Gertrude Elion's journey in scientific exploration began at age 15 when she lost her grandfather to cancer, inspiring her to study science with a chemistry focus. Despite family financial barriers from the economic crash of 1929, Elion was able to attend Hunter College and excelled in her program. She hoped to continue working in laboratories following graduation; however, open positions at the time were not available to women. Instead, she gained experience through teaching and industrial positions during World War II, while saving money to pursue a higher degree.
After successfully completing her Master of Science degree as the only woman in her chemistry class, she found her way into a pivotal collaboration with George Hitchings at a pharmaceutical company. Elion simultaneously pursued her doctoral degree on evenings and weekends for several years. When the program later mandated full-time attendance to complete the degree, Elion opted to forgo this pursuit in favor of continued research with Hitchings, a choice that ultimately proved worthwhile.
Together with Hitchingsand Sir James Black, Elion explored the potential of nucleic acid biosynthesis, particularly purines, for selective inhibition of cancerous cell growth using an innovative method of rational drug design (sometimes referred to as “reverse pharmacology”) instead of the standard trial-and-error technique of the time.
Using this process, Gertrude synthesized two compounds, diaminopurine and thioguanine, that facilitated latching of metabolic enzymes to prevent DNA production. These compounds were used as a first treatment for leukemia. When they were found too toxic for long-term efficacy, Gertrude developed another compound, mercaptopurine, which continues to be used today for the treatment of childhood leukemia and acute myelocytic leukemia in adults.
Elion’s research not only impacted the field of oncology, but she also made discoveries for the treatment for chronic illnesses and the over-all advancement of medicine. She discovered several drugs in her research: allopurinol—a treatment for gout, pyrimethamine—a treatment for malaria, and trimethoprim—a treatment for meningitis, septicemia, and urinary and respiratory infections. A particularly monumental discovery was a zathioprine, which was used as a novel immunosuppressant agent to facilitate organ transplants in immuno compromised patients.
After Hitchings’ retirement, Elion assumed the role of Head of Experimental Therapy and focused her efforts on antiviral work. Her team pioneered the creation of compounds that are able to interfere with replication of the herpes virus, which ultimately led to the synthesis of the first antiretroviral medication used to treat and prevent HIV/AIDs. In 1988, through implementation of rational drug design, this discovery led to Elion, Hitchings, and Black receiving the Nobel Prize in Physiology or Medicine for “discoveries of important new principles of drug treatment.”
Throughout her illustrious career, Elion published 225 papers, served on numerous institutional boards, and garnered several awards, including the Garvan-Olin Medal (1968), the American Cancer Society Medal of Honor (1990), the National Medal of Science (1991), and the Lemelson-MIT Lifetime Achievement Award (1991). In 1991, Elion was the first woman ever inducted into the National Inventors Hall of Fame. Despite her earlier trajectory change, Elion has been awarded 26 honorary doctorate degrees as of 2021.
Elion died in 1999, leaving an enduring legacy as a trailblazer in scientific innovation through her lifelong commitment to medical research and the treatment of cancer and disease.
In recent years, cell and gene therapies (CGTs) have transformed from scientific breakthroughs into commercial realities. These advanced modalities are redefining how we approach rare, genetic, and otherwise untreatable diseases—offering patients options that were once unimaginable. Yet, while the science is groundbreaking, the CMC (Chemistry, Manufacturing, and Controls) pathway remains one of the most significant obstacles to timely regulatory approval.
When it comes to bringing new therapies to patients in the U.S., two key FDA pathways are often discussed: New Drug Applications (NDAs) and Biologics License Applications (BLAs). While both serve the same ultimate goal—getting safe and effective treatments to market—they follow very different paths. Understanding the differences between NDAs and BLAs is crucial for companies developing new therapeutics, particularly as the biotech landscape continues to evolve.
For sponsors developing new therapies, one of the most critical milestones on the regulatory pathway is preparing and submitting a New Drug Application (NDA). While biologics require a Biologics License Application (BLA), small-molecule drugs follow the NDA process.
