When physicist Alex Zhavoronkov looked in the mirror one day and saw alarming swelling around his eyes, he guessed it was an allergic reaction to a drug he’d taken. But he couldn’t just ring up his doctor and ask — the drug was one his own company was developing, and he was its first guinea pig.

As CEO of biotech company Insilico Medicine, Zhavoronkov routinely subjects himself to his own medicine. Self-experimentation lets him quickly see whether lab predictions hold true in a human subject, or whether there are any safety issues — say, a potential allergy. (He now thinks the swelling may have been caused by a drug interaction with some tomato juice he drank.) And he thinks it has a place in the scientific literature, too: He plans to launch a journal of self-experimentation later this year.

Such experiments are one of medicine’s oldest traditions. Many a vaccine or poison was first tested on its developer; most of the earliest work on psychedelic drugs was conducted by intrepid scientists tripping in the name of research. As medicine has progressed, however, self-experimentation has become less popular in academic labs, to be taken up instead by a growing biotech industry. Those changes have given rise to a new breed of self-tester: the guinea pig CEO.

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The medical annals are filled with self-experiments, some more successful than others. To try to prove that Vibrio cholerae didn’t cause cholera, in 1892, Max von Pettenkofer drank a broth of it. (He did come down with symptoms.) In 1929, Dr. Werner Forssmann carried out the first cardiac catheterization, threading a catheter from his arm all the way into his heart to prove it wouldn’t be fatal. Dr. Barry Marshall proved, in the 1980s, that bacteria are the cause of ulcers by drinking an elixir of Helicobacter pylori bacteria — which earned him several days of vomiting, an endoscopy and stomach biopsy, and the 2005 Nobel Prize.

But in recent decades, executives have gotten in on the practice. Stanford University geneticist Mike Snyder, for instance, has founded several biotech companies, including the genomics-based medicine company Personalis. In 2012 he and coauthors published the “Snyderome”: an in-depth look at his own genome, combined with profiles of his RNA, proteins, and other factors over time.

Snyder’s study of himself has been running for over six years now, and he’s added elements such as the epigenome and microbiome. “We make billions of measurements every time I am sampled,” he said. That’s coined another epithet for his work: the “narcissome.”

Other execs have also become “N-of-1” experiments. As president of Celera Genomics, Craig Venter raced to sequence the first human genome in 2001 — his own. Liz Parrish, CEO of the biotech BioViva USA Inc., was injected last September with two of the company’s experimental gene therapies. Rob Rhinehart, CEO of Soylent Corporation, tweaked the formula for his meal-replacement drink by testing it on himself. David Whitlock, the founding scientist behind AOBiome, uses the company’s bacteria products instead of showering. Josiah Zayner — whose company, The ODIN, sells DIY science equipment — recently performed a fecal transplant on himself.

Self-experiments may hold particular appeal for biomedicine these days. Alongside the rise of personalized medicine — in which treatments are tailored to a single person — experiments on just one person suddenly become just the right size. “N-of-1 experiments are what precision medicine is all about,” Snyder said.

They’re also a potential cost-saving measure. William Bains, a scientist and entrepreneur who teaches about biotech startups at the University of Cambridge, said self-experimentation could let a researcher “leapfrog” millions in developmental costs by getting a new drug straight into human testing and avoiding rounds of tests on cells and animals.

But it can be a risky strategy for a CEO. “If you do test [a product] out on yourself,” Bains said, “conventional investors will regard you as a cowboy, conventional scientists will regard you as an idiot, and then you blow your chance of getting conventional funding.” This is a potential difficulty Parrish, the BioViva CEO who’s receiving her company’s gene therapy, might face with her business, Bains said.

The practice tends to blur lines between genuine research and marketing stunt. BioViva, for example, announced positive results of Parrish’s treatment in the same week that it announced a partnership with investment fund Deep Knowledge Life Sciences. Soylent creator Rhinehart published his blood work shortly before launching a crowdfunding campaign.

And the amount of insight from an N-of-1 test is limited. The placebo effect is a major problem for people experimenting on themselves, Bains points out, since there’s no control group.

After BioViva announced that Parrish had been injected with two of its experimental anti-aging gene therapies, she did an Ask Me Anything on Reddit. “I am patient zero,” Parrish wrote there. “I have aging as a disease.” Many commenters applauded what they saw as her bravery and pioneer spirit.

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One commenter remarked, however, “That is not how an unbiased experiment works.”

“N’s of 1 have had their value through history, and will,” said Dr. Lawrence Altman, author of “Who Goes First? The Story of Self-Experimentation in Medicine.” “But you’re not going to license a drug based on an N-of-1.” To be valuable, he said, an experiment should have a defined protocol and a plan for expanding into a larger number of subjects.

Meanwhile, Zhavoronkov plans to bring more rigor to self-experimentation by starting a journal to peer-review and publish these kinds of studies. “People are doing it anyway; why not have it published?” Zhavoronkov said. “What we’re really trying to do is unlock the knowledge which is already there.”

By combining lots of N-of-1 studies, Zhavoronkov said, it might be possible to draw larger conclusions from the field.

But academia hasn’t been as welcoming as the biotech world: Despite early interest, Zhavoronkov is finding it hard to recruit an editorial board for the nascent journal because, he said, scientists have decided that participating might harm their academic reputations.

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  • I knew Rob Warren when he was a pathologist in Royal Perth Hospital. He saw black “dots” in the biopsies of resections from patients with stomach ulcers. These black dots ONLY appeared in stomach ulcer resections. He cleaned his microscope; he cleaned his slide. The dots were still there. It was then Rob came to his hypothesis that the specks MUST be bacteria because he had excluded every other possible explanation.

    Everyone laughed at him. He finally convinced Barry Marshall to plate the material to test his (Rob’s) hypothesis. From my memory, the plates were forgotten over Easter so were incubated longer than usual, which resulted in abundant bacterial colonies.

    Then Barry did the showman trick, and got (mis) remembered in history for his part. But it was self-effacing Robin Warren’s careful observations and deductions over the years that led to the discovery of Helicobacter pylori. All Barry did was help Rob test his hypothesis because Robin is a pathologist, not a microbiologist.

    PLEASE stop rewriting history. Stop giving the showman sole credit for work that was the result of years of original and patient intellectual work by the introverted lab guy.

    The same thing happened to Howard Flory who after years of meticulous lab work tested something Flemming had looked at once and rejected years before. It was Flory who discovered penicillin as an antibiotic and conducted the clinical trials. Flemming was more or less peripheral to this process, but was a better showman than Flory.

    For both quiet, conscientious Robin Warren and Howard Flory, history has been rewritten by a showman.

    • Fascinating article by Elizabeth Preston, and an illuminating comment by Kathy about Rob Warren as the true discoverer of the bacterial cause of stomach ulcers!

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