New Hope For Cancer Patients Accessing Leading-Edge Unfunded Drugs

Peter Fergusson, CEO of Leukaemia & Blood Cancer New Zealand is heartened to hear that Pharmac has announced plans to back an Early Access Scheme which could potentially give cancer patients access to new and emerging medicines and treatments.
“Few cancer patients have time to wait, and many are prematurely dying because they do not always have access to the most optimal medication at the right time in their treatment cycle. Many of these emerging medicines and treatments are being fast-tracked, funded and made available in other OECD countries,” says Fergusson.

Leukaemia & Blood Cancer New Zealand is part of a coalition of cancer NGOs called CANGO. A collaborative group of patient-focused cancer organisations that have proposed a Rapid Access Scheme to the government as part of the broader solution for improved cancer treatment in New Zealand.
Rapid Access Schemes have been introduced, and trialed, in the UK, and some European countries, initial versions of these schemes have not proven successful however recent iterations of these schemes have been streamlined and improved dramatically. New Zealand has the unique opportunity to learn from these experiences and adopt leading-edge best practice processes based on the successes from these proven and successful structures.

“We are looking forward to receiving the pending formal reviews of the UK Early Access Scheme to benchmark those learnings against the draft proposal submitted to the Health Minister. My hope is for the government to actively ring-fence funding for a pilot scheme from the May budget. It is hard not to ponder how many lives might be improved, prolonged and saved this year if a pilot Rapid Access Scheme was implemented within the next few months?” says Fergusson.

Since being elected, the government has confirmed cancer is one of its major health priorities.

“Cancer is New Zealand’s biggest killer and is responsible for over 30% of all deaths each year. It is encouraging to hear Pharmac backing an innovative concept that could reduce cancer-related deaths. It begins to give hope that this will open the door to new and leading-edge treatment options. It shows that the government may be listening to the growing voice of concern and may also be willing to take action against a disease that has reached epidemic proportions in our country,” says Fergusson.
Some medical experts say Phil Kerslake is New Zealand’s longest surviving and most prolific cancer patient in New Zealand. Phil has been living with different cancers and its treatments since his teens – over 40 years.

Kerslake is encouraged by the prospect of a Government approved Rapid Access Scheme.

“In my case, there are few remaining treatment options, and my current treatments are viewed by doctors as palliative. The rapid evolution of new targeted therapies and immunotherapy has given me, and other patients hope for a better future.

I feel there is every chance that early access to some of the unfunded drugs in the pipeline will prove the difference between premature deaths and otherwise long, highly productive lives. I love my life and my family, and I hope to live to benefit personally from an emerging treatment. But if my good fortune is to end, I would dearly like to believe those following me have the opportunity to live on,” says Kerslake.

Molecular breakthrough for AML Patients

Earlier this year, the gene panel developed by the Leukaemia & Blood Cancer New Zealand Research Unit (LBCRU) for testing acute myeloid leukaemia (AML) patient samples became available to cancer clinicians in Auckland. Now all adult patients with newly diagnosed and relapsed AML have samples collected for DNA preparation and analysis using this 70-gene myeloid panel.

HK, a man aged 57, was diagnosed with AML in August 2015. Molecular studies showed that his leukaemia cells had two abnormal genes: the NPM1 mutation and the FLT3 ITD mutation. The second carries a high risk of relapse so, in addition to chemotherapy, this is usually addressed with a bone marrow transplant.

But transplants are risky. The LBCRU estimates that between 15 and 20 percent of patients are at risk of dying in the first year from complications. It also commits the patient, family and caregivers to four weeks in hospital and two months of follow up care. It’s usually a year before they resume a normal life.

As HK was in remission after the first of four treatments, instead of going straight to a transplant, his blood and bone marrow were re-tested at the LBCRU using a super-sensitive new test at three-monthly intervals. Using the NPM1 marker, which is seen in about 30 percent of AML cases, the team’s new molecular test can find one leukaemia cell in 100,000 normal cells.

Every test after treatment was negative. Clinicians say HK is probably cured. However, if the tests had turned positive, a transplant could have been arranged before a relapse without the need for further chemo or radiotherapy.

HK’s good news story will soon be that of many New Zealanders living with blood cancers thanks to this new molecular test for a common leukaemia.

Find out more about how your donations fund research

How your Donations Fund Research

Your donations to research, channelled through LBC, now total $7 million since the first grant in 2011. Your donations remain one of the main sources of haematology research funding in New Zealand.

The Leukeamia & Blood Cancer Research Unit (LBCRU) sits within the University of Auckland’s Faculty of Medical and Health Sciences, and was established in 2013 with an initial commitment of $1,250,000 from LBC.

The team is identifying blood cancers’ molecular markers that will allow more targeted medical care, avoid aggressive treatments when they are not necessary, and pave the way to new therapies for blood cancer and blood conditions.

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A vision to cure – Research in New Zealand

For our patients, the most visible aspect of LBC’s work is the support and information it provides them and their families. As a clinician, I am often aware of LBC’s Support Services Coordinators in our day-stay unit and our ward at Auckland Hospital. Patients exercise in the LBC-funded Support and Wellbeing Room or come to see me with an LBC booklet in their hand. Having spoken at LBC-run support groups and the patient forum, I know how well attended and important they are for patients and their families.

As LBC’s patient support services have grown, so has its support for research into better treatments and cures for blood cancer. LBC started out as a small organisation funding travel grants for New Zealand health professionals working in the blood cancer field. Now, it is a major funder of research on blood cancers and other blood disorders, with competitive research grants nationwide, and the recent establishment of the specialised Leukaemia & Blood Cancer Research Unit within the University of Auckland.

Blood cancer research in New Zealand is being carried out in a number of areas, including:

  • Establishing basic models of leukaemia in mice and zebrafish that allow us to interrogate what causes leukaemia and test novel treatments.
  • Genomic landscape studies – the ability to dissect the molecular basis of blood cancers to further understand their biology, as well as improving strategies for diagnosis, treatment, and monitoring response to therapy.
  • Clinical trials for different blood cancers, including participation in international, multicentre studies, for example, the UK NCRI AML19 trial for adult patients with acute myeloid leukaemia (AML) and local studies such as the recently opened KISS study for patients with chronic myeloid leukeamia (CML). Participation in clinical trials not only improves patient care, but is vital in testing new treatments.
  • Improving health outcomes and quality of care, regardless of health status, is an area of research which is beginning to be explored.

In the last decade, genomic (DNA) analysis in particular, has improved as a result of advances in technology.

Sequencing the first human genome took the world’s scientists 13 years and 3 billion dollars to achieve. Today in the Leukaemia & Blood Cancer Research Unit, we have a DNA sequencing machine the size of a desk printer that can sequence genes in a matter of days. Instead of looking at only single genes, now we can look at 70 or 100 genes in a leukaemia. It has opened up the whole area of personalised medicine.

At a basic science level, it’s telling us what caused a patient’s leukaemia. At the clinical level, whereas we may have treated all these people in a similar way, their different mutations may have different prognoses. Equipped with this detailed information, we may decide on transplant or chemo based on their specific mutation.

Future diagnoses may put together all the molecular or genetic data and the clinical information of the patient, i.e. type of cancer, age of patient and other illnesses present, so that a complete picture can be used to develop an individual’s treatment strategy.

Research has also provided advances in the treatment of cancers and the development of targeted therapies. Haematology has led the way in blood cancers and the overall treatment of cancer.

LBC has helped put blood cancer firmly on the map in the field of cancer research in New Zealand.

Why is research here important?

Much blood cancer research is carried out overseas, but the development of research in New Zealand is important for the following reasons:

  • To answer questions related to the New Zealand population.
  • To make a uniquely New Zealand contribution locally and internationally. For example, the CML clinical trial being carried out here is the only one of its kind in the world. The findings will add to our knowledge of managing CML patients here and worldwide.
  • To develop the technology and expertise here in New Zealand, we have to invest in it and our people. The 18 students and scientists working in the Leukaemia & Blood Cancer Research Unit are part of a growing workforce of people who are interested in blood cancers and conditions. Instead of leaving for overseas or going into different fields, these researchers are more likely to stay in New Zealand and potentially develop their own blood cancer research group in another New Zealand city.

A number of these advances have been game changing. Before 2000, the life expectancy for most patients with CML was four to five years. When Glivec was first trialled in 2000, we thought it might improve outcomes by one to two years. Eighteen years later, I still see the first patient who entered the Glivec trial in clinic and attending LBC support group meetings. The drug, and its next generation versions, have transformed the way we treat CML such that a 50 year old diagnosed with CML now has a similar life expectancy as a person of the same age without CML.

Research and clinical trials are providing new and better treatments on a yearly basis. In myeloma, several new treatments that use targeted drug or antibody strategies have been evaluated in clinical trials, and three targeted drugs for AML were approved by the US Federal Drug Administration last year.

A treatment holding much promise is immunotherapy, which is the ability to harness the body’s own immune system to assist in treating a blood cancer. Bone marrow transplants involving donor bone marrow are a form of immunotherapy using the donor’s immune system cells to recognise residual leukaemia or lymphoma cells and mop them up.

The immunotherapy field is growing in two key areas:

Checkpoint inhibitors
Our immune system typically recognises abnormal cells and destroys them before they can develop into full-blown cancer. Patients with a suppressed immune system, however, have a higher chance of cancer than the general population. Cancers often produce molecules that block the body’s immune response, so the checkpoint inhibitor drugs work to unblock that response and allow the immune system to react.

Modifying immune cells
Immune system cells can be collected from the blood of patients and manipulated by inserting a gene that generates a protein to help these cells recognise cancer cells. These modified killer or CAR-T cells, are returned to the body to kill the cancer.

Immunotherapy for blood cancer is only just beginning. It’s currently being used in acute lymphoblastic leukaemia and lymphoma, but research groups are starting to look at myeloma and AML, and colleagues at the Malaghan Institute in Wellington are working at bringing this technology to New Zealand. LBC has helped put blood cancer firmly on the map in the field of cancer research in New Zealand. As a result, our research is being recognised nationally and internationally. More importantly, the ability of LBC to fund more and better research is making a difference to our own patients and families.

Dr Peter Browett is a Professor of Pathology and Head of the Department of Molecular Medicine and Pathology at the University of Auckland School of Medicine, Consultant Haematologist at Auckland City Hospital, and Consultant Haematologist at Diagnostic Medlab. Peter has had a long association with LBC, acting as Medical Director and Trustee for many years.

Manifesto launched to address key cancer issues

Leukaemia & Blood Cancer New Zealand LBC were part of the CANGO manifesto launch in Wellington this week, alongside 9 other cancer patient organisations. The panel discussion at the Bolton Hotel aimed to bring attention and change to key cancer issues, and ensure the Minister and MPS across the political spectrum listen.

In particular CANGO (Cancer Non-Governmental Organisations) is asking all political parties to commit to cancer prevention, diagnosis, treatment, research and survivorship as key components of health policies.

CANGO was formed in 2007 and is an alliance of prominent New Zealand cancer charities including: Breast Cancer Foundation NZ; Bowel Cancer New Zealand; Leukaemia & Blood Cancer New Zealand; New Zealand Gynaecological Cancer Foundation; Prostate Cancer Foundation of New Zealand; Unicorn Foundation New Zealand; Melanoma New Zealand; Lung Foundation New Zealand; and the Cancer Society of New Zealand.

New Zealand’s aging population means that the incidence of cancer is growing exponentially, and over 65s have the highest rate of cancers requiring treatment. The effects are far reaching – from the impact on the patient, their families and friends, to the providers, the budget and broader economy.

Chair of CANGO Graeme Woodside said “CANGO’s purpose to is collaborate with cancer charities, and lobby the Government strategically especially in election years. More than 22,000 New Zealanders will be diagnosed with cancer this year, and it’s the country’s single biggest cause of death. One in three cancer patients will be cured, but we want to see that statistic improved through better research and treatment, and more education about cancer prevention”.

From ensuring all New Zealanders have access to diagnosis, care and treatment to addressing disparities in care for Maori, access for rural communities, and responding to the rapidly changing environment by ensuring faster decision making by PHARMAC, CANGO will be lobbying unashamedly until the election.

CANGO is seeking increased investment in clinical trials to address ongoing needs of cancer survivors and the aging population. Another of CANGO’s focuses is reducing modifiable risk factors – ensuring New Zealanders eat healthy and balanced diets, and participate in regular exercise.

Tiny fish may hold key to blood cancer treatment

Leukaemia & Blood Cancer New Zealand have invested over $1 million to establish the Leukaemia & Blood Cancer Research Unit at Auckland University.

The unit has attracted 18 scientists from across the world conducting ground-breaking research into blood cancer.

The researchers hope that by mapping their DNA they will be able to pinpoint which genes are causing different types of the disease to grow in humans, paving the way for personalised drugs to treat them.

A new trial using Zebra fish aims to help unlock the genetic key to blood cancers like leukaemia.

The genetic make-up of the fish is 84 per cent the same as humans and the fact they reproduce very quickly and are see-through means researchers can genetically-engineer them to replicate and mimic cancer cell growth in humans.

Professor Peter Browett says genetic information will be gathered from newly diagnosed patients and affected cells will be injected into the fish to see if they develop a leukaemia and also to see if other genes are involved.

The unit also utilises New Zealand’s first genome mapping machine which has the power and technology to look at a whole panel of genes.

LBC’s chief executive Pru Etcheverry says:

“This new research is hugely exciting as we are entering the era of precision medicine which sees medicines matched to patients’ individual genetic characteristics.”

For more information on the Leukaemia & Blood Cancer Research Unit please view this story on One News.