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FORBES.COM MARCH 20 20THETRENDDesperate, he researched possible therapies and discov-
ered articles about fecal transplants wiping out the infec-
tion. But his gastroenterologist refused to perform the pro-
cedure. So he took matters into his own hands. He asked
his roommate to supply a stool sample, bought an enema
kit from CVS, pulsed the mixture in a blender, strained
it through a coffee filter and pumped it into his gut. As
though a wizard had cast a spell, he made a full recovery
within days.
Welcome to the most promising new frontier in medicine:
poop. By focusing on what’s coming out of patients’ rear
ends, a growing body of scientific research over the last 15
years has highlighted the crucial role the microbiome plays
in human health. That new understanding could lead to
breakthrough treatments for a huge range of illnesses, from
obvious ones like digestive ailments and food allergies to sur-
prising ones like cancer and autism. A microbiome-derived
drug is already in the works to prevent childhood asthma.
Put crudely, the idea is to use gut bugs as drugs. More
than 50,000 scientific papers in the last five years have ex-
plored the microbiome’s effects. Various kinds of gut bac-
teria appear to stimulate or suppress immune responses
in the body, while others seem to fight off disease-causing
microbes. A groundswell of cutting-edge research has the
potential to deliver a burst of new therapies that will vastly
reduce human suffering—and generate huge paydays for
the field’s pioneers.
When scientists transferred gut microbiome cells from
obese mice into lean ones, the recipients gained weight. In
one study, melanoma patients with the most diverse mi-
crobiomes had the best response to immunotherapy. And
mice injected with gut bacteria from marathon runners ranlonger distances. A new drug for obesity alone could be
worth more than $20 billion.
So far, the most compelling microbiome-derived therapy is
a live fecal transplant for C. diff, which strikes half a million
Americans annually, killing 15,000. In 2013, the New England
Journal of Medicine published a paper that caught the scien-
tific community by surprise and jump-started investment in
microbiome drug development. In a randomized trial, 94%
of recurrent C. diff patients recovered after receiving fecal
transplants. To put that in context, cancer drugs with efficacy
rates as low as 10% have been approved by the FDA.
Billions of dollars are pouring into microbiome medicine.
Gbola Amusa, a medical doctor and partner at Chardan, a
health care–focused investment bank in New York, pegs the
total amount invested since 2014 at more than $5 billion.
Techie billionaires including Bill Gates, Salesforce founder
Marc Benioff and Silicon Valley venture capitalist Vinod
Khosla are funding microbiome startups, and Gates, Be-
nioff and Mark Zuckerberg have all made donations to sup-
port microbiome research at institutions including Stan-
ford, Washington University in St. Louis and the University
of California, San Francisco.
The race is on for FDA approval of the first drug made
from gut bacteria. But the science is young and unproven.
At Oppenheimer in New York, Mark Breidenbach says
investor enthusiasm in microbiome companies is on a
downswing because “there is no consensus about what the
microbiome can do.”
Amusa is more bullish. “The science is turning,” he says.
“When it comes through with proof, these biotech compa-
nies will be worth not hundreds of millions of dollars, but
billions.”→ Sharp pains shot through the
patient’s stomach, and he had
constant diarrhea. Seven rounds
of antibiotics over 18 months had
only made him feel worse.
A previously healthy man in his 20s who wishes to remain anonymous, he had contracted a recur-
ring case of Clostridium difficile, or C. diff, after having his gallbladder removed in 2012. Hospital
patients are prone to C. diff since antibiotic treatment for other maladies decimates the infection-
fighting capacity of what scientists call the gut microbiome, the trillions of cells that move through
the human digestive system. “It didn’t just affect my gut,” he says. “I was exhausted all the time.
I had really bad brain fog. I couldn’t concentrate.”