unit 1 - med chem

• Medicines are amongst the most widely used products on the planet
• The average cost of developing a new candidate drug is £515 million
• Only 10% of molecules make it to market due to high attrition rates
• Potential pitfalls in drug development include severe side effects, delivery complications, or lack of intended efficacy
• Medicinal chemists update drug design rules and tactics to maximize a molecule's success
• Medicinal chemistry is an interdisciplinary field combining aspects of organic chemistry, physical chemistry, pharmacology, microbiology, biochemistry, and computational chemistry
• It focuses on the discovery, design, synthesis, and interactions of pharmaceutical agents with the body
• Mainly concerned with small organic molecules, both natural and synthetic
• Compounds in clinical use are primarily small organic compounds
• Other compounds used in medicine include organometallic compounds, biopharmaceuticals, and inorganic compounds
• Chemotherapy is the therapeutic use of chemical agents to treat diseases
• It involves administering cytotoxic drugs to destroy or inhibit the growth of malignant cells, commonly used in cancer treatment
• Chemotherapy can be used alone or in conjunction with surgery or radiation
• Discovery and Design in medicinal chemistry
• A typical pharmacy contains about 2000 medications
• Most medicines contain a single active ingredient, usually an organic compound
• Medicinal chemists aim to find compounds with potent effects on diseases and minimal side effects
• Successful drugs must be selective, reach the correct cells, and react as intended
• Sources of drugs
• Herbs used historically can provide starting points for medicines, like quinine from cinchona tree for malaria
• Marine chemistry is a promising source of drugs, with compounds like Bengamide A showing anti-tumor properties
• Rainforests and venoms are rich sources of potential drugs, with plant species and snake venoms providing lead compounds
• Microorganisms like bacteria and fungi have been sources of antibacterial drugs since World War II
• Drug targets in the body
• Main drug targets are macromolecules like nucleic acids (DNA, RNA) and proteins (enzymes, receptors)
• Drugs bind to targets in regions called binding sites, interacting through intermolecular or covalent bonds
• Lead compound in drug development
• Lead compounds serve as prototypes for designing biologically active compounds
• Analogues of lead compounds are synthesized to improve therapeutic properties or reduce side effects
• Molecular modification involves changing functional groups and structure to enhance drug effectiveness
• Computational chemistry in drug discovery
• Computer-aided molecular modeling helps biochemists determine precise enzyme and receptor structures
• Computer-generated models of active compounds are matched with enzyme sites to identify target molecules for synthesis and testing
• Drug Development Process
• Drug discovery involves identifying potential new medicines through various scientific disciplines
• Drug development phases include discovery, preclinical studies, clinical development, and market approval
• Critical steps in the FDA drug development process include discovery, preclinical research, clinical development, FDA review, and post-market safety monitoring
• Pre-clinical studies in drug development
• Preclinical phase involves in vivo research to determine drug efficacy and safety
• Researchers assess absorption, distribution, metabolism, excretion, benefits, side effects, and interactions with other treatments