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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
Download Full Article

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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Cell-based Gene Therapy is finally here!

Oct 2017 | Archives

The first-ever approval of a Cell-based Gene Therapy by the Federal Drug Administration in the US has finally come – after many years of research and development into our long-held belief that DNA holds the key to a possible cure for disease.

Our own Professor John Rasko is Head of Cell and Molecular Therapies at RPA Hospital in Sydney and President-Elect of the International Society for Cellular Therapy – he is delighted that the day has arrived.

“This is a day we’ve been waiting for” he says “… the thought that we could ‘turbo-charge’ the immune system has been around for decades – so to have a working and approved treatment based on these ideas is fantastic.”

With this new ‘Car-T’ therapy, blood from a patient is removed and the T-cells genetically modified to help them identify the diseased Cancer cells and destroy them. These Car-T cells are reintroduced to the patient’s blood stream through a normal blood infusion, where they massively expand and go to work – killing the tumour cells… in just the same way that your body identifies and deals with bacteria and viruses.

“Just one single modified T-cell is capable of killing thousands, or tens of thousands, of Cancer cells” says Professor Rasko.

 

Cure The Future are actively involved raising funds for Car-T research (see below)

Hear the full ABC Radio interview about Car-T therapy with Prof John Rasko.

Exciting News as 1st Cell & Gene Therapy approved by FDA

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

Car-T Project

Professor Rasko & RPA Hospital are part of a global research initiative to expand the potential for Car-T therapy, in collaboration with leading researchers at other centres in USA, Canada & Sweden.

Cure The Future is the leading Cell and Gene Therapy Charity in Australia & New Zealand – Join us now & help support this fantastic project.

Let’s find a cure for cancer and other inherited diseases. 

Learn More about the Car-T Research Project
Donate to Car-T Research NOW