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International Cell & Gene Therapy Collaboration

Jul 2018 | News

Dr Xuiyan Wang is a well-respected scientist from the prestigious Cell Therapy and Cell Engineering Facility, at the Memorial Sloan Kettering Cancer Center, New York.

Her expertise is in the area of gene and cell therapies, specifically using a patient’s own immune system to fight diseases, including cancer.

(L-R) Dr Zlatibor Velickovic, Cell & Molecular Therapies PM | Dr Xuiyan Wang, Sloan Kettering Memorial Centre | Prof John Rasko AO
Working Together
Besides being a delightful colleague, her sharing of knowledge about clinical scale cell therapy manufacturing was invaluable. Xuiyan toured our facility, the Centenary Institute and Sydney University – while en route to Melbourne for the International Society for Stem Cell Research Conference.

Xuiyan contributed to our understanding with constructive information and insider knowledge – knowledge which would not be accessible from just reading published Journal articles. Her visit was not only productive, but has been instrumental for establishing a rapport and network between internationally renowned Laboratories, both of whom are currently involved together in the International Car-T Mesothelin research project.

Xuiyan also attended the Thalassaemia Society of NSW 40th year celebration, where our Director Professor John Rasko gave an address.

Our laboratories look forward to a continued collaboration into the future – to establish a service where the latest therapies are available to Australian patients.

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Computer biologist receives research accolade

Jun 2018 | News, Uncategorized

Computer biologist receives research accolade

Dr Ulf Schmitz, a bioinformatician within the Gene & Stem Cell Therapy Program at the Centenary Institute, received a prestigious accolade for his recent published work on novel gene regulation published in the journal Genome Biology.

Ulf’s paper received a recommendation from the F1000 Faculty which identifies and rates the best published research articles in the world.

Dr Schmitz, who uses computer code and high performance computer clusters to make sense of big data, discovered that intron retention, which is a form of alternative splicing of messenger RNA, is an evolutionarily conserved mechanism of gene regulation.

Ulf and colleagues studied white blood cells from 5 different vertebrate species (human, mouse, dog, chicken and zebrafish). They concluded that intron retention acts to increase gene regulatory complexity in vertebrates. Ulf is now extending this study to examining leukaemia samples to determine if intron retention regulates the expression of cancer-specific genes.

World-first breakthrough for Thalassaemia

Apr 2018 | News

In a world first, Royal Prince Alfred Hospital clinicians are among an international team to have found a gene therapy cure for Thalassaemia, a condition where sufferers must undergo regular blood transfusions and treatments for iron-overload, for life.

Thalassaemia is the most common genetic disorder in the world, affecting more than 280 million people. Almost half a million people worldwide and perhaps 1000 Australians suffer from the severe form of this disease. Those affected produce fewer red blood cells and less haemoglobin, which means less oxygen is delivered around the body.

 

In a breakthrough clinical trial, published this week in The New England Journal of Medicine, 22 participants with severe beta thalassaemia across three sites (United States, Thailand and Australia) had their own blood stem cells extracted.

 

Scientists in the United States then inserted a healthy copy of the affected gene into the stem cells that were maintained outside the body. Back home the Australian patients were given chemotherapy prior to receiving their own genetically modified cells by intravenous infusion.

Following the trial, 15 of the participants no longer needed blood transfusions. The remaining 7 still received transfusions but only needed one quarter of the volume compared to their previous requirements. The follow-up since the time of infusion is two years.

 

Trial lead and head of Cell and Molecular Therapies at RPA, Professor John Rasko AO, said the results build on many recent breakthroughs in gene therapy to treat immune deficiencies, blindness, haemophilia and leukaemia

“People with thalassaemia often have a poor quality of life. Not only do they need to undergo monthly lifelong blood transfusions, but those transfusions result in excess iron overload, which in turn require regular medications that can have significant side effects,” Professor Rasko said.

For participant Jack Saenprsert, 38, from Rockdale, the trial has been life-changing.
A chef and father of four, he underwent monthly blood transfusions at RPA for nine years. Since receiving his own gene-modified cells about two and a half years ago, he has not needed a blood transfusion.

“I had to take the day off work every month, plus come in the day before for cross-matching of the blood. It was very difficult with work. Some bosses don’t understand, or don’t want to understand. I tried going without the transfusions but I’d get so tired and yellow.

Now, I feel fresh and healthy.”

 

 Stacey Wong, 28, from Blacktown, also participated in the trial. She still needs transfusions but requires less units of blood less frequently.

“I think the doctors were a little disappointed that I still need transfusions, but I’m not. The number of times I visit the hospital is incidental because my quality of life has significantly improved. I feel so much better than I did before and I’d do it all again in a heartbeat.”

 

She is now planning her wedding in October and – for the first time ever – can leave Australia for a month of holiday.

 

Download NSW Health Media Release
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Car-T Therapy trials to get $2m Cancer Council funding

Apr 2018 | News

Cure The Future are proud to announce that Prof John Rasko and his team at the Centenary Institute at Royal Prince Alfred Hospital in Sydney are to receive a major grant from the Cancer Council to help fund the new CAR-T therapy trials, which are also a focus of our current fundraising at Cure The Future.

This comes as part of over $10 million of funding into a number of ground-breaking cancer research projects announced by Cancer Council NSW, which includes a seperate grant to Assoc. Prof. Jeff Holst, who is also a member of Prof. Rasko’s team.

Winning grants revealed at 2018 Research Awards

28th March 2018

Cancer Council NSW has awarded over $10.6 million to 17 outstanding cancer research projects. The grants help fund future breakthroughs in cancer research – the awarded research teams are leading the charge towards a cancer free future by investigating new ways to treat the disease.

The 2018 grants were announced and awarded at Cancer Council NSW’s annual Research Awards, this year held in the evening of 27 March at Westpac’s Barangaroo Towers in Sydney.

“We are excited to announce a round of extraordinary projects – all 17 recipients are extraordinary scientists who do essential and highly innovative work,” said Dr Jane Hobson, Research Grants Manager at Cancer Council NSW.

The majority of the 17 projects announced last night – awarded to the Centenary Institute, Children’s Cancer Institute, Garvan Institute of Medical Research, UNSW Sydney, University of Sydney and UTS – are three-year Project Grants. One Infrastructure Grant is going to Children’s Medical Research Institute.

Two projects received a Pathways to a cancer free future grant. The recipients of those grants are Professor John Rasko at Royal Prince Alfred Hospital (grant for $2m) and Professor Jennifer Martin at the University of Newcastle (grant for $1.96m).

Professor Rasko and team will be trialling a potential new treatment for pancreatic cancer known as CAR T-cell immunotherapy. The treatment involves taking a patient’s white blood cells, growing them in the laboratory, genetically modifying specific cells to attack only cancer cells, and then returning them to the patient. The grant provides much-needed hope for patients who receive the devastating diagnosis of pancreatic cancer (which has one of the lowest survival rates).

Professor Martin and her team will test and validate how new technologies could be used to monitor the concentration of chemotherapy in a patient’s blood in fast-tracked timeframes to provide optimal treatment dosing. It is hoped that this research will lead to direct and significant benefits in patient dosing, across wide population groups and in rural and remote areas, to improve quality of life, reduce side effects and increase chance of survival.

“The broad range of projects that we fund – across many types of cancers and stages of the cancer journey – shows Cancer Council NSW’s commitment to work across every area of every cancer,” Dr Hobson said.

“Projects like Professor Rasko’s and Professor Martin’s wouldn’t be possible without our supporters – as an organisation that is over 95% community funded, these grants have been made possible by the community,” Dr Hobson concluded.

 

Learn more about CAR-T Therapy

Pancreatic Cancer

– did you know?

  • Pancreatic Cancer has the highest mortality rate of all cancers
  • The survival rate has not improved in 40 years
  • Since 1996, 1 million families have lost someone to Pancreatic Cancer
  • 91% of patients die
  • By 2020 Pancreatic Cancer will be the 2nd leading cause of cancer-related deaths
  • It receives only 2% of federal cancer funding in the US
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About Cancer Council NSW’s Research Awards
Cancer Council NSW is proud to be one of the largest non-government funders of cancer research in Australia. We believe cancer survivors, carers and members of the general public should be at the heart of cancer research, and the community plays a vital role in supporting our work – not only by raising money for research, but also in selecting which projects we fund. That’s why Cancer Council NSW ensures that we award funding on the basis of both scientific merit and value to the community we represent.Cancer Council NSW’s annual Cancer Research Awards were held at 6pm on Tuesday, 27 March at Westpac’s Barangaroo Tower in Sydney. At the Awards, we recognise the researchers who were successful in the latest round of funding grant applications with an award to signify the start of their research.

The event is also an important opportunity to celebrate the research achievements of Cancer Council NSW over the last year, and to thank staff and donors for their ongoing support of cancer research.

Cancer Council NSW 2018 Pathways to a Cancer Free Future Grant recipients:

  • Professor John Rasko, Royal Price Alfred Hospital: Can a revolutionary new immunotherapy fight pancreatic cancer?
  • Professor Jennifer Martin, The University of Newcastle: Improving patient outcomes through personalised chemotherapy dosing

Cancer Council NSW 2018 Infrastructure Grant recipient:

  • Professor Roger Reddel, Children’s Medical Research Institute: Building research infrastructure to enable clinicians to choose the right treatment for the right patient

Cancer Council NSW 2018 Project Grant recipients:

  • Professor Peter Hersey, Centenary Institute: Exploring the role of T cells when immunotherapy for melanoma fails.
  • A/Professor Jeffery Holst, Centenary Institute: A targeted approach to triple-negative breast cancer
  • Dr Umaimainthan Palendira, Centenary Institute: Improving immunotherapy for melanoma
  • Dr Jessamy Tiffen, Centenary Institute: Improving drug treatment for melanoma
  • A/Professor Tao Liu, Children’s Cancer Institute: Investigating a new therapy for a problematic childhood cancer
  • Dr David Gallego Ortega, Garvan Institute of Medical Research: A ‘molecular microscope’ approach to develop immune therapy for breast cancer
  • Dr Ruth Pidsley, Garvan Institute of Medical Research: Improving prostate cancer treatment to minimise side effects
  • Professor Susan Ramus, UNSW Sydney: Moving towards personalised treatments for ovarian cancer
  • Professor Barbara Fazekas de St Groth, University of Sydney: Predicting responses to cancer immunotherapy
  • Professor Jacob George, University of Sydney: A new approach to fighting drug resistance in liver cancer
  • Professor David Gottlieb, University of Sydney: The Susan and John Freeman Research Grant: A new approach to boosting resistance to fungal infection in cancer patients
  • Professor Elizabeth Sullivan, UTS: Cancer and Outcomes in Pregnancy – A NSW Evaluation (COPE)

Recipients of a Cancer Australia PdCCRS project grant, co-funded by Cancer Council NSW:

  • Dr Marina Pajic, Garvan Institute of Medical Research: Improving pancreatic cancer treatment through a ‘personalised medicine’ approach
  • A/Professor Ilona Juraskova, University of Sydney: Enhancing carer involvement in a patient’s cancer care

 

See the full article at Cancer Council
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Haemophilia Breakthrough

Dec 2017 | News

World-First Breakthrough treatment for Haemophilia

Cure The Future is proud to have supported researchers at Royal Prince Alfred Hospital as part of an international team who have developed a gene therapy for the life-threatening blood disorder, haemophilia.

The breakthrough, led by our friend and colleague Professor John Rasko, is a major milestone in the quest to cure the bleeding disorder and to the treatment of 4000 other genetic diseases.

The finding, published in the New England Journal of Medicine this month, is the culmination of more than 20 years’ work and opens the door to using gene therapy to treat more than 4000 other genetic disorders, including blindness , cystic fibrosis and thalassemia .

“We are very excited about the results, as those people in our trial have previously had to live with the risks of spontaneous bleeding every day . To prevent potentially life – threatening bleeds they have typically had to inject themselves with clotting factors every few days ,” Professor Rasko said.

 “This trial has targeted haemophilia B, which affects about 500 males in Australia – with about 100 experiencing a severe form of the condition, but our next focus is targeting haemophilia A, which affects more than 2300 people .”
The director of NSW Office of Health and Medical Research, Dr Tony Penna, said the discovery demonstrated the importance of supporting medical research in NSW.

“This extraordinary international collaboration could potentially save the lives of thousands of people around the world and was only possible because of the dedication and determination of our brilliant researchers,” Dr Penna said.

 “NSW has state-of-the-art gene and cell therapy facilities with outstanding clinician researchers like Professor Rasko who are leading this cutting-edge research and its translation into clinical care. This is a world first that all Australians can be proud of and highlights the need for both public and private investment in medical research.”

The NSW Government is investing a record $1.25 billion over four years in medical research across the state.

Haemophilia B is an inherited disorder where blood does not clot properly due to missing or defective clotting factor nine (IX) . Sufferers experience a wide range of bleeding episodes, usually into the joints or muscles. Episodes can often occur spontaneously, without an obvious cause, or as a result of trauma or injury. Over time, bleeding can cause severe arthritis, chronic pain and disability.

The research involved 10 adults who were injected with a gene therapy designed to produce the clotting protein Factor Nine (IX).

 

 Trial participant Mark Lee, 38, had undergone infusions up to three times a week since birth and lost two brothers to complications from haemophilia B when they were children.
 Since receiving the experimental gene therapy, he has not had any bleeds.

“This is life – changing for me” Mr Lee says, “I spent my childhood wrapped up in cotton wool, unable to play football or do any of the things my mates could. I would always remind myself that there were people worse off than me, but it was still disappointing.”

“I have two daughters who are carriers for haemophilia, but now I know that if they have affected children, it will be one injection and they can live normal lives. This goes beyond our little fa mily currently. It will have a positive impact on all generations to come.

“And my mum now knows she won’t see her only surviving son die from haemophilia .”

Professor Rasko has spent the past two decades fine – tuning the gene therapy and says it is the beginning of the end of this lifelong bleeding disorder.

“We now know how to beat the immune response to achieve what may be a permanent cure . ”

The vice president of Haemophilia Foundation Australia, Daniel Credazzi, who has a son with the condition, welcomed the breakthrough, saying: “ The real potential of a cure with safe and effective gene therapy is very exciting for people living with this chronic condition, and for their families. ”

Haemophilia Breakthrough

by Fran Kelly | ABC RN with Prof. John Rasko AO November 2017

Download Sydney Health Press Release