Clare Scott, ANZGOG MBBS PhD FRACPPrimitive Neuroectodermal Tumor . Spinal cord . Mucinous adenoca...

Collecting specimens across international boundaries

Clare Scott, ANZGOG MBBS PhD FRACP

55% of gynaecological cancers are rare

Best practice

Experience from TCGA particularly with governance

Infrastructure for coordination of Rare Cancer research

eg IRCI, TCGA NEED A RARE CANCER MTA TO AID PRE-CLINICAL

RESEARCH

Identification of patients with rare cancers Cart-Wheel.org

IRCI is a joint initiative: • National Institute for Health Research (NIHR) Cancer Research

Network (NCRN) and Cancer Research UK (CR-UK) the National Cancer Institute (NCI) European Organisation for Research and Treatment of Cancer (EORTC).

• The aim is to facilitate the development of international clinical trials for patients with rare cancers in order to boost the progress of new treatments for these patients.

• The initiative hopes to encourage the use of innovative methodologies to maximise the potential for answering research questions and to identify and overcome barriers to international trials to allow international collaborative trials to run smoothly

• Other partners

Criteria for inclusion in the IRCI programme Rarity • Not fixed, ~a total incidence of less than 2/100,000 • Occasionally, rare clinical scenarios have also been considered. • So far, excluded rare molecular sub-types of common cancers, because

this embraces most of cancer. However, a rare molecular sub-type could be considered if it is a distinct, prospectively identifiable rare sub-group with a strong rationale for separate research.

Lack of existing trials Potential for an interventional trial • Priority is given to cancers with potential for an interventional – usually

randomised – trial (not an audit, registry or non-trial tissue collection). • sufficient patients for an international trial to be feasible. Enthusiastic champions

Governance and Ethics

Ethics – HREC/IRB • Institutional variation • Coordinated central approval in some regions Governance • Linked to Ethics, institutional variation • Use of tissue or data inevitable “ownership” issues • PI vs Institution vs company • Results in FAILURE OF RESEARCH PROJECTS

Best practice

Experience from TCGA particularly with governance

Infrastructure for coordination of Rare Cancer research

eg IRCI, TCGA, Clinical trials NEED A RARE CANCER MTA TO AID PRE-CLINICAL

RESEARCH

Identification of patients with rare cancers Cart-Wheel.org

There are many more patients with rare ancers than an be coverd by sepcific clinical trials How do we ensure they are not left totally in the dark? How do we generate a starting point for research for other rare cancer types?

Identification of patients with rare cancers Cart-Wheel.org

Approved by Melbourne Health Human Research Ethics

Committee (HREC / IRB)

HREC approved (Melb Health)

Provides information to consumer about research Designed with consumers Patients or their representative can: • register, complete questionnaire and consent form

How does CART-WHEEL.org work?

Data obtained focuses on: • accurate diagnosis • location of pathology report • location of biopsy / surgical block Collection of treatment, toxicity, follow-up data General morbidity, family history of cancer Molecular testing of patient/family or tumour

How does CART-WHEEL.org work?

Not for profit organisation A secure research platform and infrastructure that

provides access to real-time clinical, imaging and biospecimen data across jurisdictions, institutions and diseases.

The web-based platform provides ethical access while protecting both privacy and intellectual property.

Developed by BioGrid Australia Ltd

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Patient or their proxy: Download the PICF Read and choose level of consent Print, sign and post PICF (to BioGrid)

Choice of level of consent

Bone 7%

Connective tissue 20%

Neuroendocrine 8%

Endocrine 4%

Exocrine 1%

Fibromuscular 2%

Gastrointestinal 8%

Breast 14%

Haematology 11%

Liver 1%

Neurological 7%

Ovarian 8%

Gynaecological (non-ovary)

3% Skin 2%

Urogenital (non-gynae)

4%

Consented patients in Cart-Wheel.org

Sarcoma Waldenstrom macroglobulinemia

Neuroendocrine Breast Triple Negative

Breast other Phylloides

GIST HEARD

Thyroid Desmoid

Primitive Neuroectodermal Tumor Spinal cord

Mucinous adenoca incl OC Granulosa cell tumor ovary

Ovarian cancer Fallopian Tube carcinoma

Vulval carcinoma Bone (non-sarcoma)

Cholangiocarcinoma Adrenal

BCCs / Gorlins Bladder

Pilot study of first 100 patients consented

Pilot study of first 100 patients consented

49 patients (51%) received chemotherapy

Pilot study of first 100 patients consented

29 patients (30%) stated they had undergone genetic testing 8 patients (8%) had a known mutation

Quality assessment of consumer-entered information

(comparison with a quaternary hospital Petermac sarcoma database)

22 patients:

Reasonable accuracy of data entered by 22 patients (pt aware of updated data)

Quality assessment of consumer-entered information

(comparison with sarcoma database)

Details of histologic diagnosis of “sarcoma”

18 patients (83%) with “correct” diagnosis despite “sarcoma” diagnoses being complex

Advantages for Consumers

Learn about research Contribute their data for research Signal their wish to be involved in research Contacted for a clinical trial or research study Print off their pdf summary anytime Help to drive the direction of research into areas

which currently are “too hard”

Access to patient data and location of tissue: Includes field for Biobanking Routinely available FFPE blocks will become more and more

useful… Patient-specific consent, data-entry are streamlined Need to have an HREC-approved study: can include an

additional consent form to be sent to pt for access to tissue OR request IRB waiver of consent to access tissue for low impact study of rare cancer (which wouldn’t be possible otherwise)

Advantages for Researchers

Process for data access Researcher applies to BioGrid for access to CART-WHEEL data: • IRB-approved project (incl international IRB) • Description of type of patient, type of data/tissue • Data can be made available on a regular basis eg nightly,

once approved Project review – BioGrid/Cart-wheel committee Approval – help with preparation of access proposal, google

clicks to improve patient identification

Challenges

Encourage consumers and researchers to use this approach - uptake

Ascertainment bias – but alternative approaches

make identification and coordination of rare cases even more difficult

Cart-Wheel project: High-grade mucinous ovarian cancer

CART-WHEEL on Facebook

CART-WHEEL on Twitter: @CARTWHEELcancer

• Vehicle for rare tumor research

• Existing projects which need increased numbers or data-entry

• Projects which previously were not feasible due to small samples size

CART-WHEEL.org

Try out the questionnaire

Logon demo Password testuser

CART-WHEEL.org

The team at CART-WHEEL and BioGrid Our Consumers

• Email: contact@cart-wheel.org • Web: www.cart-wheel.org • Twitter: @CARTWHEELcancer • Facebook page: CART-WHEEL Rare Cancer

• Clare Scott scottc@wehi.edu.au