In 2016, the US pharmaceutical sector was valued at an impressive $446 billion. The industry's spending on research and development per employee is unmatched by any other. Interestingly, the creation of groundbreaking drugs and treatments often comes from unexpected discoveries, accidental findings, or sheer luck.
10. Chlorambucil

Chlorambucil, an FDA-approved drug for leukemia, was introduced in 1957. Leukemia, a cancer affecting blood cells, is particularly prevalent in children under 15. The drug's origins can be traced back to the use of mustard gas during World War I.
Years after World War I, and with the looming threat of World War II, Yale researchers studying mustard gas poisoning found that soldiers exposed to the gas had abnormally low white blood cell counts.
This observation led to the realization that nitrogen mustard-based compounds could be used to treat leukemia by targeting and destroying cancerous white blood cells and halting tumor growth. Further studies led to the creation of chlorambucil, which continues to be used in cancer treatment today.
9. Viagra

There's no question that Viagra is one of the most widely used and appreciated drugs around the world. Its fame is especially amplified through its frequent references in pop culture, particularly in Hollywood.
However, the discovery story behind Viagra is a bit more understated. It began in Merthyr Tydfil, a quaint Welsh town, where scientists testing a new drug for angina prevention received unexpected reports from volunteers.
Male participants were experiencing more frequent erections. Further research indicated that it was indeed the angina medication causing this effect, and thus, the “little blue pill” was born.
It was marketed as the first oral treatment for erectile dysfunction in 1998. Now, 20 years later, it has become one of the most prescribed drugs in the world.
8. Botox

Almost everyone has heard of Botox and its infamous face-freezing effect, leaving countless “celebrities” devoid of facial emotion. But fewer are aware of the drug’s surprising origins. Botox is actually a neurotoxin that is made from a purified form of the botulinum toxin that causes botulism.
It was first used as a drug to prevent muscle spasms within the body, specifically for people suffering from eyelid or vocal cord spasms. However, its usage around the eyes had some unexpected effects. The drug began to diminish wrinkles around the brow area of the face, which led to its use in cosmetic surgery to reduce facial wrinkles and to smooth skin.
The rapid advancement of this treatment has made Botox cosmetic procedures one of the most sought-after medical treatments, with over seven million procedures performed in the United States alone in 2016.
7. Smallpox Vaccination

In the late 18th century, Edward Jenner made groundbreaking contributions to the development of the smallpox vaccine. Smallpox was feared for its high fatality rate and the severe facial scarring it left on survivors.
Before Jenner's work, the only method of prevention was variolation, where pustule material from someone infected with smallpox was used for inoculation. While variolation resulted in a milder illness, it still carried the risk of death.
While practicing in a small rural town in England, Jenner observed that milkmaids who contracted the mild cowpox seemed immune to smallpox. This observation of using a less dangerous virus for protection paved the way for the development of vaccination. (Vacca is Latin for “cow.”)
The significance of the smallpox vaccine cannot be overstated. In 1980, the World Health Organization officially declared smallpox eradicated, making it the first and only disease to ever achieve this milestone.
6. Lithium

The treatment for bipolar disorder (formerly called manic depression) has evolved significantly throughout history. Unfortunately, until the late 20th century, many patients were confined to asylums. In 1948, however, Australian psychiatrist Dr. John Cade introduced a groundbreaking treatment using lithium salts.
This discovery stemmed from Dr. Cade's investigation into the old belief that mania was linked to urea. Cade took an unconventional approach by collecting urine from patients and storing it in his own refrigerator to later inject into live guinea pigs.
Over time, Dr. Cade began injecting uric acid directly into guinea pigs, using lithium urate due to its high solubility. The lithium salt had a calming effect on the animals, leading Cade to try it on himself, with similar results. His pioneering work laid the foundation for the modern treatment of bipolar disorder.
5. Penicillin

While many are familiar with Alexander Fleming's accidental discovery of penicillin in 1928, few are aware of Ernst Chain and Howard Florey, the men who transformed penicillin into a usable drug. The three men were awarded the Nobel Prize in Medicine in 1945 for their contributions to penicillin.
In the 1930s, while at Oxford University, Chain, Florey, and their team began investigating Fleming’s work on penicillin and developed a method to mass-produce the penicillin mold for treating infections.
Their innovative culturing techniques included repurposing old dairy equipment and even using Marmite as a growth medium. One of their initial approaches involved cantaloupes, which proved successful enough to proceed to clinical trials.
The efforts of the two men did not go unnoticed by the US military. During World War II, the armed forces recognized the potential of penicillin to treat battlefield infections and began mass-producing the drug for the D-Day landings. Penicillin went on to save countless lives throughout the war and in the years that followed.
4. LSD

Lysergic acid diethylamide (LSD) is a Schedule I psychoactive substance known for inducing powerful hallucinatory experiences in users. While not traditionally viewed as a pharmaceutical drug, recent studies have indicated its potential in treating post-traumatic stress disorder.
LSD was first synthesized in 1938 by Swiss chemist Albert Hofmann while attempting to develop a blood circulation stimulant. However, the substance failed to produce the desired effects, and was consequently abandoned for five years.
In 1943, Hofmann resynthesized the compound and accidentally absorbed a small amount through his fingertips, triggering an experience he later described as 'an uninterrupted stream of fantastic pictures, extraordinary shapes with intense, kaleidoscopic play of colors.'
Three days later, on April 19, Hofmann intentionally took a larger dose of LSD and had what is now known as 'Bicycle Day.' The name comes from the fact that Hofmann rode his bicycle home during his hallucinatory experience. With this, LSD was officially born and quickly became an iconic symbol of the 1960s counterculture movement, 'flower power.' Even today, the drug remains in use.
3. Warfarin

Warfarin's origins are tied to a bizarre series of events involving dead cattle, rodents, and a failed suicide attempt. It eventually became the world's most widely used anticoagulant, prescribed to as many as 1 percent of adults in the UK.
In the 1920s, a mysterious epidemic struck cattle in northern US and Canada, causing them to bleed uncontrollably. The cause was traced to moldy silage made from sweet clover, but the matter seemed resolved at the time.
However, in the 1940s, scientists Karl Link and Harold Campbell in Wisconsin isolated the compound in sweet clover responsible for its anticoagulant effects. This compound was developed into warfarin, which was first introduced as a rat poison in 1948, causing severe internal bleeding in the rats that ingested it.
Warfarin is now most commonly used to prevent and treat blood clot-related conditions such as strokes. This drug wasn't approved for human use until 1954, following a failed suicide attempt by a US military recruit that revealed warfarin's potential as a non-lethal treatment. One of the first patients to receive it was then-President Dwight D. Eisenhower.
2. Cisplatin

Cisplatin is a key treatment for testicular cancer, with success rates estimated to cure up to 90% of cases. This remarkable effectiveness has made it a cornerstone in cancer care.
The drug's discovery as an anticancer agent was largely serendipitous. US chemist Barnett Rosenberg stumbled upon it in the 1960s while exploring the effects of strong electrical fields on E. coli, when he observed it inhibited bacterial cell division.
Rosenberg discovered that the effect was caused by the platinum electrodes, not the electrical current itself. He had synthesized a compound called 'Peyrone's chloride,' first isolated in the 1840s, but its potential for cancer treatment had not yet been recognized.
Since its breakthrough in the 1960s, cisplatin has emerged as one of the most widely used anticancer drugs across the globe.
1. Disulfiram

Commonly sold under the name Antabuse, disulfiram is a medication used to combat alcohol addiction. It reacts with alcohol to induce nausea and elevate heart rate, acting as a deterrent for consumption.
Two Danish researchers, Jens Hald and Erik Jacobsen, initially tested the drug as an antiparasitic. To observe its side effects, they took small doses themselves, but an after-work cocktail party proved to be an unfortunate and unintended experiment.
After ingesting small quantities of alcohol, both men quickly became severely ill. This led them to conclude that the two incidents were related, suggesting that their antiparasitic drug might serve another purpose.
Curiously, further investigation revealed that workers in the rubber industry had been aware of this connection for some time. They observed similar reactions to alcohol among individuals handling chemicals used in rubber production, although the connection had not yet been made.
