Peter Mac News

Improving outcomes for patients with Cancer of Unknown Primary

01 August 2024

A Peter Mac-led international clinical trial has found a way to improve outcomes for CUP patients – these are patients diagnosed with a cancer that has spread but from where remains unknown.

Patients with Cancer of Unknown Primary (CUP) are currently treated with a broad-acting chemotherapy, though their long-term survival is usually poor and their optimal treatment pathway is uncertain.

The CUPISCO trial involved 636 CUP patients who were treated at Peter Mac and other specialist hospitals across 34 countries. All received initial chemotherapy, and for most this temporarily halted the growth of their CUP tumours.

Among this group with controlled cancers, 25% were randomly assigned to a control group who received further chemotherapy while 75% received a more targeted treatment with immunotherapy or other molecularly-guided therapy based on comprehensive genomic profiling of the patient’s tumour and possibly also their blood (liquid biopsy).

For the patients treated with the molecularly-guided therapy, the average length of time before their cancer got worse was significantly increased compared to the group of patients treated with standard chemotherapy alone.

In addition, more shrinkage of tumours was seen in patients treated with molecularly-guided therapy than with standard chemotherapy treatment.

Professor Linda Mileshkin, who founded Australia’s first dedicated clinic for CUP patients at Peter Mac and was the co-lead for the study along with her colleague Professor Alwin Kramer from Germany, says the study confirmed the benefits of molecularly-guided therapy as compared to just using standard chemotherapy.

“The clear benefit from molecularly-guided therapy in the investigational arm means that this should be a new standard of care for CUP patients,” says Prof Mileshkin, Director of Medical Oncology at Peter Mac.

“We need to find a way to make both the comprehensive genomic profiling as well as the relevant targeted therapies available for CUP patients to access.”

Molecularly-guided therapy involves testing cancers to look for genomic biomarkers which, if found, can match patients to receive one of an emerging class of targeted drugs that are effective against cancers with specific genomic characteristics.

The CUPISCO results are published today in The Lancet. The paper is titled "Molecularly guided therapy versus chemotherapy after disease control in unfavourable cancer of unknown primary (CUPISCO)", and you can read it in full here.

There is also an accompanying editorial in The Lancet, which you can read here.

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For more information contact the Peter Mac Communications team on 0417 123 048.

About Peter Mac

Peter MacCallum Cancer Centre is a world leading cancer research, education and treatment centre and Australia’s only public health service dedicated to caring for people affected by cancer.