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World-first breakthrough for Thalassaemia

Apr 2018 | Archives

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 | Archives

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 | Archives

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

FDA Approves 2nd Car-T Therapy – for Adult Lymphoma

Nov 2017 | Archives

On October 18th, the U.S. Food and Drug Administration approved another cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or who have relapsed after, at least two other kinds of treatment.

 “Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,” said FDA Commissioner Scott Gottlieb, M.D. “This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”

Yescarta (axicabtagene ciloleucel), is a chimeric antigen receptor (CAR) T cell therapy – and is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma (NHL).

Diffuse large B-cell lymphoma (DLBCL) is the most common type of NHL in adults. NHLs are cancers that begin in certain cells of the immune system and can be either fast-growing (aggressive) or slow-growing. Approximately 72,000 new cases of NHL are diagnosed in the U.S. each year, and DLBCL represents approximately one in three newly diagnosed cases. Yescarta is approved for use in adult patients with large B- cell lymphoma after at least two other kinds of treatment failed, including DLBCL, primary mediastinal large B- cell lymphoma, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.

Each dose of Yescarta is a customized treatment created using a patient’s own immune system to help fight the lymphoma. The patient’s T-cells, a type of white blood cell, are collected and genetically modified to include a new gene that targets and kills the lymphoma cells. Once the cells are modified, they are infused back into the patient.

10/11/2017 Press Announcements > FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma

“The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options – those adults with certain types of lymphoma that have not responded to previous treatments,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER).

The safety and efficacy of Yescarta were established in a multicenter clinical trial of more than 100 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after treatment with Yescarta was 51 percent.

Learn More about Car-T Therapy
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Cure The Future helps 1st time gene discovery

Nov 2017 | Archives

First-time discovery of a common pattern in gene regulation could provide clues for new treatments for cancer and other diseases.

Dr Ulf Schmitz and Centenary Institute’s Gene & Stem Cell Therapy Program, led by Professor John Rasko AO, have – for the first time – discovered a common pattern in the process of gene regulation in humans, mice, dogs, chickens and zebrafish.

A better understanding of this common process will help scientists identify abnormal behaviour and help develop direct therapeutic interventions to correct the ‘mistake’ and find possible cures for diseases. The extreme significance of this work has been recognised, with the research published today in the highly renowned scientific journal, the highly ranked, Genome Biology.

The team of scientists used a state-of-the-art experimental system and high performance computing to find that this mechanism of gene regulation evolved over time into a highly calibrated system, facilitating smooth functioning of the animals’ immune systems.

“Comparing patterns of gene regulation between species is important because we have learnt in the past that mechanisms which are evolutionarily conserved are often essential for the survival of a species. We found a mechanism and associated characteristics that are well conserved, which suggests that it is important and gives species a selective-advantage” said Dr Ulf Schmitz. This is a world- first study of this type, “What is special about our study is that we used a single, highly purified cell type with highly preserved characteristics,” Dr Schmitz said.

To come to their ground-breaking conclusions, the team studied the genetic code imprinted in our DNA, which is organised like a train line. The valuable cargo is lined up at stations that are connected by interspersed tracks of variable length which have long been referred to as ‘junk DNA’. Genetic trains load valuable cargo from the stations onto carriages named ‘exons’ and nonsense-cargo from the tracks onto carriages named ‘introns’. Intron carriages are usually removed from the train during their journey to the protein production factory.

Cancer and many other diseases are caused by mistakes in the body’s gene regulation process, which can occur when damaged cells do not properly self-regulate. Understanding the process which determines whether or not genes are successfully controlled, is the key to treating and finding cures for cancers and many other diseases.
Scientists were astonished to find that the systematic phenomenon of intron carriage removal was preserved in at least 400 million years of evolution. This is the estimated time at which a common ancestral organism lived from which all the animals studied in this project descended.

To study this evolution more closely, they retrieved white blood cells from human, mouse, dog, chicken, and zebrafish – all important model organisms which help scientists understand how cells work and how diseases emerge.

Researchers determined that the number of intron carriages in genetic trains and compared their characteristics. They found that preserved intron carriages are abundant in white blood cells of all these species and have very similar features such as their length, composition and location within the genetic train.

The discovery of a clear pattern in gene regulation in the group of vastly different animal species, could bring researchers around the globe, a step closer to understanding the reasons some damaged cells repair themselves and others become diseased. This understanding is highly significant as it could inform future treatments for deadly diseases including various types of cancers

See Article in Genome Biology Journal
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Genetic Testing & Life Insurance

Nov 2017 | Archives

Bioethicists are calling for tighter regulation of the insurance industry when it comes to genetic testing.

Researchers say that some Australians have been unable to take out insurance policies because of results contained in their genetic tests.

They say this is having an effect not only on individuals, but also on the progress of genomic research in Australia.

Learn More

Hear the full ABC Radio interview with Prof John Rasko.

The use of genetic testing for Life Insurance

by Claire Slattery | ABC RN PM with Prof. John Rasko AO + others November 2017

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