“When we break cholangiocarcinoma down. We understand it. Then build the response that beats it.”
— Steve Holmes
Standardisation of Australian Treatment Pathways for Cholangiocarcinoma
This Article
Webinar Review: Cholangiocarcinoma in Focus — Genomics, Guidelines, and the Road Ahead
Update on MoST / CaSP-Specific Cholangiocarcinoma Data and Standardisation of Australian Treatment Pathways
- Hosted by: Cholangiocarcinoma Foundation Australia
- Presented by: Dr Natalie Rickers,
CCF-AU Medical and Research Team - Date: January 2025
- Registered Audience: 142 Clinicians only event.
Watch the Full Webinar
This article summarises key insights from the national webinar. To watch the full session, please view the video below:
Introduction
This Cholangiocarcinoma Australia webinar review covers the 2025 national update on genomic data, treatment standards, and the move toward standardising Australian pathways of care. Cholangiocarcinoma remains one of the least understood and most difficult-to-treat cancers in Australia. Yet for the first time, national efforts are converging around a common goal: to deliver earlier, smarter, and more consistent treatment options for patients with this rare disease.
Speaker Highlights and Key Messages
Professor David Thomas
Founder and Chief Strategy Officer, Omico
Topic: Real-World Genomic Insights from MoST and CaSP
Professor Thomas presented updated real-world genomic data from 606 patients with biliary tract cancer. His key findings included:
60% of patients had a targetable genetic mutation
Only 15% received matched therapy — via clinical trial, private access, PBS, or compassionate use
The primary reasons for this gap include late testing, low-quality biopsy samples, and system delays
Professor Thomas also walked through Omico’s internal data portal — now tracking over 20,000 patients — which is already being used to guide real-time clinical decisions.
“We now know the mutational profile of cholangiocarcinoma in Australia. The challenge is not scientific — it’s operational.”
Professor John Zalcberg
Head of Cancer Research Program, Monash University
Topic: Australia’s Path to National Guidelines and Quality Measurement
Professor Zalcberg addressed the absence of formal national guidelines for the management of cholangiocarcinoma and biliary tract cancers in Australia.
He outlined a national effort to:
Review 7 existing international guidelines
Convene multidisciplinary expert working groups across states.
Identify consensus and areas of clinical variation.
Develop a set of quality indicators to be used in a future national registry.
Key focus areas under discussion included stenting practices, biopsy timing and quality, and eligibility for liver transplantation.
“Without national standards, care remains inconsistent — and variation without explanation leads to preventable inequity.”
Professor Juan Valle
Chief Medical Officer, Cholangiocarcinoma Foundation USA
Topic: Beyond First-Line Treatment — What’s Emerging in Targeted Therapies
Professor Valle offered a comprehensive overview of new and emerging treatment options, particularly beyond the first-line regimen of cisplatin, gemcitabine, and immunotherapy.
Key points included:
High response rates from targeted therapies for IDH1, FGFR2, HER2, BRAF, and KRAS mutations.
The importance of molecular profiling at diagnosis, not after first-line failur
The need for re-biopsy or ctDNA testing to identify resistance and guide second- or third-line therapies.
MSI-high patients show exceptional response to immunotherapy and must be identified early.
“The shift is already happening. What we see globally — and increasingly here — is that timing and testing now determine whether a patient lives long enough to access what works.”
Steve Holmes
CEO Cholangiocarcinoma Foundation Australia
Topic: Crosscutting Insights
“Cell damage causes signal chaos. That’s how cancer begins.
— SteveH”
1. Timing is Critical
Early sequencing matters. Waiting until late-stage progression often means patients are too unwell to benefit from targeted therapies — even if their mutation is known.
2. Australia’s System Gaps Are Fixable
The tools are here. What’s needed is coordination: reflex testing, better sample quality, faster turnaround times, and clinician confidence in the process.
3. Guidelines Must Catch Up to Reality
A national standard of care will give both clinicians and patients a clearer, evidence-informed roadmap. Without it, treatment quality will continue to vary by postcode and provider.
4. Equity Remains a Challenge
Access to matched therapies — especially those without PBS listing — remains inconsistent. Unless the system shifts toward tumour-agnostic approvals and real-time trial enrolment, patients with rare cancers will continue to fall through the cracks.
Foundation Perspective: What Happens Now
At Cholangiocarcinoma Foundation Australia, we believe that awareness has never been enough. This webinar confirms that:
Matched treatments exist
Sequencing works
Patients can benefit now — if systems align in time
Our work focuses on bridging this gap through the Optimal Patient Response (OPR) Framework initiatives:
- The Patient Navigator Journal
- The Mutational Translator
- The Patient-Endorsed Medical Registry
We are committed to turning these insights into action.
That means embedding this knowledge in everyday clinical practice and ensuring no patient is left behind because of a missing test, a slow process, or a postcode disadvantage.
In Conclusion
This Cholangiocarcinoma Australia webinar review makes one thing clear: the science, data, and treatment options for cholangiocarcinoma have advanced — but system change is lagging.
The speakers demonstrated that genomic profiling can identify targetable mutations in the majority of patients, and that proven treatments already exist. They also showed that national guidelines, faster sequencing, and earlier access to targeted therapy could significantly improve survival outcomes.
The challenge is no longer whether we can act — it is whether we act quickly and consistently enough. Without urgent changes to testing practices, clinical pathways, and drug access, many patients will continue to miss out on life-extending options.
For the Cholangiocarcinoma Foundation Australia, the priority is clear: turn these insights into action by embedding early testing, aligning national care standards, and ensuring every eligible patient has the opportunity to benefit from the best available treatments.
This review is a reminder that the future of cholangiocarcinoma care in Australia will be decided not by what we know — but by how well, and how fast, we apply it.
This review
By Steve Holmes
August 7th 2025
Source: CholangioToday.com
Disclaimer
This article is provided for general educational and informational purposes only. It does not replace professional medical advice, diagnosis, or treatment.
The views expressed by guest speakers are their own and do not necessarily reflect those of the Cholangiocarcinoma Foundation Australia.
Patients should always seek the guidance of their own qualified healthcare providers with any questions about their medical condition or treatment options.
Mention of specific drugs, clinical trials, or medical procedures does not imply endorsement, recommendation, or guarantee of outcome.
While every effort has been made to ensure accuracy at the time of publication, medical knowledge and available treatments may change over time.
