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New clinical trial with genomics focus may offer fresh hope for blood cancer patients

Wednesday August 4, 2021

A new clinical trial research program partnering Australian researchers and medical institutes will offer people living with blood cancer access to genomic screening of their individual disease, potentially leading to accessing precision medicines.

High-grade lymphomas and acute leukaemias are aggressive blood cancers, with few treatment options available to patients living with relapsed or refractory disease. By linking genomic profiles with a series of research treatments, this new program could offer fresh hope for those living with these advanced cancer forms.

The Blood Cancer Genomics Trial (MoST-LLy) will leverage the experience, infrastructure and novel clinical trial design powering the Molecular Screening and Therapeutics (MoST) Program. This program is pioneering the way that solid tumours could be treated with precision medicine. MoST-LLy represents the first time this platform will be used to screen and treat people living with blood cancer in Australia.

The research program aims to match patients’ tumour profile with available and promising treatments. This could be a standard treatment, a treatment offered by MOST-LLy, or a treatment offered by another clinical trial. The first treatment to be tested in MOST-LLy is for aggressive lymphoma, and other treatments for other blood cancers will follow later this year. The program will also research biomarkers of response or resistance to treatments to further develop precision-based therapy options.

The research program will provide a new model of rapid clinical translation and access to novel treatments for high-risk blood cancers in Australia, with findings that may change the management of advanced blood cancers and improve patient outcomes.

The program is a collaboration between the Royal Brisbane and Women’s Hospital, Royal Adelaide Hospital, QIMR Berghofer Medical Research Institute, SA Pathology, Garvan Institute of Medical Research, Omico (the Australian Genomic Cancer Medicine Centre) and the NHMRC Clinical Trials Centre at the University of Sydney. The research has ethics approval from St Vincent’s Hospital Human Research Ethics Committee and the University of Sydney is the regulatory sponsor of clinical trials undertaken through MoST-LLy.

The pilot $1.8 million program allows the screening of 240 patients initially and is proudly co-funded  by the Leukaemia Foundation and Tour de Cure. Screening is now open to patients at Royal Brisbane and Women’s Hospital, with South Australia expected to open to recruitment later this year and SA Pathology providing molecular screening for the program across both states. The program is also supported by the Australian Government, the NSW Office of Health and Medical Research, and pharmaceutical partners[1].

The program has also recently been awarded an additional $2.7 million through the Australian Government’s Medical Research Future Fund (MRFF), which will be used to expand the pilot project to 240 more patients and sites across Australia in 2022 and beyond.

With more than 50 Australians diagnosed with blood cancer every day and blood cancers combined now ranking as the second largest cause of cancer-related death in the country[2], Leukaemia Foundation CEO Chris Tanti said the importance of improving access to genomic screening and clinical trials could not be overstated.

“If we are to have any hope of reducing blood cancer mortality in this country, we must shift our focus away from treating blood cancer subtypes with a ‘one size fits all’ approach and towards targeted therapies. Genomic testing illuminates the best treatment path for each individual patient, and the potential for delivering precision medicine tailored to them may result in improved survival, less side effects than some traditional treatment options, less time spent in treatment and greater quality of life,” he said.

“This research program will provide a new model of rapid clinical translation and access to novel treatments for high-risk blood cancers in Australia. Importantly, findings through this program may improve patient outcomes and ultimately change the management of advanced blood cancers.”

MoST-LLy is led by Professor Steven Lane at the Royal Brisbane and Women’s Hospital, Professor Hamish Scott from SA Pathology, Associate Professor David Ross at the Royal Adelaide Hospital, Professor David Thomas as the Molecular Screening and Therapeutics Program (MoST) lead, and Professor John Simes as the co-chair of MoST from the NHRMC Clinical Trials Centre at the University of Sydney.

Prof. Lane, head of the Cancer Program at QIMR Berghofer and Consultant Haematologist at the RBWH, said the program provides a systematic way of understanding genetic changes occurring, developing a clinical report assessing these changes, and using that information to determine a better treatment pathway for the patient.

“This program firstly involves providing access to genetic sequencing, then bringing together those with knowledge and understanding of these genes to determine which of these genetic changes occurring are significant in terms of driving cancer development, and which changes are simply incidental,” he said.

“We can use the sequencing information to inform the patient of a treatment type which may work best for their specific situation – and that tailored treatment may be accessible through an available clinical trial in Australia.”

Interested patients should discuss this trial with their haematologist. Further information on the trial is available through the Leukaemia Foundation website.

The National Strategic Action Plan for Blood Cancer, developed by Australia’s first Blood Cancer Taskforce and released by Federal Health Minister The Hon. Greg Hunt MP last year, contains a number of actions and recommendations regarding improving use of – and patient access to – genomic testing, clinical trials and precision medicine in Australia.

“Together, genomic screening, clinical trials and precision medicine have the power to revolutionise the way we treat blood cancer in Australia and bolster the national blood cancer community’s concerted work to achieve zero lives lost to this complex set of diseases by 2035,” Mr Tanti said.


Genomic screening and precision medicine in blood cancer 

  • There are over 120 different types of blood cancer and research shows that even within each of these groups, there are many sub-groups with unique genetic mutations that affect both disease development and treatment options.
  • Genomic screening has revolutionised understanding of blood cancers and paved the way to a new generation of new precision medicines, where treatments are targeted based on an individual’s blood cancer genomic profile. Until recently it has been almost impossible to target treatments to a person’s unique genetic makeup, resulting in a ‘one size fits all’ approach to treatment.
  • Access to accurate and timely diagnostics is fundamental to treatment, care and survival for people living with blood cancer. Early and accurate diagnosis is critical to understanding what type of blood cancer an individual has, and to understanding what treatment pathway will offer the best survival and quality of life outcomes.
  • Genomic profiling is required for accurate subtype diagnosis and the subsequent treatment of blood cancer patients. Through genomic profiling, clinicians can more precisely match patients to targeted therapies. These efforts are critical to the expansion of precision medicine and the realisation of curative therapies.
  • Despite blood cancers being at the forefront of genetic characterisation and precision medicine, genetic testing is not standard of care in Australia and less than 30% of Australians facing blood cancer undergo a genomic screen to inform their diagnosis and treatment pathway.[3]
  • The National Strategic Action Plan for Blood Cancer includes recommendations around genetic screening, including developing guidelines for diagnostics and reviewing Australia’s capacity to meet these guidelines, as well as recommendations to enable access to novel and specialised therapies, including actions to improve equitable access to these therapies and making precision medicine the standard of care.

Blood cancer clinical trials 

  • Research has shown that the systematic enrolment of patients in clinical trials is one of the single most important actions to reduce the number of lives lost to blood cancer, however despite the demonstrated benefits of clinical trials, patient participation in blood cancer clinical trials in Australia remains low.
  • Patients on clinical trials are shown to have improved outcomes, and that access remains the greatest obstacle to beating blood cancer. Less than 20% of Australians living with blood cancer today report having the opportunity to participate in a clinical trial, and only 1 in 5 Australians with blood cancer who want to enrol in a clinical trial have access to one.[4]
  • The National Strategic Action Plan for Blood Cancer includes recommendations to increase access to clinical trials, including through improving discussion of clinical trial options with patients and increasing access in regional and remote areas, such as through a national approach to prioritising tele-trials.

[1] This research was conducted with support from AstraZeneca Pty Ltd and BeiGene Ltd.

[2] Cancer data in Australia, Summary – Australian Institute of Health and Welfare (

[3] State-of-the-Nation-Blood-Cancer-in-Australia_Leukaemia-Foundation.pdf

[4] State-of-the-Nation-Blood-Cancer-in-Australia_Leukaemia-Foundation.pdf

Blood cancer signs and symptoms:
Symptoms of all blood cancers can sometimes be subtle or even similar to other conditions, such as a flu. However, ongoing symptoms like recurrent infections, increased fatigue, night sweats, bone pain, bruising or enlarged lymph nodes should be immediately discussed with your GP or specialist. Early diagnosis can play a key role in surviving blood cancer, so it is crucially important that you are examined and treated properly. If you or someone you love is diagnosed with a blood cancer, reach out to the Leukaemia Foundation on 1800 620 420. Accessing our support is free of charge.

Last updated on January 3rd, 2023

Developed by the Leukaemia Foundation in consultation with people living with a blood cancer, Leukaemia Foundation support staff, haematology nursing staff and/or Australian clinical haematologists. This content is provided for information purposes only and we urge you to always seek advice from a registered health care professional for diagnosis, treatment and answers to your medical questions, including the suitability of a particular therapy, service, product or treatment in your circumstances. The Leukaemia Foundation shall not bear any liability for any person relying on the materials contained on this website.