QIMR Berghofer Medical Research Institute – Blood cancer treatment could be transformed by discovery

QIMR Berghofer Medical Research Institute researchers have discovered a potential new treatment for Acute Myeloid Leukaemia and other cancers.

Watch their exciting news HERE

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Thrombosis in MPNs – a retrospective German study

A German study has just been released which looks back at arterial and venous thromboembolic complications in 832 patients with Myeloproliferative Neoplasms (MPNs).

This study aims to add some additional data to what is currently quite limited information about the most common sites, incidences, and risk factors of MPN-associated arterial and venous thrombotic embolisms (blood clots).

A relatively high incidence of MPN-associated blood clots was observed in this retrospective study.
The most frequent arterial sites were strokes and transient ischemic attacks.
The most frequent venous sites were deep vein thrombosis (with or without pulmonary embolism) and splanchnic vein thrombosis.

Patients with polycythemia vera had a significantly higher risk of a blood clot than the other MPN subtypes whereas patients with a CALR mutation had a significantly lower risk compared with JAK2-mutated MPN patients.

The full study is available HERE.

If you’d like to know more about blood clots, how to prevent them and also how to recognise them, further information is available on our MPN AA website  HERE.


(Pictorial courtesy of ‘Stop the Clot’)

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MPNs and inflammation

 

Image: Visual abstract shows overlap between MPNs and chronic inflammatory disease.

An interesting perspective on MPNs from respected US haematologist and researcher, Dr Angela Fleischman and her colleague was recently published.

The article highlights the overlap between MPNs as cancer and/or chronic inflammatory disease.

‘….. In addition to elevated blood cell counts, MPN typically presents with increased inflammatory signaling and inflammation symptoms. Therefore, while being a clonally derived neoplasm, MPN has much in common with chronic non-cancerous inflammatory conditions, such as rheumatoid arthritis, lupus, and many more. MPN and chronic inflammatory disease share similar chronicity, symptoms, dependency on the immune system, environmental triggers, and treatments.’

The article highlights the similarities between MPNs and chronic inflammatory disease, arguing that ‘while MPN is classified as a cancer, its behaviour is more aligned to that of a chronic inflammatory disease.’
‘We propose that MPN should inhabit a fluid/spectrum between auto-inflammatory disease and cancer.’

The article is available in full HERE.

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Questionnaire: patient awareness of blood clot risks

The International Society on Thrombosis and Haemostasis (ISTH) is conducting a survey to find out more about patient awareness of cancer-associated thrombosis (blood clots) and how to help prevent and/or identify blood clots if they occur. The ISTH is keen to obtain feedback from patients from Australia and New Zealand as there has been a low response rate to date.

As MPN patients need to be vigilant about blood clots, you may wish to add your views.  Responses are anonymous and the questionnaire takes less than five minutes to complete.

The ISTH advises that ‘Patients with cancer have a high risk of blood clot formation in their veins or arteries due to the cancer itself and anti-cancer treatments. Patient awareness of these complications is key for prompt recognition of symptoms and signs, risk factors, timely referral to hospital for diagnosis and treatment, as well as for informed discussion with doctors about use of blood thinners for prevention.’

‘This questionnaire is intended for patients with cancer and aims at assessing their knowledge and satisfaction about the information received by health-care providers on the potential risks of cancer-associated blood clots and use of blood thinners.’

The survey can be assessed via the link below.

https://redcap.isth.org/surveys/?s=APAPWWEWRA

 

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Iron link offers new treatment hope for incurable blood cancer

Read more about the newly published findings on an iron link which offers a new treatment hope for polycythaemia vera.

“A landmark discovery linking iron regulation to a rare blood cancer has led to clinical trials of a potential new treatment for patients with the incurable disease.

The study focused on polycythemia vera (PV), a blood disorder causing excessive red blood cells, and found that restricting iron access to the bone marrow could reduce the production of red blood cells in this disease.

The research, led by WEHI (the Walter and Eliza Hall Institute of Medical Research) in collaboration with the University of Melbourne, the Peter MacCallum Cancer Centre, the University of Cambridge and Silence Therapeutics (UK), has led to new clinical trials of a drug that has the potential to control iron regulation in patients with PV.”

A link to the WEHI news release is HERE.

The Blood journal article referred to in the above news article is HERE.

 

 

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New treatment hope for PV

 

Dr Cavan Bennett (R) is pictured in the lab with Nathalie Cook OAM  (a PV patient) from the MPN AA who is part of the WEHI patient consumer program

A ground-breaking new treatment for polycythaemia vera (PV) has been developed here in Melbourne by researchers Dr Cavan Bennett and Professor Sant-Rayn Pasricha from the Walter and Eliza Hall Institute (WEHI). This treatment appears to avoid the need for venesections. Phase 1 clinical trials begin shortly. There will be 7 trial sites in Australia and more detailed information will be provided shortly.

A report on ABC Radio’s Health Report of 3 April this year outlines the potential benefits of this treatment for patients. The report is available to listen to HERE.

More detailed information about the research is available below.

” SLN124: ironing out a new treatment for Polycythaemia Vera (PV)

The average adult has approximately 3-4 grams of iron stored in their body. Large changes in the amount of stored iron can have drastic consequences, with too little iron (iron deficiency) causing chronic fatigue and brain fog. Two-thirds of the iron in the body is contained within red blood cells. As such, venesection (blood withdrawal – the first-line treatment of Polycythaemia Vera) induces iron deficiency by removing iron-containing red blood cells from the body. Consequently, chronic fatigue and brain fog are not uncommon in venesected patients.

A small amount of iron travels around the body in the liquid compartment of the blood (the plasma). Plasma iron supplies the body’s cells with their iron needs, most notably for producing new red blood cells. Iron in the plasma comes from two main sources: dietary iron and recycling iron contained within dying red blood cells in the spleen. The amount of iron entering the plasma is controlled by the levels of a hormone called hepcidin. Hepcidin prevents iron exiting the cells of the intestine and spleen and entering the plasma. Therefore, increases in hepcidin result in less plasma iron and, as such, less iron is available for producing new red blood cells. Hepcidin is produced in the liver and the biological pathway that leads to hepcidin production has an internal brake, which limits hepcidin production.

Researchers Dr Cavan Bennett and Professor Sant-Rayn Pasricha from the WEHI (Melbourne, Australia) therapeutically silenced hepcidin’s internal brake, TMPRSS6, in a new clinically relevant mouse model of Polycythaemia Vera. In doing so, they discovered that hepcidin levels increased and plasma iron levels went down. Furthermore, they showed that the increase in hepcidin acted as a sort of medical venesection and reduced the haematocrit of the Polycythaemia Vera mice by nearly 20%. However, they have revealed that unlike traditional venesection, therapeutically increasing hepcidin levels did not physically remove iron from the body and therefore did not lower the total body iron levels. Because of this, it is thought that compounds that silence hepcidin’s TMPRSS6 could replace traditional venesection. This would offer an alternative to venesection and importantly offer hope to the population of Polycythaemia Vera patients intolerant to venesection who currently have no alternative therapeutic options.

Dr Bennett and Professor Pasricha used a short interfering (si) RNA developed by Silence Therapeutics Plc to silence hepcidin’s TMPRSS6. SLN124 is a siRNA in development by Silence Therapeutics as a treatment for haematological conditions. SLN124 has a potential to modulate hepcidin by ‘silencing’ TMPRSS6 to increase hepcidin levels to restrict iron in the bone marrow thereby reducing red blood cell production. SLN124 is expected to replace venesection and reduce thrombotic risk while reallocating iron away from the bone marrow to improve PV symptoms including fatigue. SLN124 has demonstrated safety in healthy volunteers and is currently being assessed in patients with thalassaemia. A new study for SLN124 is starting in Australia (and other locations) to investigate the safety and efficacy of SLN124 in patients with Polycythaemia Vera. This is a two-part study:  the first part will look at different doses and the second part will compare a single dose with placebo. All patients participating in the phase 1 are eligible to participate in the phase 2. ”

The photo above was provided with kind permission of WEHI, and is Copyright:© WEHI (Walter and Eliza Hall Institute of Medical Research) 1G Royal Parade Parkville 3052

 

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MPN Horizons conference 2022

Reflections on the 2022 MPN Horizons meeting, Netanya, Israel
from Ken Young, MPN AA team member

The MPN Horizons meeting was held this year in Israel and online.

It was a great success with many excellent presentations.
Ken recommends in particular watch the sessions from Medical Session 3 which feature Dr Gabby Hobbs, the MPN AA’s Nathalie Cook OAM and Dr Ruben Mesa.

Here is the link to the videos from MPN Horizons Hybrid Conference 2022

Ken Young watches on whilst Nathalie Cook presents at the November 2022 MPN Horizons Conference in Netanya, Israel.

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Leukaemia Foundation – survey of blood cancer in Australia 2022

The Leukaemia Foundation launched the National Strategic Action Plan for Blood Cancer in 2020. Its overarching principle is that ‘every Australian with a blood cancer should have equitable access to the best information, treatment and care wherever they live and whatever their background.’

As part of this plan, the Leukaemia Foundation is working towards a goal of zero lives lost to blood cancer by 2035.

To help get there, they need your knowledge and around 20 minutes of your time, to complete this survey.

All responses are anonymous.

Click HERE to begin the survey and be a part of change for the better to help people living with a blood cancer.

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MPN AA Fellowship: Engineering and Pre-Clinical Validation of CALR+ directed CAR-T cells

Dr Chloe Thompson-Peach (R) accepting the cheque from the MPN AA’s Jolanda Visser

MPN AA is thrilled to announce the grant of $30,000 for an MPN Alliance Australia Fellowship titled Engineering and Pre-Clinical Validation of CALR+ directed CAR-T cells.  The grant has been made to an early career researcher from the South Australian Health and Medical Research Institute (SAHMRI) and the University of Adelaide, Dr Chloe Thompson-Peach. Chloe has previously been part of the team under Dr Daniel Thomas researching MPNs in Adelaide at SAHMRI (see separate story).  The fellowship will last for a year, and we will update you during that time.

The grant has been made possible by a generous donation of funds from a Sydney MPN patient, Sarah Gardner, following her recent fund raiser.
We are extremely grateful to Sarah for making the Fellowship grant possible and to the Leukaemia Foundation for facilitating provision of the grant. For Chloe, it enables her to pursue her research passion which has the potential to improve lives of MPN patients into the future.

Sarah Gardner. Sarah’s fund raising efforts have made this MPN Fellowship possible

Research aims for the project – Dr Chloe Thompson-Peach

Myelofibrosis is an insidious condition of the bone marrow, which disrupts the normal production of blood cells. It is characterised by painful inflammation, low blood counts and fibrous tissue building up in the bone marrow. In some patients, myelofibrosis can also develop into acute leukaemia over time. The lack of effective therapeutic options for these cancers has led us to the development of a new immune treatment for these individuals. We have developed a novel immune treatment, a biological therapy, that is a monoclonal antibody directed against the mutant CALR gene, which the second most common cause of myelofibrosis or essential thrombocythemia. We have shown this to be effective in cell lines and patient cells harbouring pathogenic CALR mutations in the laboratory. Additionally, our treatment has also been shown to have no effect on cell lines and primary cells that do not carry mutations within the CALR gene, suggesting it is truly selective, which means it will not have side effects.

In this fellowship, I propose to make and test a specific “CAR” T cell for these patients, a chimeric antigen receptor T cell, using the knowledge we have gained from our antibody therapy. CAR T cells involves removing the good white cells from a patient’s blood and then inserting the CAR gene into these T cells in the lab. These cells are then reprogrammed to target the mutated CALR protein on the surface of the cancer cells. These cells will initially be tested in mouse models of CALR driven myelofibrosis to determine if the CALR specific CAR T cells are able to seek out and destroy cells carrying the pathogenic CALR mutation. I am hopeful that by combining antibody therapy, interferon therapy and specifically designed CAR T cell we may move towards demonstration of long-term remission or cure in patients with myelofibrosis.

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Leukaemia Foundation’s July MPN newsletter

The Leukaemia Foundation has released its latest newsletter for MPN patients and it’s an excellent read.
There is so much happening in the MPN space in Australia.
Individual links to each story in the July newsletter are provided below.

1.  A tool predicting the potential progression of MPNs is something under development from Professor Wendy Erber, a world leading diagnostic haematologist and MPN specialist, and her team of dedicated researchers.

2.   An antibody for myelofibrosis – “that’s a true discovery”
A new monoclonal antibody discovered by Adelaide researchers could become the world’s first effective treatment for primary myelofibrosis.

3.   Expert interview with Dr Cameron Curley on transplantation and CAR-T therapy.

4.  Patient stories 

–  Living with PV has become “a normal way of life” for Debbie

–  Charlie’s blood cancer went undiagnosed for a decade

5.    Clinical trial comparing allogeneic stem cell transplants vs best available non-transplant therapies for myelofibrosis (MF).
Associate Professor Nada Hamad is the Australian lead on this international trial which will compare best available non-transplant therapies with stem cell transplant for MF patients.

6.    Optimal care pathways being developed for blood cancer patients.

7.   WEBINAR: Exploring real life with blood cancer: The mind and body experience

This is a brilliant webinar, packed full of valuable advice featuring:
–  haematologist Dr Cecily Forsyth
–  psychologist Jane Fletcher
–  physiotherapist Julie Allen

8.   PODCAST: Talking Blood Cancer podcast series from the Leukaemia Foundation has been a great success and is filling a need for people living with blood cancer.

9.   More MPN stories from this and earlier newsletters are available

10.  Need support?
The Leukaemia Foundation encourages you to find out about the different ways they can help
– by visiting their  website or
– by calling 1800 620 420 to speak with a Blood Cancer Support Coordinator.

 

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