- Person
- Gertrude Elion
- Lived
- 1918–1999
- Field
- Pharmacology
- From
- American
The story
In the late 1940s a diagnosis of acute leukaemia in a child usually meant death within months. Gertrude Elion had joined the Burroughs Wellcome laboratory in Tuckahoe, New York, in 1944 as assistant to George Hitchings. Hitchings wanted to develop drugs by deliberately imitating natural compounds rather than by trial and error, and he hoped that cancer cells could be tricked into taking up false building blocks for their growth and destroyed without harming normal cells.
Elion was assigned to the purines, two of the building blocks of DNA and RNA, and she began making antimetabolites, molecules similar enough to the natural purines to be taken up by dividing cells yet different enough to jam their machinery. Very little was then known about how cells made nucleic acids, and she later compared each series of experiments to a mystery story. In 1948 she and Hitchings found diaminopurine, which showed promise against experimental leukaemia, but it proved too toxic for patients.
They kept going. In the early 1950s Elion synthesised two new compounds, thioguanine and 6-mercaptopurine. Working with the Sloan-Kettering Institute, doctors tried 6-mercaptopurine in leukaemia patients who no longer responded to methotrexate, and about a third of them went into complete remission. The drug, sold as Purinethol, was approved in the United States in 1953 and was patented in 1959. The National Inventors Hall of Fame describes it as the first major medicine to fight leukaemia.
Used at first on its own, 6-mercaptopurine produced remissions that had rarely been seen before, and it became part of the combination regimens that doctors built up over the following decades. Those combinations changed the odds completely. A disease that was once almost uniformly fatal in children is now one that most survive, and 6-mercaptopurine is still used, usually together with methotrexate, in the treatment of acute lymphoblastic leukaemia.
The drug also led to others. Efforts to improve and prolong its action produced azathioprine for transplant patients and allopurinol for gout. Mercaptopurine is on the World Health Organization's List of Essential Medicines, and it remains one of the clearest examples of how Elion's purpose-built chemistry saved lives.
Why it matters
Generations of children have survived leukaemia with the help of a drug Elion designed, and her approach showed that cancer could be attacked with purpose-built chemistry.
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
- 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.
- Elion's chemistry also fought malaria and infectionsGertrude Elion's work with George Hitchings contributed to pyrimethamine against malaria and trimethoprim against bacterial infections.
- 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 Elion Jewish Women's Archive Encyclopedia
- Gertrude B. Elion – Biographical NobelPrize.org
- The Nobel Prize in Physiology or Medicine 1988: Press release NobelPrize.org
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