Rare disease funds to boost pancreatic and blood cancer studies: Rare Disease Day feature

Adj A/Prof Charles Pilgrim and Prof Andrew Spencer describe

their newly funded MRFF research and how it will help patients.

Early this month Central Clinical School researchers were awarded funding under the MRFF’s 2021 Rare Cancers, Rare Diseases and Unmet Need (RCRDUN) program. 

As part of Rare Disease Day today, we asked two CCS researchers - Charles Pilgrim from the Department of Surgery, and Andrew Spencer from the Australian Centre for Blood Diseases - working with rare diseases about the research being funded.

Study to streamline treatment for pancreatic cancer patients

Adjunct Associate Professor Charles Pilgrim from the Department of Surgery was awarded nearly $3 million for a project aiming to improve the accuracy of reporting of CT scans in pancreatic cancer to optimise care for patients.

Charles Pilgrim 

Approximately 4,000 people are diagnosed with pancreatic cancer (PC) annually in Australia. The five-year survival rate is 11%.

People with PC are treated according to the extent of their disease at diagnosis. However, one of the problems that doctors face is adequately distinguishing exactly how locally infiltrating a cancer is, and therefore identifying which patients who should receive chemotherapy before surgery versus surgery alone.

Unfortunately, in two-thirds of patients, the cancer has already spread to organs such as the liver or lungs by the time they’re diagnosed. For the third of people where the cancer hasn’t obviously spread, some have tumours that are clearly resectable (surgically removable), but for others this is not possible as the tumour has advanced locally and infiltrated important nearby organs. There is also a group in between the clearly resectable and locally advanced groups, who are therefore called ‘borderline resectable’.

“It’s important to be able to clearly differentiate because those who could have surgery, should have surgery, and those where it is never going to be safe or successful to remove the tumour should be spared a futile operation,” Adjunct Associate Professor Pilgrim said.

For those able to have surgery, clinicians need to know whether they should have surgery first (if the tumour is clearly resectable) or chemotherapy first for those ‘borderline’ cases.

Patients with locally advanced tumours would generally have chemotherapy and radiotherapy and only on rare occasions proceed to surgery at all.

“It has a significant impact on what treatment patients should be offered as to what the ‘resectable’ status is – the resectable status is completely identifiable on a CT scan,” he said.

Adjunct Associate Professor Pilgrim has been funded over five years to conduct a multi-centre stepped-wedge randomised trial to test whether a structured radiology report can improve the accuracy of reporting of CT scans in PC patients.

“If we can more accurately separate the borderline resectable and locally advanced group from those who are clearly resectable, we will end up taking fewer patients to theatre only to find their tumours are not able to be removed, while at the same time improving the outcomes for those borderline patients who are able to undergo complete surgical removal following an initial course of chemotherapy,” Associate Professor Pilgrim said.

“Additionally, by ensuring clinicians across Australia are all ‘talking the same language’ when it comes to exactly what we mean when we say ‘borderline resectable’ or other stages of disease, we will make comparisons between outcomes and identification of variations in care meaningful and should ensure benefits from trials of different treatments are able to be fast-tracked into clinical practice,” he said.

The project, which will build on a successful pilot study at The Alfred and the Austin hospitals supported by the Victorian Government, will recruit 40 different hospitals across Australia.

Multiple myeloma scan showing its

distribution in bones throughout the body.

Grant will help optimise treatment outcomes in blood cancer

Treatment of the blood cancer multiple myeloma (MM) is complex and costly. Professor Andrew Spencer from the Australian Centre for Blood Diseases (ACBD) was awarded a $1.68 million RCRDUN grant over five years to develop a more efficient use of the high-cost therapies.

Myeloma is a type of blood cancer that develops when plasma cells, a type of white blood cell, spread abnormally in bone marrow. Most people have multiple bone lesions at the time it is diagnosed, hence the term multiple myeloma. The five-year survival rate is 51%. Around 2400 Australians are diagnosed with the disease each year.

New therapies being tested in clinical trials are achieving survival benefits but these are not being seen in clinical practice, in part because many MM patients stop therapy early, often due to side-effects, Professor Spencer said.

“We hypothesise that if treating clinicians are made aware of emerging patient symptoms, thus informing timely intervention, duration on therapy can be optimised, enhancing treatment effectiveness and making more efficient use of these high-cost therapies,” he said.

Professor Spencer and his team will test a technique called patient-reported outcome measures (PROMs) to do this.

Routine use of PROMs, including feedback to treating clinicians, has been shown to improve outcomes in non-haematological cancer populations, but has not been evaluated in MM care.

Professor Spencer’s team showed in the pilot MY-PROMPT randomised controlled trial, that real-time feedback of patient-reported outcome measures (PROMs) in MM is feasible and acceptable to patients and clinicians.

The MY-PROMPT-2 trial will build on this by testing whether real-time feedback of PROMs to clinicians improves event-free survival in patients with relapsed MM receiving standard of care (SoC) treatment with PROMS, compared to SoC alone.

“If successful, it will improve and optimise the use of high-cost tax payer funded therapies,” Professor Spencer said.

This Rare Disease Day feature is presented by the CCS Community and Researcher Engagement (CaRE) committee. See more about CaRE: www.monash.edu/medicine/ccs/community-engagement