Same cancer, same drug but our access to treatment was anything but consistent
They’re three women all battling an incurable type of blood cancer, but Neda, Nikki and Shirley have felt hardship in very different ways.
Before lenalidomide was listed on the PBS, the breakthrough medicine could cost more than $194,000 for single course of treatment.
But it was an option that could help these three Australians to prolong their disease in remission.
Unable to pay the $1,000 monthly cost, Neda Masters (pictured above) from Queensland was on the verge of moving to America where she could get affordable access the drug.
The 46-year-old mum said: “We didn’t have a choice. Thankfully, my husband had a job lined up but I didn’t know what I was going to do if my family couldn’t live and work in America with me.“
Getting access to the best treatment was going to come at huge cost. We were unable to make the move to America because of the COVID-19 pandemic.
“Thankfully, the PBS listing in March means I can access the drug here at an affordable price.”
Nikki Wagner from New South Wales could only initially afford three boxes of lenalidomide, calling them her “princess pills” with each small tablet costing around $250.
Thankfully, her brother intervened and raised the $18,000 needed to start maintenance on this drug.
The 56-year-old said: “It cost about $5,000 for each box of 21 tablets. There was no way we could continue to afford them.
“However, following the initial costs outlayed, I was told I would be getting the drug on compassionate grounds through the drug company. From then on, I was able to get a box for less than $8, a life-changing discount.”
Shirley Irwin was not so lucky. The 71-year-old from Victoria used $54,000 from savings to pay for her lenalidomide which she took for eight months.
The mother-of-two said: “I’ve had some difficulty coming to terms with spending the kid’s inheritance on this medication. But they assured me, ‘we don’t want the money, we want you’.”
Daratumumab – first new myeloma agent PBAC-recommended in years
For the first time in 13 years, a new agent with a different mode of action against myeloma, daratumumab (Darzalex®), has been recommended for Pharmaceutical Benefits Scheme (PBS) reimbursement.
At the July 2020 meeting of the Pharmaceutical Benefits Advisory Committee (PBAC), daratumumab received a positive recommendation for PBS listing as a second line treatment, in combination with bortezomib (Velcade®) and dexamethasone for patients with myeloma.
In a submission to the PBAC in June 2020, the Leukaemia Foundation provided consumer comments in relation to daratumumab, based on the everyday experiences of people living with myeloma.
Peter, a myeloma patient from Western Australia, said:
“This drug is proving revolutionary in the fight against this terminal blood cancer and should be available as a permanent line of therapy [as] it can extend or save lives.”
The Leukaemia Foundation’s submission also stated that, based on the summary clinical trial information provided, “we understand daratumumab-based combinations have demonstrated efficacy in comparison to other agents tested in head-to-head relapsed or refractory myeloma trials in terms of delaying disease progression and response rates, and have also been shown to lead to molecular remissions in a greater number of patients with relapsed multiple myeloma”.
The PBAC noted in its recommendation that daratumumab monotherapy would be provided by the sponsor (Janssen), on a compassionate basis, to all eligible relapsed and/or refractory (R/R) myeloma patients who have no other PBS-funded treatment options.
A study, published in an August 2020 online issue ofJournal of Hematology & Oncology, confirmed and extended the previously known effectiveness and tolerability of daratumumab plus standard of care (bortezomib/dexamethasone) as a treatment for R/R myeloma.
The Leukaemia Foundation also made a submission to the PBAC for carfilzomib (Kyprolis®).
“We received eight responses to the request for feedback on this submission,” said Emily Forrest, the Leukaemia Foundation’s Head of Policy and Advocacy.
“Views amongst patients and their families/carers were mixed, with some concerned about the side-effects of the treatment,” said Emily.
“There is a high unmet need for new treatments for myeloma, which is currently an incurable disease which becomes progressively harder to treat after each relapse as patients become refractory to different treatments.
“We believe that the listing amendment for carfilzomib would provide clinicians with additional choice in therapy regimens for patients with refractory myeloma.”
Carfilzomib also received a positive recommendation to amendments to the dosing regimen and a change to a streamlined authority level, as sought by the sponsor (Amgen Australia).
Emily said the Leukaemia Foundation would provide submissions to the PBAC for consideration at its November 2020 meeting for elotuzumab (Emplicit®) and ixazomib (Ninlaro®) for patients with R/R myeloma.
Read Shirley Irwin’s experience with myeloma and a range of treatments including carfilzomib and daratumumab. (add link to story)
Shirley’s “so well” on her latest treatment – daratumumab
Shirley Irwin has had radiotherapy and two transplants, she’s dipped into savings to access treatment, had surgery, five different lines of treatment, and relapsed twice.
Despite all that has happened since her diagnosis with high-risk myeloma in May 2018, Shirley says, “I would say I have had a milder journey”.
Now on the monoclonal antibody, daratumumab (Darzalex®), which she started in July 2020, Shirley doesn’t know what her next option will be, but she’s prepared to personally pay for access to new therapies again, if they are not available on the Pharmaceutical Benefits Scheme (PBS). She and her husband, Bruce*, have a second property that they are prepared to sell, if necessary.
Now though, Shirley says she’s “so well, feels so good and is absolutely normal except, except weak to walk”. The daratumumab is working for her, she has no side-effects and hopes to continue this treatment on an ongoing monthly basis.
“If I could coast along like this, it would be lovely,” said Shirley.
Myeloma diagnosis and initial treatment
Myeloma came out of nowhere for Shirley, appearing as a sudden pain “that was scary”, while on holiday. She and Bruce had been away for six weeks in their caravan, had met up with friends from Perth, and were at Hall’s Gap.
They continued down the coast and up to Canberra before Shirley decided the pain was too great to go any further.
“It was leaving me uncomfortable and affecting my body. I said to Bruce, ‘I want to go home, straight home’,” said Shirley about returning to Ocean Grove, the seaside Victorian town where they have lived all their married life – 45 years.
She went to the doctor and after some tests a lesion was found on her spine and she was told she had high-risk myeloma. She was 69 and knew nothing about myeloma.
“I’d never had surgery, I’d never been ill; it was a big learning curve,” said Shirley, now 71.
Treatment began immediately. Radiotherapy on her spine, to shrink the lesion, before induction treatment with bortezomib (Velcade®) and a stem cell harvest, in preparation for an autologous stem cell transplant in October.
“I didn’t have any big hiccups, no big infections or anything like that. All was well,” she said, describing her 16 day-stint in hospital at Geelong for the transplant.
“Then I rallied around to have a luncheon for my 70th.”
That was in November, and her condition then was “only just strong enough” and she wasn’t eating much.
“I slept a lot,” she said. “I slept on the terrace in the sun, on the couch, in the caravan. I went from place to place until I got stronger. It was tiring, but it was fine. You just do what you have to do.”
Her second stem cell transplant
Shirley “took it as it came” until March (2019), when she had her second transplant. By then, her paraprotein level, which the first transplant had brought down to two, had risen to four.
“When I was first diagnosed, it was 75, which was ridiculous,” she said.
Due to her myeloma being Stage 3, she knew she was to have another ‘piggyback’ transplant.
“They had harvested enough stem cells to give me two transplants and it’s not uncommon to have two,” said Shirley.
“By the time I was due to have it, I’d forgotten the first one… but it didn’t take long to remember!
“I think I did the transplant very bravely and I rested for another couple of months after that.”
Shirley relapsed after two transplants
“Then in June, I relapsed,” said Shirley.
The myeloma had come out through the soft tissue in her left buttock.
“I thought… that’s a muscle or something… and took myself to the physiotherapist who manipulated it. Little did I realise that was the relapse.”
Shirley had expected to go through a course of treatments, starting with thalidomide, then lenalidomide, “then the next one, and the next one, and the next one… until where I am now”, she explained.
But her haematologist said, “thalidomide wouldn’t do anything, we’ll jump straight into lenalidomide with carfilzomib, but you can’t have both on the PBS”.
The decision to pay for therapy
Therefore, to have both treatments together, as a combination therapy, Shirley would have to pay for one of them.
“The lenalidomide was the cheaper of the two,” she said.
“My daughter, who was with me at the time, said, ‘well, if that’s the option, that’s what’ we’ll do’.”
But Shirley wasn’t comfortable with this decision initially.
“It meant spending our money for retirement. There wouldn’t be any cash left and there would be less when we’re gone.
“It caused me a lot of stress and took me a while to come to terms with spending the kids’ inheritance on this medication, but the family assured me, ‘we don’t want anything, … we want you’.”
Bruce had worked out that the cost of Shirley’s treatment to that point, accessed via the public medical system [the Irwins don’t have private health cover or receive a pension], was around $450,000. He convinced her and she subsequently agreed, that paying $60,000 so she could have two treatments at the same time, which her specialist said, “was the best recipe for her”, was a “pretty fair go”.
Shirley got access to the more expensive treatment, carfilzomib, through the PBS, and began the lenalidomide/carfilzomib regimen in June.
“I settled into that really well,” she said.
By October, Shirley had gathered strength. She and Bruce went on a “special family holiday” to Noosa in Queensland, along with her sister, Heather, and husband, John, daughter, Paige and her husband, Dan, and their three boys, and son, Saul.
Both Paige and Saul, live close-by to their parents.
“One’s just across the bridge at Barwon Heads and one’s round the corner,” said Shirley.
Another relapse and more treatment
For nine months, Shirley did “really well” on the combination therapy until the myeloma appeared again, in April 2020, this time in her thigh bone, causing a fracture.
“I’d relapsed again,” said Shirley.
She was told the myeloma had cloned and was no longer responding to the lenalidomide/carfilzomib. She needed a different treatment, but first she had to have more radiotherapy, and before she could have that, she needed surgery to put a pin in her right leg, from her hip to her knee, to secure the bone.
That happened within days, “which was super”, said Shirley as she couldn’t weight-bear and “it was so painful”.
“It took six weeks to get over the surgery, but then after a week or so I could have the radiotherapy. Now I’m fine in that leg.
The protocol to access daratumumab
“Then they had to start me on something else. Pomalidomide was next on the list, so I had that for a month. Even though they felt it wouldn’t do anything, you have to go through it to get the next, daratumumab.
“That’s the only time I felt anger, when the carfilzomib stopped working. It made me angry that I had to wait before I could go on the daratumumab,” said Shirley.
Scans showed spots on Shirley’s ribs and one at the top of her arm, so she had two rounds of chemotherapy to treat these, along with six cycles of daratumumab.
“I’m absolutely fine on the daratumumab,” said Shirley.
It’s a four-hour infusion every Thursday in the day ward, and she hasn’t had any of the side-effects she experienced on previous treatments – constipation, diarrhoea, tummy or bowel pain.
“On the first day my body races a bit and I get a bit tired but that’s all and of course, I’ve lost my hair again from the chemo.
“But I think I’m at the end of the line of what I can have,” said Shirley matter-of-factly about what treatment she may have next.
“I’m just going along. I feel well and I’m happy being at home with my husband.
“You know with COVID, you can’t do anything anyway, I still don’t feel I’m denied anything. This time that I have can’t be a high time because of COVID.
“I don’t walk so much anymore, well, not a long way, I haven’t got that strength, but we’ve got a light wheelchair, which I loathe, and I have been in it to go along the river.
“And we’re near the beach and it’s quite lovely and my husband likes to walk.”
Shirley loves to quilt and has sewn since her childhood. She and her sister, Heather, ran a dedicated quilt shop together for many years at Geelong, then Shirley worked at a haberdashery at Ocean Grove prior to retiring.
“I’ve got plenty here [at home] to sew. I love all my fabrics and there is so much that I want to do. I have a sewing workroom but find it very hard to go in there,” said Shirley.
After her myeloma diagnosis, Shirley couldn’t see the point, knowing the treatment “was just to keep you alive and as well as could be”.
“I did try to ignore the term, but it really has been palliative care,” she said.
“When I was diagnosed, the diagnosis was terminal. I know there’s no cure for myeloma, but I wasn’t expected to get 15 years out of it.”
After Shirley’s first relapse, she pushed for a prognosis and was told, “well, without treatment, maybe six months”.
“But it didn’t worry me, it was just a fact,” said Shirley, who is now heading towards three years down the track.
Dealing with a loss of choice in her life
“So that’s why if I went into my sewing room, it was sad,” she said, describing a sadness that was “different… not scary… not complaining… just not good”.
“I didn’t like that my choice had been taken away and it’s hard knowing that.
“At first, I thought, ‘oh, what’s the point of looking out the window? What’s the point of pulling out that weed? What’s the point of… ?
“But it’s okay. I go along with it now happily, and what will be, will be.”
“I’ve brought things out of the sewing room and have done a bit of handwork downstairs,” said Shirley.
And she’s teaching her three grandsons to sew.
“When they were little, they would sit on my knee while I sewed.
“I’ve got my eight-year-old [Finn] and six-year-old [Darcy] on the sewing machine. It’s just delightful, and they are so precise,” said Shirley about her daughter’s children.
“I put a box under the foot press for Darcy, so he can reach. His chin’s just above the machine.
“I show them where to sew and where to line it up, and they love it because they’ve got strips of fabric that become something.”
Their younger brother, Reuben, aged three, watches on.
They call her Grandma and sometimes, when Shirley is well enough, all three stay the night.
“It’s lovely. I follow them to their bedrooms and kiss them goodnight and the oldest one loves to cook. When he comes to stay, he cooks my egg and asks, “how would you like your egg this morning Grandma?”.
What will be, will be
“My thing has always been, what will be will be, so let go of the fear because someone is looking after you.
“The doctors are wonderful, the treatment you’re getting is great, and it changes if you change. You only have to let them know if you’ve got a side-effect and they can make an adjustment.
“I feel for my husband in the future, but he’s really strong and is busy doing things.
“We don’t worry about the ‘when’ or the ‘what after’.”
“We have a property down at Wye River. He was a builder and he’s nicely preoccupied with plans for that. If we do sell it, it needs to be renovated and extended, so that’s what he’s doing.”
** Shirley’s sister attended a Leukaemia Foundation Wellbeing Seminar at Geelong while Shirley was undergoing active treatment, which enabled her to be well informed about how to support Shirley during her recovery. Blood Cancer Support Coordinator, Linda Saunders, conducted a home visit to Shirley to provide one-on-one education, and visited her several times to provide support when she was an outpatient.
Research aims to improve QOL by decreasing treatment toxicity
Research aims to improve QOL by decreasing treatment toxicity
Exciting research is examining whether a new inhibitor can be used to prepare myeloma patients for bortezomib and other myeloma therapies, to increase the efficacy and potentially decrease toxicity of these therapies.
Dr Vandyke was awarded a Priority-driven Collaborative Cancer Research Scheme grant in 2019 to build on her finding that the novel N-cadherin inhibitor, LCRF-0006, can dramatically increase the antitumour efficacy of low dose bortezomib (Velcade®) treatment. This grant was co-funded by the Leukaemia Foundation, Cure Cancer Australia, and Cancer Australia.
“The project has been a long time in the making,” said Dr Vandyke.
“We’ve been working on a N-cadherin inhibitor (LCRF-0006) as a potential target for myeloma for almost 10 years.
In this project, Dr Vandyke is looking at one particular drug (bortezomib) that’s used in the majority of myeloma patients and which causes peripheral neuropathy in about a third of these patients.
“This is a really horrible side-effect affecting the nerves in the extremities. It causes pain in the hands and feet, pins and needles, numbness, and burning sensations – it’s really nasty,” she said.
“Bortezomib, as well as targeting the myeloma tumour cells, also targets some of the long nerve fibres that go into the hands and feet. It targets the long axons of these nerve fibres and causes damage.”
Dr Vandyke’s project will look to increase the way the drug is delivered intravenously from the bloodstream to the cancer cells.
“The way bortezomib moves out of the blood and into the cancer cell is limited by the blood vessels as they’ve got a strong boundary around them that stops the blood from seeping out,” said Dr Vandyke.
“We think that this N-cadherin inhibitor is just opening up that barrier a little bit to allow the drug to get through more efficiently. It’s working more on blood vessels in cancer rather than normal blood vessels.
“You’re getting more of the drug into the cancer and can decrease the amount that’s going elsewhere in the body which should decrease those side-effects.”
The research team has just published a paper in the medical journal, FASEBBioAdvances, using a pre-clinical model showing that using LCRF-0006 effectively increases the effect of the bortezomib.
“This means we can actually use a lower dose of bortezomib and therefore we hope side-effects will stop because you’ll get a better anti-cancer effect with a lower-dose drug,” said Dr Vandyke.
The next steps will be to see if the side-effects are actually decreased and to optimise the drug to be ready for human clinical trials.
“Because this drug hasn’t been used in patients before, it would have to go through Phase I trials and safety studies before going on to larger clinical trials,” said Dr Vandyke.
“Also, because we don’t have a pharmaceutical company that’s making the drug, the next big hurdle would be funding – always a challenge.”
Dr Vandyke is motivated by the people who are living with myeloma and whose quality of life could be improved significantly by her work.
“As a scientist, you can get bogged down in the finer details of what you’re doing and you forget the big picture,” she said.
“We have a lot of engagement with patient groups that come for tours and education sessions at the lab.
“While advancements in myeloma therapies have improved survival rates significantly, toxic side-effects and treatment-related quality of life (QOL) have become increasingly important factors for patients.
“Many patients won’t want to take the drug if it’s going to have such a detrimental effect on their quality of life, regardless of the long-term survival outcome.”
Dr Vandyke is also passionate about fostering the next generation of researchers.
“I really enjoy the teaching and supervision side of things. I’ve got quite a few PhD students that I co-supervise with Prof. Zannettino and I’m mentoring a couple of junior researchers as well,” she said.
“One of them, Dr Krzysztof Mrozik, has been a real driver for this project – we couldn’t have done it without him.
“It’s also great for them to be able to talk to the patients and their carers and families and see this is why we’re doing what we’re doing.”
Dr Vandyke said she was, “incredibly grateful to Leukaemia Foundation supporters for enabling me, and my team, to do the work we do”.
“They have made this project possible.”
Dr Vandyke has used this funding support to expand her research group; to hire a technical support staff member, and support the research of a junior researcher.
“These grants are given to some of the very best emerging talent from around Australia, and I am proud to be included in this group.”
Hi, I’m Neda and I’m just one of the 110,000 Australians currently living with blood cancer.
Neda, who recently stayed in one of our accommodation centres while undergoing blood cancer treatment, has a special message to share with Leukaemia Foundation supporters.
On behalf of every family battling this devastating disease – I want to say thank you and tell you about the incredibly vital role you’ve played in all our blood cancer stories this past year.
My own story started nearly a decade ago. Complications after my pregnancy resulted in abnormal bleeding, chronic pain, bad headaches and constant respiratory issues.
But I just kept being told I had severe anxiety and needed to sleep – well, of course I was anxious, I knew there was something seriously wrong with me…
Years later, finally a (brilliant) doctor asked for MY story and that’s when I was set on the right path to diagnosis.
Just days before boarding a plane to the U.S. I received the phone call;
“Mrs Masters, do not get on that plane, if you do…you might die.”
I was diagnosed with the incurable blood cancer, myeloma, and I had the worst type. I would need chemotherapy and then a tandem stem cell transplant to survive.
This is the first way you are making a difference in my life. Your donations are hard at work supporting research in early detection and prevention.
The funny thing was, I wasn’t even scared at this point. I was just relieved to know I wasn’t going crazy and I could finally start working towards a healthy life.
Myeloma is not an easy disease to diagnose either. At first I was told I had lymphoma however, the diagnosis changed to myeloma an hour before my first treatment.
Your support is giving me hope that others like me could be diagnosed sooner. Because of you, right now a Blood Cancer Taskforce is hard at work, concentrating on better diagnostics and educating more health care professionals about the disease so it can be picked up sooner.
After my diagnosis I was told I needed to be in the closest capital city as soon as possible to begin treatment.
So many questions. How were my son and husband going to cope? Where were we going to stay? How sick am I going to get?
Thankfully, in my first week in hospital I received a visit from my Leukaemia Foundation Blood Cancer Support Coordinator, Sheila.
My first ‘Angel of Mercy’, Sheila helped me navigate all the big questions. She told me what was coming, the best haematologists to seek out and all the amazing services I could access through the Leukaemia Foundation.
Because of you, in the past year other individuals and families like me have been visited by someone like Sheila over 26,542 times offering emotional support and assistance through the shock of a blood cancer diagnosis.
Then there’s the accommodation – could you imagine if my husband or mum had to pay for a hotel for weeks on end while I received treatment?
We were offered a safe and clean apartment, completely free of charge and close to the hospital, with plenty of people around to assist us with whatever we needed.
But it’s not only about the money, it’s about keeping families together – you ensured that 837 families in the past year weren’t kept apart by a diagnosis and could stay close while their loved one faced the biggest challenge of their lives.
My 15-year-old son, Sana, also wanted to be close to me while I had treatment.
Sweet Nicole from the Leukaemia Foundation, another angel you sent me, told me about the hospital high school he could attend close to our accommodation and sorted out the enrolment for me.
That really meant the world to my husband, Todd, and to me – it was so important we all stay together during these tough times.
Speaking of Todd, he’s also benefitted greatly from your kind support attending carer sessions, myeloma forums and support groups hosted by the Leukaemia Foundation.
And it doesn’t matter where you live with so many resources now available online to help people living with blood cancer and their families navigate a diagnosis. Again, this was all made possible by you.
At the time of writing this, I am recovering from my second stem cell transplant and am full of hope and strength knowing you are behind me all the way.
I am now on the drug, Revlimid® which earlier this year was listed in on the Pharmaceutical Benefits Scheme (PBS) here in Australia.
Before, I wouldn’t have been able to afford the $1000 a month it costs for the drug and my doctor said I will need it to stay in remission.
Thank you for your incredible support in getting this drug listed on the PBS, with the Leukaemia Foundation advocating for the medical needs of people like me.
You might not realise, but last year your support also helped three new medicines to be listed on the PBS, ensuring people living with blood cancer can access affordable treatment.
So that’s my story so far. Thank you for being part of it.
Davina’s selfless generosity could spark research breakthrough
Before tragically losing her life to incurable blood cancer in 2018, Davina Sickerdick made a lasting commitment to others facing this devastating disease.
Davina was diagnosed with myeloma after persistent back pain raised concern with her GP.
Myeloma, also known as multiple myeloma, affects more than 140,000 people worldwide each year – last year more than 2,000 Australians were diagnosed.
Davina’s partner of 22 years, John, remembers the relentless radiation, chemotherapy and stem cell harvesting that followed her diagnosis.
“There was never really a break with it,” said John. “When you have myeloma you are permanently on chemotherapy, meaning hospital visits every month and blood tests at least twice a week.
“She really had a terrible time with it, various complications along the way and she was allergic to some of the drugs.”
With her health getting worse and faced with the reality she may not survive, Davina decided she wanted to help others.
“Davina was that sort of person,” remembers John.
“She couldn’t do enough for anyone, and nothing was too much trouble.”
Davina always considered herself to be an ordinary person but her family and friends thought of her as extraordinary, full of life, colourful and loved anything that had a little “sparkle”.
“She was the one who took the initiative and contacted the Leukaemia Foundation to discuss the process [of leaving a Gift in Will].
“They armed us with the correct wording and made sure we were well supported to make our wishes clear and formalised.
“My Will reflects the same thing as Davina’s – I will be directing money towards helping more people battling blood cancer through the work of the Leukaemia Foundation.”
Davina’s kind gift has funded strategic research projects looking to better understand myeloma and why so many patients relapse after initial treatment.
With Davina’s gift, Professor Andrew Zannettino from the University of Adelaide has embarked on a three-year study to target certain cells which could eliminate this disease.
“The impact of this gift cannot be underestimated,” said Professor Zannettino.
“Davina’s generosity means we can look for new ways of beating blood cancer and hopefully ensure no one else has to experience the same struggle.”
John is looking forward to seeing real outcomes for families battling blood cancer and encourages others to consider updating their Will.
“I just hope more research can be done, and not just in myeloma, I know there are many other blood cancers needing the same type of support,” said John.
“I feel very proud and happy to think that her gift could spark a real breakthrough, we always spoke about making a difference with our estates and I hope this can make a really positive change for society.”
Darwin mother-of-two Jeanette Kingston, 41, never takes a single day for granted.
When she was diagnosed with a blood cancer called multiple myeloma on August 14, 2018 she thought death was knocking at her door. With two young children, Brayden, nine, and seven-year-old Tahlia, Jeanette made up her mind to fight for her life.
With husband Matthew by her side, she won the first round, taking on chemotherapy at the Darwin Hospital before being sent to Adelaide for an autologous stem cell transplant. Right from the start she knew she’d be faced with a second with specialists advising back to back transplants would give her the best chance of a cancer free future.
Stem cell transplant
“The biggest thing I have learned from it all is that before cancer, I just took life for granted,” Jeanette said.
“But I quickly learned tomorrow isn’t promised. The biggest blessing is now – I don’t take anything for granted. I have young kids, I want to be around as long as I can.”
Jeanette first began experiencing flu like symptoms after an overseas trip home to Denmark. She thought she had picked up the typical bug on the plane. But – days later she still wasn’t feeling better. In fact her symptoms were getting worse. She went to the doctor but at first, they didn’t suspect anything too menacing.
Cancer blood test
When she woke with her heart racing a few nights later – once again a doctor’s visit didn’t awaken any suspicions. Her blood counts were a bit low. The ECG was normal. But a few days later, Jeanette could barely get out of bed. On August 7 she was taken to hospital, and blood tests revealed she had “some kind of cancer”.
“The doctor explained my chances were a bit like rolling a six-sided dice – five out of the six sides would mean I had some kind of cancer.” The sixth option was that it wasn’t cancer but some kind of false indication. “Unfortunately I didn’t roll a six,” Jeanette said. “I was diagnosed with myeloma on August 14.”
Like many people, she first thought her diagnosis was melanoma. She had never heard of myeloma. She then undertook a treatment regime of tablets and intravenous chemotherapy, but was told her best chance of long term survival were back to back, autologous stem cell transplants (the second to be undertaken in May 2019).
Jeanette explains her cancer diagnosis has changed her perspective on life. She went to a psychologist to help her gain a more positive mindset. In fact, the whole family went to see a psychologist. She said this was an important part of her journey. Having a strong mindset helped her to cope with the uncertainty of the diagnosis, and to realise she could beat the disease.
“It gave me a kick in the butt,” she said. “We live life a little differently. We live a different type of ‘normal’, but we know I am lucky to still be here to live it.”
“That’s been the biggest blessing. Now, we don’t take anything for granted. We live in the moment and enjoy what we’ve got.”
CAR-T cell therapy
Jeanette and her family are busy making plans for the future and ticking things off the bucket list. They are hoping to have a family holiday in Queensland and Jeanette is hoping to rejoin her friends for her annual Bali escape in December – which she missed in 2018 due to her diagnosis.
She is interested in the breakthroughs happening in blood cancer treatment including immunology and CAR-T cell therapy and is optimistic about her future should she relapse at any time – knowing that medicine is progressing and is on her side.
First ‘next generation’ genetic test available in six months
Australian patient outcomes in the rapidly changing fields of genetic testing and precision medicine, where a therapy targets a particular genetic mutation, has been accelerated by Leukaemia Foundation funding.
Within six months, an accredited diagnostic gene test, that sequences 38 genes at the same time, will be available across Australia for people with a range of different myeloid neoplasms.
And this time next year, clinicians also will have access to a second test, for myeloma, with a third cutting-edge test for lymphoid malignancies well into development at that stage.
Each test will use a patient’s bone marrow sample to screen a disease-specific panel of gene mutations for diagnostic purposes and to track treatment.
The single test for the myeloma gene panel will test 15 genes including deletion of TP53 – the major prognostic indicator – and mutations that predict response to standard of care immunomodulatory agents, as well as recently identified mutations for which targeted therapies are available.
“We’ve designed what we think is the next generation of genetic tests and they can be updated as genetic discoveries continue,” said Dr Anna Brown, Head, Molecular Oncology in the Department of Genetics and Molecular Pathology at SA Pathology (Adelaide).
She is on the SA Genomics Haematology Malignancies Node development team which recently received a $200,000 grant from the Leukaemia Foundation; an investment that enables the appointment of a medical scientist and bioinformatician for two years.
“Having these dedicated staff members will accelerate our progress and take these tests all the way from a research finding and concept, through development and the rigorous NATA* accreditation pipeline, to a standard of care diagnostic test,” said Dr Brown.
The test results will be delivered within a week to clinicians along with clinical guidelines for target therapies, to help them choose the best therapy in the care of their patients.
“Our ability to sequence and understand the mutations in large numbers of genes, using current technology, has revolutionised blood cancer diagnosis,” said Dr Brown.
“We can see things in tumours we couldn’t see even a few years ago.”
Treatments can be designed that target a particular aspect of a cancer.
Dr Brown said researchers are identifying treatments that will attack or target a specific type of gene mutation and every targeted therapy will be different, depending on the mutation.
“The same gene mutations occur in a range of different types of cancer and rather than separating cancers into different types, based on the organ they affect, we can separate out cancers using a genetic classification based on the mutations they have.
“That means the way we treat cancer can be approached differently and precision medicine is changing the way we do clinical trials,” said Dr Brown.
“For example, recent studies have shown gene mutations in myeloma that are the same as gene mutations in leukaemias and solid tumours, like melanoma.
“As they all have the mutation that matches the therapy, you can trial it.
“These are called basket trials, where entry for a trial is based on a genetic test that shows you have the mutation that matches the therapy.
“It’s the beginning of this still, but groups in the U.S. have been getting big numbers [on basket trials] and are showing that the targeted therapies give you a better outcome than whatever the standard of care is,” explained Dr Brown.
“Our group is focused on setting up systems where people can access those new cutting-edge targeted therapies though a clinical trial and one of our jobs now is making sure we get clinical trials coming to Australia.”
Precision medicine also provides more treatment options, especially in the relapsed setting, where the identification of mutations could mean there are potentially more therapies to try.
“Patients who progress on therapy have the poorest outcomes,” said Dr Brown.
“A lot of trials are looking at this group of patients and are finding good responses from those on new targeted therapies, which is really exciting.”
“And another aspect of precision oncology is being more precise in knowing who is and who isn’t going to respond to a particular therapy.
“We’re really grateful to the Leukaemia Foundation for partnering with us and the timing is really good,” said Dr Brown.
“The extra resources will spur on a lot of extra activity we wouldn’t have been able to do it without them.
“This grant is covering more than just developing the test technology. It’s developing the entire pipeline and having dedicated staff members to do this work for the next couple of years will accelerate our progress.
“I think we will reach a stage, and I don’t know how far away that is… not too far… where everybody will have genetic testing of their tumour,” said Dr Brown.
* National Association of Testing Authorities (Australia).
This article was published in the April 2019 issue of Myeloma News.
The tale of ‘the terrible twosome’ #kickingmyelomasbutt
Their nicknames for each other are Curly and Spikey. They’re also known as the ‘terrible twosome’ and, while they’ve walked the same path since their diagnoses with myeloma, they’ve each had vastly different experiences.
They are one year apart in age, 53 and 54, both are married and have only children – daughters.
One is a theatre nurse at a hospital, the other is a carer at a nursing home. They live in Geelong.
Both were fit, well and asymptomatic until a sudden pain event struck out of the blue. This led to medical appointments, tests and finding out they had blood cancer within two months of each other.
Neither of them had ever heard of myeloma.
They have the same haematologist, both had the VCD* chemotherapy regimen, and their stem cell transplants were scheduled for May 2018.
As neither of them had great veins, they had a vascath [a catheter similar to that used for dialysis] so they could be hooked up to the apheresis unit for their stem cell harvests.
One breezed through her stem cell transplant, describing it as “a piece of cake”. For the other, “everything that could go wrong, did”.
Both achieved a remission, went back to work, and got on with life.
After their transplants, they went on maintenance thalidomide. One continues to take the drug and tolerates it, no worries.
The other took thalidomide for two weeks and got so sick she thought she was going to die, so she’s had no maintenance treatment. She found out the day before speaking to Myeloma News that she may be relapsing and potentially faces a second transplant.
How Kellie met Rita
This story is about Rita Mauri and Kellie Devlin. They’ve become the best of friends since meeting at the Leukaemia Foundation’s Barwon Blood Cancer Support Group at Geelong in April 2018 and have provided support and encouragement to each other since.
“I’m thankful that we’ve got each other,” said Rita, who was at the support group with her daughter, Laura, now aged 26.
“We were in a circle. Everyone was talking about their illness, where they were up to, and how they were feeling,” said Rita.
“Laura spoke about my illness for me, because I was too emotional. I was crying all the time.
“I was frightened of everything, the unknown… what’s going to happen to me, am I going to live or am I going to die.
“I wasn’t going to go. Linda (Saunders, the Blood Cancer Support Coordinator for Barwon South West Region) said come along, because I was crying on the phone with her for a long time. I said okay and I went along and thank god that I did.
“We got talking,” said Rita about meeting Kellie that day.
“Rita was walking with a cane and was really not in a good place,” said Kellie.
“When she asked what I was there for, I said, ‘I’ve got myeloma and I have to have a transplant’. She said, ‘I do too, but I’m not going to have a transplant. I’m too frightened to have it’.
“She was very distressed and polarised with fear, which is understandable. Having a transplant is big and the reality is you may not come home.
“I basically said to her, ‘listen, you’re walking with a stick, you’re hunched over like an old woman, you’re 52 years old. You’re probably stuffed if you don’t have it, you might be stuffed if you do, so it’s probably the lesser evil.
“I said, ‘come on, you’ll be alright. We’ll be in there together, it’ll be fine’,” said Kellie.
“Looking back on it now, I’m very fortunate that Kellie was there with me and a few weeks ahead of me,” said Rita.
“Seeing her go through it put me at ease a little bit and I thought, ‘it’s not that bad after all’.
“Seeing how she coped with it so well, and thinking hopefully, I’ll do the same thing as she did.
“Mine didn’t turn out that well. I had all these hurdles to go over but I’m still here to tell the tale,” said Rita.
Kellie “had no symptoms and was perfectly well” when she was diagnosed with kappa light chain myeloma on 16 November 2017. She had her transplant six months later, on May 9.
Her myeloma journey began when she got the “most horrific pain” in her leg while getting ready for work one morning.
“Then I lost the feeling in my leg and thought, ‘I’m having a stroke’. It turned out to be a big lesion and my femur was about to snap,” said Kellie.
All her bloods were normal, but a bone marrow biopsy confirmed she had myeloma.
“I only had about 25% infiltration in the marrow which, considering some people have 100%, was pretty good. I was standard risk which was good. I was well which again was good.”
After four cycles of VCD, Kellie “breezed through the transplant”.
“I was out in 15 days, didn’t vomit, nothing. It was a piece of cake really, and I went back to work, and everything has been cruisey.”
Rita was diagnosed on 10 January 2018 and her story began in early-December 2017. She thought she had gastro and had hurt her back vomiting. She went to an osteopath who put it down to a pinched nerve.
“I persevered for a couple of weeks. Then the pain was so severe I ended up going to the emergency department on Christmas Day,” said Rita.
After a two-week stint in hospital being treated for a renal infection, she went back to the ED on January 10 because “the pain was still so so bad”.
She had more tests, including a light chain test that she didn’t know about.
When Rita was told she had myeloma, she was more worried about the pain in her back.
“The pain got to me more than the diagnosis,” said Rita.
“The pain was so severe I didn’t comprehend what was going on. I was in la la land. Everything was a blur. I wasn’t worried about the myeloma at that time.”
She had three fractures in her spine and her light chain reading at 20,000 “was the highest they’d ever seen, she said.
From January to April 2017, Rita was in and out of hospital for her treatment. In May, a blood clot in her neck set her transplant back a week, and when her stem cell harvest produced only 1000 stem cells, it was further delayed.
To ‘float’ her stem cells for a second harvest, Rita was given “an injection which was very expensive – $7000”, which resulted in a bounty of 16,700 stem cells.
She had 6000 of them infused during her transplant on 5 June 2018, and the rest are in storage.
Straight after her transplant, Rita got colitis and once that was treated she spent another 2½ weeks in rehab.
“I eventually came home in August. It took me a while to get back on my feet. I lost my mobility and had the shakes. I didn’t drive until November and went back to work in February 2019,” said Rita.
“Kellie was in and out in 2½ weeks for her transplant but I was in hospital for 2½ months. It was bizarre.
“And that’s why we laugh together sometimes – we can’t believe that I was so sick, and she got out of it so lightly.”
When Rita was in rehab regaining her strength, Kellie would visit her and that’s when they gave each other their nicknames.
“My hair came back crazy curly and hers came back all spikey; we were Curly and Spikey,” said Kellie.
“And if we’re at the hospital together, the nurses laugh and say, ‘oh, here they are… the terrible twosome’. We’re a bit of a celebrity in a crazy sort of way,” said Kellie.
Rita described them both as “very similar in character – she’s the same daggy as I am”.
“We get on really well. We go out for coffees and we message each other. We’d like to catch up more often,” said Rita.
“Kellie’s very busy. She has an 11-year old daughter and takes her to basketball, swimming and dancing, and she works a couple of days a week.
“We often go to the doctor together. We have a coffee and talk about the situation we are in,” said Rita.
“It’s amazing to have this person there with you, doing the same thing as what you are, and knowing that she knows what you are going through.
“It’s just the best thing that has ever happened to me,” said Rita.
“We’re walking the same path. We understand each other,” said Kellie.
“I can say, ‘I’m anxious’ and she knows what that means and how it feels. If I say, ‘I feel fantastic’, she knows what that means and how that feels as well, and vice versa.
“I think that’s the common thread with patients with blood cancers if you have a blood buddy. They get what you’re saying when other people don’t,” said Kellie.
Leukaemia Foundation support
Kellie said the Leukaemia Foundation “does blood cancer really well”.
“The support and the information and everything offered is phenomenal.
“And Linda, honestly, she’s an angel. She’s very sympathetic and empathetic and very kind and helpful,” said Kellie.
Where they are at now
“I’m on thalidomide at the moment – it keeps me in remission,” said Rita.
“I take one day at a time and enjoy the moment.”
“Rita’s gone back to work. She looks amazing and she feels amazing. And I feel amazing, but my bones are clearly telling another story,” said Kellie about the medical results she received in February.
“Rita came with me to the haematologist. My light chains had jumped. When I was told I had tears in my eyes and Rita had tears in her eyes, and it was like ‘golly, here we go’.
“My story now is perhaps going to take a different road, but I’ve got the hashtag, #kickingmyelomasbutt. You can’t focus on the ‘what wills’ and ‘maybes’, just focus on life and keep going.
“I don’t want to sound blasé, and this is going to sound absolutely crazy, but I just didn’t feel death in me [when Kellie was diagnosed] and I just knew… ‘it’s not my time, so bring it on and let me get back to work, and get back with my family, and back to life’.
“And I feel like that now. This is a bump in the road that I’m not particularly happy about but let’s just get it over with, so I can get back on with my life,” said Kellie.
* VCD or CyBorD: bortezomib (Velcade®), cyclophosphamide and dexamethasone.
When this article was published, Kellie had started a second course of treatment– four cycles of lenalidomide (Revlimid®) to be followed by a stem cell transplant, earmarked for late-July 2020.
Novel combination therapy now available for myeloma patients through PBS
Wednesday June 3, 2020
The Leukaemia Foundation has welcomed news that a new novel combination therapy option will now be available to more myeloma patients through the Pharmaceutical Benefits Scheme (PBS).
Federal Minister for Health, The Hon. Greg Hunt MP, has announced an expansion to the listing of lenalidomide (Revlimid®) on the PBS Highly Specialised Drugs Program to allow use in combination with bortezomib (Velcade®) and dexamethasone for newly-diagnosed multiple myeloma patients.
Minister Hunt said it is estimated up to 2,300 patients may benefit from this listing each year.
Lenalidomide is an oral immune-modulating medicine that helps a patient’s immune system recognise and destroy myeloma cells, as well as preventing new cancer growth.
This announcement follows news on April 1 that the medicine would be made available through the PBS to newly-diagnosed myeloma patients receiving maintenance treatment who have already undergone an autologous stem cell transplant.
Leukaemia Foundation General Manager of People Living with Blood Cancer Kathryn Huntley said the Leukaemia Foundation had advocated for both PBS listings, with lenalidomide being one of six myeloma medicines the organisation has actively advocated for increased access to in the past year.
Ms Huntley said she understood the combination is the first novel triplet therapy reimbursed in Australia for these patients, and a highly effective treatment option with generally manageable side effects.
“The Leukaemia Foundation is grateful this triplet combination therapy will now be a more readily accessible and affordable treatment option for Australians living with multiple myeloma who need it most,” she said.
“There is a high unmet need for more treatment options for people living with myeloma and the Leukaemia Foundation’s priority is to ensure that all Australians living with blood cancer have access to the best therapies and treatments available.
“New, improved and more timely treatment options like these that can reduce toxicity and side effects, improve survival outcomes and a patient’s quality of life are always welcome.
“This announcement is another step in improving the lives of myeloma patients, and we look forward to seeing further progress to improve survival and quality of life for all Australians living with this blood cancer into the future.”
Around 18,000 Australians are currently living with myeloma, also known as multiple myeloma. It is a complex and relatively rare blood cancer affecting the body’s plasma cells, which produce antibodies. Myeloma develops when plasma cells undergo a cancerous change and multiply at an increasing rate, taking over the bone marrow. Patients experience weaker bones which are more prone to breaking, as well as bone pain, kidney damage, frequent infections, anaemia and increased bleeding and bruising.
Currently, 2,074 people are diagnosed with myeloma each year, however it is expected 4,952 people will be diagnosed with this type of blood cancer in 2035. Sadly, 1,098 Australians already lose their life to myeloma each year, and this figure is projected to increase to 3,037 people by 2035.
“While treatment options and survival rates for some blood cancers are improving, the sad reality is that myeloma is not curable, and the impact of a diagnosis of myeloma on peoples’ lives and the ongoing affect to their health remains severe,” Ms Huntley said.
“That is why the Leukaemia Foundation is committed to empowering Australians living with myeloma with access to the best information, support, treatment and care so they can manage its impacts and live well with their blood cancer.”
Late last year the Leukaemia Foundation released the State of the Nation: Blood Cancer in Australia report, which led to Minister Hunt supporting the Leukaemia Foundation to establish a Blood Cancer Taskforce. The Taskforce is now working towards delivering Australia’s first National Strategic Action Plan for Blood Cancer, bolstering the Leukaemia Foundation’s goal to see zero lives lost from blood cancer by 2035.
The Blood Cancer Taskforce unites Australia’s leading haematologists, researchers, patients and members of the blood cancer ecosystem for the first time to work with the Leukaemia Foundation to develop the National Action Plan, which will provide a blueprint to tackle key issues facing people affected by blood cancer, today and into the future.
Ms Huntley said the formation of the Taskforce and development of the National Action Plan together mark a major milestone for the blood cancer community and will set the national agenda around blood cancer for many years to come. The National Action Plan is due for release later this year.
The Leukaemia Foundation provides free practical, emotional and educational support to Australians diagnosed with a blood cancer including myeloma. The Leukaemia Foundation produces a series of disease specific newsletters including Myeloma News, and invites all Australians living with the disease to subscribe to ongoing information here.