Medicines

Medicines

  • Medicines are substances or preparations used in treating disease.
  • The development of a medicine involves multiple stages including discovery, testing, and approval.
  • Discovery often involves identifying a Target Molecule which has a key role in the disease condition.
  • Once a target molecule has been identified, Lead Compounds that can interact with it are developed. These are often discovered by screening multiple compounds for their effect on biological systems.
  • Once a potential lead compound is identified, it undergoes several rounds of optimization, to enhance its effectiveness and decrease any harmful side effects. This process involves making modifications to the chemical structure of the compound.
  • The medical compound is then subject to Preclinical Testing, where its safety and effectiveness are evaluated. This often involves testing in laboratory animals.
  • If the results of preclinical testing are satisfactory, the compound proceeds to Clinical Trials. These involve testing on small groups of human volunteers to assess its safety, dosage, and efficacy.
  • Clinical trials are conducted in several phases, each with an increasing number of participants. Phase I tests for safety, Phase II tests for effectiveness and continues safety assessments, and Phase III compares the new medicine to current treatments or placebos.
  • If the medicine shows positive results in clinical trials, it is submitted for approval by regulatory authorities, who decide if the medicine can be made commercially available.
  • After a medicine has been approved and released to the market, it continues to be monitored for any potential side effects or long-term impacts in a stage known as post-marketing surveillance.

Pharmacophores

  • A Pharmacophore is an abstract description of molecular features which are necessary for molecular recognition of a ligand by a biological macromolecule.
  • They are usually represented as a three-dimensional arrangement of features in space.
  • The pharmacophore concept is used in the design and optimization of new drugs by identifying the characteristics of the drug necessary for it to bind effectively to its target.
  • The concept of a pharmacophore can also be used to compare different drugs which have similar effects to identify common structural features.

Quantitative Structure-Activity Relationships (QSAR)

  • Quantitative Structure-Activity Relationships (QSAR) are mathematical models that relate a quantified physiological effect of a drug to quantifiable features of its chemical structure.
  • QSAR models are used to predict the activity of new drugs, based on their similarity to known drugs.
  • The goal of QSAR modeling is to identify a relationship or pattern between structural features of drugs and their physiological effects. This can then be used to design more effective drugs or improve the characteristics of existing drugs.
  • QSAR modelling involves the use of statistical methods and machine learning algorithms.

Drug Delivery Systems

  • The effectiveness of a medicine is not only determined by its ability to interact with its biological target, but also by the efficiency of its delivery to that target.
  • There are several types of drug delivery systems that have been developed to improve the delivery of medicines to their respective targets.
  • These include: Oral delivery systems (taken by mouth), Intravenous delivery systems (injected directly into the bloodstream), Inhalation delivery systems (inhaled into the lungs), and Topical delivery systems (applied directly to the skin or mucous membranes).
  • The type of drug delivery system used depends on the specific medical condition being treated, the type of medicine, and the desired outcome of the treatment.

Drug Resistance

  • Drug resistance is a significant challenge in the treatment of many diseases. It occurs when a disease-causing organism or cancer cell changes in a way that reduces or eliminates the effectiveness of a drug.
  • Mechanisms of drug resistance include alteration of drug targets, activation of drug efflux pumps, and metabolic inactivation of the drug.
  • Strategies to overcome drug resistance include the development of new drugs, combination therapies, and strategies to block resistance mechanisms.