Eugene Goldwasser, the University of Chicago biochemist whose agonizingly long but ultimately successful search for a single protein helped launch the biotechnology industry, died Friday in Chicago after a brief illness. He was 88.
The immediate cause of death was renal failure associated with advancing prostate cancer, which he’d lived with for over 20 years. When his kidneys began to fail shortly after Thanksgiving, Goldwasser opted for hospice care instead of dialysis, a procedure revolutionized by his discovery.
Goldwasser, whose government-funded research began as a Cold War experiment to cure radiation sickness, found and purified erythropoietin, or EPO, which is a naturally-occurring hormone produced by the kidneys to stimulate new red blood cell production. Today, genetically-engineered versions of EPO cure anemia in dialysis and cancer patients and generate billions of dollars in sales for Amgen, Johnson & Johnson and Roche.
Unlike its discoverer, EPO has generated numerous headlines over the years. Its high price, which like Goldwasser’s long quest is mostly paid by the government, has generated anger and intense lobbying on Capitol Hill and at the Centers for Medicare and Medicaid Services. It has been used illegally by athletes like Tour de France bicyclists to provide more energy by expanding oxygen-carrying capacity. And, more recently, regulators have issued warnings against EPO overuse after clinical trials showed overdosing raises the risk of heart attacks and strokes in chronic kidney disease patients and premature death in cancer patients.
But none of that was foreseen by Goldwasser when he decided in 1980 to provide California-based Applied Molecular Genetics, later Amgen, with the world’s sole supply of purified EPO, which it had taken him nearly a quarter century to find. Though he disclosed his findings to the University of Chicago as required by his federal funders, the university never patented the discovery.
That decision allowed Amgen scientists to identify the gene that produced EPO, patent its use, and manufacture the protein using the then new technology of recombinant engineering. The company also financed the clinical trials in dialysis patients that led to Food and Drug Administration approval of the first EPO product ’ Epogen ’ in 1989.
But as far as Goldwasser’s long search for EPO that predated those efforts, ’private companies rarely support that kind of research. It takes too long, and the odds of success are even longer,’ I wrote in 2004 in ’The $800 Million Pill,’ whose first chapter documented the biochemist’s quest. ’The Goldwasser-Amgen story provides an excellent opening snapshot of the complicated relationship between basic and applied research in the public and private sectors and shows how private firms rely on public research to come up with important new drugs.’
Born in 1922 in Brooklyn, New York, Goldwasser’s father moved the family to Kansas City during the depression after his clothing manufacturing business failed. Inspired by books like Sinclair Lewis’s ’Arrowsmith’ and Paul de Kruif’s ’Microbe Hunters,’ the teenager studied science at a local community college, which he attended for free, and won a scholarship to the University of Chicago, where he majored in biological sciences.
During World War II, he worked on top-secret programs to discover antidotes to chemical warfare agents and in 1944, moved to Fort Detrick, Maryland, to work on the army’s anthrax program. After the war, he returned to Chicago to get his doctorate in biochemistry, and went to work in Argonne Cancer Research Hospital, which was developing novel approaches to treating leukemia using derivatives of mustard gas, the chemical warfare agent that wiped out infection-fighting white blood cells.
In 1955, the Atomic Energy Commission awarded Goldwasser a grant to identify and isolate the protein that stimulated red blood cell production. ’This was a time when everyone was scared to death and children in the schools were taught to crouch under their desks,’ he told me in an interview in 2002. ’It was a time of foolish panic, but it gave me every young investigator’s dream. I had all the money and space I needed. And I didn’t have to write any reports. I thought it would take about three months.’
Instead, it took 22 years before he published his seminal paper documenting his discovery in the Journal of Biological Chemistry. His efforts to convince Midwestern firms like Parke-Davis in Michigan and Abbott Labs in North Chicago to work on commercializing his discovery fell on deaf ears, thus leading him to Amgen.
In the decades following his major discovery, Goldwasser’s research focused on the cells inside the kidney that produced EPO. He hoped to discover mechanisms for repairing damaged kidneys, which would make use of artificial EPO unnecessary. In 2003, he closed his lab and retired after the National Institutes of Health lost interest in his work.
Goldwasser is survived by his second wife Deone Jackman; two stepchildren, Tara and Tom; and three sons by his first marriage, Thomas, a rare book dealer in San Francisco, Matthew, an education consultant in Chicago, and James, a rare book dealer in New York. He also has five grandchildren. His first wife Florence died in 1981 of leukemia.
Merrill Goozner is an award-winning journalist and author of “The $800 Million Pill: The Truth Behind the Cost of New Drugs” who writes regularly at Gooznews.com.