Drug discovery
Drug discovery identifies new medications through screening and optimization.
Drug discovery is the process by which new candidate medications are identified, operating at the intersection of medicine, biotechnology, and pharmacology. Historically, drugs were found by isolating active ingredients from traditional remedies or through serendipitous observations, as with the discovery of penicillin. This early approach, known as classical pharmacology, involved screening substances—whether crude extracts or purified chemicals—in intact cells or whole organisms for a desired therapeutic effect, without prior knowledge of the biological target. Only after an active substance was identified would researchers attempt to determine its target. The modern era began when scientists realized that drug effects result from specific interactions between drug molecules and biological macromolecules like proteins or nucleic acids, leading to the use of pure chemicals rather than crude plant extracts. Examples include morphine from opium and digoxin from *Digitalis lanata*. Later, targeted synthesis of small molecules against known physiological pathways emerged, yielding successes such as Gertrude Elion’s purine analogues (which led to the first antiviral, immunosuppressant azathioprine, and treatments for leukemia, malaria, and gout), James Black’s beta blockers and cimetidine, and Akira Endo’s statins. Following the human genome sequencing, the ability to clone and produce large quantities of purified proteins enabled high-throughput screening of vast compound libraries against isolated biological targets hypothesized to be disease-modifying—a process called reverse pharmacology, now the most common approach. Hits from these screens undergo medicinal chemistry optimization to improve affinity, selectivity, metabolic stability, oral bioavailability, and potency. Modern drug discovery is capital-intensive, funded largely by governments and philanthropic organizations for basic research, and by pharmaceutical companies or venture capitalists for late-stage development. Despite technological advances, it remains a lengthy, expensive, and inefficient process; by 2010, the cost per new molecular entity was about US$1.8 billion. Successful compounds proceed through clinical trials and a regulatory approval process, such as the New Drug Application in the United States. The process also involves complex interactions among investors, industry, academia, pate
- field
- Medicine, biotechnology, pharmacology
- known_for
- Process of identifying new candidate medications
- cost_per_new_molecular_entity_2010
- US$1.8 billion
- common_targets
- Proteins such as GPCRs and protein kinases
- funding_sources_21st_century
- Governments and philanthropic organizations for basic research; pharmaceutical companies or venture capitalists for late-stage development
Lore & Background
Historically, drugs were discovered by isolating active ingredients from traditional remedies or through chance observation, such as the discovery of penicillin. This early approach, known as classical pharmacology, involved screening crude extracts or purified chemicals in intact cells or whole organisms without prior knowledge of the biological target. Only after an active substance was identified would researchers attempt to determine its target. Later, scientists began synthesizing small molecules to specifically target known physiological or pathological pathways, a strategy that produced notable successes in areas like purine metabolism, beta blockers, and statins. After the sequencing of the human genome enabled rapid cloning and synthesis of purified proteins, the field shifted toward reverse pharmacology: high-throughput screening of large compound libraries against isolated biological targets hypothesized to be disease-modifying. Hits from these screens are then tested in cells and subsequently in animals for efficacy. Modern drug discovery involves identifying screening hits, then optimizing them through medicinal chemistry to improve affinity, selectivity, efficacy, potency, metabolic stability, and oral bioavailability. Once a compound meets these requirements, it proceeds to drug development and, if successful, clinical trials. The process remains capital-intensive, lengthy, and inefficient, with low rates of new therapeutic discovery. Basic discovery research is primarily funded by governments and philanthropic organizations, while late-stage development is funded by pharmaceutical companies or venture capitalists.
Reader's Guide
Drug discovery is a capital-intensive process involving large investments from pharmaceutical corporations and national governments. Despite technological advances, it remains lengthy, expensive, and inefficient, with a low rate of new therapeutic discovery. Basic discovery research in the 21st century is funded primarily by governments and philanthropic organizations, while late-stage development is funded by pharmaceutical companies or venture capitalists. Drugs must pass several phases of clinical trials and a new drug approval process, such as the New Drug Application in the United States. The process involves complex interactions between investors, industry, academia, patent laws, regulatory exclusivity, marketing, and the need to balance secrecy with communication. For rare disorders, the orphan drug funding process provides hope for pharmacotherapeutic advances.
Did You Know?
- Historically, drugs were discovered by identifying active ingredients from traditional remedies or by serendipitous discovery, as with penicillin.
- In the 2020s, qubit and quantum computing started to be used to reduce the time needed to drug discovery.
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