Gertrude Elion: Forging the path towards a cure for cancer

by Isabella Fraschilla

Despite never receiving a PhD, Gertrude Elion was awarded the 1988 Nobel Prize in Medicine. She shared this prize with Sir James Black and her mentor George Hitchings for revolutionizing pharmacology by rejecting the standard trial-and-error approach for rational drug design. Their critical work in understanding drug metabolism and physiological processes resulted in the rare Nobel Prize awarded to employees of a pharmaceutical company. Furthermore, the Nobel Prize Committee believed that each of the drugs Elion developed was worthy of a Nobel Prize, underscoring her role in modern medicine.

Born in 1918 in New York City, Gertrude “Trudy” Elion was raised by her Lithuanian and Russian immigrant parents and close grandfather. When she was a teenager, her grandfather died of stomach cancer, prompting Elion to study science in search of a drug to cure cancer. She attended Hunter College on a full merit scholarship – a necessity as the stock market crash of 1929 left her family bankrupt. After obtaining her bachelor’s degree in chemistry, Elion wanted to pursue a doctoral degree. However, at this time during the Great Depression, the limited fellowships available were offered to men. Resolute in her scientific goals, Elion worked as a secretary, taught biochemistry to nurses, and worked as an unpaid lab assistant to support herself and gain experience. Then World War II created labor shortages in the pharmaceutical industry, which became more willing to hire women scientists. Elion went “full speed ahead.”

Dr. George Hitchings hired Elion as a research assistant and mentored her at Burroughs Wellcome, now GlaxoSmithKlein. They hypothesized that they could treat diseases based on differences in the types of DNA building blocks used by human cells versus bacteria, viruses, and parasites. Furthermore, they thought that the demand for these DNA building blocks would be greater in rapidly growing cancer cells compared to non-dividing healthy cells. In the 1940s, the DNA structure had not been published yet, but they knew that one key component of DNA was a purine molecule. Elion developed the synthetic purine, termed 6-MP, to trick rapidly growing cancer cells into incorporating this purine-like molecule into their DNA. In this way, 6-MP could obstruct cancer growth while sparing most healthy cells that do not grow as quickly. Unfortunately, normally proliferating cells, such as cells of the immune system, hair follicles, and digestive system, are also temporarily affected by this drug, explaining many of the common side-effects associated with chemotherapy. Elion’s 6-MP propelled the early development of chemotherapy, as it was the first drug to successfully treat childhood leukemia, and it is still commonly used in chemotherapy regimens today.

In addition to 6-MP, Elion discovered many other life-saving compounds based on the purine structure. For example, she designed a drug that suppresses overactive immune systems during organ transplantation, enabling doctors to perform the first kidney transplants. After her early success in Hitchings’s lab, Burroughs Wellcome promoted Elion to lead the new Department of Experimental Therapy. In this role, she shifted to studying how pathogens rely on purine derivatives that human cells don’t need. Using this knowledge, Elion discovered drugs to treat malaria, bacterial infections, and genital herpes.

After retiring in 1983, Elion continued to teach and mentor scientists until she died in 1999. Indeed, her former team at Burroughs Wellcome credited her mentorship when they discovered the first drug that could prevent HIV replication in AIDS patients. She enjoyed mentoring young scientists and advised them that “They must not get discouraged. They must not let other people discourage them, and they must not get discouraged by themselves.” Dr. Elion received 26 honorary doctorate degrees, the National Medal of Science, admission into the Royal Society, and the Nobel Prize in Medicine. She was the first woman inducted into the National Inventor’s Hall of Fame.

Isabella Fraschilla is a Ph.D. student in the Immunology Program at Harvard Medical School. She is studying how immune cells regulate commensal gut bacteria.

Photograph of Gertrude Elion is licensed under CC BY 4.0.

Cover image by Arek Socha from Pixabay.

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