RNA that, by matching up
with a mirror copy present
in Cameron’s cells, allows his
body to correctly assemble a
protein his nerve cells need.
Last year, two drugs that
work along these lines were
approved in the U.S. The
other, Exondys 51, developed
by Sarepta Therapeutics, was
approved to treat muscular
dystrophy after becoming the
focus of impassioned lobbying by parents of affected
boys, who prevailed on the
U.S. Food and Drug Administration to allow it on the
market despite limited evidence of its benefits.
There are no such questions about the drug Cameron receives. Its effects are
“Lazarus-like,” according to
one expert, but it is not simple and is not a permanent
cure. It’s delivered through
a spinal tap once every four
months, and Cameron will
need it for the rest of his life.
The cost of the injections:
$375,000 a year. “This has given us a
glimpse into a future that’s really quite
profound, at least in serious disorders of
the central nervous system,” says Michael
Ehlers, executive vice president of Biogen.
What happens next for Cameron is not
clear. Because Spinraza just started being
tested in children five years ago, doctors
aren’t sure what will occur as Cameron
grows older. C. Frank Bennett, senior vice
president of research at Ionis, says the
company is now studying the results if the
drug is given even earlier, just after birth,
and before symptoms start. So far, he says,
these kids are meeting their developmental milestones by rolling over and crawling
on time. —Karen Weintraub
“Software is eating the
world, but AI is going to eat
— Nvidia CEO Jensen Huang on how the
machine-learning revolution is just beginning.
“We have no interest in
creating more survivors
with impaired quality of life.”
— Alan Flake, a surgeon at Children’s Hospital
of Philadelphia, where researchers kept eight
premature baby lambs alive in artificial wombs
“I would block its attempts
to patrol my facilities, to
prevent it from monitoring
— 24-year-old Byun Hyun Woo, a champion
Starcraft player, who does not believe artificial
intelligence will master the game in his lifetime.
BY THE NUMBERS
Number of new cars that China’s BYD, the largest electric-vehicle manufacturer in the world, is
making per day, down from 900 before the government cut subsidies for EVs.
Number of hours a home can be powered by a
single revolution of the world’s largest wind turbines, 195 meters tall, which are operating off the
north west coast of England.
Factor by which the cost of DNA-based data
storage needs to fall before it can become commercially available, according to Microsoft,
which plans to be ready to offer it when it does.
Annual cost of the immunotherapy Keytruda, the
first drug approved to treat cancer on the basis
of its genetic makeup rather than its
location in the body.
sages, has spent about $1.5 billion since
being founded in 2002 but is still working toward its first product. In a January paper, it showed that one of its drugs
could lower cholesterol levels for six
months with one shot.
Rachel Meyers, previously a senior
executive at Alnylam, says one problem
facing RNA drugs is that companies have
too often tried to use them to treat diseases where other options exist. Instead,
she believes, RNA therapeutics are more
likely to find success in situations, like
Cameron’s, that can’t easily be treated in
any other way. Spinraza works through
a mechanism called “antisense.” The
drug is a chemically enhanced strand of