Putting your medicine where your mouth is

Last week was science’s High Holy Days, as Nobel Prizes were awarded for significant contributions in physiology or medicine, physics and chemistry (among other awards for literature and peace). Part of the prize for physiology or medicine was awarded to Ralph Steinman, a noted cell biologist from Rockefeller University, for his discovery of dendritic cells, a critical part of the immune system. There was some controversy regarding his inclusion in the prize, as Steinman had died a few days before the announcement and Nobel Prizes are not awarded posthumously. In the end, the committee decided that since they were not aware of Steinman’s critical medical condition before a decision was made, his estate would keep the award.

In light of this brief controversy, it was revealed that Steinman had been battling pancreatic cancer — a cancer with a five-year survival rate of only 6 percent — for four years. What’s more, Steinman had created some experimental treatments based on the very dendritic cells he had discovered more than three decades ago and was having them administered to him in the hopes of curing his cancer. Although it’s unclear how much his treatments actually helped him (he was also receiving traditional cancer treatments such as chemotherapy and radiation), it’s clear that living four years with pancreatic cancer is outside the norm.

Steinman’s self-experimentation is the latest in a long line of famous scientists who believed in their research enough to test it on themselves. Nobel laureate Barry Marshall (a 2005 physiology or medicine Nobel laureate, along with Robin Warren) is probably the most famous self-experimentalist in recent history. Marshall’s motivation stemmed from the medical community’s rejection of his idea that peptic ulcers are caused by the bacteria Heliobacter pylori. He was unable to overcome the widely accepted notion that stress or overproduction of stomach acid are to blame for ulcers, so after failing to infect laboratory animals, Marshall did what any self-respecting scientist would do. He drank a bacterial culture containing H. pylori and within five days, he developed symptoms of gastritis, a precursor to ulcers. After a course of antibiotics, Marshall’s H. pylori infection was resolved and so was his gastritis. While it was a sensational finding (the tabloid Star even wrote an article about it titled “Guinea-pig doctor discovers new cure for ulcers … and the cause”), it took some time before other scientists corroborated his finding and the scientific community honored him with its highest prize.

Another famous Nobel Prize-winning self-experimentalist was the German physician Werner Forssmann (a 1956 physiology or medicine laureate, along with Andre Cournand and Dickinson Richards), who was the first to develop a technique for cardiac catheterization. Forssmann had the idea that a catheter directly into the heart could be used to deliver medicine or other treatments without damaging the heart itself. So against the advice of colleagues, Forssmann gave himself a local anesthetic and put a catheter into his arm and snaked it all the way into his heart. He then proceeded to have an X-ray taken, showing the catheter in his heart. The fact that he was still alive and well had provided evidence that this procedure could be useful and safe in the clinic.

So what’s the lesson here? Well, it’s clear that human testing is a critical part of validating the efficacy of a new procedure or medication. However, the subjects enrolled in these studies are usually those who have tried more traditional treatments without success. The experimental treatment tends to be their last chance to stave off death, as it was in the case of Steinman. But if you’re healthy and are thinking of self-experimenting just to prove your point, then you had better make sure you’ve got it all figured out. Chances are, there are some self-experimentalists that weren’t quite right about their science and didn’t make it into the history books when their experiments went wrong!

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