Precision medicine doubles progression-free survival in high-risk cancer

Pharma News: The development of next-generation sequencing in conjunction with targeted anticancer therapies has allowed the delivery of precision medicine, selecting for the molecular drivers of a patient’s cancer. Australian researchers and clinicians have demonstrated that precision medicine significantly improves outcomes for children with high-risk cancer.

“These are very exciting results which we believe have important implications for the treatment of children with cancer,” said Professor David Ziegler, Chair of Clinical Trials for the Zero* Childhood Cancer Program and senior study author.

An impressive 55% of children receiving personalised treatments achieved complete or partial remission or had their disease stabilised for at least six months. This is particularly notable given that these children had aggressive cancers that had failed standard therapies.

“We’ve already shown that precision medicine can help identify new treatment options for many high-risk patients. Now we’ve shown that it not only can shrink their tumours, but also lead to a significant improvement in long-term survival for those patients,” Professor Ziegler added.

Notably, the two-year progression-free survival (PFS) for children treated with precision medicine was more than double that of those receiving standard therapy (26% vs. 12%) and five times higher than those receiving unguided treatments.

Associate Professor Loretta Lau, a paediatric molecular oncologist at Kids Cancer Centre and researcher at Children’s Cancer Institute, and Professor Glenn Marshall AM, Clinical Lead of ZERO and co-senior author, emphasised the transformative potential of precision medicine.

“Our study provides important new evidence that response to precision-guided therapy translates into improved survival,” they stated. “This work provides hope to families where none previously existed in a new model of treatment for high-risk childhood cancer, which is changing national and international clinical practice.”

Professor Ziegler, who also serves as a paediatric oncologist at the Kids Cancer Centre and a research leader at Children’s Cancer Institute, reinforced the importance of molecular analysis in treatment.

“We’ve shown that matching a therapy to a patient through molecular analysis of their cancer, and implementing that targeted therapy early on, are key to achieving the best possible results,” he said.

While the study showed that precision medicine significantly improves PFS, its impact on overall survival (OS) is less clear.

“The difference in 2-year OS of 38% in precision medicine treatment did not reach statistical significance when compared with the 2-year OS of non-precision medicine treatment (24%), unguided therapy (20%) and standard of care (23%). This may have been impacted by patients receiving multiple lines of therapy and different salvage therapies; longer follow-up may be required to further assess the impact on OS,” the authors commented.

Federal Minister for Health Mark Butler praised the advancements, saying, “Australia’s health and medical researchers are world-class, and we acknowledge the remarkable advances in the use of precision medicine through the Zero Childhood Cancer program. The Government pays tribute to the researchers, clinicians, and specialists at Children’s Cancer Institute and Kids Cancer Centre at Sydney Children’s Hospital. Their outstanding work in cutting-edge treatment has given very sick children a new beginning to survive and thrive.”

Supported by the Australian Government and the Minderoo Foundation, and sponsored by the Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG), the ZERO program’s first national precision medicine trial began in 2017. This trial focused on identifying new treatment options for children with high-risk cancers.

The success of this clinical trial led to joint funding of $67 million from the government ($55.8 million through the Medical Research Future Fund) and Minderoo Foundation ($12.2 million) in 2020. This funding enabled the expansion of the program to be available to all 1200 Australians aged 0-18 years diagnosed with cancer, a milestone achieved in 2023.

It marks the first time precision medicine has been made available to every child diagnosed with cancer, not just in Australia, but in any country worldwide.

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*ZERO is jointly led by Children’s Cancer Institute and Kids Cancer Centre at Sydney Children’s Hospital, Randwick and involves 9 child cancer treatment centres in the country. This study involved more than 100 scientists and clinicians working together across all these 9 child cancer centres.