Scientific Self-Experimentation: Five Discoveries That Changed Medicine
Myron Yaster MD, Mark Rockoff MD, and Alan Jay Schwartz MD MSEd
Prospective randomized controlled trials (RCTs) are considered the highest level of evidence in evidence-based medicine (EBM) for establishing causal relationships between interventions and outcomes. RCTs typically have the greatest regulatory oversight and most robust study processes, including on-site data monitoring, independent event adjudication, data safety monitoring boards, and blinded core laboratories. On the other hand, historically, some of the most important discoveries in clinical medicine were done without Institutional Review Board (IRB) oversight. IRBs are a federally mandated committee that reviews, approves, monitors, and oversees biomedical and behavioral research involving human participants, with the primary goal of protecting the rights, safety, and welfare of research subjects.
In today’s PAAD, reporter Tony Dunnell retells the story of 5 discoveries by research scientists who took the ultimate research risk when it comes to proving the efficacy of their work: experimenting on themselves often when their institutional IRBs did not approve the studies because they thought the experiments were too dangerous to perform on human subjects. This is an absolutely fascinating article that I thought many of you would enjoy. Myron Yaster MD
Original article
Tony Dunnell: 5 Scientific Discoveries Born From Self-Experimentation
https://historyfacts.com/science-industry/article/scientific-discoveries-self-experimentation/
Throughout the history of medicine, self-experimentation has served as a catalyst for transformative scientific advances. Long before modern institutional review boards and formal research ethics, physicians and scientists occasionally assumed the role of both investigator and research participant, accepting substantial personal risk to validate novel hypotheses. Although contemporary ethical standards rarely permit such approaches, several landmark discoveries arising from self-experimentation fundamentally altered medical practice and improved patient outcomes worldwide.
One of the most celebrated examples is the development of the polio vaccine by Jonas Salk. Following successful animal studies in 1953, Salk vaccinated himself, his wife, and their children before initiating large-scale clinical trials. The absence of adverse effects and demonstration of protective antibody responses helped establish confidence in the vaccine, which ultimately led to the near elimination of paralytic poliomyelitis in many countries. Salk’s decision not to patent the vaccine further accelerated its widespread adoption and remains an enduring example of scientific altruism.
Self-experimentation also advanced the field of anesthesiology and surgery. In 1799, Humphry Davy self‑experimented with nitrous oxide, documenting its effects on the body. In 1921, surgeon Evan O’Neill Kane performed an appendectomy on himself under local anesthesia to demonstrate that many abdominal procedures could be performed safely without general anesthesia. At a time when general anesthesia carried substantially greater risk, Kane’s dramatic demonstration helped increase acceptance of local anesthetic techniques and contributed to safer surgical care for patients with significant comorbidities.
Another remarkable example involved Air Force physician John Paul Stapp, who repeatedly subjected himself to extreme acceleration and deceleration forces using rocket sleds during the 1950s. In one experiment he endured more than 46 times the force of gravity, sustaining rib fractures and temporary visual impairment. His investigations overturned prevailing assumptions regarding human tolerance to high gravitational forces and directly influenced the design of aircraft restraint systems, ejection seats, and ultimately modern automotive seat belts, saving countless lives through improved crash protection.
Perhaps no act of self-experimentation has had greater influence on modern gastroenterology than that of Barry Marshall. In 1984, after repeated skepticism from the medical community, Marshall deliberately ingested a culture of Helicobacter pylori. Within days he developed acute gastritis, and subsequent endoscopy confirmed bacterial colonization. Successful antibiotic treatment completed the demonstration that H. pylori causes gastritis and peptic ulcer disease. This work overturned decades of conventional teaching that attributed ulcers primarily to stress and diet, replacing chronic acid suppression with curative antimicrobial therapy. Marshall and colleague Robin Warren received the 2005 Nobel Prize in Physiology or Medicine for this discovery. Another example of self experimentation advancing the understanding of disease transmission occurred in 1900 when Jesse William Lazear allowed a mosquito to bite him to test the hypothesis that mosquitoes were the vector for yellow fever transmission. He contracted the disease, ultimately succumbing to the disease.
Similarly, as a surgical trainee, German physician Werner Forssmann revolutionized cardiovascular medicine by inserting a catheter into his own antecubital vein then walked to the X-ray department on the floor below where under the guidance of a fluoroscope he advanced the catheter into his right ventricle. This was then recorded on X-ray film, documenting the catheter lying in his right atrium. His daring experiment demonstrated the feasibility of cardiac catheterization, laying the foundation for modern diagnostic angiography, electrophysiology, structural heart interventions, and interventional cardiology. Like Marshall’s work, Forssmann’s initially controversial experiment was eventually recognized with a Nobel Prize.
Collectively, these examples highlight the extraordinary commitment of investigators willing to place themselves at personal risk in pursuit of scientific knowledge. While contemporary ethical oversight appropriately emphasizes participant protection, informed consent, and rigorous study design, the legacy of these pioneering self-experiments underscores an enduring lesson: carefully conceived scientific inquiry, coupled with exceptional personal courage, has repeatedly transformed clinical medicine. Their contributions continue to influence vaccination, anesthesia, trauma care, cardiology, and gastroenterology, demonstrating that individual acts of scientific conviction can yield profound and lasting benefits for public health.
We are certain that there are many other important studies that were performed by researchers on themselves that changed the practice of medicine. If you know of any that you’d like to share, send to Myron (myasterster@gmail.com ) and he will post in a Friday reader response.
Addendum From Mark Rockoff MD
I suspect there are many other examples of individuals who experimented on themselves (or their family members), but there are a few that stand out to me.
The article appropriately mention (Edward) Jenner since he was the first to demonstrate (in 1796) that vaccination of individuals with cowpox prevented them from getting the much more lethal smallpox. (indeed the word vaccine is derived from from the Latin word vacca, which means “cow”) Jenner first tried this on the child of his gardener and later extended this to his own child and many others. He, therefore, is rightly considered the physician who pioneered the concept of vaccines. However, what is less well know is the story of Zabdiel Boylston. He was one of the first physicians to perform surgery in the new American colonies removing gall stones in 1710 and a breast tumor in 1718. However, he also remarkably was the first physician in North America to inoculate individuals with pus from a smallpox sore which greatly reduced the mortality from naturally-acquired smallpox. He first did this in 1721 with two of his African slaves as well as his own 6-year-old son. This was met with widespread hostility at the time; he was initially arrested and had his life threatened. But this procedure proved to save many lives and was importantly used by George Washington with his troops during the American Revolution which helped them survive the smallpox epidemics that ravaged the continent at that time.
Another remarkable physician worth noting is William Halsted. Halsted was one of the four Founding Fathers of Johns Hopkins Hospital and its first chief of surgery. In his early career, he performed gallstone surgery on his own mother on the kitchen table in their home in NYC and he also performed what was possibly the first successful blood transfusion in the country on his own sister whom he injected with blood from his arm after finding her unconscious from a postpartum hemorrhage. Perhaps he is best known for experimenting with cocaine, then touted as a wonder drug because of its pain-numbing abilities when injected into peripheral nerves. He and some colleagues used themselves as test subjects beginning in 1884, thereby advancing the concept of local anesthesia, a critical leap for medical procedures, including dental work. Unfortunately, Halsted became addicted to cocaine then morphine which he used to try to wean himself from his cocaine addiction. Morphine addiction haunted him throughout his life, with him using nearly 200 mg/day before his death in 1922, just before his 70th birthday.