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PREFACE
In the fall of 2008, the hot-air balloon that hovered in the distance along Interstate 76
bore tiger stripes, marking the Philadelphia Zoo below. As it loomed, the tethered
balloon provided a reprieve for parents stuck in the ever-present traffic, giving restless
and excited children something to focus on other than escaping the car. Once they
got to the zoo, youngsters scampered up the path leading to the entrance, seeking
the strings that held the giant, colorful balloon aloft.
For the family of three from Upstate New York, the approach on the path to the zoo’s
entrance was slower. Even if they hadn’t been holding hands, it would’ve been clear
that they were a unit, all three with light that they were a unit, all three with light
reddish-brown hair and blue eyes. It was just four days after Corey’s gene therapy,
and the eight-year-old, still using his silver-tipped cane out of habit, stepped hesitantly,
looking down at the few orange and yellow leaves on the path. When the family
reached the iron entrance gate, Corey stopped, and like the other kids, couldn’t help
but gaze up at the enormous balloon. Then he let out a shriek.
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Ethan, Nancy and Corey Haas
“It hurts!” Corey cried, shielding his eyes with his hands.
His parents were momentarily stunned. Then it very slowly dawned on them what was
happening. Could it really be? So soon? They gently pulled Corey to the side of the
pathway so others could pass.
“Are you okay? What do you mean, it hurts your eyes?” asked Nancy, smoothing his
hair, her face a mix of concern and joy. To this day she cannot tell this tale without
crying.
“The light! It hurts!” Corey repeated, slowly lowering his hands and squinting. He was
terrified because this had never happened to him before. He’d so often stared right
into a lit bulb with no reaction at all, even as an infant.
Corey had been born with an inherited condition called Leber congenital amaurosis
type 2— LCA2. A single gene had a glitch that prevented his eyes from using vitamin A
to send visual signals to his brain. Legally blind, he was headed toward a world of total
darkness by early adulthood. But a groundbreaking medical experiment had sent
viruses bearing healthy genes into his left eye, with the spectacular results realized not
at the hospital or in a lab, but at the zoo— in just days. Gene therapy was sorely in
need of good news, for in that same city, nine years earlier almost to the day, an
eighteen-year-old had died shortly after such an experiment.
Hands shaking, Ethan fumbled with his cell phone, finding the direct line to Dr. Jean
Bennett, impatient as the seconds passed. What was taking so long? Why didn’t she
pick up right away? He nervously shifted his weight from foot to foot as his gaze
remained on his son, who now had tears streaking his cheeks. When “Dr. Jean”
answered, Ethan could barely get the words out.
“It’s Ethan,” he stammered. “The sun … It hurts his eyes!”
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For the very first time, Corey Haas could really see. And in that moment, a
biotechnology was reborn.
Four days earlier, Corey had been legally blind, heading toward a word of total
darkness.
Dr. Jean Bennett
PART I
THE BEST THAT CAN HAPPEN
The lightning bugs were out tonight, their butts all lit up. Corey could see them!
—ETHAN HAAS, Facebook, June 28, 2010
Corey Haas
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1 MEETING COREY
When I met nine-year-old Corey Haas, it was hard to believe that just over a year
earlier, he had been well on his way to certain blindness. As a geneticist, I’d followed
the incredible story of his gene therapy, and as a mom, I was curious about the young
man who had made medical history.
Nancy, Ethan and Corey Haas
On a dazzling Saturday morning in early December 2009, I drove forty-five minutes
north to meet Nancy, Ethan, and Corey Haas, who live in the Adirondack Park. This
spectacular expanse of nature is not a park in the conventional sense, but a vast
swath of New York State peppered with small, older houses and log cabins nestled
among towering pines, with the occasional noticeably newer vacation home of a
down stater. The meandering Hudson River, only a few hundred feet across this far
north, snakes through the Haas family’s small town of Hadley, where Corey’s parents
grew up. The river is only a block away from their Cape Cod home, flooding their
basement in bad snow years. I’d been in the area before, so I found it easily, but some
of the reporters coming to interview the family over the past month had grown uneasy
when the towering mountains blocked even the most powerful cell phone and GPS
signals. But they made it— Ethan gives good directions.
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Although it took only days for the researchers to realize that Corey’s gene therapy had
worked, they had waited a year to publish the results, to be sure the effect lasted and
hadn’t caused any problems. They had learned from past gene therapy fiascoes, and
also knew Corey’s newfound sight would be big news. And they were right, with Good
Morning America setting the slightly hyped tone by describing “revolutionary surgery
that could give the gift of sight to the blind.” When the research paper appeared in
The Lancet in November, the Haas family traveled to New York City for the very first
time, where they made the rounds of the major TV news programs. Despite the intense
interest, though, only a few journalists ventured north to meet Corey on his own turf.
The morning of my visit, the scent of the season’s first snowfall was in the air. In the
Adirondacks, you don’t need a weatherman to know which way the wind blows, to
quote Bob Dylan. You just have to watch the squirrels. I had to hopscotch around
them as I walked up to the Haases’ front door, my hands laden with my laptop, books,
and a large box. The squirrels scurried everywhere, grabbing and stashing acorns in
the heart-shaped crevices at the bases of the tree trunks, occasionally dashing the
wrong way and bashing into the large picture window in the living room that reached
almost to ground level. The squirrels knew snow was coming.
When Corey opened the door, our eyes went first to the scampering squirrels, then to
each other. He gave me a huge grin, his blue eyes dancing behind thick glasses. He
took my leg and guided me to a coffee table, where I dropped my armload. As Ethan
and Nancy introduced themselves, Corey began rummaging around the rocks and
fossils in the box, a subset of the collection I’d begun at his age. Knowing he’d only
recently been legally blind, I knelt down and tried to show him how to feel the
indentations of the trilobites and brachiopods. But he proudly refused all assistance,
peering closely at each fossil, as I had at his age, slowly turning each one to see what
different angles might reveal.
I looked around. The décor was a welcoming blue and white, the rooms unusually tidy
for the home of a small boy. His toys were neatly aligned on low shelves and stacked
in corners, without the clutter that continually grows in my own home. But perhaps this
was a habit from the time when Corey had to memorize where every single item in a
room was in order to move about. Above the couch where I sat and typed as the
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family talked, a shelf held a dozen of Nancy’s 110-plus dolls. These were the special
ones, the others inhabiting closets and a storage room, packed in with Nancy’s
scrapbook collection. The other walls held what can only be described as a gallery of
Coreys. My favorite was a photo of Corey at about one year old, with a broad smile
and thick glasses, “Corey” stitched in needlepoint below, framed in red. The earliest
photo on the walls, taken when Nancy and Ethan had yet to learn that Corey had
inherited an eye disease, showed baby Corey stuffed into railroad overalls, a yellow
shirt, and white socks, looking off to the side as if he’d rather be anywhere else. A wall
in the dining room had the words live, laugh, and love spelled out in wood, with three
photos beneath each— all of the resident celebrity, Corey.
As Ethan talked and sorted copies of medical reports to give me, Corey jumped up
every few minutes to show his mother a rock. I glanced at them, trying, but failing, to
imagine this energizer bunny of a boy stumbling around the same living room, crashing
into the furniture like the squirrels outside careening from tree to tree. But that day he
rocketed around the cozy living room with ease. He showed me his handheld
Nintendo and how to extract a fart noise from a container of Silly Putty– like goop, and
then he asked me to name the minerals in his rocks. Periodically he’d demonstrate the
hardest part of the gene therapy for him— lying ramrod still on his back. I noted subtle
signs of what the family had been through. Once in a while Corey would lose his
footing briefly, and Nancy’s arm would instantaneously zap out to steady him,
sometimes hugging him close. He didn’t squirm away. Corey’s independence was still
new to all three of them. For years he could see only shadows, and in dim light,
nothing at all.
On September 20, 2008, two days before Corey’s eighth birthday, and fifteen months
before I met him, the Haas family had traveled to Children’s Hospital of Philadelphia—
CHOP. That week, Corey underwent a battery of what had become familiar tests, plus
a few new ones, and on Thursday morning was finally prepped for the procedure.
Then, as Corey lay anesthetized, the eye surgeon Al Maguire snipped open a tiny flap
in the left eye, the worse one, and carefully injected 48 billion doctored viruses into a
tiny space just above a thin layer of colored cells that resembled patterned bathroom
tile. This space and the layer of cells hug the rods and cones at the back of the eye.
Corey’s colored cells weren’t doing their job of nourishing the precious rods and
cones, which are called photoreceptors because they capture incoming light. His rods
and cones had been slowly starving for years, gradually becoming too weak to send
light signals to his brain to paint an image. As a result, before Corey lay down on the
operating table, he saw only blurry, dark shapes. But that would change— sooner than
anyone had even dared imagine.
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Al Maguire