The Cure the Future Service Awards where announced recently at the end of year party for both the clinical and research teams of Professor John Rasko AO.
The teams from the Centenary Institute and Department of Cell and Molecular Therapies, enjoyed a well deserved afternoon of relaxation and reflection after a big year of successful grant applications and commencement of ground breaking clinical trials.
The Awards are given to one staff member in each on the teams who have demonstrated excellence throughout the year. The winners of the awards for 2018 were:
Dr Carl Power for excellence in pursuing ethical questions and raising public awareness in the field of Gene and Stem Cell Therapy, in particular for support with the preparations for the ABC Boyer lectures.
Dr Zlatibor Velickovik for excellence in pursuing and developing philanthropic and government sponsored research grants, resulting in several successful grants supporting clinical trials.
Both recipients are pictured below with Cure the Future representatives.
Cure the Future Service Award Presentation – Dianne Langmack OAM(Chair), Professor John Rasko AO, Dr Carl Power (recipient), Jennifer Andrews(Vice Chair), Dr Zlatibor Velickovik (recipient)
Congratulations to Professor John Rasko and his team, who have recently been recognised for their excellent in research and clinical work with funding from the Li Ka Shing Foundation, for a world-first immunotherapy trial for patients with mesothelioma and pancreatic cancer.
The Li Ka Shing Foundation has funded the newly established Li Ka Shing Cell and Gene Therapy Initiative at the University of Sydney to the tune of $4.5 million over 4 years.
The funding will enable clinical trials on both patients with mesothelioma and pancreatic cancer. The trials will involve the reprogramming of CAR T cells, healthy immune cells, which will be reproduced and re injected into to patients to fight and destroy cancer cells. The impact of this technology is profound on participants in the clinical trials.
About CAR T Research
Early clinical data from leading research centres around the World highlights the promise of Chimeric Antigen Receptor (CAR) T cells for the targeted treatment of cancers such as pancreatic cancer, muscle cancer, ovarian cancer, some breast cancers and asbestos-related lung cancer.
A collaboration of international researchers agree it is the best possible promise of a cure we have seen – so they have got together to do clinical trials of this treatment. Australia’s team, led by Professor John Rasko, Australia’s leading Cell & Molecular Therapies specialist, will leverage the existing cell manufacturing infrastructure and expertise at the Cell & Molecular Therapies department at Royal Prince Alfred Hospital.
International collaborators will work together to develop these CAR-T cells – and initiate Phase I clinical trials to treat each of these cancer conditions.
In Australia, the initial focus will be on the treatment of advanced pancreatic cancer. This treatment option will be a first for Australian patients – and cellular immunotherapy of the type proposed here may revolutionise outcomes for patients and their families living with many types of cancer.
Our “CAR-T mesothelin” cancer immunotherapy is potentially capable of targeting pancreatic, ovarian, synovial and mesothelioma (a subset of lung cancer caused by exposure to asbestos) and well as other lung cancer & breast cancer subsets.
Cure The Future’s mission is to support and enhance the research and clinical work of Professor John Rasko and his team. We are proud support the promotion this initiative.
This year marks the 60th anniversary of the ABC’s Boyer Lectures. Delivered by Professor John Rasko, the 2018 Life Engineered lectures explore ethical and other issues around gene therapy and related technologies, and their potential to cure disease, prolong life and change the course of human evolution.
The first lecture will be broadcast on RN’s Big Ideas at 8pm tonight. In light of this, we’ve asked Merlin Crossley to explain what gene therapy actually is and how we got to where we are with it.
Over the last few centuries, infectious diseases have been understood and tackled, through advances in sanitation, anti-microbial medications and vaccination. One day we may also be able to tackle genetic diseases – lifelong conditions arising from mutations that we inherit from our ancestors or that occur during our development.
We’re over the foothills but we still have mountains to climb in treating genetic diseases.
Step 1 – understanding genetic disease
The key step to tackling infectious diseases was to truly define the nature of the microorganisms that caused them. Similarly, with genetic diseases the first step was to understand and define the nature of a gene.
Our red blood cells carry oxygen with the help of the protein haemoglobin. shutterstock.com
Around the same time in 1949, US chemist Linus Pauling demonstrated that the disease sickle cell anaemia was caused by a chemical change in haemoglobin. He called this the first “molecular disease”. With the advent of DNA sequencing in the 1970s, the actual mutation in the globin gene was identified.Scientists, including Watson, Crick and Franklin, determined the structure of DNA in 1953. Gradually it became clear that a gene was a stretch of DNA that encoded a functional product, such as the oxygen-carrying protein haemoglobin.
Rapidly after this, the genetic lesions responsible for other inherited diseases – such as haemophilia, cystic fibrosis and muscular dystrophy – were identified. From this moment on, the idea of replacing defective genes or correcting them captured people’s imaginations, and this is the basis of what we now call “gene therapy”.
Step 2 – replacing defective genes
Patients suffering from genetic diseases either have a defective gene or may altogether lack a key gene. In the early stages of gene therapy there was no way of correcting genes, so researchers focused on supplementing the body with a replacement gene.
In the 1980s, recombinant DNA technology (where chosen DNA molecules are transferred between individual organisms) was developed by harnessing the miniature machinery bacteria and viruses use to move DNA around. This allowed researchers to isolate individual human genes and encapsulate them in harmless viruses to deliver them into human cells.
In somatic gene therapy, the therapeutic genes can be put inside a virus and transported into the body. shutterstock.com
It was possible to get the genes into certain blood cells and other accessible tissues. This process was termed somatic gene therapy (from “soma” meaning, “the body”) and was distinct from germline gene therapy where eggs or sperm, or early embryos, would be modified and whole people and their offspring changed forever.
Human germline gene therapy is widely outlawed and there is no evidence it has ever been seriously attempted.
But somatic gene therapy has been attempted and, in some cases, has been successful. Viruses really can be made harmless and filled with human DNA, which they deliver into the patient’s cells.
A handful of people have now been successfully treated in this way for haemoglobin deficiencies, haemophilia, and for immune disorders, such as so-called “bubble boy” disease (where victims are particularly vulnerable to infectious diseases).
Step 3 – improving replacement gene therapy
Attempts at these forms of gene replacement therapy began in the 1990s but early results were disappointing. It proved difficult to get the genes into enough human cells, and when the genes did get in they were often turned off after a few weeks.
More worryingly, it was not possible to determine where in the chromosome the replacement gene would land. Often it integrated harmlessly in an unimportant part of the genome, but sometimes it landed near to, and activated, growth control genes called “oncogenes” that drive cellular proliferation and cancer.
Some of the first children treated for “bubble boy” disease developed leukemias. These leukemias were treatable, but the complications, together with immune reactions, such as led to the death of Jesse Gelsinger in an early gene therapy trial in 2000, led to caution.
Over the years, researchers have developed better viruses, systematically improved the gene delivery protocols and found control switches that aren’t turned off by our body’s anti-viral response. In recent gene therapy trials for haemophilia, haemoglobin disorders, and also for specific inherited forms of blindness, many of the patients treated have benefited.
Step 4 – gene correction
The advent of new techniques, most notably CRISPR-mediated gene editing, has led to the idea of correcting a mutant gene rather than adding a replacement. CRISPR is a system that bacteria use to identify and cut invading viral DNA. It has now been used by researchers to direct DNA modification machines to chosen human genes.
We can now develop miniature chemical tools to convert harmful mutations back into normal sequences. News that this technology was being used on human embryos in China created a storm of controversy but so far those experiments have only involved embryos that were known to be non-viable and the research has been purely experimental.
Elsewhere researchers aren’t exploring modifications of whole embryos. Instead the somatic gene therapy approach is being followed, for example, to see if genes can be corrected in a high proportion of blood stem cells and whether these cells can then be transplanted back into the patient to cure their disease.
Step 5 – The future
We will soon see an increasing number of patients helped by both gene replacement therapy and by CRISPR-mediated gene correction. But the work is likely to be focused on a few specific diseases rather than there being a broad advance across all genetic diseases.
An increasing number of patients will soon be helped by CRISPR-mediated gene correction. shutterstock.com
The diseases treated first will share some key characteristics: the genetic defects would be well-understood; they must affect a tissue we can get at easily (blood will be easier than brains and bones); the conditions would be serious and have no other effective treatments.
And they must be so costly in terms of human suffering and economic burdens that a complex and expensive treatment such as gene therapy becomes a viable option.
This means common blood and immune disorders are likely to feature in the first generation trials. Cancer is also a genetic disease, but one typically caused by mutations that accumulate in our cells over time rather than by inherited mutations, and somatic gene therapy, in the form of immunotherapy, involving enhancing the capacity of our immune systems to fight cancer may also become common.
A Nobel Prize was just awarded for anti-cancer immunotherapy, and it is likely that the genetic modification of the immune system will increasingly be used to treat cancers.
The age of gene therapy is arriving but it will be gradual, not sudden. But incrementally, more people will benefit from these treatments.
In the long term, as we all become aware of mutations we carry in our own genomes that may affect our offspring, there may be pressure to correct more and more genetic lesions. This will remain too risky and expensive for many years so gene therapy will likely remain a niche and specialist treatment for the foreseeable future.
Merlin Crossley, Deputy Vice-Chancellor Academic and Professor of Molecular Biology, UNSW
Our own Prof. John Rasko AO has delivered the ABC’s prestigious Boyer Lectures for 2018 – in which he explains something about the research we are doing into Cell and Gene Therapies.
He examines the social and moral impact of these revolutionary genetic and cell-based technologies on our lives and the power of gene therapy to cure disease, prolong life and change the course of human evolution.
This link will take you watch the lecture on ABC iView. Please come back soon!
“We stand at the start of a revolution that may alter the very fabric of our being and the essence of what it means to be human, Reproductive technologies and gene and cell-based technologies offer the possibility of prolonging life and curing disease – even controlling our evolution as a species.”
Across this series of four outstanding lectures, Professor Rasko explores issues such as:
How advances in biomedicine, such as prenatal testing and gene therapy, have revived the debate around eugenics – a concept poisoned by its association with the Nazis. How gene therapies will not only enable us to cure inheritable diseases but to re-engineer ourselves at the most basic level. Why stem cell research has had such a scandal-prone history, promising the dawn of regenerative medicine but delivering few therapeutic wonders. And whether having the power to re-engineer life gives us the right to do so — a question Mary Shelley posed 200 years ago in her novel Frankenstein.
Prof. John Rasko AO and Mercia Bush with her painting ‘Dreams do come true’
– inspired by the cells of a human retina, in celebration of a cell and gene therapy which can cure an inherited form of blindness.
Nearly 100 guests gathered at the new Luke’s Kitchen in Waterloo on Thursday 16th August for some delicious canapes by Luke and his ‘Test Kitchen’, paired with some fabulous wines by top Margaret River winery and long term CTF supporters Pierro.
MC Tim Gilbert welcomed existing supporters, new faces, Board members – and some of the scientists & researchers from the Centenary Institute – whose work Cure The Future sponsor.
Our ever popular Chairperson Diane Langmack presented a Cure The Future/Rotary Fellowship to Dr. Gerard Chu for his research studies into Cancer.
We also raised nearly $25,000 at the auction thanks to our generous sponsors, with prizes including 3 rooms at the new Sheraton Four Points Opening night, weekends at The Byron & Cloud 9 and a painting of retina cells by Mercia Bush called ‘Dreams Do Come True’.
Prof. John Rasko AO spoke abut the great success achieved in Cell and Gene Therapy over the last year – describing it as “A pivotal moment in the history of human medicine”.
He also announced that he has been invited to give the prestigious
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