Clinical trials are the engine of cancer research. Virtually every cancer treatment available to people with cancer today is the direct result of a clinical trial. People with cancer who make an informed decision to participate in a clinical trial provide the gift of information that has helped scientists and doctors develop new ways to slow, halt and prevent cancer.
The following stories highlight the personal experiences of some of our current and former consumer representatives who have participated in a clinical trial. Each story gives insight as to what their lives have been since completing active treatment and what survivorship means to them. We hope these offer encouragement and support to help patients and their families suffering from blood cancers.
Acute Myeloid Leukaemia survivor
“The drugs and protocols offered to patients during clinical trials come at no cost and participation in trials for some patients may give them opportunities and hope, that may not have been accessible otherwise. Participation in a Clinical Trial also means regular and careful attention from Health Professionals in addition to the care offered by your own Specialist.”
Travelling through the UK and Europe in November 2006, it was cold as you would expect that time of the year and I picked up a respiratory infection, that to say the least, was persistent. After returning home and multiple packets of antibiotics, which seemed only to take the edge of the infection, a blood test revealed some abnormalities.
I was diagnosed with Acute Myeloid Leukaemia (AML) in the week before Christmas 2006; as a fit and relatively healthy individual this came as a shock. I failed induction chemotherapy and progressed to an Allogenic Bone Marrow Transplant on 5 April 2007 with my brother, my only sibling, as my donor.
Pulmonary Graft versus Host Disease has diminished my lung capacity significantly; long term steroids depleted my muscle tone developed previously from being a daily walker. Other things changed in my life, my ability to work full-time and fatigue like l had never experienced, to mention a few.
Today my efforts are directed to returning to my previous level of fitness through walking and gym, working part-time and most satisfyingly using my Blood Cancer experience in my role as a Consumer Representative to ALLG.
In addition to activities with ALLG, I also work with Cancer Australia, The Leukaemia Foundation, The Alfred Hospital and the Walter & Eliza Hall Institute as a Consumer Representative.
I find it most satisfyingly using my blood cancer experience in my role as a Consumer Representative to the ALLG. The contributions I make to the ALLG are many and varied and are done in the hope that, through my experience, patients diagnosed with a blood cancer travel that well-worn path just a little bit easier.
As a result of my employment l became familiar with reviewing and understanding clinical papers, trials and studies. I have participated in many clinical trials as a patient through my treatment for AML and understand the processes and follow-up required from patients.
The benefits of participating in Clinical Trials are many and varied. Importantly, often participation in trials allows patients access to drugs used in combination or prior to their general availability. The drugs and protocols offered to patients during clinical trials come at no cost.
Participation in trials may give some patients opportunities and hope, that may not have been accessible otherwise. It also means regular and careful attention from health professionals associated with the trials, in addition to the care offered by a patient’s own specialist team. Contributing to research today in this way, may also mean saving lives in the future and giving hope to other patients.
All of this would not have been possible without the care I received from my own specialists, family and friends whose unflinching support during this difficult time, never wavered.
Myeloproliferative Neoplasm survivor
“As a passionate advocate for people living with leukaemia, I know serving as a consumer representative to assist in providing patients with opportunities to participate in clinical trials ultimately helps improve access to new medications for others afflicted with blood cancers”.
In 2008 after years of seemingly unrelated symptoms including headaches with visual disturbance (which I assumed were migraines), sore hands and mouth, abdominal pains and bone pains, I was eventually diagnosed with essential thrombocythaemia, a subtype of the rare chronic leukaemia myeloproliferative neoplasm (MPN). A bone marrow biopsy confirmed MPN because I was positive for the JAK2V617F mutation and had bone marrow changes characteristic of ET, in addition to a high platelet count. Initially, I was treated only with low dose Aspirin, to prevent blood clots. Then in 2010, my disease progressed to polycythaemia vera (PV) and I had to decide between the two available treatment options, the immune modulator Roferon (the original form of Interferon Alfa-2A) or the chemotherapeutic agent Hydroxurea (HU or Hydroxycarbamide).
At the time of my diagnosis, information on MPN was scarce in Australia and online. After learning of a biennial Doctor/Patient MPN conference held at the Mayo Clinic in Scottsdale Arizona, I travelled to the US in 2011 and attended this conference to learn more about international MPN research, to assist my treatment decisions.
At the 2011 Mayo Clinic MPN conference I learned about a clinical trial for PV conducted in Paris using Pegasys, the slow release, better tolerated formulation of Interferon Alfa-2A. Data from this study showed Pegasys was effective in controlling blood counts and improving bone marrow status in PV, and had lower toxicity than the original form of interferon Alfa-2A, Roferon.
I decided to start interferon (Roferon) rather than HU, because research showed interferon is capable of inducing haematological and molecular remission and improving long term outcomes in MPN. Data presented at the 2011 Mayo Clinic conference showed long term treatment of HU is associated with an increased risk of progression to acute leukaemia, so I was reluctant to take HU long term. In 2011, however only the original form of Interferon Alfa-2A, Roferon was available on the PBS for treatment of MPN. Pegasys, (the better tolerated form of interferon) was only accessible to MPN patients ‘off label’ at a cost of about $30,000 per year, therefore it was unaffordable for most MPN patients.
After commencing Roferon treatment in 2011, I suffered all the typical toxicities associated with interferon – ‘flu-like symptoms, fatigue, hair loss, lowered mood etc. I reported the adverse effects I experienced on Roferon treatment to Roche (the manufacturer of Roferon and Pegasys), and enquired about the company’s plans to apply for Pharmaceutical Benefits Scheme (PBS) listing of Pegasys for MPN. Roche had no plans to seek PBS listing of Pegasys for MPN at that time and had not applied for Therapeutic Good Administration (TGA) listing for Pegasys treatment in MPN. (TGA approval of a drug for treatment of a specific disease is usually required before a PBS approval can proceed.). Roche however, offered me compassionate access to Pegasys. After switching from Roferon to Pegasys the adverse effects I had suffered on Roferon, quickly diminished. Treatment with Pegasys enabled me to get on with my busy home life as a mother of teenaged children and my professional life as a dietitian working in Community Health, with improved quality of life and minimal interferon side effects.
From 2011 until 2018, I advocated for Pegasys to be included on the PBS for Australian MPN patients by regularly contacting Roche, my Federal Member of Parliament, and the Australian Government. Over this time, I updated Roche on my positive response to (low dose) Pegasys, in terms of disease control and tolerability. In 2016, I met with Prof. Andrew Wilson, Chair of the Pharmaceutical Benefits Advisory Committee (PBAC) and discussed the issue of PBS access to Pegasys for people with MPN. After this meeting, I wrote a submission directly to Prof. Wilson, requesting inclusion of Pegasys on the PBS for treatment of MPN. I included information on my personal experience of treatment with both Roferon and Pegasys and references to published scientific studies showing safety and efficacy of interferon for treatment of MPN. My submission was accepted for consideration by the PBAC in early 2017. On 1 August 2018, Pegasys was finally listed on the PBS for treatment of MPN, enabling hundreds of MPN patients around Australia to have affordable access to this less toxic form of interferon. In 2018, Dr Cecily Forcyth asked me to be a coauthor with seven Australian haematologists of an article titled ‘Recommendations for the use of pegylated interferon in the treatment of classical myeloproliferative neoplasms’. This article was published in the Internal Medicine Journal in Nov 2018.
After several years on a low dose of Pegasys my blood counts are stable and within normal ranges and I do not require venesection to control my polycythaemia vera. A recent bone marrow biopsy (BMB) showed regression of fibrosis compared with previous BMBs, thanks to Pegasys treatment!
Since my diagnosis I have volunteered with the Leukaemia Foundation as a member of the MPN National Working Group and have presented at the two of the Foundation’s patient conferences, in my professional capacity as a dietitian. I regularly write articles for the Leukaemia Foundation’s newsletter, MPN News and have been a guest speaker on the Foundation’s MPN patient telephone forums.
In 2016 I became a member of the MPN Alliance Australia (MPN AA), a group of MPN patient volunteers affiliated with the Leukaemia Foundation, who advocate for people with MPN and raise funds for MPN research in Australia. My fellow MPN AA members and I co-hosted the first ever Australian/International MPN doctor-patient conference in Melbourne, in April 2017, together with the US-based MPN Advocacy & Education International (MPN A&EI). This conference included presentations from both Australian and US-based haematologists with expertise in MPN, and over 150 MPN patients from around Australia and New Zealand attended.
In 2016 I joined the ALLG as a consumer representative, until 2021. As a passionate advocate for people living with leukaemia, I look know serving as a consumer representative, to assist in providing patients with opportunities to participate in clinical trials, ultimately helps improve access to new medications for people afflicted with blood cancers.
Mantle Cell Lymphoma survivor
“Life is good, and I am living it, but now I feel like I need to give something back. I’ve been doing some fundraising and a little public speaking, which has led me into ALLG as a Consumer Representative. I am hoping I can do some more good work and help others to get through it a little easier than I did. Clinical trials are the only way we get new and better treatments. That’s what the ALLG is all about.”
In February of 2010 I was snow skiing in Japan, I had lost a bit of weight before the trip and was feeling fatigued a lot but like most men blew it off and didn’t go to the Doctor when I should have.
On the last day of our trip I had a fairly decent stack on a steep black run and managed, I think, 2 or 3 cartwheels beating myself up pretty good. Back home and the bruising was starting to go away but I had a severe pain in my stomach which was getting worse, I finally made an appointment with my GP for the following week but never got there as the pain become so bad I went to the Emergency room. White cell count was through the roof and the pain was found to be from a very enlarged spleen.
Initially I was told it was Chronic Lymphatic Leukaemia (CLL) but after more tests a confirmed diagnosis of Mantle Cell Lymphoma (MCL), stage 4, which was the scariest news of my 44 years.
I had no idea what Lymphoma was, much less this rare sub-type and of course Dr Google gave me the worst prognosis possible. I went straight into Nordic Protocol chemotherapy for 6 months of hell before getting my first remission. At first remission we harvested my stem cells in case a future Autologous Stem Cell Transplant (SCT) was required.
In remission I tried to go about my life, but MCL is a nasty one that is known to relapse, and sure enough in July 2011 I did relapse. Straight back into Chemo hell and my haematologist gave me the worst news after 2 rounds – the disease was becoming resistant to the treatment and it was recovering between chemo rounds quicker than I was. Time for the heavy artillery and I underwent some really horrible treatment, I spent long periods in hospital much of which I can’t remember but I am told I just laid there moaning a lot, I lost a third of my body weight but eventually achieved a second remission and had the Autologous SCT. I got out of hospital just in time for Christmas 2011.
At my monthly checkup in May 2012 I got the best news ever, a matching bone marrow donor had been identified on the international donor registry and given my resistance to treatment it was strongly recommended I have the transplant without delay. I had my Allogenic SCT on Friday the 13th of July 2012.
When I had my 50th Birthday, it was a milestone that if Dr Google was to be believed I should never have reached. July  marked the 10th anniversary of my life-saving bone marrow transplant. It was my only chance after the initial treatment had failed.
Life is good now, and I am living it. When I returned from another skiing trip in Japan, I had completed that steep black run again without a fall.
I now also have another wonderful annual event to celebrate – my wedding to my love Tracy that was in March .
Now I feel like I need to give something back. I’ve been doing some fundraising, and a little public speaking for Lymphoma Australia, which has led me into ALLG where I am hoping I can do some more good work and help others get through it a little easier than I did.
Acute Myeloid Leukaemia survivor
“If I had acute myeloid leukaemia (AML) 30 years earlier it would have been a death sentence, but research has made survival not only possible but has improved survivors’ quality of life. Hopefully in another 30 years’ time a blood cancer diagnosis will just be a minor inconvenience.”
In 2005 I was diagnosed with acute myeloid leukaemia and between chemotherapy and a stem cell transplant I spent five long, hard months in hospital. It was an overwhelming time physically and emotionally and I was extremely happy to finally come back home to my husband and young children.
My hope is that the journey will be easier for each person diagnosed with blood cancer. That’s why research is so important. If I have had AML 30 years earlier it would have been a death sentence, but research has made survival not only possible but has improved survivors’ quality of life. Hopefully in another 30 years’ time a blood cancer diagnosis will just be a minor inconvenience.
Mantle Cell Lymphoma survivor
“Now 10 years on from stem cell transplantation, with thanks to medical research, new drugs and techniques I have once again reached a point where my life is fulfilling. I have a whole new outlook on life, now with the realisation none of us are invincible – cancer doesn’t care who it strikes.”
Prior to my cancer diagnosis, I had not thought much about cancer – why should I? I was invincible, I had reached the prime of life – my career and personal life were fulfilling – I had everything to live for. Then WHAM! I was diagnosed with mantle cell lymphoma a rare form of non hodgkins lymphoma. Before I knew it my whole world had changed.
Fortunately for me medical research was my saviour. I was told by medical experts that if I had been diagnosed 12 months earlier I would not be here today. Treatment was a struggle, and even with treatment my median survival didn’t look good.
Now 10 years on from stem cell transplantation, with thanks to medical research, new drugs and techniques I have once again reached a point where my life is fulfilling. I have a whole new outlook on life, now with the realisation none of us are invincible – cancer doesn’t care who it strikes.
I became involved with the ALLG, because the bottom line is, I owe my life to cancer research. Having travelled the cancer journey, I want to help make the road less bumpy for those who must reluctantly follow. In fact, I want to help improve this critical area of cancer research, so that we can one day wipe out this insidious disease.
John Stubbs AM
Chronic Myeloid Leukaemia survivor
“On 1 March 2001 I underwent a Bone Marrow Transplant. As a former cancer patient who underwent a clinical trial, I now see it as my job to promote, engage and encourage access to cancer research and clinical trials.”
You always remember the date of diagnosis – it is a bit like a birthday – it comes around every year. So, the 2nd December 1999 remains embedded in our lives. I was told that I had Chronic Myeloid Leukaemia.
From a small eye infection which did not respond to anti-biotic cream – so after 2 weeks I had a blood test and bingo, 2 days later found out I had leukaemia. In hindsight, I did have bruising, was very tired (but working in a cafe and catering company) I thought tiredness went with the territory.
A bone marrow extraction confirmed the diagnosis.
The feelings at diagnosis are common for all cancer patients – shock, horror, why me – my wife cried in the arms of the haematologist, we drove to a park and I lost it!! I did not really feel sick and certainly did not look sick! Not overweight, fit healthy, and in fact a bit guilty that I had such a serious disease with little or no symptoms and certainly no visual signs!!
On 1 March 2001 I underwent a Bone Marrow Transplant – my donor was an American woman.
Today, I do feel physically and mentally great, back in the gym, have muscle, am fit and healthy, but I do suffer from some peripheral neuropathy in both feet which affects my balance slightly. But after all that my body has endured, this is “small fry”.
As a former cancer patient who underwent a clinical trial, I now see it is as my job to promote, engage and encourage access to cancer research and clinical trials.
My goal and focus is to ensure that cancer patients and their families’ word is heard and delivered at the highest government level. My other priority is to ensure politicians are aware that a lot more can be done at little or no cost to improve the lot of cancer patients!
I am a committed and passionate advocate for people affected by cancer as a regular speaker at medical conferences and seminars (nationally and overseas) on cancer policy, advocacy, clinical trials and related issues. I contribute to booklets and research papers as an Associate Investigator, edit consumer booklets for the Cancer Council and advise on government policy on cancer, cancer services and the value of consumer involvement.