Biorepositories help research institutions by providing tissue samples for clinical studies. Human tissue samples play a critical role in disease research by enabling assessments of molecular expression, prediction of toxicity, and identification of biomarkers. They help clarify and expand field-of-use claims, selection of appropriate species for preclinical studies and they assist in the clinical trial stages of drug development. Below is a list of key areas where availability of tissues (both from humans and preclinical species) can support pharmaceutical and other research.
Assessment of Molecular Expression
Biobanks contain vast libraries of human tissue samples, allowing for the assessment of expression levels of biological target molecules such as proteins and RNA. Methods include immunohistochemistry, in situ hybridization, western blotting, PCR and tissue microarrays, all of which can be applied to both normal and diseased tissues to assess expression levels of target molecules. Determining expression levels in a large volume of tissue samples provides critical information to drug developer, allowing an assessment of the appropriateness of a potential drug target. The exclusion of inappropriate drug targets saves millions in funding and years of wasted research.
FFPE DNA and RNA analysis leads the way as a source of comprehensive tissue information. It enables the stratification of tissues, thus advancing our understanding of heterogeneous diseases like cancer that were previously treated without an appreciation of their inherent molecular heterogeneity.
By illuminating altered levels of target molecule expression in organs and tissues outside of those targeted by a drug, data gathered from testing tissue samples can warn researchers of unanctipated toxicity in drugs under development.
In addition to assessing expression levels, human tissue samples provide an excellent source for the identification and clarification of biomarkers. Well-annotated tissues offer an opportunity for disease stratification that can help identify appropriate personalized therapy for patients exhibiting similar biomarker profiles.
Field of Use Claims
By accurately identifying drug targets in tissue samples, targets can then be searched for in well-classified samples from patients with different diseases. The enormous quantities of well-annotated FFPE blocks could serve as a means to expand the use of existing drugs for diseases that exhibit similarities in biomarkers.
Preclinical Species Selection
The selection of appropriate species for preclinical evaluation of pipeline drugs can be aided by tissue procurement from biorepository collections, particularly procurement of FFPE tissue. This is done by analyzing differences and selecting species with the most similar target compound expression profiles, as determined by tissue arrays. Efficiently modeling human diseases assists drug developers to avoid investigating costly, dead-end avenues, testing compounds in preclinical stages that will prove ineffective or toxic in human trials.
Once drug development reaches the clinical stage, embedded tissue blocks can continue to play a critical role in furthering research. Tissue samples enable patient stratification, prognostic assessments and pharmacological studies that would be impractical to perform by acquiring large numbers of trial participants.
While in vitro studies lay the foundation for a biochemical, molecular and genetic understanding of the biology of diseases, human tissue samples provide a source of information from which fundamental knowledge is transformed into actionable information.
Conversant Bio. Well-Annotated Tissue Samples: An Essential Part of Drug Discovery.
Roswell Park Cancer Institute Blog. The Importance of Tissue Samples in Research.