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Exceptional Responders Program

Studying those who have exceptional responses to treatment and using this knowledge to help others.

Collagen fibres running through a cancer tumour, created using a multiphoton microscope.

The Exceptional Responders Program is a collaborative study between the Garvan Institute of Medical Research, The Kinghorn Cancer Centre, and clinicians and patients nationwide. By analysing unusual individual cases and unexpected responses to treatment, we can understand more about how treatments work – and how we can make them work better.

The primary aim of Exceptional Responders is to improve the lives of people with cancer. Our new sub-study, ERAD, uses the same approaches to improve the lives of people with autoimmune or inflammatory diseases.

We aim to engage the oncology and clinical community, to recruit and analyse outlier patient cases and exceptional responses to cancer therapy, so that we can learn more about mechanisms driving disease and responses to treatment. By working with clinicians nationally, we develop partnerships to advance our research into more effective biomarkers for predicting patterns of disease and response to treatment, and identifying new cancer therapies.

1Identify biomarkers

In collaboration with clinicians nationally, examine exceptional individual patient cases or responses to treatment, to identify predictive and prognostic biomarkers and new cancer therapies.

2Create a database

Create a public repository of data to advance the use of genomics in treating cancer.

Similar to the study of rare disease, studying rare responses to treatments can provide valuable information and insights. By analysing unexpected and unusual responses to treatment, we can better understand how therapies work, and how we can make them more effective.

Research of treatment outliers can provide information about:

  • new treatment targets
  • predictive biomarkers of toxicity
  • predictive biomarkers of response and non-response
  • prognostic biomarkers

Our program

We partner with clinicians, institutions and patients who are passionate about improving cancer care through precision medicine.

Patient cases that are included in the study form an ongoing collaboration with the referring clinician, to capitalise on the treating clinician’s knowledge and insight. Research output and publications are co-branded and/or co-authored, in recognition of this collaboration.

Sequencing data generated from the project is ultimately made available in a public repository as a research tool and reference dataset, to contribute to the use of genomics in precision medicine.

The Exceptional Responders in Autoimmune/Inflammatory Diseases (ERAD) Program is co-led by Dr. Meg Barnet and Dr. Etienne Masle-Farquhar. ERAD utilises genomics to help understand and better treat immune diseases, in close partnership with the HOPE Program.


We invite clinicians to refer patients they believe meet the selection criteria after discussing the program with their patient. The clinician can refer a patient to us via a standard medical referral. Please refer to the recruitment process for more information.

We seek referrals from clinicians for patients they believe meet the selection criteria, after discussing the program with their patient. The clinician can refer patients to us using a standard medical referral. Referrals can be made via the following:


Fax: 1800-PATIENT

After receiving a referral, we will contact the referring clinician to discuss the case. Following this, we will arrange a telehealth consultation with the patient, to discuss the study and gain their consent.

We will then collect relevant clinical details regarding the case and review the case for inclusion in the study (clinical sub-committees).

If the case is clinically suitable for inclusion, we will contact the patient to obtain a single (one-off) blood sample (2x10mL tubes). We will contact the treating institution to arrange retrieval of archival tissue at the time of analysis.

Once we have the biological materials, we will biobank the samples and review cases with our scientific and steering committees as sub-groups develop, or on a case-by-case basis in some instances.

We will communicate with the referring clinician following the patient consent and at the time of analysis.

What is an exceptional response?

There are many variables of treatment response (related to drug, host and cancer) and mechanisms of response are often multifactorial.

Generally, cases should meet these quantitative criteria:

  • A CR or PR to a drug or other treatment, where <10% of patients would be expected to respond
  • A CR or PR that lasts >3 times the median/expected PFS
  • Best response PD where >90% of patients would be expected to respond.

Qualitative assessment is tumour specific. Cases will be discussed by a tumour-specific sub-committee, taking into consideration aspects such as the natural history of the disease and previous treatment response.

Selection criteria

While there are no firm inclusion or exclusion criteria, the following principles generally apply to patients entered into this protocol:

  • The patient must have a clinically appropriate exceptional response as noted by their treating clinician and agreed to by the steering committee. The exact definition of this is adaptable to the disease.
  • The patient must have normal tissue (e.g. blood, buccal swab) and preferably tumour
  • Patients with non-favourable exceptional response will also be considered for inclusion (e.g. chemotherapy resistance in conventionally chemo-sensitive tumours).

Please contact us for further clarification of the above.



+61 (0)2 9355 5655