MBCHB, FRACP, FRCPA, CEO, Myeloma New Zealand
Multiple myeloma is a blood cancer that resides in the bone marrow. It is called ‘multiple’ because it often affects multiple sites in the body when the condition presents. Patients may experience pain from disease in the skull, spine, pelvis, ribs, shoulders, and hips.
Patients with myeloma may also experience serious problems such as bone and kidney disease. Infections are also a feature of this condition because of a patient’s reduced immunity.
The bone lesions are called lytic because the calcium is reduced and so as well as “pathological’ fractures, sometimes patients can present with high levels of serum calcium which can lead to confusion and weakness.
Many will not have heard of myeloma but it is the second most common blood cancer in New Zealand, behind non-Hodgkins lymphoma.
I established Myeloma New Zealand in 2016 to bring more attention to this incurable disorder. There were already well-established groups overseas like Myeloma UK, Myeloma Canada and Myeloma Australia and some had been going for over 20 years. I believed New Zealand needed an advocacy group too, so I established it myself.
As a haematologist, I have always had a strong interest in the condition which tracks back to when I was working as a Renal Registrar in Christchurch in 1978. In those days, if patients presented with ‘casts’ blocking up their kidneys from light chains secreted by the plasma cells, they weren’t offered any treatment and were basically left to die.
I was disturbed by this and believed that surely more could be offered. In 1980, I went to further my training at St Vincent’s in Sydney at the first bone marrow transplant unit established in Australia. This was very exciting and new because patients with conditions such as acute myeloid leukaemia could be cured of the disease.
This was over 40 years ago and when myeloma patients presented at that time, we had to explain they might expect to survive for only two to three years. All we could offer were simple oral drugs such as melphalan or cyclophosphamide. Once a patient became drug resistant, we had little to offer.
Thankfully in 2021, we have more choice of therapies and now the majority of patients can expect to live for around 8 to 10 years. There is still a group of around 25% of people presenting with what is termed “bad risk” genetic signatures such as a double P53 mutation, and some of these people have a very bad outlook and only last 6 to 9 months. This group has what we refer to as ‘an unmet clinical need’.
In 2019, Myeloma New Zealand, with the aid of an educational grant commissioned a very detailed report called “The Burden of Multiple Myeloma” which is the most detailed analysis of any cancer ever done in New Zealand.
It looks at real world data, across a whole population, and we identified there are now over 3000 patients living with myeloma in New Zealand. Annually, there are around 400 new patients diagnosed annually. Every year, 200 people will die. The incidence is increasing and sits at 8.2 new cases per 100,000 population.
The other interesting finding was a higher rate in Maori (7.2 per 100,000) and a strikingly higher rate in Pasifika peoples. The reason for this is not clear but some work from the Middlemore group suggests that it may relate to an increased incidence of a high-risk genetic signature.
In New Zealand, patients who present with newly diagnosed myeloma will be offered a three drug combination of bortezomib, cyclophosphamide and dexamethasone which is a strong steroid. This is a reasonably effective treatment and after about four cycles, the patients deemed ‘transplant eligible’ will be offered an auto transplant to induce a deeper remission.
We set this up in Wellington in 1995 after purchasing a Kobe cell separator using private funding. Initially the machines were run by our dedicated haematology nurses but now the New Zealand Blood Service has taken over this role throughout the country.
The rationale for the auto transplant is to be able to give a high dose of that trusty old drug melphalan to confer a high tumour kill. It’s not a pleasant experience as there is gut toxicity.
In the Burden of Disease study, we found that the actual uptake of the auto-transplant option was lower than expected at a rate of only 42% up to the age of 70 years.
This was a matter of concern as high dose therapy has been shown in many studies to confer a survival benefit. There are several reasons for the reduced uptake, including co-morbidities such as heart and lung disease making the procedure too hazardous. There was also a reduced uptake in the Maori and Polynesian population which could also be partially explained by co- morbidities such as diabetes and kidney disease.
At present in New Zealand, there is significant pressure on the various bone marrow transplant units because of COVID-19, and also because of increasing referral numbers.
The unit at Auckland hospital is under stress and is having to send some cases to other DHB’s such as Wellington and Palmerston North. There is only one bone marrow transplant unit in the South Island which serves a million people.
The most frustrating part of setting up Myeloma New Zealand in 2016 has been the glacial pace of obtaining Pharmac funding, and buy in for the plethora of novel therapies that are now available for treating myeloma.
Bortezomib was funded in 2011 and was considered quite expensive but a generic is now available and so haematologists have good access to this drug for induction and also at relapse. Lenalidomide is a second generation IMID or immune modulator and has been funded for use in a third line situation for some time, and is a very useful addition for people who relapse at any time. There has been an odd situation in which the very old and toxic drug thalidomide was funded as second line, and so patients had to have this drug for some months and, if they developed neuropathy, they could go on to the superior agent Lenalidomide.
In the last six years or so there have been a host of new myeloma drugs which have been extensively trialled and have then entered mainstream therapy in the US and the UK.
I read with some degree of envy that such novel agents as carfilzomib, pomalidomide and more recently the anti CD38, monoclonal antibody, daratumumab have been funded in Australia.
We are constantly talking about how our drug budget is down at about 20 in the OECD rankings. It is then absolutely galling to hear the politicians go on about how New Zealand has a world class health service. It is just untrue. To promote that narrative is wrong.
Myeloma New Zealand appeared before a Health Select Committee in 2019 to table six novel therapies for myeloma that we thought had merit. The questioning from this committee, which was made up of both National and Labour MP’s, was of average quality. There was little medical expertise amongst them.
I waited patiently for a response which took over 12 months. The response was vanilla, so I concluded that appearing before these health committees was a waste of time.
Three clinicians in my team had taken a morning off work and we felt that it had achieved nothing.
I am somewhat heartened by my recent experience with Pharmac in that they have taken the time to drill down into what is needed to improve the current issue which is a lack of effective myeloma drugs in the relapsed/refractory space.
They have given a high priority ranking to at least two drugs. However, I know from experience not to jump for joy at the high priority rankings because sometimes these drugs can still sit unfunded for years.
We would dearly love the monoclonal antibody daratumumab to be funded. In a media release three years ago, I considered it to be a “paradigm shift” and a “gamechanger”.
Let us then hope that the beginning of 2022 will bring in some good news for our 3000 myeloma patients who tend not to complain, but are aware that New Zealand is about five years behind our Australian neighbours in access to effective medicines.