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Ken isn’t ruled by his MPN and is optimistic about his future

Ken isn’t ruled by his MPN and is optimistic about his future

Ken at a Fluro Friday mental health promotion at Phillip Island in 2016 at which he was a speaker.
Ken at a Fluro Friday mental health promotion at Phillip Island in 2016 at which he was a speaker.

Had Ken Young been home, rather than in Melbourne’s CBD when his spleen spontaneously ruptured, he mightn’t have lived to tell us about his 20+ years living with an MPN.

“I nearly died,” said Ken, 64, describing the life and death incident in December 2005.

“I was travelling on the train when I had this incredible pain on my left side and shoulder. I thought I was having a heart attack.”

This happened on his way to the city to buy clothes for his first grandchild. He got off at Flinders Street Station, hailed a guard, explained he was unwell and to call an ambulance, which took him straight to hospital.

“Had I been home having a snooze, I probably wouldn’t have woken up. It was very confronting,” he said.

Seven years prior, Ken, was diagnosed with polycythaemia vera (PV), aged 43, after going to his GP about a sore foot. It turned out to be an ischaemic toe that was pre-gangrenous, and he was told to go straight to hospital. That’s where he spent Easter 1998, not camping as planned!

“I was very lucky to find, quite early in my journey, someone who said, ‘yeah I think I know what it is’,” said Ken.

But first, he “fell into the hands of surgeons” who looked at how to remove his toe. They tried to find the cause and thinking there may be a blockage, Ken had an angiogram. He was also told, ‘your blood is like jam’.

“It never clicked…why it was like jam?” said Ken.

Ken and two of his rescue dogs
Ken and two of his rescue dogs

“When they couldn’t work out what the problem was, I was sent to a hospital physician. She took one look at me and my blood results and said, ‘I think I know exactly what the situation is’.

“I had that classic ruddy complexion of polycythaemia,” said Ken, and his haematocrit “was quite high”.

She referred him to a specialist professor but when the result of a bone marrow biopsy was ‘MPD unspecified’, Ken went back to the physician.

“She said, ‘I’m pretty sure it’s polycythaemia’ and referred me to a specialist haematologist,” said Ken, who was originally diagnosed with an MPD.

“I was told I had a myeloproliferative disorder and said, ‘how do you spell that’? It wasn’t named a neoplasm until a few years later.

“When I was first diagnosed, I was quite shocked,” said Ken, because his haematologist had no information to give him and he was strongly advised against looking on the internet.

“I was working in IT at the time, so I went on what was then Alta Vista (before Google) and it came up with the backend of an insurance company that basically said, ‘do not insure – dead within five years of diagnosis’, so I thought, okay this is serious.”

Not much online support was available either, but there were online email lists in the UK and U.S.

“Because I was in the tech industry, I quickly joined up,” said Ken, who came across a fellow Australian with the disease “who was very knowledgeable”.

“He helped me with the ins and outs of the MPDs and as there was no Australian email list, he encouraged me to set one up,” said Ken, which he did in September 1998.

“That’s how I got to be an advocate in MPDs.”

Ken has since run the Australian MPN email list “in various forms and on various platforms”. It started with 10 subscribers and at the last count had 233 Australians and New Zealanders.

Over the intervening years, Ken has had a strong connection to people with MPN and has been very active with the MPN Alliance Australia which is a Leukaemia Foundation-associated patient group.

“A lot of people just don’t know what’s available to them and many say, ‘I’m not happy with my specialist’.

“I refer people to the Leukaemia Foundation’s support services, and talk about advocacy and what their rights are as patients. A lot of them have absolutely no idea that you can actually ask for a second opinion and simple things like asking a doctor to write things down.”

Ken’s professional background is secondary teaching and he’s worked in youth homelessness and disability. Now he works part-time – four days a week – for a primary care partnership in Victoria in the area of patient advocacy within health services.

“Because I’ve got the lived experience, I’ve got a lot to contribute in that area.”

Ken’s treatment protocols

After being diagnosed, Ken had a venesection every two to three months until the spleen incident in 2005, when his medication was changed to hydroxyurea (Hydrea®), which Ken reacted to “very badly”.

“Within four months, I had massive ulceration from my lips down through my trachea into my upper respiratory area, and incredible ulceration on my legs.

“They quickly took me off Hydrea and put me on interferon. For six to seven years I was on quite a high dose, which I self-injected at night five times a week.

“I felt incredibly exhausted, and I think it was the cause of peripheral neuropathy in my feet.”

Ken was on interferon from 2006 until January 2019 when he went on pegylated interferon alfa-2a (Pegasys®).

“I worked closely with Nathalie Cook [a fellow MPN advocate] to get pegylated interferon on the Pharmaceutical Benefits Scheme,” he said.

“Now I’m on 45mg of Pegasys once a week. My bloods are controlled, I’ve got a lot more energy, I don’t feel tired, and I find it much easier to take than Roferon®-A [interferon alfa-2a].

“I’ve got a routine. Every Saturday night I do my injection; subcutaneously either into my stomach or thighs, where there’s a bit of meat, and it’s very straight forward.”

Over his 20 years with MPN, Ken has had three bone marrow biopsies; in 1998, 2001 and 2018.

“There’s been very little progress of fibrosis. It’s still polycythaemia and it’s still controlled,” he said.

Ken Young with his wife, Claire, standing near the ocean.
Ken Young with his wife, Claire.

Living with an MPN 

“I’ve had MPN for a significant chunk of my life. It’s just part of who I am, and I just get on with it,” said Ken.

“In retrospect, having the diagnosis hasn’t limited my enjoyment in life. It’s extended it in a way that makes me want to be part of a wider community of people, and it’s quite a sharing and positive experience.

“You get to a point in life where different things are important and for me it’s being able to make a difference.

“Part of the problem with getting a blood cancer is the sense of helplessness,” said Ken.

“I can’t change the diagnosis – that’s happened. I can’t change the treatment – that’s just going to happen, but I can change other things for other people… making sure there’s information and support services.

“For me it’s working out what you can change and focusing on that. There’s so much of life you can’t control – it’s about being active where you can.

Ken’s advice

“Educate yourself,” said Ken.

“It’s not about becoming an academic and reading scientific papers. There are ways of getting information that are quite accessible and there are some wonderful YouTube presentations from MPN specialists that you can sit and listen to.

Here’s Ken’s ‘go-to’ list:

“And this may be a blokey comment… but a lot of males won’t address health issues and tend to have a ‘she’ll be right’ attitude.

“It’s really important to share when you’re not feeling well, and things change.

“I know a lot of people, myself included in some respects, who tend to be a bit stoic.

“The important thing is to know your changes, notice what’s happening to your body and to yourself, and share this with the people around you.

“There’s probably a reason that you should investigate.”

How the MPN field has progressed

“When I was first diagnosed no one knew what caused MPN,” said Ken.

“The whole thing has changed. Understanding at the genetic level has changed dramatically and the understanding of the JAK2 and other genetic mutations has opened up all sorts of treatment options, particularly with the JAK2 inhibitor drugs.

“There are discoveries that MPNs have links to inflammation. There’s the development of immunotherapies, and the potential for targeted treatment in the CAR T-cell area is something that is completely revolutionary.

“None of that was around 20 years ago. Where we’ve come from, to where we are now, is quite amazing,” he said.

“Now in Australia there are dedicated MPN research groups like the work Steven Lane is doing in Brisbane and Kate Burbury at Walter and Eliza Hall Institute and Peter McCallum Cancer Centre, Andrew Perkins at Monash, David Ross in Adelaide, and Wendy Erber and her team in Perth.

“And there’s more research in the U.S. and UK looking at the whole notion of what’s driving these diseases that offers a whole new way of looking at them and new treatment patterns.

“The main treatment option for people at the moment – if you can do it – is a bone marrow transplant, which has incredible risks.

“There may well be different treatment options. Who knows where it’s all heading. It’s an exciting time,” said Ken.

Where Ken is at with his MPN

“My disease is stable, and I’ll probably live my three score and ten years,” he said.

“I’m quite optimistic about my future actually. I’m not ruled by my disease or defined by it.

“I do other things that are positive contributions to the community,” said Ken, who with his wife, Claire, are foster carers.

New classifications of MPN mean better diagnostics and more definitive prognoses

New classifications of MPN mean better diagnostics and more definitive prognoses

How the MPNs are classified is set to change with eight new classifications based on the underlying mechanisms that drive the mutations, according to international MPN expert, Professor Tony Green.

His Green lab at Cambridge University has been pivotal in identifying the new genetic groups that are expected to replace the “conventional way” of categorising people with MPN used “for 100 years or more”.

Professor Tony Green
Professor Tony Green

Early in his career, Professor Green found haematology “fascinating” because it allowed him to do research that he couldn’t have done in other areas of medicine.

“You get blood cancer in the blood and in the bone marrow, where the action is. You can’t do that [research] in the brain very easily… patients tend to complain!” he said.

Although a consultant haematologist and “clinician by trade”, Prof. Green has spent 80% of his 30-year career in research leadership roles.

He heads his own research group, is the Head of the University of Cambridge Department of Haematology, Professor of Haemato-Oncology at the University of Cambridge, and Director of the Cambridge Stem Cell Institute. He also is a recent president of the European Hematology Association.

Research at the Green laboratory at Cambridge

The driver for most scientists, he says, is understanding how things work.

“Once you understand how things work, or how things have gone wrong in the case of a blood cancer, you can use that knowledge to manage patients with those diseases better. Whether it’s to diagnose them better, treat them better, or give them better information about their prognosis,” said Prof. Green whose lab works in three broad areas.

One uses information “we now have” to make a difference in the clinic, and the second is “more biological” – trying to understand how mutations cause the changes they do.

“How does a mutation in a particular gene, like JAK2, result in the sort of diseases that it does? How does it cause the stem cells to go wrong or the red blood cell progenitors to increase?” said Prof. Green.

“There’s a lot we don’t understand about the mechanisms there.

“Quite often, when you study something abnormal, you learn stuff about the normal, and vice versa,” said Prof. Green about the third area – the unexpected insights that are stumbled across, “such as discovering how the normal regulation of blood cell formation works while trying to understand how the mutations work and the pathways they are part of”.

Referring to the clinical impact of his research, Prof. Green highlighted two messages he thought MPN News readers might want to know from a paper titled Classification and Personalized Prognosis in Myeloproliferative Neoplasms, published in the New England Journal, in October 2018.

A new classification system for MPNs

The first was a new way of classifying the MPNs.

“Until now, we have used the old-fashioned terms to describe patients. We put them into categories – polycythaemia vera (ET), essential thrombocythaemia (ET), myelofibrosis (MF)… based on describing what we see in the patient… too many red cells, too many platelets. It’s a phenotype*-based classification,” Prof. Green explained.

“There are lots of different causes of too many red blood cells and lots of different causes of too many platelets.

“What you really want is a classification based on the underlying mechanisms. The mechanisms that drive these diseases are all the different mutations.

“We’ve looked at 2000 patients with many, many different mutations,” he said.

Based on genome classification, eight separate genomic groups were identified according to the mutations each patient had and covering the gamut of MPNs. And each of those eight groups included some people with what would have been called PV, some with what would have been called ET, and some with what would have been called MF.

“Those eight groups are defined by the real biological causes of these diseases,” said Prof. Green.

“I think this is going to be a much more robust and objective way of categorising patients, which will make it easier when it comes to doing clinical trials, which will have patients stratified into those eight groups rather than the rather subjective names that we give these diseases at the moment.”

The second aspect of the paper was the result of using the “very large database” of 2000 people and “all our clinical and molecular data to generate a way of giving an individual patient a personal prognosis, which is something we couldn’t do”.

“Because what tends to happen is… we put people into groups. We say you are low-risk or high-risk. But low-risk has a whole mixture of people in it and high-risk has a whole mixture of people in it. So, we didn’t know where an individual person fitted in.

“Now we have been able to do that,” said Prof. Green.

“Comparing data from a patient in the clinic to the 2000-patient data bank, we can say with some sort of confidence, ‘well Mrs Jones, you have approximately a 20% chance of developing AML five years from now’.”

Prof. Green expects patients would have polygenic tests** and those results would be combined with their age, gender, laboratory data, blood counts, etc.

“Put that all together and you can give somebody a much more personal idea of what their outlook might be,” he said.

The question now, Prof. Green said, was how this new form of classification would be implemented by individual clinicians and applied in clinical trials.

“It will take a little time for change to occur, and it will be interesting to see how quickly this is picked up and how rapidly it is used,” he said.

“I suspect it will be used more in clinical trials to begin with because you can stratify your patients going into a clinical trial into these eight groups.”

He also expects, initially, that this genomic approach will be compared with the conventional way of categorising patients in a controlled, clinical setting.

Prof. Green said applying the genomic classifications in the clinic would mean genomic sequencing of all MPN patients.

“There are quite a few steps that need to be put in place before this can be set up and run routinely in the clinic.”

World-leading MPN research

In 1995, the Green lab set up “the largest randomised clinical trial ever in any MPN” – the PT1 study.

“The group in Cambridge is unusual. We span everything from large clinical trials through to identifying the mutations, through to understanding what the mutations do,” said Prof. Green.

“Our main strength is trying to understand the disease, and the diagnosis and prognosis stage, which I think is really essential.

“I don’t work at the bench anymore,” said Prof. Green, who has up to 15 researchers in his lab – a mixture of a PhD students, technicians and post-docs.

“I try to sit down with them as a group once a week, and individually, which is variable but ideally is once a week.

“My role is to provide a bit of strategic direction and help keep the focus in the right place, rather than going off on tangents.”

He said some people on his team were good at making mouse models. One post-doc was “absolutely superb” at all the protein signaling-type approaches and transcription regulation – the molecular biology, and others have expertise in methylation changes – the cell biology of what’s going on.

“To understand a disease properly, you really need people to understand the cell biology,” said Prof. Green.

“Sometimes you get taken into apoptosis, or cell death. Sometimes you get taken down into mechanisms of receptor signaling, and other times you are down in the nucleus asking questions about transcription and chromatin.

“And, depending on where our results lead us and the sort of question we are trying to answer, the group evolves.”

What a difference 20 years makes

Prof. Green said the field “had moved fantastically over the last 20 years”.

“If you look across the blood cancers, the number of advances that have made an enormous difference to patients has been very exciting.

“One can point to several diseases where, 20 years ago, a patient with a particular cancer, would almost invariably be dead within five years.

“Now, they are alive and well 20 years later. They may not be completely cured, but what we have achieved has made an enormous difference.”

A word of advice

Prof. Green offered people with MPN some advice.

“This is so easy to say and very difficult to do… those with a very positive approach to their disease, who are just getting on with it and letting it interfere with their lives as little as possible – that has got to be the way to go.

“There is a lot of life to live out there, family to spend time with, all that stuff. Don’t let it [an MPN] get in the way. Easy to say, difficult to do.

“MPN has a relatively slow and long trajectory. They do progress; ET and PV slowly but surely over many years,” he said.

“If you follow somebody for sufficiently long, many will develop either MF, or the unlucky ones will develop acute myeloid leukaemia. But it is such a long trajectory that a large proportion of them will die from something else.

“Most come with chronic phase PV, or ET, and one of the things I absolutely say up front is… if you have got to have a disease, this is the one to have, because most people with chronic phase disease will die of something else, not their disease. They will die with their disease, but not from it.

“I try and give them the information we have about what their personal outlook might be. It’s too easy for people to think they have got a blood cancer and they are going to die, it’s not like that for MPNs,” said Prof. Green.

Professor Green’s holy grail

Prof. Green has two holy grails which he would like to see achieved during his career, and the first is “a targeted therapy that works”.

“The JAK2 inhibitors are not targeted to the mutation,” he said.

“We need a better way of targeting. What we want is a JAK2 inhibitor that only hits the mutant. That is a long way off, but it would be nice.

“And, then, in terms of the consequences, I’d like to really understand in humans how this mutation [JAK2] arises. We are starting to get this information. We can now do lineage tracing in man, so we can look at a patient and we can say ‘your JAK2 mutation arose when you were five’.

“But what we don’t know is how that mutation changed your stem cells back then. So, getting a better understanding of what is going on in the patient looking back is the second holy grail.”

The physical appearance or biochemical characteristic of an organism as a result of the interaction of its genotype and the environment.

** A polygenic score, also called a polygenic risk score, genetic risk score, or genome-wide score, is a number based on variation in multiple genetic loci and their associated weights.

European amyloidosis meeting highlighted the need to improve education

European amyloidosis meeting highlighted the need to improve education

Vince O’Donnell is a patient advocate for the Australian Amyloidosis Network.
Vince O’Donnell is a patient advocate for the Australian Amyloidosis Network.

Vince O’Donnell learnt of the concerningly low rates of referral for people at risk of amyloidosis when he attended the second European meeting for ATTR amyloidosis for doctors and patients.

The meeting, in Berlin (Germany) last September (2019), is held every two years by the Amyloidosis Alliance: The Voice of Patients, a global entity dedicated to improving the quality of care and experience of amyloidosis patients by exchanging experiences and constructive interactions at an international level.

Vince and his partner, Terri, link closely with the Australian Amyloidosis Network (AAN) and have been active in the Australian amyloidosis community since Vince was diagnosed with hereditary amyloidosis polyneuropathy with cardiomyopathy in 2014.

In his advocacy role, Vince said he represents Australian patients’ concerns and encourages the inclusion of the patient-perspective in improving diagnosis, treatment and care for all types of amyloidosis.

“The three days of the meeting were full of lectures, talks, networking and information sharing,” said Vince, 64, of Queensland.

The Alliance taskforce presented its findings from the 2018 initiatives, including the improvement of education, accessibility of medications and an upcoming trials report.

Vince said a pilot study presented at the meeting found most GPs didn’t give their patients a referral despite an alert showing the possibility of an amyloidosis diagnosis appearing on their computer screens.

“Ideally, every medical school across all primary healthcare should be educated going forward,” said Vince.

“The AAN does this in some capacity but patients need to be more involved and at the very least made aware this is happening.

“Advancements across the whole sector also need to be promoted to ensure the continued education of general practitioners, specialists, primary health carers and the wider community,” said Vince.

In 2019, new Alliance taskforces started working on Standards for Cure and Care, Research and Trials, Advocacy, and AL Amyloidosis.

“Of particular interest was the Nutritional Aspects report by Dr Anna Hüsing-Kabar (Münster),” said Vince.

“Terri and I have asked to join the Lifestyle taskforce should the initiative be voted in this year.”

At a dinner at the Bode Museum, Vince spoke with fellow members of The Asia-Pacific Regional Network; a subgroup under the Amyloidosis Alliance for patients from Australia, South Korea, Japan and New Zealand.

“We hope China, Thailand, Vietnam and India, along with other Asia Pacific countries will join our regional network,” said Vince.

“Terri and I will continue to advocate for Australia’s formal membership of the Amyloidosis Alliance as we are only classed as an Associated Patients Group.

“We will also continue to meet with our regional network and seek others who want to work with us flying the flag for all Australians living with amyloidosis and their families,” he said.

Read Vince’s story of living with hereditary amyloid polyneuropathy with cardiomyopathy, from the January 2019 issue of Amyloidosis News

New amyloidosis drugs and clinical trials update

New amyloidosis drugs and clinical trials update

A new class of drug may soon be available to Australian patients with significant cardiac AL amyloidosis in a Phase III clinical trial, expected to open in mid-2020.

The monoclonal antibody, currently called CAEL101, was designed by the Columbia University Amyloidosis group in the U.S. to bind on to the amylogenic protein and help clear it from the body.

Dr Hasib Sidiqi
Dr Hasib Sidiqi

“It’s a different class of drug,” said Dr Hasib Sidiqi, a consultant haematologist at Fiona Stanley Hospital (Perth). Last October, he returned to Australia from a two-year scholarship at the Mayo Clinic (Rochester, U.S.) where his focus was myeloma, amyloidosis and bone marrow transplantation.

Speaking to Amyloidosis News about treatment developments and clinical trials in the pipeline for AL amyloidosis, Dr Sidiqi said treatments generally targeted the plasma cells that produce the amyloidogenic protein. The aim being “to try to kill them off, so you’re shutting the factory of production”.

“CAEL101 targets the actual amyloidogenic protein, to try and help clear it from the body and hopefully even help clear it from the organs that are affected.”

Early Phase I/II studies of CAEL101 showed efficacy, particularly for patients with cardiac involvement, said Dr Sidiq.

Now CAEL101 it is being considered for a Phase III trial, where it would be used in combination with standard therapy – cyclophosphamide, bortezomib and dexamethasone (CyBorD); the regimen commonly used for newly diagnosed amyloidosis patients.

This trial is undergoing feasibility assessment at several centres in Australia, including the Fiona Stanley Hospital.

“If selected, we hope to open the trial later on this year,” said Dr Hasib Sidiqi.

The link between myeloma and amyloidosis treatment

Treatments used for AL amyloidosis tend to be borrowed from the treatments that work for myeloma.

“If we look at amyloidosis pathophysiologically*, abnormal plasma cells produce the amyloidogenic light chains that are then deposited in tissues and cause organ disfunction,” said Dr Sidiqi.

“So, the goal of treatment is to try and stop production of those light chains and we’ve realised that the same treatments that we’ve used for myeloma – a cancer of the plasma cells – work quite well in amyloidosis.

“Amyloidosis patients have unique challenges because organ involvement with cardiac and kidney dysfunction is more common than what we see in myeloma patients.

“The key treatments we have tried over the last 5-10 years have used bortezomib (Velcade®)-based treatment,” he said.

“It works very well for AL amyloidosis and the best data on that treatment comes from the UK National Amyloidosis Consortium.

“They recently published a trial of 900 patients treated with Velcade/bortezomib-based regimens up front for amyloidosis and they showed very promising results.

“Most people will respond to this therapy and those who achieve good responses can go on to have several years without the disease relapsing. That gives us further evidence that the treatment we’ve been using for several years now actually has very good outcomes.”

Dr Sidiqi said some of the other myeloma drugs that are available pose unique challenges in patients with AL amyloidosis and gave the example of carfilzomib.

“It can cause cardiovascular issues such as high blood pressure and, in some patients, heart failure although this is less common,” he said.

“But in our amyloidosis patients, who may already suffer from heart disfunction, carfilzomib is very poorly tolerated, so that is a drug we try and avoid.

“Likewise, other drugs like the immunomodulatory drugs – lenalidomide (Revlimid®) and pomalidomide (Pomalyst®) – tend not to be so well tolerated in the amyloidosis patients as the myeloma patients. Although they have been looked at in smaller studies, overall their use is limited in AL amyloidosis.”

Daratumumab and AL amyloidosis

What Dr Sidiqi said had emerged as a success story in the last 12-24 months was the use of daratumumab (Darzalex®), a monoclonal antibody to CD38, in patients with AL amyloidosis.

“We know daratumumab is a highly effective treatment in myeloma. It’s used in all stages of the disease, from upfront to relapsed settings,” said Dr Sidiqi.

“The first early trials of daratumumab used in AL amyloidosis were reported at the 2017 annual meeting of the American Society of Hematology. One trial from Boston (U.S.) and one from the French group, Intergroupe Francophone du Myélome (IFM), showed that patients with AL amyloidosis treated with daratumumab do exceptionally well.

“They respond, and they respond very quickly, with the majority of responses seen in the first cycle of therapy, and there have been some really amazing responses with people achieving a deep response even after one dose of the drug!

“Those early positive results then translated into an expansion of clinical trials,” said Dr Sidiqi.

One trial is looking at daratumumab in combination with bortezomib, cyclophosphamide and dexamethasone compared to bortezomib, cyclophosphamide and dexamethasone as upfront therapy.

“So, we’re looking at whether the addition of the daratumumab to the existing triplet of drugs that we were using for newly diagnosed patients would add any benefit. That trial opened in a few Australian centres but is now closed to recruitment.

“I think it’s going to show some promising results. We’re eagerly awaiting that because it could really help improve our options for treating AL amyloidosis.

“Daratumumab is a really exciting drug for AL amyloidosis,” said Dr Sidiqi.

“Currently our access to daratumumab in Australia is limited. Myeloma patients are able to access this through Janssen’s compassionate access program. I believe daratumumab is being resubmitted this year to the Pharmaceutical Benefits Advisory Committee for consideration.

“Unfortunately, as far as I am aware there aren’t any trials with daratumumab targeting the AL amyloidosis patient population that are currently open in Australia.”

Dr Hasib Sidiqi, right, with one of his “Mayo mentors” and amyloidosis specialist, Dr Morie Gertz, at the American Society of Hematology 2018 annual meeting.
Dr Hasib Sidiqi, right, with one of his “Mayo mentors” and amyloidosis specialist, Dr Morie Gertz, at the American Society of Hematology 2018 annual meeting.

AAN-initiated clinical trials  

However, Dr Sidiqi said the Australian Amyloidosis Network (AAN) – a group of physicians from across the country with four amyloidosis centres – is considering two new Phase II trials for people with AL amyloidosis.

“One is to consider daratumumab in combination with a couple of medications that we sometimes use for AL amyloidosis – melphalan (Alkeran®) and dexamethasone – in patients who have relapsed disease,” he said.

“If they’ve already had CyBorD, that would be a very good trial to have open. It would give these patients access to what we know is a really effective drug for AL amyloidosis.”

Dr Sidiqi said plans are for this trial to be run in collaboration with the Australasian Leukaemia & Lymphoma Group and to recruit 40-60 Australian patients.

The second trial is for isatuximab, a monoclonal antibody to CD38 which has a slightly different structure to daratumumab but is in the same class. It already has been tested and found to be effective in treating myeloma.

“We are working in collaboration with the French group, IFM, to see if we can open a clinical trial using isatuximab in combination with pomalidomide and dexamethasone,” said Dr Sidiqi.

This trial also would be in the relapsed setting, “so if you’ve had treatment before and your disease has come back, that’s what we would be targeting”.

“We hope to open the isatuximab trial in Australia at two or three AAN centres. Again, that would allow us to access what we hope is going to be a very effective drug for AL amyloidosis.”

Both trials are in the negotiation and discussion phase with the industry sponsors that would support the clinical trials.

Dr Sidiqi said autologous stem cell transplantation also was a treatment option for a select group of patients.

“You have to have very good organ function and you need to have a good performance status to be considered for that but that’s another option.”

Dr Sidiqi is a member of the AAN, which covers all the amyloidoses.

As good data is not available on the incidence sand prevalence of amyloidosis in Australia, Dr Sidiqi said the AAN also was looking at doing epidemiology studies to gather this information.

Members of the AAN catch up once a month by teleconference.

“It’s a good forum for us to discuss tricky patient cases and come up with a consensus on a treatment plan,” he said.

“While most people with amyloidosis are treated at one of the AAN’s four city centres – in Brisbane, Sydney, Melbourne and Perth – that doesn’t capture all of Australia.

“It’s likely that a lot of other patients are being treated elsewhere by our haematologist colleagues. We are very accessible for opinions as a group and are happy to discuss cases with anyone who wishes to,” said Dr Sidiqi.

* The study of structural and functional changes in tissue and organs that lead to disease.

“It’s an ongoing journey you’ve got to manage”

“It’s an ongoing journey you’ve got to manage”

Cath Armstrong felt “absolute relief” to be diagnosed with amyloidosis in February 2017 because it meant she finally knew what she was dealing with… and it wasn’t all in her head!

Looking back, she believes she’d had amyloidosis for the previous three years; it just hadn’t been diagnosed.

Cath Armstrong, right, and her eldest daughter, Rebecca.
Cath Armstrong, right, and her eldest daughter, Rebecca.

In 2014, she’d spent a week in a Brisbane hospital having tests to find the cause of a range of symptoms – pain up the side of her face, weight loss (12kg in a month) and problems with her digestive tract.

“They couldn’t find out what was wrong and gave me sedatives. I think they thought it was a nervous disorder,” said Cath, 63, who lives at Tamworth (NSW).

“That’s fairly common with amyloidosis if you read about it. People sometimes do one or two stints in a mental health unit before finding out it’s not in their head… there’s actually something physically wrong with them,” she said.

Cath was “out of action” for nearly four months when she couldn’t drive or work, and it took her a long time to recover – over 18 months. She was initially treated by a neurologist for a nerve condition called trigeminal neuralgia. She then saw a musculoskeletal therapist who was also a cranialsacral therapist.

“I had to be very careful what I ate and could only eat really small meals. I didn’t know why but thought I could be gluten intolerant,” said Cath.

A gastroenterologist who did an endoscopy saw evidence that she had stomach ulcers that were healing.

“They didn’t run the Congo red test on the biopsies that is required for a diagnosis of amyloidosis, so didn’t pick up that’s what I had,” said Cath.

Then, in late-2016, she got “really sick” again in the lead-up to Christmas. A bookkeeper, Cath was working as PA for a developer, “doing all the paperwork for 46 residential blocks”. It was challenging and she was under a lot of pressure.

“Normally I don’t have any trouble operating under pressure, but it was too much with my system being unwell,” said Cath.

“I knew there was something wrong, but I didn’t know what it was and because I couldn’t get any answers, I just tried to manage it myself.”

Cath had severe weight loss again, along with bleeding from the bowel and ongoing problems with her digestive tract.

Her GP recommended another endoscopy, but her earliest opportunity to have the procedure in Tamworth was a couple of months later, in February 2017.

She also had a colonoscopy and this time biopsies from both investigations revealed amyloidosis.

“The gastroenterologist did 34 biopsies and every single one was positive to amyloidosis,” said Cath.

“He didn’t know what he was looking for and when he saw me, he said ‘this is not what I was expecting to find’.

“He said, ‘do you know what myeloma is?’ and I said ‘yes’. My sister-in-law had it and had passed away 10 years previously. Then he said, ‘well, amyloidosis is similar’.”

Cath was referred to a haematologist in Tamworth who had a special interest in myeloma and amyloidosis.

“He did a bone marrow biopsy and aspiration and gave the definitive diagnosis of AL amyloidosis, and myeloma as well! Then he ran through the treatment options, saying ‘think about what you want to do’,” said Cath.

“I made the decision pretty quickly because I went away and did a heap of research.”

She started treatment on the CyBorD regimen – cyclophosphamide, bortezomib and dexamethasone, the initial goal being to lower her lambda and kappa light chain ratio readings as much as possible. At that stage they were so high and she wasn’t suitable for an autologous stem cell transplant (SCT) but later that year she was put forward as a candidate for a SCT.

Cath with her granddaughters, Remy and Odetta.
Cath with her granddaughters, Remy and Odetta.

“I wanted to have the transplant in Brisbane because all my family were up there,” said Cath who has three adult children and seven grandchildren.

“It’s really important to have a good network around you. All my kids had different roles in supporting me when I got my diagnosis.

“My eldest girl [Belinda] used to fly down to Tamworth and see me. My second daughter [Rebecca] took time off – 2½ months – when I had the transplant, and my son [Matthew] would come down to see me and filled in if my daughter couldn’t come to the hospital.

“I had somebody there just about every day.”

Cath Armstrong with her granddaughter, Zara, in her garden at Tamworth before her stem cell transplant.
Cath Armstrong with her granddaughter, Zara, in her garden at Tamworth before her stem cell transplant.

In November 2017, Cath went to Brisbane for lead-up tests prior to her stem cell harvest, but during the gastroscopy, she “aspirated on the table”, and her children were told she ‘probably wouldn’t make it’.

Cath pulled through and recovered in ICU. But plans for the transplant were delayed. First, she needed two heart stents, but because her lungs were “burnt” from the aspiration and she had a “terrible cough”, she had to wait for her lungs to recover.

In January 2018, Cath’s stem cells were harvested but they didn’t get enough stem cells, so she had a second harvest the following month, and the Leukaemia Foundation helped out with accommodation in Brisbane.

Her transplant was in March. She was hospitalised for seven weeks during and after the transplant and went home to Tamworth in May.

Cath described the transplant as “reasonably successful”.

“The light chain numbers went down to virtually normal. My lambda light chains, which had been really high (about 320 at diagnosis) came down to 20-something (after the transplant).

“One of the problems was my kidneys. They had a lot of trouble recovering after the transplant and even now my kidney function is compromised.

“For a short time after the transplant I didn’t have any chemo, but then the light chain numbers started going up again, so I went back on maintenance chemo – thalidomide and two other drugs.

“That bopped along okay,” said Cath, but the drugs affected her cognitively.

“You’re not as sharp as normal, so number crunching was at times quite challenging. I had the advantage of working from home. If I didn’t feel well, I could have a rest and work later into the evening.

Cath with her three children, from left, Rebecca, Belinda and Matthew in December 2018 when Cath was being treated with prednisone.
Cath with her three children, from left, Rebecca, Belinda and Matthew in December 2018 when Cath was being treated with prednisone.

“I never really stopped working,” said Cathy, but she didn’t work much for two months during the transplant.

Then, at the end of the 2019 financial year, she gave up bookwork for the time being when she got “a nasty dose of shingles”. The nerves in her bladder were affected and she had a suprapubic catheter for four months.

This coincided with starting a new drug, lenalidomide, her fifth variation in treatment since her diagnosis.

“It’s expensive because it’s not on the PBS – $6000 a month,” said Cath, who was “very fortunate” to access the drug on compassionate grounds because she had myeloma as well.

She was on lenalidomide until the end of November 2019 when her immune system “had a bit of a crash and burn”.

“My platelets got down to 30; the normal range is 150-400,” she said.

In January (2020), Cath went back on lenalidomide at a reduced dose – one tablet every three days – and when she spoke to Amyloidosis News, she had just found out her immune system was starting to recover.

“My platelets were up to 130 today,” she said.

In mid-2019, Cath decided it was “a bit confusing” to have two haematologists – one in Brisbane, with a team of six or seven specialists including a nephrologist and cardiologist, and one at Tamworth.

“Now I’m just running with the team in Brisbane, where they all work together, which certainly isn’t available in Tamworth,” said Cath, who flies to Brisbane for her four-weekly reviews.

“Usually for a couple of days, before getting the okay to go home. Sometimes I’ve had to stay for up to 2½ weeks to do more blood tests, while they try and figure out what they are going to do with me!

Cath’s approach to managing amyloidosis

“You’ve got to learn to not have too many expectations about anything, to go with the flow and remember that amyloidosis is a disease you’ve got to manage, like diabetes.

“It’s all relative. I read this case today about a guy in the States with exactly the same as me; AL amyloidosis and myeloma and he had a SCT. He was in his early-30s and a personal trainer. He ended up having a heart transplant because his amyloidosis was in the heart and it also went to the kidneys and he had a kidney transplant. Then the myeloma kicked in and his spine started to crumble, so he had two steel rods put in his back. So I think you’ve got to keep it all in perspective and say, ‘this story doesn’t sound too bad does it’?”

“I just deal with things as they arise. I take it as it comes,” said Cath who describes herself as “a bit of a problem solver”.

“If something doesn’t work, I go to plan B, and if that doesn’t work, I go to C, D and E. You’ve just got to have that sort of attitude, not to be negative, and to look for solutions all the time.”

Cath doesn’t have a bucket list.

“You realise after something like this that all of that is irrelevant. What’s most important is family. I network with my friends and have friends stay and I enjoy myself.

“A lot of people probably don’t realise it takes a lot of effort to cope with this illness. It’s ongoing management, and I still get tired,” said Cath.

Her days revolve around daily physio exercises, visiting a physio once a week, exercise classes, regularly seeing her local GP for “other bits of treatment’’, injections to keep her bones strong and post-transplant immunisations.

“My goals now are eliminating as much stress as possible, physically to keep building in strength, and catching up with family and friends,” she said.

Cath’s advice based on her experience

“My advice is to seek help and, if you possibly can, to be well supported by family, and keep them informed. Whoever your support network is, take them on the journey, don’t block them out. You’ve got to include them if you want them to help you. This is a terminal disease, so we have to deal with it together.

“When I was in hospital, and they didn’t think I was going to make it, one of the doctors said to me, ‘you know, not many people of your age (early-60s) come in with power of attorney done, so their kids can do things and make decisions’.

“I did my power of attorney in the nine months when I was being prepared for the SCT, and I’ve since done an Advanced Health Care Directive and made it very clear what my wishes are.

“Don’t to be afraid to ask your specialists questions, even the basic terminology in a blood test. Ask them to explain things to you so you feel you can make an informed decision and you’ve got a clear understanding of what’s happening to you.

Number of Australians living with blood cancer to more than double

Number of Australians living with blood cancer to more than double

Tuesday, 4 February 2020

Twenty Australian children, adults, parents and grandparents are losing their lives every day as blood cancer becomes one of the nation’s most diagnosed and deadly killers.

Sadly, that number is projected to more than double by 2035, and around 186,000 Australians could lose their lives to the disease in the next 15 years, according to the Leukaemia Foundation’s recently released State of the Nation: Blood Cancer in Australia[1] report.

Today, on World Cancer Day, Leukaemia Foundation CEO Bill Petch has launched the annual World’s Greatest Shave campaign urging Australians to continue to support the fight against blood cancer as the organisation prepares for a massive jump in demand for its services and begins paving the way for a new national, collaborative approach to help save the lives of Australians fighting the disease.

“The first of its kind, State of the Nation report looks at the impact of blood cancers across the country and provides evidence showing that blood cancers are also under-reported,” Mr Petch said.

“We now know that the true size, scale and impact of blood cancer in Australia has been significantly underestimated, potentially leading to inconsistency and inadequacy of funding and service delivery.”

The State of the Nation: Blood Cancer in Australia report shows that not only is there an urgent call to better meet the needs of those currently living with blood cancer, but that demand on support services is going to grow substantially in the next 15 years.

Every day, 41 Australians will be told they have blood cancer and 110,000 Australians are currently living with the disease. According to the report, these numbers are also projected to more than double by 2035 to 100 people diagnosed with blood cancer every day and more than 275,000 living with the disease.

Mr Petch said the report also found the cost to the health system of treating and caring for people with blood cancer is projected to increase to over $10.9 billion in 2035 – up from $3.4 billion annually today. The total cost to the Australian economy is also expected to reach $71.9 billion a year by 2035 – more than triple today’s annual estimated cost of $22.9 billion.
“Blood cancer is and will continue to be an issue for all Australians and there is an urgent need for collaborative action to help meet the needs of those living with the disease, today and into the future.”

With the support of the Federal Minister for Health Greg Hunt, the Leukaemia Foundation has established the  Blood Cancer Taskforce, which includes 30 of Australia’s leading blood cancer experts and stakeholders, to deliver Australia’s first National Strategic Action Plan for Blood Cancer.

“It’s the first time in Australian history that the issue of blood cancer has been put under the national microscope, and now we are working collaboratively on a  National Action Plan to catalyse health system reform, help accelerate research, enable access to novel and specialised therapies and most importantly, to empower patients. The aim is to create a future where all Australians receive the best treatment available and not only survive their disease,but thrive with all of the support they need.”

Co-chaired by Mr Petch and Professor John Seymour AM (Director, Department of Haematology, Peter MacCallum Cancer Centre and the Royal Melbourne Hospital), the Taskforce includes leaders of the Australian blood cancer community such as Prof. Sanchia Aranda AM (Cancer Council), Richard Vines (Rare Cancers Australia), Elizabeth De Somer (Medicines Australia), and Prof Andrew Roberts (WEHI) and Prof David Gottlieb ( Westmead Hospital).

“The Leukaemia Foundation is proud and privileged to stand with Australia’s incredibly talented and diverse blood cancer community to make sure everyone has access to the best care, to accelerate research delivering rapid advancements and to empower people with blood cancer to live well,” he said.

“We’re not going to stop until together, we have conquered one of Australia’s most prevalent and deadly cancers.”

Mr Petch said the Leukaemia Foundation was determined to achieve its bold new goal to see zero lives lost to blood cancer by 2035, and the organisation is calling on the national community to help make this vision a reality.

“The funds raised by World’s Greatest Shave are vital to improve and save lives, so we urge all Australians to take a stand against blood cancer by rallying together, signing up and getting sponsored to bravely shave their heads or cut or colour their hair in 2020,” he said.

Join the fight against blood cancer by registering for World’s Greatest Shave at

Learn more about the Leukaemia Foundation’s path to zero lives lost to blood cancer hereOpen this document with ReadSpeaker docReader.



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Government’s commitment to blood cancer

Government’s commitment to blood cancer

Monday 3 February, 2020

The Leukaemia Foundation welcomes the commitment from the Federal Health Minister Greg Hunt to establish a Blood Cancer Taskforce and Action Plan.

The Blood Cancer Taskforce will work with leading clinicians, researchers and patient groups in the blood cancer community to develop a blueprint and recommendations for Government to improve blood cancer survival rates.

Currently, close to 13,000 Australians are diagnosed with a blood cancer like leukaemia, lymphoma and myeloma every year (1), however recent analysis showed the number would increase to around 17,000 people (2) by 2025. This is close to 50 Australians projected to be newly diagnosed, every day, by 2025 – or two people every hour.

Leukaemia Foundation CEO Bill Petch said that this commitment from Government is welcome news for the blood cancer community.

“We are pleased that the Government has acknowledged the need for national leadership and action on blood cancer,” Mr Petch said.

“There are significant differences in treatment and survival outcomes depending on where a person lives, so it’s really important that we work together with Australia’s leading experts, Government and the blood cancer community to develop a plan to beat blood cancer.”

“For people living with blood cancer this is really significant, because we now have bipartisan commitment from both sides of politics to develop a blueprint for action on blood cancer.”

The Leukaemia Foundation looks forward to working with the incoming Government, our members and partners to progress this important work.

The Leukaemia Foundation provides practical and emotional support to Australians diagnosed with a blood cancer at no cost, thanks to the generosity of the community through our fundraising efforts. We invite all Australians living with a blood cancer, their families and carers to subscribe for ongoing information at or call 1800 620 420.

[2] Incidence data from 2005 – 2015: