PRECLINICAL
STUDIES
Simranjeet Kaur Diwan - 156
Msc II, Sem 4
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Preclinical Studies: Laying
the Foundation for Clinical
Trials
Preclinical studies are the foundation of clinical trials. They are conducted before
human trials. The goal is to assess the safety and efficacy of a therapy. A key
question is if the compound is promising enough for human testing. These
studies follow FDA and IRB guidelines, with ethical considerations.
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Types of Preclinical Studies: In Vitro Assays
Cell-Based Assays Biochemical Assays Advantages
Cell-based assays examine drug effects on Biochemical assays analyze interactions Advantages include high-throughput
cells in petri dishes. Examples are cancer between drugs and molecules. Interactions screening and mechanistic insights. These
cells (HeLa, MCF-7, A549) and neurons. with proteins and enzymes are tested. assays are also cost-effective.
These in vitro assays are experiments outside a living organism. Testing a new antiviral compound's ability to inhibit viral replication in cultured
cells is an example.
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Types of Preclinical Studies: In
Vivo Animal Models
1 Animal Models 2 Endpoints Measured
Non-Rodents
Animal models use animals to Pharmacokinetics,
simulate human diseases. Mice, pharmacodynamics, toxicology,
rats, and pigs are commonly used. and efficacy are measured. These
indicate the safety and
effectiveness of the treatment.
Rodents
These in vivo assays are experiments conducted on living organisms. This provides a
more complete picture.
Zebrafish
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Preclinical Tests Overview
1 Pharmacokinetics (PK) 2 Pharmacodynamics (PD) 3 Toxicology Studies
ADME: Absorption, Mechanism of action and Acute, sub-chronic, chronic
Distribution, Metabolism, biological effects toxicity testing
Excretion
4 Genotoxicity Tests 5 Reproductive Toxicity
Evaluates DNA damage and cancer risk Effects on fertility and fetal development
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From Preclinical to Clinical: The Transition
IND Application
1 Submit to the FDA
Go/No-Go
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Is drug safe?
Data Integration
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Combine all data
Data from in vitro, in vivo, and toxicology studies is integrated. Challenges include predicting human response and scaling up
manufacturing.
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The Importance of Rigorous
Preclinical Research
Crucial for Drug Optimize Drug Design
Development
Helps in improving clinical trial
Provides essential information protocols for better results.
about safety and efficacy.
Ethical Considerations
Regulatory compliance are of utmost importance in research.
Investing in high-quality research increases clinical trial success. Ethical
considerations and regulatory compliance are essential.
Referen
ces
• Vermetten, E. and Bremner, J.D. (2002), Circuits and systems in stress. I. Preclinical studies.
Depress. Anxiety, 15: 126-147.
• FDA: Step 2 – Preclinical Research:
https://www.fda.gov/patients/drug-development-process/step-2-preclinical-research
• NCATS Toolkit: Cell and Animal Models:
https://toolkit.ncats.nih.gov/module/discovery/developing-translational-research-tools/cell-a
nd-animal-models/
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