The discovery of reliable blood biomarkers to diagnose cancer in a non-invasive way remains one of the critical challenges in cancer research. Furthermore, a main problem of existing biomarker research is the lack of standard design for clinical trials for biomarkers evaluation, unlike the standard randomized control trials for therapeutics evaluation. Since a primary objective of Project AiPBAND is to establish diagnostic and prognostic biomarkers for brain cancer, a clinical trial will be designed to be a tool for the clinical validation of biomarkers that will be discovered during the project.
This goal carries a lot of hope to improve the diagnosis of the disease, but ensuring that biomarkers are clinically useful is a complicated process.
The clinical validation process will be divided into several steps. Initially, a cohort of samples will be analyzed in an attempt to discover a new biomarker. Then, the outcomes from the first step will be validated in an independent sample cohort. After that, further analysis will be done to confirm that the clinical decision-making process will be enhanced as a result of the introduction of the biomarker and it will have a meaningful impact on the lives of brain cancer patients.
Two of the ESRs are in the process of designing a pilot study for one of the biomarkers for brain cancer diagnosis, and we have updates from both of them:
My project is focused on the identification of novel circulating/serum miRNAs as non-invasive diagnostic biomarkers for brain cancer patients. In an initial pilot experiment, we have identified some microRNAs dysregulated in the serum of GBM patients with respect to controls, which could be a potential diagnostic signature. Now, we are validating these miRNAs and studying their role in brain tumors. Moreover, I am setting a new NGS analysis in serum samples, of a larger cohort of IDH1 wild-type versus IDH1 mutant brain tumor patients (including different glioma grades, relapses and meningiomas) with the aim to identify serum miRNAs as non-invasive diagnostic biomarkers able to distinguish glioblastomas from lower grade gliomas.
Ana Belen Diaz, ESR-6 at National Cancer Institute Regina Elena, Rome, Italy
The ultimate objective of my project is to provide clinical data regarding the newly identified biomarkers. To achieve this goal, a clinical trial will be designed to compare the concentration of the biomarkers in the blood between brain cancer patients and healthy individuals. This study must follow the Good Clinical Practice (GCP) principles. GCP is an international ethical and scientific quality standard for the design, conduct, performance, monitoring, auditing, recording, analyses and reporting of clinical trials. It also serves to protect the rights, integrity and confidentiality of trial subjects. As part of my project, I am spending two months secondment at Plymouth University, UK. The objective of this secondment is to design a pilot study for some of the discovered biomarkers; this will give us the chance to assess the suitability of the study protocol and the statistical analysis which will be used for the clinical validation of the biomarkers.
Ahmed A. Ali, ESR-14 at the MTA Group in Italy