Patients with CalR mutation – promising ASH update

The American Society of Hematology’s December 2024 conference included developments in treatments for MPN patients with the CalR mutation. A range of Australian and international immunotherapy research is underway and some treatments are already in clinical trials. It is too early to present results.

A fascinating video about CalR treatment research is linked below.
In the video, Drs Alex Rampotas and Zoë Wong explain that there are already specific immunotherapies being trialled against the CalR mutation, all of which may well be effective.  They specifically mention a ‘B specific T cell engager’ and ‘a blocking antibody against it’.

However their collaboration is about a third type of immunotherapy option, a novel second generation CAR-T cell therapy. They advise that CAR-T is the ‘strongest immunotherapy so potentially  ……. able to overcome some of the immune suppression of myelofibrosis and directly eliminate the malignant stell cells.’

The full video is 8 minutes long, unfortunately with background noise.  However if you have the CalR mutation, watching the video will give you a Christmas present of great promise!

https://www.vjhemonc.com/video/qsoln_mbhbk-development-and-evaluation-of-a-first-in-class-car-t-therapy-against-calreticulin-mutant-neoplasms/

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MPN Horizons meeting 6-8 September 2024 Warsaw – A personal reflection from Sharon MacIntyre

 

Well let’s just say it was an honour to attend the MPN Horizons conference this year.  There was representation from MPN specialists, many MPN advocacy groups, PV, ET & MF patients, pharmaceutical companies and more!  The title “Shifting the treatment paradigms of MPN” was very apt. Since I first attended in 2017, sponsors have grown from 3 to now 11! And patient advocacy groups now include Thailand, Korea, and other nations which previously were not represented.  It is such a growing global family to address MPN needs.

The almost 70 participants in Warsaw, Poland were from an amazingly geographically spread of 26 countries, including Australia, India, Chile, to Europe, America and Asia! All were very vocal in sharing new developments for MPN from research to trials, to advocacy and patient stories. All sharing a key purpose – to make life better for MPN patients.

Before we dived into a massive 3 day program, it was refreshing to attend a breakfast session where Pharma was the lead and seemed genuinely keen to collaborate and bridge the access barrier different countries and patients in need have. The session was called “Navigating the Path to Equal and Equitable Care” to discuss the most important barriers for MPN patients.  I learnt a lot, particularly helpful that there is a special consideration element to access which can be sought with the Pharma company directly for review/access to medications.

This was the third time I have attended Horizons and as a highlight for myself being diagnosed at 18 years of age. I was particularly impressed with the inclusion of a session solely for young MPN patients by Alice Watson from UK. Under the auspices of MPN Voice she has started a group for under 40’s MPN patients to ask questions, address issues such as study/work life balance, starting a family and pregnancy, long term MPN and progression.

The sessions from world leading specialists were very informative.  Dr Claire Harrison spoke on state-of-the-art news for Myelofibrosis – including the new prognostic model; how there are more options available for lower risk MF patients; and how the success of Haplo transpants as an option could be explored. A haematologist from Germany, Dr Susanne Isfort, talked about the 3 common MPNs and the drugs she typically uses to treat them, and about data showing that interferon in young patients may be looked at as an option to stop MF progression.

Jon Mattias from MPN Voice presented a session on a great new application “Health Unlocked” which is in development. This app tracks patient symptoms and data that hopefully could be useful for medical appointments and could be integrated into a wearable device.  This type of app could be rolled out globally – but more needs to happen on 3rd party permissions and how to protect personal data.

Dr David Ross from Adelaide Australia, spoke on the differences in an individual’s height and gender etc which often isn’t taken into consideration in treating patients. For example, spleen size, depth and volume in a female 5’2 and a man 6’2 can be completely different in what is considered large! He felt spleen volume is more important than just length – something I had never heard of before. There were pictures from Dr Wendy Erber’s lab in Western Australia on machine learning for precise fibrosis scoring which was very interesting.

Elena Greschner from Austria talked about fatigue and how around 80% of MPN patients suffer fatigue.  Yet only about 30% of haematologists ask their patients about fatigue. She stressed the fact that Quality of Life is important (as well as the blood cell numbers). Dr Patrick Harrington mentioned patient data in his MPN research that showed a fifth of MPN patients can only work reduced hours or need to stop work early before retirement age.  Emphasis was placed on being aware of increased clotting risk, infection and organ failure.

Another brilliant session I thought was valuable were the regional breakouts. Being an MPN AA advocate from Australia – ideas for more Asia/Pacific collaboration were bounced around and very positive. I’m looking forward to seeing some of these ideas come to fruition. Perhaps “Chai for Cancer” events at workplaces in Australia borrowed from advocacy group ‘Friends of Max’ in India’s success.

One of the advocates talked about their “March for Cancer” event and how citizen participation was important!  Perhaps we can bring back a lantern walk for MPN and other blood cancer advocacy.  The kids love this!

The importance of a healthy diet and exercise were again highlighted.  I for one will be trying to implement when my symptoms are ok, a more Mediterranean Diet of real foods (hopefully more than packaged and takeaway) and some gentle exercise for 30 mins a few times a week.

All in all the MPN Horizons conference was absolutely brilliant! A wonderfully informative and collaborative 3 days in Warsaw. I’m looking forward to seeing these developments turn into new application and drugs (some in clinical trials already) and much more positive patient outcomes for the future.

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Ruxolitinib available for some PV patients? PBAC call for submissions by 29 January 2025

In March 2025, Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) will consider an extension to the existing listing of Ruxolitinib (known as Jakavi®) onto the Pharmaceutical Benefits Scheme (PBS). The extension sought will be for the treatment of adult patients with PV who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).

NOTE: CLOSING DATE FOR PATIENT INPUT – 29 JANUARY 2025

To help the Government in its considerations, PBAC is taking consumer comments until 29 January 2025.
* See below for advice on making a submission.

The MPN AA is supportive of ruxolitinib being available on the PBS for PV patients. It would provide another treatment option when first line therapy fails. Thanks to the MAJIC-PV trial conducted in the UK, Ruxolitinib has now been established as a viable second line treatment option for PV.

For more information please see our News post about the Majic-PV trial.

*Next steps if you’d like to make a submission

Making a submission to the PBAC is a way for PV patients to have a voice by explaining how having access to this treatment could impact on their own quality of life.

If you follow the instructions below you will be able to make your comments via a simple process.

  1. To comment for the  2025 PBAC meeting, start by clicking on this LINK.
  2. On Page 2, enter your name and contact details and select the category that best describes your reason for input, ie:
    – Individual who would like to access the medicine to treat own health condition
    The drug you are commenting on is ‘Ruxolitinib Jakavi®.
    You will see that this is a ‘Resubmission to request a General Schedule Authority Required (STREAMLINED) listing for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).’ This is because the drug company unsuccessfully sought approval in 2019. However as more results are now available from the MAJIC study, the pharmaceutical company is resubmitting its application.
  3. On Page 3, provide your input by answering the questions in the free text boxes OR by attaching a PDF or Word file at the bottom of the page.
  4. On Page 4, please declare any conflicts of interest relevant to the responses provided in Pages 2 or 3.
  5. When you have completed the form, select ‘Submit Response’ on Page 5. You will be sent a confirmation receipt and link to a PDF copy of your response to the email address you provided on Page 2.

If you’d like any support in making your submission, you can contact the Department of Health at HTAconsumerengagement@health.gov.au or watch one of their facebook Q&A recordings HERE.

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‘Majic-PV’ clinical trial comparing ruxolitinib vs BAT for hydroxyurea resistant/intolerant PV

The MAJIC-PV trial  studied ruxolitinib for PV patients against best available therapy (BAT) which in this case was mainly hydroxycarbamide (hydroxyurea).  It concluded that ‘ruxolitinib treatment benefits hydroxyurea intolerant or resistant patients with superior complete [haematological] response, and event free survival as well as molecular response; importantly also demonstrating for the first time, to our knowledge, that molecular response is linked to event free survival, progression free survival, and overall survival.’

Watch Dr Claire Harrison MD, FRCP, FRCPath, Guy’s and St Thomas’s NHS Foundation Trust discuss the results from the MAJIC-PV trial: ruxolitinib vs BAT for hydroxyurea resistant/intolerant PV.

Jean-Jacques Kiladjian further explains in a recent talk about ‘State of the art in PV’, that the MAJIC study also showed that ‘Patients having a complete haematological response during the first year of treatment had a significantly better event free survival than those who did not.  So this is for the first time, an evidence that normalising the blood count, results in benefits in terms of less complications during the follow up. And the other point is …… that this event free survival was also better in patients who achieved a molecular response (reduction in allele burden) compared to those who did not. …….
So to summarise, …….we have to optimise [PV] management by recognising resistance in tolerance to first line therapy, and switch quickly to second line therapy because….achieving complete haematological response within 12 months results in better long term outcomes.’

Image: screenshot of the MAJIC trial outcomes showing: superiority of ruxolitinib in event free survival for patients intolerant or resistant to HU; and also the benefits to PV patients of achieving a complete haematological response within 12 months

While not specifically addressed in the MAJIC study, pegylated interferon is also an existing alternative first line treatment to hydroxycarbamide for PV patients. However about 20% of patients are unable to tolerate interferon and so a need remains for effective additional treatments. Of interest is that the MITHRIDATE trial now underway in the UK and France is comparing ruxolitinib with hydroxyurea and with pegylated interferon.

 

 

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Message for patients taking anagrelide

The MPN AA has become aware of a warning placed on the MPN treatment Anagrelide.

While haematologists would already know about the warning, and be managing patients’ treatment accordingly, the MPN AA wants to also inform any MPN patients taking anagrelide.  The warning states:

SPECIAL WARNING AND PRECAUTIONS FOR USE

Do not stop using anagrelide suddenly without checking first with your doctor.  Rather you may need to slowly decrease your dose before stopping it completely. Stopping suddenly will cause the platelet level in your blood to increase quickly. It should be noted that there is risk of thromboembolic events during this rebound phase which may lead to potentially fatal thrombotic complications, such as cerebral infarction. Platelet counts should be monitored closely when anagrelide is ceased.
We will update the other languages as promptly as possible, hopefully within a couple of weeks.

 

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Inflammation and MPNs

This presentation by MPN specialist Dr Hans Hasselbalch from Denmark is of great interest to MPN patients.
Whilst the topic is inflammation, and the importance of reducing inflammation, Dr Hasselbalch’s talk is comprehensive.

He explains the latest MPN research findings, including the extraordinary discovery that 11.3% of stroke victims have the Jak2 mutation. He also reminds us of the huge numbers of undiagnosed MPN patients around the world and the possibility of targeting and eliminating the Jak2 mutation when it is in its early CHIP stage.
He explains about the risk of inflammatory bowel disease in MPN patients, the benefit of early intervention with interferon and even a potential role for statins for MPN patients, and so very much more.

His talk is available HERE.

 

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MPN patient conference videos available

The videos and presentations from the inspiring MPN Horizons 2024 conference in Warsaw, Poland, are now available.
The conference shared the latest advancements in MPN research, treatment, and advocacy from a range of MPN experts.
Two Australian researchers David Ross from South Australia and Belinda Guo from Western Australia were amongst the presenters.  We have linked to a few of the presentations most immediately relevant to MPN patients:

ET

PV
  • State of the Art in PV
    Jean-Jacques Kiladijan (Virtual) – Video
  • Pipeline for PV
    Susanne Isfort  –VideoPDF
MF
And if you’re interested in research and future MPN drug treatments, the rest of the videos and presentations are available to watch HERE.
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Pegasys® delivery update for regional patients

The company delivering Pegasys®, JustMeds, has advised that they are now able to deliver Pegasys® to regional patients in cold chain validated shippers.

These shippers are specially made to keep your Pegasys® between 2-8 degrees for 96 hours.

The shippers need to be returned for your next delivery and the instructions for their return are on the box – see photo below.

And just a reminder that Pegasys® for all Australian patients can now be ordered and reordered online at
https://www.justmeds.com.au/send-your-script

         

 

 

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Pegasys: new way for Australians to order via online link

The company dispensing and arranging delivery for Pegasys® has set up an online ordering link to speed up the ordering process for Australian patients.

This should save patients time and effort and enable patients in Australia who need their Pegasys urgently to get through to the dispensing team.

The link to order can be accessed HERE.

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Additional genetic mutations in MPNs: impact on prognosis

An important study of genetic mutations in MPNs and their impact on prognosis has just been released in ‘Leukaemia’, titled “Characterization of myeloproliferative neoplasms based on genetics only and prognostication of transformation to blast phase.”

The authors state “By conducting a thorough genetic analysis, we’ve developed a model that relies on 12 genetic markers to accurately stratify MPN patients. The model can be simplified into a decision tree for routine use.”

“Our data suggest to perform a broader genetic screening at diagnosis and also at clinical progression, as driver mutations may change and the MPN-driver mutations present at diagnosis may disappear.”

Their conclusion is “Consequently, expanding genetic analysis beyond JAK2CALR, and MPL at diagnosis is crucial for accurate MPN classification, early high-risk patient identification, and timely intervention.”

This important study can be freely accessed HERE.

 

 

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