Shortly after Cameron Harding’s one-
month check-up, his mother, Alison, saw
that her newborn seemed to stop moving. She’d unwrap him from a swaddle
and his arms would flop to one side. He
wouldn’t kick his legs or turn his head.
The diagnosis: spinal muscular atrophy.
The inherited illness, which destroys the
motor neurons that control movement,
often kills children before they turn two.
In Cameron’s case, it seemed he’d probably never even have a birthday.
But when he was seven weeks old,
Cameron’s parents enrolled him in a clini-
cal trial for an experimental drug. In vid-
eos shot two months later, he could move
his head and reach for a toy. No child with
his condition had ever made such a recov-
ery before. The drug Cameron received,
Drugs made of
RNA have been in
development for more
than 20 years. But Cameron’s remarkable
trajectory is a sign they could be ready
to join familiar pharmacy chemicals and
biotech proteins as the next great class of
drug types, proponents say. “Right now,
RNA therapeutics—that’s the future of
medicine,” says Steven Dowdy of the Uni-
versity of California, San Diego, School
of Medicine. Dowdy admits
to being a partisan—his lab
works on RNA—but says
advances in chemistry have
finally made this type of drug
feasible. “We still have a lot
of work to do, but it’s going
to be huge,” he says.
Some investors think so,
too. One private company,
Moderna Therapeutics, has
reportedly managed to raise
nearly $2 billion to develop
what it calls a “platform” for
inventing new RNA treatments. Overall, more than
150 clinical trials under
way are using RNA to treat
cancer, infection, hormone
problems, and neurologic
diseases, including Huntington’s disease.
Inside our cells, the genes in our DNA
code are translated into copies made of
RNA. These copies, in
turn, float into the cell
body, where they serve
as the information template from which proteins are manufactured.
Most drugstore pills act directly on proteins. Gene therapy, meanwhile, seeks to
replace DNA instructions with new ones.
What drugs like Spinraza seek to do
instead is use RNA to block, modify, or
add to the existing RNA messages in a
cell. The most serious challenge to this
approach, says Dowdy, is that cells evolved
How a Boy’s Lazarus-like
Revival Points to a New
Generation of Drugs
Drugs made from RNA may be the next great class of medicine.
“This has given us a
glimpse into a future that’s
really quite profound.”
billions of years ago to keep RNA from the
environment out. Overcoming this deliv-
ery problem has cost company chemists
many years of effort.
While the science of RNA has
unfolded since the 1960s to a drumbeat
of Nobel Prizes, the advance into real
medicine hasn’t been quick. RNA drugs
have been beset by serious side effects,
and several have been pulled from human
tests over safety concerns. In October, the
biotech company Alnylam had to stop a
large study of one drug, Revusiran, after
unexplained deaths in volunteers being
treated for a hereditary metabolic disease,
ATTR amyloidosis. Alnylam, which works
with a technique for blocking RNA mes-
Cameron Harding is one of
the first children to survive
spinal muscular atrophy.