In the discover y of drugs, cellular and genetic factors that play a role in specific diseases are identified by scientists, chemists and pharmacologists. A search is carried out for chemical and biological substances targeting biological markers and likely to have drug-like effects.
Out of the many new compounds identified during the discovery process, a few are clinically evaluated and considered safe for testing in humans. Further clinical testing is done and effective compounds are approved for treatment.
There are many new scientific approaches being used to identify targets and obtain lead compounds. They include the use of synthetic chemistry, genomic and proteomic technology, laboratory automation, recombinant DNA and bioinformatics.
a. Physiology based drug discovery: It follows physiological readouts where compounds are screened and profiled based on the read out. Purely physiology-based approaches jump right into screening, identification of drug target and action mechanism follows in later stages. This is done through deduction based on the specific pharmacological properties that lead compounds poses.
b. Target-based drug discovery: Identification of the function of a possible therapeutic target and its role in disease is done first. This is sometimes a difficult task, given the huge number of human or pathogen genes and the different types of their gene products.
Understanding the mechanism of a disease guides research and develops a possible treatment to reverse the disease process. Disease mechanisms can be classified into genetic disorders, infection by bacteria, fungi or viruses, immune or autoimmune disease, trauma and acute disease based on organ failure or injury and multicausal disease.
Particular gene defects that cause hereditary disorders have been identified in a number of diseases. Advancements in DNA sequencing technology have facilitated prompt identification of disease genes through genetic