More Medical Researchers Engage In Self-Experimentation

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Shirley S. Wang

Shirley S. Wang
The Wall Street Journal
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Jan. 25, 2016 11:17 a.m. ET

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When Bob Hariri developed a product he thought could be useful as a human-skin replacement for burn victims, he had no trouble finding a subject willing to test it--himself.

An entrepreneur and a neurosurgeon with both a medical degree and a doctorate, Dr. Hariri is one of a number of scientists who have experimented on themselves with new or yet-to-be approved medical products or technologies, and who say such practice can be indispensable in the development of innovative biomedical treatments.

Some scientists are pushing for self-experimentation data to be reported publicly and more systematically to aid scientific progress. Alex Zhavoronkov, chief executive of an aging-research company called InSilico Medicine Inc., and others hope to start a peer-reviewed journal on self-experimentation, where scientists and other qualified individuals would publish high-quality case studies of tests performed on themselves. He plans to launch a crowdfunding operation in the next few months to fund it.

The idea is “to unlock the knowledge [of self-experimentation] that resides there anyway,” says Dr. Zhavrononkov, who takes an old diabetes drug called metformin that is supposed to have antiaging properties, even though it hasn’t been approved for that purpose.

Scientists have long experimented on themselves but often kept the practice quiet. Most of the time, an individual case report, sometimes known as an “N of 1”, isn’t considered scientifically rigorous. The gold standard with new medical treatments involves randomizing and systematically comparing groups of people in order to draw conclusions for the broader population.

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Dr. Alex Zhavoronkov plans a self-experimentation journal. Photo: Andrew Garazha

Self-experimentation, whether by scientists or patients, is becoming more commonplace. It has become easy for interested parties, including people with illnesses, to devote time to experimenting with treatments they find online, says Charles Cantor, a retired professor at Boston University, who is working with Dr. Zhavoronkov on the self-experimentation journal. He says he hasn’t tried self-experimentation but is open to the idea.

Some of the most visible attempts at harnessing self-experimentation data in recent years have focused on the genome. Michael Snyder, chair of the genetics department at Stanford University, and a team of researchers analyzed Dr. Snyder’s own genome and other personal molecular data, which they dubbed Snyderome, and published the findings in the journal Cell. Looking at Dr. Snyder’s data over a 14-month period, the team demonstrated that an “integrative personal omics profile” analysis could identify medical risks such as Type 2 diabetes, and molecular changes that occurred between healthy and diseased states.

A larger-scale effort, called the Personal Genome Project and initiated by the geneticist George Church, encourages anybody to sequence their entire DNA and make the data public for research purposes. Dr. Church was one of the first in the project to publicly release his genomic data.

Advocates say self-experimentation can yield information that is hard to get from a clinical trial. The experimenter feels what it’s like to be the patient and gets insight into how to improve testing procedures. Also, a number of individual reports, when cobbled together, can start to yield a picture of whether a new treatment is likely to work or not, though one wouldn’t rely on those reports alone to conclude safety or effectiveness.

Undoubtedly, there are ethical and scientific challenges involved in using data from self-experimentation, including privacy concerns about such highly personal information and the possibility that the experimenter’s personal experience will bias the research.

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Dr. Jonas Salk gave the polio vaccine to himself, lab scientists, his wife and children before national testing in the 1950s. Photo: Bettmann/CORBIS

Dr. Church, a professor at Harvard Medical School, says he saw a decade ago how the vast amount of information resulting from sequencing individuals’ entire genomes could be useful in better understanding disease. In 2005, he proposed the Personal Genome Project. He and his team dealt with issues of anonymity and identity of personal medical data by simply telling participants that their confidentiality couldn’t be guaranteed when their data was made public.

To make sure people understood what they were signing up for, the team created a rigorous consent process, including multiple-choice comprehension questions; they also required consent from family members, because genetic information yields information about relatives.

When he proposed the project, the Harvard human-subjects ethics committee decided he should be willing to do it himself if he was encouraging other people, and Dr. Church was one of the first to have his genome sequenced. Every procedure he has asked participants to do, he has tested on himself.

“Almost every step of the way, me walking through the trenches with the patients made for a more pleasant protocol design,” he says.

An early aim of the research was to establish stem cell lines for participants, which involved taking a skin sample using a “skin punch biopsy.” The procedure turned out to be quite painful due to an unnecessarily large amount of skin extracted and poor anesthetics, recalls Dr. Church, who did it first and vetoed it. Then they discovered the physician conducting the biopsies had a better procedure that he used with children, including medicinal cream and a punch that was 36 times as small as the first. The researchers switched to that method.

Some 200 people so far have publicly released their sequenced genomes—which is thought to be the largest high-quality set of publicly available genome data, says Dr. Church. Thousands more have signed up to do so.

The database can be used as a point of comparison for any disease, says Dr. Church. Some of the projects where it is currently in use include as a control in research in age-related diseases, and in studies that test the accuracy of software interpreting the genome.

As self-experimentation gets more widespread, Dr. Church says data-sharing will become more important. “That information vanishes in the wind if there’s no sharing of the data,” he says.

Dr. Hariri, the neurosurgeon and biomedical entrepreneur, says it is appropriate and even noble for scientists to test out an innovation before subjecting a wider number of people to it.

“Insofar as safety is concerned, most pharma inventors reach a point where they have complete confidence in product they invent,” says Dr. Hariri. “If you could ask someone who invented a medication, ‘Would you be willing to take this?’ and they said no, that would be a red flag.”

Dr. Hariri tested his skin-substitute product on himself before commercialization when he suffered a second-degree burn, as well as blisters from workouts. “If you’re someone who has the confidence to try this invention on yourself and you get the results you expected, it turns you into a true believer,” Dr. Hariri says. After his self-experimentation, he recused himself from the evaluation process to reduce the possibility of bias during trials. The product now is on the market as Biovance.

“When I confirmed for myself that my inventions work, I then immediately took myself out of the review process,” Dr. Hariri. “I’m not going to be the one to sway the evaluator here. I think that protects you from the natural tendency to introduce bias.”

Write to Shirley S. Wang at shirley.wang@wsj.com