Page 99 - Forbes - USA (March 2020)

their microbiome investments. But Alex Morgan, a Khosla where 1,000 germ-free mice, delivered by Caesarean section
Ventures principal with an M.D. and Ph.D. from Stanford, in sterile conditions to ensure they are bacteria-free, live
suggests Khosla’s decision to back Viome has nothing to do inside plastic-encased rectangular bubbles. Grad students
with nutritional advice. Instead, he says, the firm invested douse the animals’ food with various gut microbes to test
because Viome hired a team of scientists from the U.S. De- which bacteria promote tremors and motor problems in
partment of Energy’s Los Alamos National Laboratory. In mice that correlate with Parkinson’s symptoms in humans.
addition, Viome had made a deal with the lab to license a In 2016, David Donabedian, a chemistry Ph.D. who was
valuable tech platform that has a unique ability to sequence then a partner at Longwood Fund, a Boston venture capital
the biochemical activity in microorganisms. firm, volunteered to raise the money and research power to 97
So even if Jain is selling snake oil, Viome might have sig- move Mazmanian’s biotech venture forward. The company,
nificant value. Indeed, British pharma giant GlaxoSmith- Waltham, Massachusetts–based Axial Biotherapeutics, has T
Kline struck a royalty deal with Viome in November 2019 to $55 million in backing and 30 employees. Under Donabe- H
use its tech to help develop microbiome-derived vaccines. dian as CEO, Axial is in the early stages of developing syn- E

Jain’s investors could make out handsomely. thetic drugs made of small molecules it hopes will absorb M
I
the particular gut-bacteria byproducts (called “metabo- C
R
A crobiologist Sarkis Mazmanian, 47, is working on a drug to treat the digestive problems suffered B
t Caltech in Pasadena, California, mi-
lites”) that appear to exacerbate autism symptoms. It’s also
O
I
O
by many people with Parkinson’s.
considered one of the foremost gurus of
M
microbiome research. In 2012 the MacAr- In the U.S., more than a million people suffer from autism, E
thur Foundation gave him a $500,000 and there are no drugs to treat it; an additional million have
“genius” grant for his work on the microbiome’s role in Parkinson’s. What would be the value of an FDA-approved
disease. Since then, he’s been exploring one of the most in- drug for either condition? “I can’t give you a market size,”
triguing connections in human health: the “gut-brain axis.” says Donabedian. “But if either one hits, it will be huge.”
The working thesis is that the bugs in your belly have a Chris Howerton, a biotechnology analyst at Jefferies, a
direct impact on your neurological health, which has pro- New York investment bank, is less shy. “If every single mi-
found implications for autism, Parkinson’s and Alzheimer’s. crobiome paper turns into a proven therapy, it could im-
In 2008, two years after joining the Caltech faculty, pact the drug markets for most major categories of disease,
Mazmanian published a cover story in Nature that docu- which together were worth $350 billion in 2018 in the U.S.
mented his successful treatment of inflammatory bowel dis- alone,” he says. “The breadth of the microbiome’s potential
ease in mice with human gut bacteria. A Caltech colleague, application is really tantalizing.” F
Paul Patterson, who was
researching autism in mice,
saw a possible connection to
the digestive problems suf-
fered by as many as 60% of
children with autism.
Together they started test-
ing whether human gut bac-
teria could induce and ame-
liorate autism-like symptoms
in mice. In the midst of their
early work, Patterson was
diagnosed with fatal brain
cancer. In a hospital room
at UCLA where Patterson
was awaiting surgery in May
2014, Mazmanian signed pa-
pers giving Patterson a stake
in a company that would de-
velop drugs from their exper-
iments. “I wanted Paul to get
the recognition of his con-
ETHAN PINES FOR FORBES Patterson died the following The Gut-Brain Connection
tribution,” says Mazmanian.

month.
Mazmanian is carrying
Caltech professor Sarkis Mazmanian in one of his Pasadena, California, labs. In a trailblazing study,
on their research in his sub-
he transferred gut bacteria from humans with autism into sterile mice who then exhibited autism-like
basement lab at Caltech,

M A R C H 2 0 2 0 behaviors. “The most rigorous clinicians and investors,” he says, “realize this is a long journey we’re on.”
F O R B E S . C O M