Pioneering the next phase in targeted therapies for thousands of Australians with advanced blood cancers

9 December 2015

Our donors are helping Peter Mac researchers pioneer the development of a new combination drug therapy to treat advanced blood cancers, which currently claim the lives of around 4,000 Australians each year.

Peter Mac’s laboratory researchers have found that by combining two drugs, survival times of pre-clinical models can be doubled, giving new hope for the development of more effective blood cancer treatments.

The researchers published the study overnight in Cancer Discovery, building on a world-first clinical trial already underway at Peter Mac, which uses the drug CX-5461 to treat patients with incurable blood cancers such as myeloma, lymphoma and leukaemia.

The trial has shown promising results to date, however the research team has found that CX-5461 could be even more effective when used in combination with another drug, Everolimus, which is already used to treat other cancers.

According to Professor Rick Pearson, Head of Peter Mac’s Cancer Signalling Laboratory, the research findings significantly enhance understanding of pre-emptive strategies to kill off cancer cells before they have the chance to become resistant to therapy.

“CX-5461 targets a particular process that is required for cancer cell survival. Our experiments show that adding Everolimous synergistically strengthens this attack, more rapidly and more effectively eradicating the killer disease.

 “We know that all cells rely on ribosomes which act like a factory producing the proteins essential for their growth and survival,” according to Professor Pearson.

“Peter Mac researchers, in collaboration with Professor Ross Hannan at the John Curtain School of Medical research, have previously shown that certain blood cancers are far more reliant on these proteins than normal cells, and that eliminating the protein production capability of ribosomes leads to the rapid death of cancer cells, while normal cells stay viable.

 “As a result of this research breakthrough, fifteen patients are currently involved in a phase 1 clinical trial of CX-5461 – a compound that our research team is developing with pharmaceutical company Senhwa.

“This novel therapy works to inhibit the ribosomes’ protein production capability, effectively starving the cancer cells of a key ingredient they need to survive and proliferate. 

“By adding Everolimus to this treatment, we have shown the potential for even more powerful results. A further study in collaboration with scientists at Monash University shows striking effects in the targeting of late stage prostate cancer through a similar strategy indicating that this approach may be generally applicable for a range of cancer types.”

Associate Professor Simon Harrison, Consultant Haematologist at Peter Mac and Principal Investigator on the CX-5461 first-in-human trial, says this new research provides further confidence that researchers are on the right track.

 “The prevalence and poor prognosis for people with advanced blood cancers demands the ongoing and intricate study of abnormal cell behaviour, which has been an indicator of cancer for over 100 years. To date 15 patients have been treated on the first-in-human clinical study with a number of patients experiencing prolonged benefit.

“With this new knowledge we can now work closely with pharmaceutical companies to potentially fast track the testing of this combination approach for the benefit of patients.”

Peter Mac’s grateful thanks go to our donors, whose generosity helped give this research team the specialised technologies and resources needed to undertake this ground-breaking work.

To help make more discoveries like this one possible, please donate now.

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