Promising finding for possible future myelofibrosis treatment

Adelaide researchers have discovered a possible new method to treat myelofibrosis (MF), that could have the potential to greatly extend lifespan without the side effects caused by current treatments.

While MF symptoms can often be controlled with the drug Ruxolitinib, side effects may be problematic and currently, the only cure for MF is an allogeneic stem cell transplant.

Dr Daniel Thomas, leader of the Myeloid Metabolism Lab at the South Australian Health and Medical Research Institute, (SAHMRI) and Associate Professor of Medicine at the University of Adelaide, led the study in collaboration with Professor Angel Lopez at SA Pathology.

Dr Thomas says it was a stroke of luck that led to the extraordinary find.
“We were actually trying to make a tool to study myelofibrosis. We didn’t realise the antibody we made would have therapeutic properties,” Dr Thomas said.
“Our drug blocked the growth of cancer cells in a very aggressive live model of the disease, significantly increasing survival rate without noticeable negative side effects,” Dr Thomas said.

Co-lead author and biochemist Dr Denis Tvorogov generated antibodies using a peptide fragment called ‘neoepitope’ that’s only present within the cancer and not on any normal tissues. What he didn’t expect was for the antibody to kill cancer cells when he tested it on patient samples, working with an early career scientist Dr Chloe Thompson-Peach.
“What is really exciting is that many other cancers have similar peptide fragments that we could also target by harnessing the immune system,” Dr Tvorogov said.
“These fragments are created by the insertion or deletion mutations within the cancer. We’ve found they not only drive cancer growth but also vulnerable for targeting without side effects.”

The new antibody is currently being prepared for early phase clinical trials set to run in South Australia later this year, supported by local biotech company, AusHealth.  Pre-clinical models have shown the drug is effective at shrinking tumours and Dr Thomas is confident the antibodies will prove to be safe and effective in humans.
“We estimate there are at least 12,000 Australians living with cancer or having had cancer that express a recurrent neoepitope similar to what is found in myelofibrosis, that could be curable with
an immunotherapeutic approach,” Dr Thomas said.

“This discovery brings us a fresh perspective. We need to build cell therapies and antibody therapies against these fragments as fast as possible.”

The full media release is available:  SAHMRI media release – Lucky find could hold key to beating rare blood cancer —

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World rare disease day

Today is RARE DISEASE DAY.

MPN expert, clinician scientist Professor Andrew Perkins, is interviewed about his team’s work on MPNs HERE.

WATCH MPN expert Professor Andrew Perkins and the MPNAA’s Ken Young speaking about MPNs.

 

 

 

 

 

 

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Momelotinib: an emerging treatment for myelofibrosis patients with anaemia

A phase 3 clinical trial has been underway for myelofibrosis patients who are symptomatic, suffer from anaemia and have been previously treated with a Jak inhibitor. The trial was using the drug Momelotinib.

Findings just released on 25 January this year are very encouraging.  Momelotinib achieved statistically significant benefit on symptoms, anaemia and spleen size.
The trial, undertaken by drug company Sierra Oncology, is a global, randomized, double-blind clinical trial evaluating momelotinib in 195 myelofibrosis patients.

Access to the full report of the trial is available HEREThe full data set will be presented at an upcoming medical meeting which we will also publish once available.

Ruben Mesa, MPN specialist  and co-principal investigator of the study said that “As a clinician, I am thrilled to see data that confirm the potential of momelotinib as a treatment option for myelofibrosis patients who are anaemic or at risk of becoming anaemic,”  “Anaemia of myelofibrosis is strongly correlated with reduced quality of life and a decrease in overall survival. Half of all myelofibrosis patients present with anaemia at diagnosis and virtually all become anaemic over time. With currently approved therapies being myelosuppressive, it’s wonderful to know that we may soon have such an effective treatment option for these patients.”

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Second versus first wave of COVID in MPN patients

European haematologists have been undertaking ongoing studies of MPN patients who have contracted COVID.  This study covers the second COVID wave of 304 MPN COVID cases from 39 different hematology centres.  Their findings are outlined in just released (January 2022) correspondence in the International Journal ‘Leukemia’.  Importantly, they found a relatively higher risk from COVID for patients over 70, more thromboses in ET patients and a need for permanent surveillance of MPN patients who have survived the acute phase of a COVID infection.

‘Patients of the second wave presented, compared to those of the first, with a less severe disease, including a lower degree of inflammation, leading to hospitalization in a smaller percentage of cases. Overall, the mortality rate was significantly lower, likely due to early COVID-19 diagnosis, facilitated by the greater availability of swabs than in the first wave, more efficient management of infected patients, better prepared health systems and preferential protection of older and higher-risk MPN vulnerable subjects.’

‘However, patients over 70 years still presented with an excess of mortality, particularly when associated with comorbidities and an MF phenotype. Unfortunately, no data are available so far in our series to support a role of vaccinations. The high thrombosis rate in patients with ET was confirmed, suggesting that in this MPN phenotype regimens of antithrombotic prophylaxis in addition to heparin should be explored. Also in the second wave, but to a lesser extent than in the first, the health consequences of COVID-19 protracted far beyond acute infection, suggesting careful and permanent surveillance of patients with MPN who have survived the acute phase of SARS-CoV-2 virus infection.’

The full article has been made available HERE.

 

 

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Expert interview with Professor Andrew Perkins

The latest Leukaemia Foundation’s MPN newsletter features a detailed and wide-ranging interview with MPN expert, Professor Andrew Perkins.
As well as his ongoing care for MPN patients as a clinical haematologist, Professor Perkins and team are focussed on improving diagnostics and treatment for MPN patients, especially those with myelofibrosis.

His interview is available HERE.

 

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Reported incidence and survival of myeloproliferative neoplasms in Australia

An update to the Australian Cancer Atlas was released earlier this year and for the very first time it included MPNs. This was such an encouraging development for MPN research.

Since that time, researchers and epidemiologists have been exploring MPN incidence further and a paper has just been released in the journal ‘Pathology’.

MPN AA thanks the authors for their thoughtful analysis and is proud to have been able to support this research through donations from the Australian MPN community.

Spatial disparities in the reported incidence and survival of myeloproliferative neoplasms in Australia

Due to the restrictions around access to medical journals, free access to the full article will be available until 3 November 2021. 

 

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Protection against COVID for people with blood cancers

Norman Swan’s Health Report has featured an item about people with blood malignancies and their response to Covid-19 vaccines.
The item relates to new research published in the Lancet Haematology.  This research found that people with blood malignancies may not produce as many protective antibodies to the vaccines as others.
This is because in blood cancers, the immune system itself is affected.

Norman interviewed haematologist, Associate Professor Kate Burbury, about this research.  A brief extract is below as well as the link to the article.

Kate Burbury:  Most of us [haematologists and oncologists] would be advocating some protection is better than none. And the only concern we would have is that they [the blood cancer patient] might not mount an adequate antibody response and therefore advocating that they continue to pursue the usual protective mechanisms such as keeping themselves safe and avoiding people that might have infective symptoms.
The interview transcript, podcast and the research in the Lancet Haematology are linked HERE.

Encouraging responses for MPN patients to Covid vaccines

Subsequent to the above article, the Leukaemia and Lymphoma society in the US has outlined the response of  different blood cancer patients to Covid vaccines and while a small sample, the results are very encouraging for MPN patients, showing some 97% of patients had detectable Covid-19 antibodies.
The graphic is below and the full article is linked HERE.

Antibody response to Covid-19 Vaccine by Blood Cancer Diagnosis Chart

 

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MPNs now included in Australian Cancer Atlas

In what we believe could be a world first, myeloproliferative neoplasm (MPN) data is now included in the latest Australian Cancer Atlas. This Atlas provides national data on how cancer diagnosis and excess death rates vary by geographical area across Australia.  This latest update incorporates details about the geographical patterns in MPN diagnoses and survival across Australia from 2007-2016.

The inclusion of MPNs is the initiative of Cancer Council Queensland, in collaboration with QUT.  The MPNAA donated $10,000 towards this initiative and wishes to thank in particular Professor Peter Baade and his team, Dr Jess Cameron as well as WA’s Professor Lin Fritschi for their dedication to enabling MPNs to be part of this important resource.

The Australian Cancer Atlas is an interactive online resource helping researchers, members of the community, medical professionals and policy makers understand how the cancer burden varies by small geographical areas across Australia. It is fascinating to see how MPNs diagnoses vary across Australia, as well as MPN survival rates.  The Atlas enables users to easily visualise those differences and offers critical insight into how the patterns of cancer and outcomes in Australia vary depending on where people live, which can be used to drive research priorities and policies going forward.

Professor Baade said that “The Australian Cancer Atlas has already proven itself to be an invaluable resource and benefit to many Australians, gaining industry, scientific and community acclaim. By updating the data contained in the Atlas ensures it remains relevant, and provides a great foundation for the next phase of development.”

MPN patient Jolanda Visser from the MPN Alliance Australia said that she was pleased to hear about MPNs being included in the Atlas.  MPN Alliance Australia’s $10,000 donation was raised through a major fundraising dinner organised by Jolanda Visser held in 2018. “Having looked at the Atlas and seeing that we can retrieve MPN stats is making me feel very good,” said Ms Visser.  “It is exciting that patients like me will be able to look at this information about MPN in their local area.” The MPNAA is keen to optimise the accuracy of MPN data, which is reliant on reporting of MPNs within each state and territory. The MPNAA will be liaising with stakeholders with the aim of maximising the integrity of this data to further inform research and health outcomes.

Co-lead on the Australian Cancer Atlas, the Distinguished Professor Kerrie Mengersen from the Queensland University of Technology said the latest editions would greatly benefit the Queensland community.  “The Australian Cancer Atlas is a shining example of what can be achieved by combining cross-institutional expertise in statistics, e-research and cancer,” said Professor Mengersen.  “This collaboration is inspiring since it not only benefits our community but also leads to new knowledge and new research.”

For more information on the Australian Cancer Atlas, please visit, atlas.cancer.org.au.
To access MPN data, MPNs are listed in the Atlas as Classic MPN.

Image from the fundraiser Charity dinner organised by MPNAA’s Jolanda Visser.

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When did I acquire my MPN?

Dr Jyoti Nangalia and a team of researchers from the Wellcome Sanger institute and Cambridge University in the UK will be presenting some fascinating findings at this years virtual American Society of Hematology (ASH) meeting.

Extensive genetic mapping by the team finds that MPNs “originate from driver mutation acquisition very early in life, even before birth, with life-long clonal expansion and evolution, establishing a new paradigm for blood cancer development. Early detection of mutant-JAK2 together with determination of clonal expansion rates could provide opportunities for early interventions aimed at minimising thrombotic risk and targeting the mutant clone in at risk individuals.”

Information about this presentation is available HERE courtesy of MPN Hub.

Dr Ann Mullally’s lab in the US has recently announced similarly fascinating findings. This research which further explores the cellular origins and development of mutations giving rise to JAK2 and MPNs, provides further evidence that mutations can develop decades prior to clinical presentation of MPNs, and can arise from a single hematopoietic stem cell.

More information on this important research is available HERE

 

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MPN Alliance Australia donates $15,000 towards groundbreaking MPN research at University of Western Australia

MPN AA is very proud to announce that with the help of our supporters, we have donated $15,000 for MPN research in Australia to Professor Wendy Erber and her colleagues, Dr Kathryn Fuller, Dr Henry Hui and Dr Belinda Guo at the University of Western Australia. The team are undertaking research on a new technique to see if they can detect which MPN patients may be at risk of progressing to leukaemia or marrow fibrosis. It is hoped this may ultimately provide an alternative to the current method of utilising bone marrow biopsies for the purpose of detecting the first signs of disease escape or progression. Although only a minority of MPN patients are affected, it is not currently known who this might affect or when this will occur. By the time symptoms appear, it is generally difficult to cure. Hence a predictor of progression may assist in providing treatment earlier and possibly providing cures.

The method the team will use is their own invention, and the one that won a Eureka Award (affectionately known as the “Oscars of Science”!) in 2018. As you will see from the Youtube video it is a flow cytometry method that enables the study thousands of cells to see if the cell-of-interest has the chromosome change that may predict progression.

We wish Prof Erber, and Drs Kathy Fuller, Henry Hui and Belinda Guo every success with their research and look forward to sharing their progress in the future.

 

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