Nobody has probably ever heard of Eugene Goldwasser, a retired professor from the University of Chicago. This is not strange as he is neither a Nobel laureate nor an eminent figure in his field of science. However, his name definitely sounds more familiar if we mention that he dedicated several decades of his scientific career to finding the molecule of erythropoietin, commonly known as EPO. Erythropoietin is a hormone that promotes the formation of red blood cells within an organism or, in simpler terms, provides cells with a better supply of oxygen. This, in turn, improves the endurance of athletes, making the hormone a popular prohibited stimulant which has recently also caused problems for the cycling champion Lance Armstrong.
In search of a cure against the effects of radioactivity
The story about Eugene Goldwasser, recently popularized by Merrill Goozner in his resounding book The $800 Million Pill – The Truth Behind the Costs of New Drugs (University of California Press, 2004), is interesting from several points of view.
On the one hand, it tells the story of a typical scientist who was so much drawn to his research that he persisted in continuing his work despite decades of failed attempts and dead ends until all the long years of hard labor finally led him to an important scientific discovery. In the late 1970s, after decades of experimenting with sheep blood and after collecting and dehydrating 2550 liters of urine, he succeeded in producing 8 milligrams of pure natural human erythropoietin.
On the other hand, it also tells the story about one of the first great commercial breakthroughs resulting from the biotechnological revolution in the field of drug production. Today, pharmaceutical companies produce synthetic EPO in large quantities and sell it for similarly large amounts of money. The biotechnology company Amgen, one of the main producers of EPO, creates more than a half of its multibillion dollar yearly profit on this hormone alone.
Unfortunately, Goldwasser does not receive his share of this enormous heap of money, founded on the production of “his” molecule, even though with more than twenty years of enduring work he made the largest contribution to isolating this molecule from the mass of others, found in the human body. As he failed to patent his discovery at the time (he wrote to state financiers, asking them to take care of his patents, but received no response), he has to be content with the thirty thousand dollars graciously deposited to the account of his laboratory at the University of Chicago by Amgen each year.
The story about the discovery of erythropoietin, not unlike many other stories of science in the 20th century, begins during the Cold War, an era extremely generous to scientific research. Soon after the Second World War, young Goldwasser was invited to join a research group commissioned by the state to discover ways of defending against the consequences of a nuclear war. The government was interested in finding an antidote to the deadly effects of nuclear radiation on the human body. It was in the beginning of the 20th century when scientists had already come to understand that blood has to contain a substance which instructs the bone marrow to produce red blood cells that carry oxygen around the body. Scientists named this molecule erythropoietin after the term for the process of red blood cell formation called “erythropoiesis”, even though they knew nothing about the molecule itself at the time.
Looking for a needle in the haystack
In 1955 Goldwasser was entrusted with a research mission to find the erythropoietin molecule and discover a way to produce it in large quantities, so it could be used to cure radiation sickness. The search took longer than they expected, it extended over more than two decades. However, if one takes a closer look at how demanding the job Goldwasser and his colleagues had taken on, the time spent to complete the task does not seem to be quite as long anymore. A healthy person produces two to three million red blood cells each second which adds up to nearly half a ton of blood in a lifetime, yet in the same period barely enough erythropoietin to make a tiny pill goes through the human body. The search for a molecule they knew almost nothing about was therefore far from simple and could be compared to, for instance, looking for a lost coin on a long sandy beach.
During the first years of his research, Goldwasser wanted to find out which part of the body actually created EPO. His assistants carefully removed organs from lab rats until they were certain that it was the kidneys that were responsible for the bad blood. In the next phase they injected sheep, waiting to be slaughtered, with a chemical that destroyed all the red blood cells in the test animals. They were convinced that the destruction of the red blood cells would result in a mass production of EPO which could in turn be detected in the blood serum of these “sacrificial lambs”. They then injected the serum into the anemic sheep and looked for an increase in the number of blood cells in their blood streams. Unfortunately, there were no visible results, even though the experiments had already been going on for more than a decade. It was obvious that they had reached a dead end. When they were already on the edge of despair another research group fortunately published its discovery, revealing that the excess EPO was not to be found in blood, but in urine. After fifteen years of hard work it must have been quite depressing to find out that you have been looking in the wrong place all along. Nonetheless, they at least had a new goal.
A stroke of good fortune from the Far East
The lucky side of this misfortune was that a Japanese researcher Takaji Mijake offered Goldwasser his help in collecting urine samples from anemic patients in his neighborhood who supposedly produced excess EPO naturally due to their illness. In just a few years, he collected and dehydrated 2550 litres of urine. In 1975, when the two scientists first met in the lobby of a Chicago hotel, Mijake bowed solemnly to Goldwasser and handed him a large package wrapped in Japanese silk. Inside the neatly wrapped container was a priceless treasure, at least for the two scientists: a great amount of dehydrated, powdered urine. After a complex process of purification, Goldwasser and his colleagues managed to isolate 8 milligrams of pure human EPO from the collected urine. In August 1977 they revealed their discovery in scientific literature. Goldwasser and his team were overwhelmed with joy.
The goose that laid golden eggs
The story does not end here, though. In order to use EPO as a medicine it was necessary to find a way to produce it in large amounts outside the human body. Goldwasser was confident in the potential of his discovery, but at first no one took him seriously. He went from company to company, from one investor to the other, but did not succeed in getting anyone’s attention. Fortunately, his search coincided with the beginning of the biotechnological revolution. It was at that time when they started to produce synthetic insulin with the help of bacteria cultures to cure diabetes patients. The more far-sighted entrepreneurs gradually came to understand that investing in biotechnology would earn them good money. Some of them are already enjoying their profits today.
After many difficulties, that over the years began to spread increasingly from the fields of science to the courts of law, EPO became one of the first biotechnological geese that steadily laid golden eggs. Unfortunately, all these heaps of money do not always lead to heaps of new scientific discoveries, but mostly to thinking up new ways to increase the already substantial amounts of cash. Amgen, after EPO’s enormous commercial breakthrough in the 90s, was once said to have been quickly transformed from a powerful research group into an outstanding law firm that just happens to hide, somewhere deep in its cellars, a department for medical research.
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