherwise have done.”

So we took a punt, and one of the things we tried was Cu(-ATSM) in models of both MND and Parkinson’s disease and it worked brilliantly in both models.”

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As we celebrate and acknowledge the 2020 Nobel Prize winners for Chemistry and Medicine and Physiology and celebrate their success, as scientists we know, in order to succeed, we must be permitted to fail. Public funding needs to be more accepting of risk; to fund scientific ‘failure’, exploration and therefore also discovery. Failing this, we continue to risk losing our brilliant young emerging researchers, that could one day bring us the greatest benefits to humankind.

And on a final note:

Thank you to the scientific team led by Paul Young, Trent Munro and Keith Chappell at the University of Queensland and CSL Behring for their work on the UQ/@CSLBehring COVID-19 vaccine. Some of the early CSL work was done at Bio21.

The UQ clamp technology, which was developed based on basic discovery research, was unique in the world and the UQ team created a powerful vaccine with a strong antibody response to the spike protein.  The vaccine was performing well in trials, but the unexpected antibody response to the clamp protein, that gives a false HIV positive test result, has led to the vaccine work being terminated.

It is a heart-breaking decision for all the teams involved in this project, who have worked so hard through this incredible year. It is an example of what we all know – the ups and downs of scientific research. As fellow scientists we really feel for them and know the sacrifices they have made.

They are heroes and we are proud of their work.

Michael Parker,

Director, Bio21 Institute