Molecular Profiling of Patients with CUP and Rare Cancers
Research type
Research Study
Full title
Personalised Medicine in Patients with Cancer of Unknown Primary (CUP) and Solid 'Rare Cancers' using the Foundation One (FM-1) Panel and Immune Biomarkers
IRAS ID
232783
Contact name
Hendrik-Tobias Arkenau
Contact email
Sponsor organisation
HCA Healthcare UK
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
Cancer of unknown primary (CUP) is a term used to describe a metastatic malignancy without a defined anatomical site of origin. Unfortunately, patients with CUP have limited treatment options and poorer outcomes when compared to patients with a defined tumour diagnosis. Despite the emergence of sophisticated imaging and immunohistochemical testing, CUP is largely treated as a single entity, often using platinum-based combinations.
Several combination chemotherapy regimens have been evaluated, and these have led to a limited range of therapies available for CUP patients. Phase 2 studies of empirical regimens showed response rates of 25 to 35% and survival ranging from 6 to 16 months. Up until now, molecular profiling has been mainly used to identify the tissue of origin when immunohistochemistry cannot clarify the putative primary tumour. Nevertheless, CUP is likely to represent a spectrum of disease subtypes rather than a distinct molecular entity. Therefore, the adoption of both robust immunohistochemical panel and biomolecular profiling may allow the development of a tailored treatment algorithm involving the use of targeted agents.
In addition, patients with solid ‘Rare Cancers’ are facing a similar dilemma, as diagnostic and treatment algorithms are often limited due to the rare frequency of such cancers. Rare cancers are generally classified as a group of rare diseases which is defined in the European Union as diseases with a prevalence of fewer than 5 cases out of a population of 10,000 or as tumours with an incidence of less than 6 per 100,000 cases per year.
Rare cancers are often inadequately diagnosed and treated in relation to both a) lack of knowledge of the biological and clinical behaviour of the disease and b) lack of clinical expertise and available treatment options. These factors can partially explain why patients suffering from rare cancers have worse overall survival when compared with more common cancers.
Improving the outcome for patients diagnosed with CUP or solid cancer is an unmet clinical need. Therefore, molecular characterisation of the disease might have relevant therapeutic implications. In addition with increasing evidence of benefit from immune therapeutics, i.e. checkpoint inhibitors, in various cancers biomarker of response are currently investigated, i.e. PDL-1, MMR status, Tumour Mutational Load and others, and may also be relevant in the biological characterisation of the disease
REC name
London - Queen Square Research Ethics Committee
REC reference
17/LO/1351
Date of REC Opinion
29 Aug 2017
REC opinion
Unfavourable Opinion