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A trial put Joe in remission for six years and counting

A trial put Joe in remission for six years and counting

Jozsef (Joe) Mulaahmetovics was among the 13 Australians with amyloidosis who participated on the international ALLG MM13 trial which he says saved his life.

Joe Mulaahmetovics and his family

When he was diagnosed with AL amyloidosis in the heart, in January 2013, Jozsef had never heard of the disease.

“Amyloid in the heart causes the heart to stiffen so it is unable to fully relax and fill up with blood,” said Joe, 65, a retiree.

“Even though the heart muscle may still be able to contract normally, blood supply to the body is less efficient, so there may be reduced blood flow to vital organs.”

The thickness of Joe’s stiffened heart wall was 12.5 mm. This has since reduced to 11 mm which he says, “is not much”, and it causes ongoing fatigue and weakness.

Soon after his diagnosis and before starting any other treatment, Joe’s haematologist, Dr Peter Mollee, mentioned a clinical trial. Within a few days Joe was accepted on to that trial and not long after that he started the treatment protocol.

“When you are diagnosed with a life-threatening disease and your survival chance is very slim, you are absolutely shocked, so I was very happy and lucky to have the opportunity to go on the trial.

“The chemo was melphalan and dexamethasone, and for a few lucky patients like me, I got the additional bortezomib (Velcade®) injections in the stomach twice a week for the first four weeks.

“When my stomach became really red and hot and covered in welts, I was administered the bortezomib in the arm from then on.

“I was thankful to be accepted on the trial and even happier when I found out my body was responding to the bortezomib.”

At the time Joe and his family were living on Queensland’s Gold Coast.

“During the trial we travelled to Brisbane’s Princess Alexandra (PA). Hospital to get the Velcade injection 2-3 times a week, sometimes more often. We didn’t need to stay overnight.

“My treatment started in June and finished in late-December in 2013,” said Joe who hasn’t had any further treatment for amyloidosis since.

“My body slowly started to respond to the treatment and my blood count started to move down slowly. It was a slow process, but positive signs started.

“Around late-November, Dr Mollee reduced the amount of Velcade because I had more and more side-effects. He was worried about permanent damage.

“After the treatment I went to PA for a check-up every 3-4 weeks, then later on, it was every six weeks.

“It took nearly two years when Dr Mollee said, ‘you are in remission’.”

“Prior to starting the trial, I also underwent a stem cell collection procedure for future treatment if necessary,” explained Joe, but he hasn’t needed to use these stored stem cells.

Now Joe, his wife Katalin, and their daughter, Sylvia, (pictured above) live at Bribie Island north of Brisbane.

“Constant fatigue forced me to stop working. I was an accommodation manager at the Gold Coast, responsible for taking care of a holiday complex and letting the apartments,” said Joe, who is still plagued by a range of side-effects.

He describes these as including “nerve pinching, joint pain, pain in my toes, shooting pain in the side of my leg, blurry vision sometimes, shaking hands, tiredness, numbness in fingers, left leg and arm, forgetting words, dizziness, derealisation, and brain fog sometimes.

“But all things considered, I feel very lucky.”

“If there is one thing I could tell other people diagnosed with amyloidosis, it would be to never give up.

“I feel extremely lucky to have been diagnosed at an early stage and that I received help immediately. After six months of chemotherapy treatment I’m still in remission. I feel happy.’

“It is very important to stay strong and positive mentally. Follow the doctor’s orders, eat well and rest as much as you can. Join groups and meet other people with the same condition.”

Joe continues to have three-monthly check-ups, which includes an amyloid blood and urine test, and once a year he has an ultrasound and x-ray.

“I’m still in remission and I am absolutely sure the treatment saved my life. I wouldn’t have survived without it, and Dr Mollee,” he said.

“Living with amyloidosis can certainly be challenging but a positive attitude and having the support of medical professionals and your loved ones goes a long way in fighting this disease.”

Joe goes to meetings with other amyloid patients at the Leukaemia Foundation.

“In the initial stages of my diagnosis they [the Leukaemia Foundation] provided a one-off rebate on one of my household utility services, which was of great assistance.”

MM13 trial results – bortezomib improves overall survival 

MM13 trial results – bortezomib improves overall survival 

A clinical trial, demonstrating the benefit of bortezomib (Velcade®) in improving response rates and overall survival in AL amyloidosis, is described as “a staggering result, almost never seen in blood cancers”.

Long-term follow up of patients enrolled in an international Phase III AL amyloidosis trial, known in Australia as ALLG MM13, and funded here by the Leukaemia Foundation, has shown that bortezomib treatment results in a ~30% absolute improvement in survival, said Associate Professor Peter Mollee, a consultant haematologist in clinical and laboratory haematology at the Princess Alexandra Hospital (Brisbane), Associate Professor with the University of Queensland Medical School, and chair of the Scientific Advisory Committee of the Australasian Leukaemia & Lymphoma Group (ALLG).

Assoc. Professor Peter Mollee
Assoc. Professor Peter Mollee

Last month, the results of the ALLG MM13 study, that tested a new standard of care for patients with AL amyloidosis, were published in the Journal of Clinical Oncology.

The randomised, controlled study of melphalan and dexamethasone versus bortezomib, melphalan and dexamethasone, investigated the haematologic response after three cycles of therapy in previously untreated patients with systemic light-chain (AL) amyloidosis.

There were 109 participants from 10 countries in the trial, including 13 Australian amyloidosis patients; just over 1/10th of the trial enrolment, which A/Prof. Mollee said was “quite a lot more than most other countries, on a per capita basis”.

The study, which opened in 2011, was run by the ALLG at four hospital sites in Australia, and was completed in January, after extended follow-up.

Read Jozsef Mulaahmetovics’ story – one of the Australians on the ALLG MM13 study 

ALLG MM13 results “staggering” and produced “amazing data” 

A/Prof. Mollee said the trial results showed not only that the proportion of patients who achieved a response was higher, but the responses were far deeper.

“The trial was powered to show a difference in the haematologic response – that is, how low you could push down the light chain that was causing the problem, by killing the cells that are making the light chain,” explained A/Prof. Mollee.

“The haematologic response rate overall was 79% in the bortezomib arm and 62% in the control arm, but deeper responses (technically known as a very good partial response or a complete response) were 64% in the bortezomib arm and 39% in the control arm, and these were highly significant differences.

“Response is important, but you’re always looking for a good improvement in the organs affected by the amyloidosis, and also that you may prolong the patient’s life,” said A/Prof. Mollee.

“And this is where there was a very surprising result; the magnitude of improvement in the number of patients alive in the group that got bortezomib.

“In the patients who received the bortezomib, the proportion of patients alive four years after completing their course of treatment was around 70%, whereas the proportion of patients alive in the arm that didn’t get bortezomib was around 40%.

“So, if you got the bortezomib, that’s almost one in three patients who were alive, who previously wouldn’t have been. When you look at randomised trials and cancer treatment, you rarely see that improvement in overall survival,” said A/Prof. Mollee.

 “I think it’s quite a staggering result.”

“These patients had their treatment between 2011 and 2017, and now we’ve got enough follow-up to know the really important outcomes; how many patients are alive four to five years down the track.

“We know that data, and it’s amazing data.”

However, despite the results of this trial, bortezomib is not approved specifically for AL amyloidosis, in Australia or anywhere else in the world. In fact, worldwide, there are almost no countries where any drug has yet been approved for AL amyloidosis.

AL amyloidosis patients can only access bortezomib on Australia’s Pharmaceutical Benefits Scheme if they also have a diagnosis of myeloma.

“So, we’ve got a great drug, but it’s not always easily available at this stage,” said A/Prof. Mollee.

 (Read more about this in our Expert Series interview with A/Prof. Mollee) 

Background to the ALLG MM13 trial 

The first-ever trial in AL amyloidosis in Australia – the ALLG MM8 study – looked at melphalan and dexamethasone in various doses and was supported with a $30,000 grant from the Leukaemia Foundation in 2005.

Several years later, when the results of the Phase II trial were presented at a European meeting and A/Prof. Mollee spoke to amyloidosis experts there, discussions raised the need to internationally collaborate. With AL amyloidosis being such a rare disease, single countries couldn’t conduct randomised studies due simply to not having enough amyloidosis patients to participate.

“Professor Giampaolo Merlini from the Pavia Amyloidosis Centre in Italy had an idea for a trial looking at bortezomib, and the possibility of Australia joining the trial came up,” said A/Prof. Mollee.

Bortezomib was highly effective in myeloma – a related disease – and looked likely to being effective in AL amyloidosis too.

“A trial was designed to add bortezomib to what was then the standard of care for most patients; melphalan and dexamethasone, to see if it improved outcomes in amyloidosis,” said A/Prof. Mollee.

However, the European Myeloma Network that was sponsoring the trial didn’t have the ability to sponsor it in Australia.

“We took a proposal to the ALLG to run the trial, with some funding and supply of the drug from Janssen (the manufacturer of bortezomib), and a $105,000 Grant-in-Aid from the Leukaemia Foundation,” said A/Prof. Mollee.

“This trial – ALLG MM13 – wouldn’t have happened in Australia without the Leukaemia Foundation grant!” he said.

“It was a substantial grant and it enabled us to have enough money to bring the trial to Australia.”

“When this trial was designed, pharmaceutical companies were reluctant to run a study in this disease.

“This is one of the advantages of investigator-initiated trials. Researchers can try to answer important questions in rare diseases.

“This trial broke new ground in AL amyloidosis.”

Delaine Smith, CEO of the ALLG
Delaine Smith, CEO of the ALLG: “The outstanding results from the ALLG MM13 trial will help deliver better lives and better treatments for patients with AL amyloidosis here and around the world.”

“Since this trial, a number of pharmaceutical companies have sponsored studies because they have seen that you can do trials in this patient group, the international amyloid community could work together, and there was a good chance that their drugs could get to market.

“But I don’t think any of that ever would have happened without this study (ALLG MM13) proving that the networks were there internationally to make studies work.”

Delaine Smith, CEO of the ALLG said, “the ALLG is proud to deliver research that makes a significant impact locally for Australian and New Zealand patients as well as having global reach.

“As Australia and New Zealand’s only not-for-profit cooperative trials group focusing on all blood cancers, the ALLG is privileged to offer a range of important trials in challenging disease areas such as AL amyloidosis.

“In fact, we have another AL amyloidosis trial in the pipeline to launch in 2021. The outstanding results from the ALLG MM13 trial will help deliver better lives and better treatments for patients with AL amyloidosis here and around the world.”

About bortezomib 

A/Prof. Mollee said that while bortezomib was very effective, it was not for everyone and was a more difficult drug to use in patients with AL amyloidosis, compared to in myeloma patients.

“Patients with myeloma have a large amount of cancer cells, but typically speaking, preserved organ function, so their heart, kidneys and liver are all normal.

“But while the tumour bulk in AL amyloidosis is very low, the organ function is compromised because of amyloid deposition in those organs.

“This means their heart, or kidneys, or liver, or nerves aren’t functioning properly; all of which are very common issues for patients with AL amyloidosis.

“So any drug is more poorly tolerated in this group of patients than it is in a population with preserved organ function.

“If you get an infection and you’ve got amyloidosis, it’s much more difficult to handle than if you’ve got a cancer but have preserved organ function,” said A/Prof. Mollee.

On top of that, bortezomib has particular toxicity on the nerves; an organ system that’s often damaged in amyloidosis to start with, said A/Prof. Mollee, so you’ve got far less reserves to deal with the side-effects of the drug.

On the bortezomib arm of the MM13 study, A/Prof. Mollee said people did get more side-effects overall, “particularly problems with peripheral neuropathy (nerve damage) which can persist, more problems with low blood counts, and heart failure in patients with cardiac involvement”.

“Patients with AL amyloidosis need to be aware of that, but while the side-effects are more in the short-term (during the treatment period), there’s a long-term payoff.”

While A/Prof. Mollee doesn’t have information on how many patients have had to go on treatment following the trial, he said, “in the majority of patients, the light chain that causes the disease will come back again at some stage, in which case they move on to the next line of treatment”.

“There are more and more options becoming available for patients whose AL amyloidosis has come back. There is no doubt that we are making real progress in this rare disease which is great news for patients,” said A/Prof. Mollee.

Associate Professor Peter Mollee discusses issues around AL amyloidosis

Associate Professor Peter Mollee discusses issues around AL amyloidosis

Associate Professor Peter Mollee is a consultant haematologist in clinical and laboratory haematology at the Princess Alexandra Hospital (Brisbane), Associate Professor with the University of Queensland Medical School, and he chairs the Scientific Advisory Committee of the Australasian Leukaemia & Lymphoma Group (ALLG).

Assoc. Professor Peter Mollee
Assoc. Professor Peter Mollee

He describes AL amyloidosis as a rare disease where plasma cells in the bone marrow make an immunoglobulin light chain, which circulates in the blood. In AL amyloidosis, that light chain misfolds and deposits as amyloid in any of the body’s tissues, except the brain. It interferes with the function of the organ/s affected, leading to organ failure and ultimately, without treatment, to death.

This condition has a poor prognosis if diagnosed late. Late diagnosis still occurs because amyloidosis is uncommon and can present in an enormous number of ways and to numerous different types of doctors and specialists. In the past, there was a lack of effective treatment options, however, treatments and outcomes are now improving rapidly.

Almost nowhere in the world* is there a drug specifically approved for AL amyloidosis and, according to Associate Professor Peter Mollee, that’s a common problem for a lot of rare diseases.  

“And the problem with amyloidosis is… it’s a rare disease,” he said.

“It’s tough to raise money and generate awareness for rare diseases. We don’t have a prime minister or film star who has it. It’s not highly visible in the media like breast cancer, heart attacks or kids with cancer. No one can spell amyloidosis, and very few people have heard of it!” explained A/Prof. Mollee.

Back in 2001, while on an overseas fellowship at the Princess Margaret Hospital in Toronto (Canada) A/Prof. Mollee was given a project, to write-up the hospital’s experience in transplanting amyloidosis patients.

“It was then that I became involved in the management of those patients and developed an interest in the disease,” he said.

Putting amyloidosis on the medical map in Australia 

When A/Prof. Mollee returned to Australia, in 2003, he found there were no amyloidosis clinics or services here, no patient information, there were a lot of problems with diagnosis management, and there had never been an Australia-wide clinical trial for the disease.

“There was no coordinated approach, so there was a gaping hole in what was required. At the time, a group of Brisbane specialists interested in amyloidosis, together with social worker Ms Pat Neely, had already identified these issues and was working towards developing solutions. I just fitted in with their plans and slowly over the years the situation has improved,” said A/Prof. Mollee.

“Initially, the only philanthropic organisation that would help us was the Leukemia Foundation in Queensland, even though a lot of amyloid is not blood cancer. They were happy to take it on and provide some support, which was invaluable.

“The Leukaemia Foundation funded the first-ever amyloidosis clinical trial in Australia in 2005, and as there was no patient information in Australia at all, they helped us with the first patient information booklet.

“Pat Neely did a research project as part of that clinical trial, interviewing patients and carers, that led to the information in that booklet.

“We specifically found out what the issues for the patients and carers were, as opposed to medical professionals just writing what they thought was important.”

Gradually multi-disciplinary amyloidosis centres were established in the state capitals – in Brisbane, Sydney and Melbourne, and more recently, Perth.

Pat Neely
Pat Neely was among those instrumental in the formation of the Australian Amyloidosis Network

The Australian Amyloidosis Network (AAN) is an affiliation of these amyloidosis centres, dedicated to the  diagnosis and management of Australian patients with all types of amyloidosis,” explained  A/Prof. Mollee who, together with Dr Simon Gibbs, Professor Graeme Stewart, Dr Fiona Kwok and Pat Neely, was instrumental in the formation of the AAN.

“The amyloidosis centres have been invaluable because, with all the organs involved with amyloidosis, you need a multiplicity of specialists besides haematologists, particularly cardiologists and renal physicians, because those organs are so often affected, and neurologists as well,” said A/Prof. Mollee.

“We’ve done a lot of work in diagnosis with pathology. That was poor in Australia, and it’s still not optimal, but is improving all the time.

“And we’ve managed to bring a number of trials to Australia, and that’s been really beneficial.

“So there’s a big – well, big for a rare disease – body of activity going on behind us.

“Overall, things are really progressing, never as quickly as you would like, but definitely better than 20 years ago.”

Accessing treatments in AL amyloidosis 

“There are a number of issues in Australia, and the main issue is that it’s often difficult to get the drugs you want,” said A/Prof. Mollee.

Despite bortezomib (Velcade®) showing improved response rates and overall survival in the ALLG MM13 trial, A/Prof. Mollee said the treatment was not yet approved for AL amyloidosis by the Therapeutics Goods Administration (TGA) or listed on the Pharmaceutical Benefits Scheme (PBS) for amyloidosis patients.

“So, we’ve got a great drug but it’s not specifically registered for patients with AL amyloidosis at this stage,” said A/Prof. Mollee.

“It’s a similar problem in most countries around the world - there is still no drug approved for amyloidosis, specifically.

In Australia, AL amyloidosis patients can only access bortezomib on the PBS if they also have a diagnosis of myeloma.

“Fortunately, a good proportion of amyloidosis patients in Australia would meet that definition of having myeloma, so they can access drugs on the PBS.”

But not all of them can though, and that’s a problem for those patients who don’t meet the definition of myeloma. It can be difficult for their doctors to get hold of bortezomib, unless they can access it through the hospitals, insurers, or by self-funding.

A/Prof. Mollee said that while there are various ways people with AL amyloidosis can access treatment, none was optimal.

“The main game in Australia, where we’ve got an egalitarian approach to healthcare, is to have bortezomib available to everyone, whether they’re rich or poor, whether they’ve got health insurance or not, no matter where they live; they should have access to this treatment.

“We need to get these agents listed, one, by the TGA to be registered for amyloidosis on the label, and two, to get them funded on the PBS.”

This applies to other drugs besides bortezomib. Many drugs that work well in myeloma also are effective in AL amyloidosis. While our access to PBS-funded drugs for myeloma is quite good, unfortunately it can be much more difficult to access such medicines for AL amyloidosis patients.

A/Prof. Mollee also noted that there is a small proportion of patients for whom it’s not appropriate to use bortezomib, “mostly because they’ve got severe nerve damage as part of their amyloid”.

“It can be dangerous to use bortezomib in such patients. That group aside, there’s also a small group of patients that would not receive initial chemotherapy but be taken straight to a transplant; that’s appropriate as well.

“Another issue in managing patients with AL amyloidosis is that, because of impaired organ function, many treatments can be difficult to tolerate. Patients often have significant heart or renal failure which can limit the type and dose of drugs that can be given. Supportive care to optimise organ function and quality of life becomes very important.”

AAN members at the AAN Amyloidosis 2019 meeting
AAN members at the AAN Amyloidosis 2019 meeting in Melbourne. In the front row, Drs Darren Lee, Peter Mollee, Prof. Angela Dispenzieri, behind, Drs Masa Lasica, Antonia Carroll, Fiona Kwok, Graeme Stewart, James Hare, Jay Baumwol, Julian Gillmore and Simon Gibbs.

Clinical trials in AL amyloidosis 

The bortezomib-based regimen (cyclophosphamide, bortezomib, dexamethasone) forms the backbone of several clinical trials that have gone forward.

One such recent trial has examined the addition of an anti-CD38 antibody that is effective in myeloma, daratumumab, to the bortezomib-based regimen. Accrual for this trial closed recently in Australia and A/Prof. Mollee said the initial results, which have just been reported in Europe, “were very promising”.

The next trial for newly diagnosed patients which is planned to come to Australia will assess a new anti-fibril antibody called CAEL-101. This new antibody binds to amyloid deposits in the tissues in order to send a signal to the immune system to remove already deposited amyloid from the organs.

The trial plans to test whether the addition of CAEL-101 to bortezomib-based chemotherapy improves the outcome of patients with AL amyloidosis involving the heart.

“Another drug combination, which we hope soon will be trialled for amyloidosis, is isatuximab (a monoclonal antibody to CD38 that’s similar to daratumumab), and pomalidomide (an immunomodulatory drug) in relapsed and refractory amyloidosis. This trial offers a new promising combination treatment to patients whose amyloidosis has come back after initial therapy.

Amyloidosis incidence and prevalence 

When asked, how many people in Australia have amyloidosis and of them, how many would have AL amyloidosis, A/Prof. Mollee’s response was, “we don’t know the exact answer”.

“We performed Australia’s first epidemiology study, which was published in 2019, using data from all the pathology laboratories in Queensland that do amyloidosis testing,” he said.

“Until then, we had no data in Australia about how common the disease was, and that’s very standard but essential information for any disease. This shows how far behind the goal posts we were on this disease – we didn’t even know how common amyloidosis was in Australia. That project took five years to complete.

“It’s quite time consuming to get all the ethics approvals and governance approvals necessary for that type of research, especially when it is mostly conducted in doctors’ spare time.”

Based on all the pathology reports that mentioned amyloidosis, the incidence of newly diagnosed amyloidosis is 12 patients per million people per year.

“Unfortunately, that study couldn’t tell us in depth information on the types of amyloid, but we looked at it indirectly and the incidence of AL amyloidosis is probably around eight per million per year. So, based on Australia’s population of 25 million, that’s about 200 new cases per year.”

And A/Prof. Mollee said there was another interesting aspect to amyloidosis.

“A complicating factor in assessing the incidence of all types of amyloidosis is that there is another type of amyloidosis, called transthyretin amyloidosis, that is currently vastly underdiagnosed and wouldn’t have been captured in our study.”

“Autopsy studies on older patients in the general population, aged 90-100, have found one-quarter of them to have amyloid deposits of transthyretin,” said A/Prof. Mollee.

“Those deposits are not enough to cause disease, such as heart failure, but these studies do show how extraordinarily common amyloid deposition is.

“That type of amyloid [transthyretin], typically causes heart problems in older men. In the past, it just hasn’t been diagnosed because doctors tended to not investigate 80-year-olds who get a bit short of breath or have a thick heart on scans. That is all changing now as effective treatments become available.”

A/Prof. Mollee said there were probably thousands of cases in Australia that are undiagnosed, and that transthyretin amyloidosis will become the most common cause of any amyloidosis by far.

“That’s a separate form of amyloidosis and a different issue from AL amyloidosis, but it makes it hard to answer the question about how common amyloidosis is.”

A/Prof. Mollee said there was no prevalence data because there’s no amyloidosis registry in Australia. Amyloidosis is an uncommon disease, but it’s not a notifiable disease like cancer, so we have no good data source to answer such questions.

Long-term survivorship

“Having said that, we’ve got a number of patients with AL amyloidosis who have been treated and their amyloid hasn’t come back. There are long-term survivors and some of them have no evidence of the disease,” said A/Prof. Mollee.

“We’ve seen patients whose organ functions returned completely to normal and, at least on the tests we’ve got available, their organs look normal.”

Read Fahimeh Taheri’s story – she’s been in remission for seven years

AL amyloidosis – blood cancer or blood disorder? 

Whether AL amyloidosis is a blood cancer or a blood disorder, A/Prof. Mollee said, “that’s a difficult question in the field of amyloidosis”.

“This partly comes back to, what is cancer? You think that is a straightforward thing, but it’s not.

“When does a disease go from a pre-cancerous position to a benign cancer, to a malignant cancer? These are concepts that are not straightforward to researchers in the field.

“And it’s very difficult to explain those concepts to the public and patients.

Most patients with AL amyloidosis have an underlying bone marrow condition called ‘MGUS’ which is a monoclonal proliferation of plasma cells and which is generally considered a premalignant condition. MGUS can progress to myeloma in a small percentage of cases.

As such, most patients with AL amyloidosis don’t have what most people think of as cancer in their bone marrow. On the other hand, most drugs on the PBS are only available for patients who have the blood cancer, myeloma. In this regard, it can be an advantage for AL amyloidosis to be considered a blood cancer because accessing treatments becomes easier if you have a diagnosis of myeloma.

“So, it’s caught up in semantics and terminology. AL amyloidosis is definitely a blood disease. It’s closely related to myeloma. Calling it a cancer causes trouble. Not calling it a cancer causes trouble. It’s hard to know what the right thing to say is,” said A/Prof. Mollee.

*  Bortezomib is approved for AL amyloidosis in New Zealand. 

Fahimeh has been in remission for seven years  

Fahimeh has been in remission for seven years  

Fahimeh Taheri’s amyloidosis was picked up very early. She can pinpoint the time when she started developing the disease. It was around February 2012, when she was 54.

Fahimeh and his family
Fahimeh Taheri, centre, with sons, Mortza and Ali, daughter, Hoda, and husband, Mohammad

She knows that because, as a registered nurse, she sometimes checked her own urine, and there was no presence of protein in the last test she did, two months before a kidney biopsy showed she had amyloid in her kidney. [Amyloid can harm the kidneys’ filtering system, causing protein to leak from your blood into your urine.]

Fahimeh, 62, of Hobart, has been in remission for seven years – since July 2013 – and believes her early diagnosis was important to achieving her disease-free outcome.

She and her family migrated to Australia from Iran in 1998, and Fahimeh, who had been a teacher, changed her career to nursing. She studied and completed her Bachelor of Nursing and had just started a new job at a hospital dialysis unit when she was diagnosed with AL amyloidosis in April 2012.

Scrubbing up for work at the dialysis unit where Fahimeh works
Scrubbing up for work at the dialysis unit where Fahimeh works

Fahimeh’s diagnosis and initial treatment 

The mother of three started feeling very tired, and by 9pm she was drowsy and sleepy, which was unusual given she was used to staying up well into the night reading. She also noticed a change in the shape of her nails.

“I was sure something was wrong with my body,” said Fahimeh, so she went to see a GP.

The doctor ordered blood tests to check her iron and asked Fahimeh for a urine sample which was tested on the spot. It showed the presence of protein.

“I told the doctor, that’s very strange. I’d never had protein in my urine,” said Fahimeh.

“Then the blood test showed no iron and I was sent to a nephrologist who did the kidney biopsy, and then I had a bone marrow biopsy as well, and that showed I had AL amyloidosis plus smouldering myeloma.”

Her initial reaction was disbelief.

“At first, I was like – it’s wrong, the tests are wrong. I have always been a healthy, happy person and very strong… what happened to me?”

“My response was – it’s a bad dream. I wanted to sleep and get up, open my eyes and say, ‘it was a bad dream’. But it wasn’t.

“My haematologist was so positive explaining the treatments, and at the same time he told me there is no cure!

“I had never heard of amyloidosis, so straight away I went to the internet and read that from diagnosis to finish is six months, maybe a year. It was so depressing.

“When I told my nephrologist this, he said not to look at the internet, and that the treatment had changed from years ago.”

Under the eyes of a nephrologist and haematologist, Fahimeh started chemotherapy in
September 2012 with a course of cyclophosphamide and dexamethasone. Next, she was treated with melphalan.

Then Fahimeh had injections to stimulate her bone marrow to produce stem cells which were then harvested, and in November she had an autologous stem cell transplant.

She went home to recover but a week after the transplant Fahimeh developed a temperature and was admitted to hospital. For the next 10 days she was confined to an isolation room and treated with blood transfusions and antibiotics.

“I was very, very sick. My platelets and haemoglobin were very low. I couldn’t even walk,” said Fahimeh.

She returned home “to a very clean and disinfected area of the house”.

“We didn’t let anybody come to our house,” said Fahimeh.

Three months later when she went to see her haematologist with her husband, Mohammad and daughter, Hoda who is a GP, Fahimeh received the shocking news. The transplant hadn’t worked.

“I burst into tears and I told my daughter, ‘the world is finished for me, that’s it’.

“But the doctor was very optimistic. There were different treatments to try that people were responding well to.”

A new treatment and remission 

Fahimeh started the new treatment on Christmas Eve – bortezomib (Velcade®) combined with cyclophosphamide and dexamethasone.

“My results showed that I had AL amyloidosis plus smouldering myeloma, so my haematologist was able to access bortezomib. At that time, it was only available for people with myeloma.”

Fahimeh was on this treatment regimen for six months, until mid-2013. During this time, she halved her work schedule to two days a week as she was exhausted and didn’t feel very well for two or three days after each treatment.

“My body responded perfectly,” said Fahimeh.

Within a year her kidney function had returned to normal, with no presence of protein in her urine, and she is thankful not to be affected by peripheral neuropathy as a side-effect, but when her hair grew back, it was extremely curly!

Fahimeh hasn’t had any further treatment for amyloidosis since but she continues to have three-monthly blood tests, at her request “because I want to make sure everything is okay”, and six-monthly appointments with her haematologist along with a urine test.

Thankful to be in remission  

She shows no evidence of either amyloidosis or smouldering myeloma being active.

“At the moment I don’t have anything,” said Fahimeh.

“I don’t know if it will come back. I try not to think about it.

“I don’t know what is going to be in the future, but every day is a bonus for me. Every day I thank god for being healed and in remission for seven years.”

There has been one not-so-small hiccup over the years – heart surgery.

In 2018, Fahimeh noticed she was short of breath when walking, which she put down to having asthma and not using her inhaler every day. Her GP referred her to a cardiologist, and she had a series of tests.

“Then the sad thing – I had to have triple bypass surgery,” said Fahimeh.

“Three months after, I went back to work and back to normal life!”

Leukaemia Foundation support 

Fahimeh received support from the Leukaemia Foundation soon after her diagnosis with amyloidosis.

“They are such a great support. I really appreciate their help, their support and everything,” she said.

“They were so nice to us. When I started treatment and twice when I was in hospital, they gave us a card, so my husband didn’t have to pay for parking. It was great he could stay with me all day. They helped with petrol vouchers too.”

The Leukaemia Foundation also connected Fahimeh with a fellow amyloidosis patient, long-term survivor and retired researcher, Carole Bartlett, who lives in Western Australia.

“She was diagnosed a year before me and is still in remission.

Fahimeh and Carole
Fahimeh Taheri with Carole Bartlett: “Carole was a great help to me, to understand I am not alone, there are other people with AL amyloidosis”

“Carole was a great help to me, to understand I am not alone, there are other people with AL amyloidosis.

“She was so positive. We are still in contact and when she came to Hobart, she came to our place for dinner.”

Fahimeh has travelled to Melbourne many times to attend the Leukaemia Foundation’s annual patient conference.

“I went with my daughter, to gather information, to find out about the new treatments. It brought me hope and happiness feeling you’re not alone.”

Life looks different to Fahimeh now 

“My life has changed. Every single day, I thank god I am still alive and it’s such a beautiful day’.

“Every morning when I get up, I go outside. I appreciate that the weather is sunny and I appreciate when it is raining.

“Life is completely different to my eyes than before. Spring is different, summer is different. It looks like I am born again.”

Helping the dialysis patients at work

Fahimeh loves her job and has no plans to retire.

“I am 62 but I feel I’m 42. I don’t think about my age. I think age is just a number,” she said.

Not only does she share her own experiences with a life-threatening disease, to help support and encourage the dialysis patients she sees every day at work, she even does a belly dance for them!

Sharing a joke or a story inevitably results in the sound of a patient’s laughter coming from any room Fahimeh is in.

“They see how positive I am, and they tell me this makes a difference and is life-changing for them.

Fahimeh’s advice to others with amyloidosis 

Firstly, unless you are accessing a recommended, trusted website, Fahimeh says, “don’t look at the internet,” because a lot of the information there is old.

“Treatment changes every day and is getting better.

“And don’t think it [an amyloidosis diagnosis] is the end of your life. It’s not. There’s lots of good treatment.

“Being positive makes a big difference. Talk to yourself. Say, ‘I am a fighter’ and ‘this is not going to kill me’.

“Being negative and down, pulls your immune system down as well. A positive attitude builds up your immune system.”

Fahimeh with her family
Fahimeh celebrating her birthday with husband, Mohammad, daughter, Hoda, elder son, Ali, and grandsons, Sami and Reza

Fahimeh also suggests having a hobby. She loves cooking and reading medical books and doing research.

“I remember, after chemotherapy, I couldn’t concentrate on reading. I’d read a page, then I had to go back and read it again. That was very frustrating.

“I tried getting up and doing a breathing exercise. After five minutes, I’d sit and read that page again. Don’t give up. It’s very important not to give up.

“Exercise is very important for everybody,” said Fahimeh who jumps on her treadmill for at least 30 minutes each day.

“And if you can’t do half an hour, break it up into 10 minutes three times a day.”

Finally, Fahimeh says living with a chronic disease or illness is a big challenge.

“It is normal to grieve and to be sad. Sometimes, I have to tell myself there are people worse than me.

“If you are positive, it brings happiness to your life.

“I try to have a very positive attitude and every day I think about the research to find better treatments for this disease and maybe one day they will find a cure.”

You stepped up for Jack and his family in their darkest hour

You stepped up for Jack and his family in their darkest hour

Jack Mcilvar in May 2020

Stem cells are primitive cells likened to ‘baby’ cells, yet to decide what they want to be when they grow up. They turn into all the different blood cells, including the cells of our immune system.

But people with blood cancer or related blood disorders often need their stem cells replaced because of their disease or toxic treatments like chemotherapy.

Most stem cells donated to Australians actually come from overseas. So, what happens when unprecedented travel restrictions during a pandemic strangle the vital supply of stem cells?

Jack Mcilvar was 22 years old when he was diagnosed with a dangerous blood disorder called aplastic anaemia. He needed a stem cell transplant this year to save his life.

His father and carer, James, recalls how his son had battled aplastic anaemia for many months, spending weeks in intensive care and coming close to death. But there was light at the end of the tunnel: a stem cell donor had been found in Europe and Jack was finally well enough for a transplant.

His new live cells were prepared for a trip to Australia, to be accompanied by a necessary human courier. Meanwhile, Jack’s doctors in Adelaide began the normal process of destroying what was left of his immune system ahead of the transplant.

But then COVID-19 began to spread. Borders closed, couriers could no longer travel, transplants were deferred. Jack’s life-giving cells remained thousands of kilometres away.

“We just couldn’t believe our luck,” James recalled.

“Our son had no immune system left and was now one of the most vulnerable people in the world.

We’d got through so much and were at last ready for a transplant, but now there was a global pandemic.

“Doctors had to stop treatment because his stem cells were no longer guaranteed to arrive.

“Our family was in complete turmoil. Who could we reach out to? It was a feeling of utter helplessness.”

Then a chance encounter in hospital. One of our team, visiting other families with blood cancer, overheard James in the patient lounge and offered to help.

Jack’s stem cells did make it through.

“We’re still not sure how it happened but Jack’s transplant arrived, we think on one of the last flights in carrying cells,” James explained.  “We were lucky. It was such an incredible relief to us.”

Jack Mcilvar after his transplant

After weeks of difficult recovery following his transplant, and against all the odds, we’re thrilled to report Jack is now on the verge of going home. Thank you so much for being there for families like Jack’s during the coronavirus pandemic.

There are lots of challenges ahead but with Jack and thousands of others like him catalysing our efforts, we’re ready to take them on together.

Thank you for keeping Gavin’s family close during a bone marrow transplant

Thank you for keeping Gavin’s family close during a bone marrow transplant

Gavin Hill outside in the garden
Gavin Hill, ALL survivor

Gavin thought leukaemia was only a children’s cancer until the day his shock diagnosis completely changed his family forever.  

 In early 2019, Gavin and his wife Jen had just returned to their home in Bundaberg after travelling around Australia.  Their beautiful daughter Dusty has just started school 

Life was good.  

“Work was going well for me, and my partner, Jen finally had the opportunity to go back to Uni,” said Gavin.   

Just like many of us might think, Gavin suspected he was simply working too hard when he became run down and struggled to shake a couple of infections 

Jen encouraged Gavin to go to the GP to get checked out.  

Before he could catch his breath or make plans with the familyGavin was flown straight to Brisbane from his family home in Bundaberg and was diagnosed with acute lymphoblastic leukaemia (ALL). 

Gavin Hill
Gavin during treatment

It didn’t really mean much to me at that stage. I knew next to nothing about it,” admits Gavin.   

This is the moment support like yours comes alive. The moment just after his wife Jen and young daughter Dusty drive hundreds of kilometres to be by Gavin’s side, not knowing how long he’d be there or just where they could stay.  

We connected with the Leukaemia Foundation and were offered a unit at the Patient Accommodation Village and lots of great information.  

“It blew me away what the foundation was providing for the people in our situation every day of the week.”  

After two full cycles of chemotherapy failed, Gavin was lucky to find a suitable bone marrow donor from the Australian Bone Marrow Donor Registry.  

“There was no option B for me – if I didn’t get the transplant, I didn’t have a hope of surviving.”  

Gavin, Jen and Dusty continued to stay at the Leukaemia Foundation Village while he completed the 100-day recovery.  

Gavin and his family recently returned home to Bundaberg and slowly but surely, they’re returning to everyday life.  

You’re there for teenagers with blood cancer through every setback and every victory

You’re there for teenagers with blood cancer through every setback and every victory

Siobhan Hoy and her mum Sally
Siobhan Hoy and her mum Sally

After suffering a devastating relapse last year, Siobhan Hoy knew her Leukaemia Foundation family would be there for her again.

Siobhan was just 14 years old when she was first diagnosed with blood cancer in 2015.

“I was a fit, healthy teenager with a few bad bruises and a couple of bloody noses,” said the now 19-year-old. “A blood test and one phone call later and my life changed completely.”

Siobhan and her family stayed at a Leukaemia Foundation Patient Accommodation Village while she received life-saving treatment.

“My first diagnosis was really hard. I didn’t want to talk to anyone and felt so embarrassed and ashamed of my illness,” remembers Siobhan.

“I wouldn’t leave the room for anything until my mum convinced me one day to go down to see the Leukaemia Foundation support staff in the office.

“I was introduced to Maryanne, a Blood Cancer Support Coordinator you help to fund, and we just clicked straight away.

“There was no shame in what I was telling her. We would talk about all my teenage issues and she would laugh and reassure me through it all.”

When Siobhan relapsed in 2019, she felt assured by her first experience with the Leukaemia Foundation, knowing she would have access to all the supportive care she needed.

“I walked in that first day and said to Maree at the front desk: ‘I’m back, buddy, let’s do this!’,” laughs Siobhan.

“Maree and my mum, Sally, also became really great friends. They would go walking and work out in the gym together.

“That was really important for my mum’s mental health as well because being a carer is not easy.”

Siobhan is grateful to you for the family she built while staying at the village and keeps in regular contact with Maree and Maryanne.

“They see you at your best and worst, experiencing every setback and every victory together.

“I consider them my lifelong friends and actually really miss them now as happy as I am to be getting on with my life.”

Siobhan is now in remission – and we miss her, too!

You helped Jenny navigate her diagnosis

You helped Jenny navigate her diagnosis

Jenny and her son
Jenny with her son, Joshua who flew in from Western Australia to Queensland to be one of her carers.

Because of kind supporters including you, Jenny von Pein connected with the vital information she needed to find her best treatment and take on her blood cancer with confidence. 

Jenny was first diagnosed with a long-developing blood cancer called chronic leukaemia after a routine blood test.

“I got in contact with Sheila, a Leukaemia Foundation Blood Cancer Coordinator,” explains the 57-year-old. “I needed to go where the most valid knowledge was and, if necessary, where the clinical trials were.

“Sheila was excellent at talking me through the terminology, what to expect with the treatment and what it means to have a chronic leukaemia.

Having to travel four hours from her hometown for treatment, Shelia also helped Jenny to access a patient travel subsidy scheme and find accommodation.

“You don’t know what you don’t know. Shelia was just excellent; she was in constant contact with me throughout the whole journey.

“She has been a real leveler, easing my anxiety over the travel to treatment and through chemotherapy.

“We’ve already started to chat about whether I’ll be well enough to go back to work.

“I now know what I’m entitled to.”

Jenny is focusing on her recovery with her medical team monitoring her condition closely with weekly blood tests.

“It’s so important to understand your illness and talk to the Leukaemia Foundation to get direction and assistance on where you’re heading next with your disease,” said Jenny.

Jenny was recently told she is blood cancer-free and is feeling grateful for all the support she received to find the right treatment pathway for her.

Long-term research breakthrough

Long-term research breakthrough

Generous support helped deliver a research breakthrough that could lead to the first drug approved for patients with the rare blood disease, AL amyloidosis.  

Back in 2011, the Leukaemia Foundation invested in an international AL amyloidosis clinical trial, headed up in Australia by Associate Professor Peter Mollee.  

Assoc. Professor Peter Mollee
Assoc. Professor Peter Mollee is a consultant haematologist in clinical and laboratory haematology at the Princess Alexandra Hospital and chairs the Scientific Advisory Committee of the Australasian Leukaemia and Lymphoma Group (ALLG).

The trial was run in Australia by the Australasian Leukaemia and Lymphoma Group (ALLG) and took place in seven hospitals around Australia and in 14 countries internationally.   

“It enabled us to have enough money to bring the international trial to Australia,” said Assoc. Professor Mollee.   

“It wouldn’t have happened without that vital support.  

“This was a trial that investigated the effectiveness of a drug called bortezomib in people with AL amyloidosis.” 

You may have heard of bortezomib because it’s highly effective at treating a related blood cancer called myeloma.  

But AL amyloidosis is quite different to myeloma. The disease sees the build-up of amyloid proteins in tissue and organs of your body and can lead to fatal organ failure.   

This year, the results of Assoc. Professor Mollee’s trial were released.   

“You’re always looking for a good response from the organs, but also that you may prolong the patient’s life,” said Assoc. Professor Mollee.   

“The trial demonstrated the significant benefit of bortezomib in improving response rates and overall survival.   

“The 30% absolute improvement in survival is a staggering result almost never seen in blood cancers.”   

The project team are now awaiting publication of their results and will then look to submit the new treatment protocol to the Therapeutic Goods Administration, the regulatory body for medicines in Australia.   

Assoc. Professor Mollee is grateful to support like yours for ensuring people living with this rare disease have access to improved treatments.  

“The Leukaemia Foundation has provided invaluable support since the beginning and none of this would have been possible without the care and dedication of the community – thank you.”  

Hope for new blood cancer therapies

Hope for new blood cancer therapies

Dr Leisl Butler
Your support is driving the work of Dr Liesl Butler (pictured) as she strives for new blood cancer discovery.

Dr Liesl Butler, a junior haematologist at Monash University, is just one of the promising young blood cancer researchers boosted by kind supporters.   

Liesl was awarded a Leukaemia Foundation of Australia/Haematology Society of Australia and New Zealand (HSANZ) PhD Scholarship in 2020. 

Your generosity will support Dr Butler over the next three years to investigate the gene mutations and biological pathways that lead to the development of chronic blood cancers called myeloproliferative neoplasms (MPNsto develop more effective targeted therapies.   

“The MPNs are a challenging disease group which cause significant health problems and limit life expectancy; new therapies are desperately needed,” explains Dr Butler.

Liesl was “thrilled” to discover that she had been offered the PhD scholarship, overcoming what she considers the biggest hurdle for researchers: funding.  

“I feel privileged to have Leukaemia Foundation supporters backing my project – I am incredibly grateful to everyone,” said Dr Butler.  

“I look forward to what I can achieve over the next three years with the assistance of the scholarship and hope to make significant advances in blood cancer research.”