- Person
- Gertrude Elion
- Lived
- 1918–1999
- Field
- Pharmacology
- From
- American
The story
Gertrude Elion is best known for drugs against leukaemia, gout, transplant rejection and herpes, but the same research programme also reached diseases of the tropics and everyday infections. Elion and George Hitchings, who worked together at Burroughs Wellcome from the mid-1940s, built their method on one idea: study how a parasite or bacterium handles the building blocks of its genetic material, then design a molecule that blocks the step it depends on without harming human cells.
In the early 1950s the programme produced pyrimethamine, marketed as Daraprim, against malaria. It works by blocking an enzyme called dihydrofolate reductase, and the Nobel Assembly noted that it is about 2,000 times more toxic to that enzyme in the malaria parasite than in the human host. Today it is used above all, together with other drugs, to treat toxoplasmosis, a parasitic infection.
The same approach led in 1956 to trimethoprim, which binds the bacterial version of the enzyme about 100,000 times more strongly than the human one. It came into use in 1962 and is mainly prescribed for urinary tract infections. The team then made an important discovery: both drugs worked much better when combined with sulphonamides, which block an earlier step in the same pathway. That principle underlies the combination drugs trimethoprim-sulfa, used against many bacterial infections and against a form of pneumonia common in people with AIDS, and pyrimethamine-sulfa, used against malaria.
Elion kept up her interest in malaria long after these drugs reached clinics. She served on several committees of the Tropical Disease Research programme of the World Health Organization and chaired its Steering Committee on the Chemotherapy of Malaria.
Pyrimethamine and trimethoprim are both on the World Health Organization's List of Essential Medicines. They have treated infections in vast numbers of people around the world, including in the poorest regions, and they remain clear examples of rational drug design.
Why it matters
These drugs have treated infections in vast numbers of people worldwide, including in the poorest regions most affected by malaria.
About Gertrude Elion
Chemist without a doctorate who pioneered rational drug design and created drugs for leukaemia, transplants, gout and herpes, sharing the 1988 Nobel Prize in Physiology or Medicine.
More about Gertrude Elion
- Gertrude Elion's 6-MP gave children with leukaemia a chanceIn the early 1950s Gertrude Elion synthesised 6-mercaptopurine, one of the first effective drugs against acute leukaemia, then almost always fatal in children.
- Elion's team created acyclovir, a pioneering antiviralIn the 1970s Gertrude Elion's group developed acyclovir, the first selective and effective antiviral drug, which attacks herpes viruses while sparing healthy cells.
- Gertrude Elion's allopurinol still treats gout todayAllopurinol, developed in Gertrude Elion's laboratory, blocks the build-up of uric acid and remains a mainstay treatment for gout.
- Elion's azathioprine made kidney transplants workableGertrude Elion's laboratory produced azathioprine, the first widely used drug to stop the body rejecting a transplanted kidney from an unrelated donor.
- Researchers Elion trained helped develop AZTAfter Gertrude Elion retired in 1983, scientists she had trained, using her methods, developed AZT, one of the first drugs used to treat HIV/AIDS.
- Gertrude Elion spent her later years mentoring studentsFrom 1971 Gertrude Elion taught at Duke University, mentoring medical and graduate students and publishing more than 25 papers with them.
- Gertrude Elion won a Nobel Prize without a doctorateTurned away from graduate funding, Gertrude Elion never completed a PhD, yet her rational drug design produced leukaemia, transplant and antiviral drugs and won the 1988 Nobel Prize.
Sources
- Gertrude B. Elion Wikipedia
- Gertrude B. Elion – Biographical NobelPrize.org
- The Nobel Prize in Physiology or Medicine 1988: Press release NobelPrize.org
- Gertrude Elion Lemelson-MIT
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