Biomarker Discovery and Validation
Biomarkers provide vital information regarding safety and efficacy during drug discovery and development. They are loosely defined as diagnostic, prognostic, or predictive and can be used to select patients for clinical trials or monitor patient response and treatment efficacy. In addition, after regulatory approval, biomarkers can be used to identify patient populations that would benefit from a particular therapy along with continuing to monitor the treatment’s efficacy. Show more
Biomarkers are used in research to design and develop more effective therapeutics, to assist in guiding decisions about which candidates to develop further, as surrogate markers for the selection of patients, and as endpoints for both benefits and risks. There are a wide range of types of biomarkers that can be monitored, such as predictive, prognostic, pharmacodynamic, safety, and efficacy markers. One class of biomarkers that is actively being researched is safety biomarkers. These markers are being developed to help determine which drug candidates are most likely to be safely tolerated in humans.
From Discovery to Validation
Biomarkers range from specific molecules such as a protein, gene sequence, or expressed RNA to physiological parameters such as blood pressure or body temperature. They may be expressed in a specific location or tissue or found in serum, plasma, and other bodily fluids. Because of this diversity, most successful biomarker programs have a broad reach and investigate cellular, proteomic, and genetic sources in order to find the most valuable biomarkers.
Validation of biomarkers requires that their levels change in response to a disease, condition, or other parameter in a consistent and statistically significant manner. Identification and characterization of biomarkers can reduce the number of candidate drugs that fail clinical trials, aid in the discovery of more effective therapeutics, and provide methods for monitoring animals in preclinical research and evaluating the response of human participants in clinical trials.
Both gene expression and the presence of specific genetic sequences are used as biomarkers for many diseases including cancer. Treatments can even be tailored to an individual patient based on these genomic biomarkers. Typically, large-scale gene expression studies are conducted to identify genes that are up- or down-regulated in a particular indication or disease. These studies rely on the ability of the researchers to design exploratory and confirmatory panels as well as on powerful software tools to analyze resulting data and identify potential biomarkers.
Interrogating biological samples to identify proteomic profiles gives rise to a plethora of potential biomarkers. Proteins are vital, functional parts of living organisms and as such reflect what is happening in that organism. Monitoring the post-translational state of proteins of interest can yield important biomarkers and indicate tumor progression.
The ability to uncover novel and rare cell types through multiparameter analysis allows researchers to characterize cell populations in greater detail and identify subpopulations that serve as markers for disease. Simple and reliable isolation of these cells for downstream assays further supports the cellular biomarker workflow by providing pure populations for genomic and proteomic analysis. Show less
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