netic disorders. Already, Minassian
and Gray have more than six months’
worth of studies lined up. “I get con-
tacted every other week by a new fam-
ily about some disease,” Gray says.
“We have people preparing to fly from
Australia to come live here,” Minas-
sian adds. “It’s happening.”The Hann family couldn’t afford to
wait for UT Southwestern’s virus pro-
duction facility. The clock was ticking
from the moment they got Joseph’s
Batten diagnosis. He lost his vision
within about a year, and shortly after
that his motor skills degraded to the
point that he could no longer walk
on his own. Around the start of this
year, he lost his ability to speak much
beyond cooing or squealing.
The sliver of hope that Minassian
offered the Hanns came at a high
price. Because of the rarity of Joseph’s
disease—there are no hard numbers
available, but Minassian estimates 1
in 500,000 is born with it—funding
for a gene therapy study was much
harder to come by than it would be for
more common diseases. If the Hanns
wanted a clinical trial to test a poten-
tial cure for their son, they’d have to
raise money—at least $1 million, they
were told, possibly more. Insurance
companies don’t pay for such scien-
tific research.
After establishing a fund called Bat-
ten Hope, Gina and Matt began reach-
ing out to corporations and founda-
tions as well as their social circles.
Within fourteen months they’d met
their goal, and to date they’ve raised
more than $1.5 million. (Minassian
marvels that one of the unexpect-
ed benefits of having set up shop in
Dallas is that it’s an excellent place to
raise money.) The Hanns eventually
connected with Vigene Biosciences,
a company that could build the vi-
ral doses sooner than Gray could. If
everything goes according to plan,
UT Southwestern’s clinical trial on
CLN7, using the Vigene doses, will
begin later this year.
There’s no guarantee, however,
that Joseph will be among the six to
ten patients who will each have a qua-drillion viral particles (a couple tea-
spoons’ worth) injected into their spi-
nal fluid in an attempt to stop or slow
their brain’s degeneration. There’s a
possibility that guidelines for the trial,
as set down by the FDA and UT South-
western, may determine that he is no
longer healthy enough to participate.
For Minassian and Gray, it’s hard to
see a patient like Joseph—one they’ve
come to know well—potentially de-
nied treatment. Gray has become
personal friends with the Hanns. But
there are good reasons for running
a study like this one strictly by the
book. “If we move in an irresponsi-
ble way and, God forbid, something
disastrous happens in the context of a
clinical trial, it doesn’t just affect that
patient. It doesn’t just affect our one
trial,” Gray says. “It affects the whole
field of gene therapy.”
The Hanns are painfully aware that
Joseph might get left out. They choose
to see their now seven-year-old’s
journey as one of hope, not despair.
Many children with rare diseases go
misdiagnosed for years—and yet, af-
ter Joseph’s first grand mal seizure,
the Hanns just happened to end up
in an ER with a doctor who just hap-
pened to have read a paper about
Batten disease. They just happened
to find Minassian at the moment he
was setting up a gene therapy cen-
ter, and Minassian just happened to
recruit Gray, who just happened to
have recently done research on the
very disease that Joseph suffers from.
The Hanns pray for the good for-
tune to continue, Gina says. As we
chat, Joseph sits a short distance
away, parked close to the TV. He can’t
see the cartoons, but he still loves to
listen along. Periodically he honks the
horn attached to his walker to playful-
ly chase off his younger sister. He’s a
happy boy. “I have all the faith in the
world this is going to come out in our
favor,” Gina says. She also knows that
the CLN7 trial is happening because
of Joseph, and even without him in
it, it could save other children’s lives.
“That’s a pretty awesome legacy.” TMEDICINE