of pediatric neurology, he made
a proposal. He wanted to build a
gene therapy center, on the cut-
ting edge of medical technology,
where researchers and physicians
could collaborate on treatments for
a whole spectrum of genetic dis-
orders. He was tired of attending
funeral after funeral, frustrated
about not being able to do more
for patients. UT Southwestern
had been expanding its research
and treatment facilities in recent
years, thanks in part to generous
gifts from Dallas philanthropists,
and jumped at the opportunity.
“They saw that this is part of the
future of medicine, which other
places haven’t,” Minassian says.
UT Southwestern’s gene ther-
apy center is expected to be fully
operational by the end of this year.
And the Hanns no longer have to
simply accept that their son’s ear-
ly death is a foregone conclusion.
After Minassian saw Joseph’s test
results, he of course told them the
prognosis was bad. But now, with
his center being built, he was able
to add, “There’s hope, maybe.”Steven Gray, a biomedical re-
searcher at the University of
North Carolina, was on Minas-
sian’s mind when he spoke to the
Hanns. Minassian was trying to
recruit Gray, who had done some
work on CLN7, to join him in Dallas.
Today, more than two years
later, the men sit side by side at
a long table in a windowless con-
ference room on the UT South-
western campus, at the end of a
hallway lined on both sides by lab
space that’s being transformed
into the gene therapy center of
their dreams. It’s a collaborative
effort between Gray’s team of re-
searchers and Minassian’s team
of physicians. While there are a
handful of other academic gene
therapy centers in the country,
most tackle just a few diseases.
“Here we are bringing in pediatric
neurologists to work with [Gray]
on twenty or more different dis-eases,” Minassian says.
Guangping Gao, the president
of the American Society of Gene
and Cell Therapy, says an academic
research institution operating at
that scale would be “incredible.” He
describes Gray as “very motivated.
His goal is to clear rare diseases off
the table.”
Indeed, UT Southwestern’s pro-
gram could soon transform many
lives. For much of modern history,
doctors have classified diseases pri-
marily based on their observable
symptoms. A patient like Joseph,
going blind and brought to an ER
after a violent seizure, might well
have simply been diagnosed as epi-
leptic. That’s not incorrect; “epilep-
sy” is a general term for any brain
disorder that causes seizures. But
since the mapping of the human
genome—the complete genetic
blueprint for building a human—
was completed, in 2003, research-
ers have been able to pinpoint the
exact causes of a host of diseases,
including the broad array lumped
together as epilepsy. Joseph’s form
of Batten disease, for instance, is
known to be caused by a mutation
in the CLN7 gene that results in
a failure to produce a necessary
protein, which in turn leads to de-
velopmental problems.
Most childhood genetic disor-
ders can similarly be traced to a
problem with a single gene, and
that’s why curing such conditions
seems so tantalizingly possible—
even if scientists still can’t entire-
ly explain the processes by which
a specific defect alters the brain’s
development. “If we could simply
fix the cause, irrespective of our
relative lack of understanding of
how the brain works, we could help
the patient,” Minassian says. “I re-
ally wanted to dedicate the rest of
my career to working on ways to
solve this problem. In so many oth-
er diseases, the problem is that we
don’t understand the cause. Here
we know the cause.”
The potential solution: in the
simplest terms possible, doctorsand could transfer to work at its
corporate headquarters there.
Soon a chain of email referrals
put them in touch with Dr. Berge
Minassian, a pediatric neurolo-
gist who had joined the University
of Texas Southwestern Medical
Center only a few weeks earlier,
in January 2017. A phone call with
Minassian, who had worked on
similar genetic disorders, sealed
the Hanns’ decision. They’d pack
up their four children and move to
Texas in hopes that it would give
their son a better shot at life.
By the time the genetic test re-
sults arrived, a few weeks later, the
Hanns were closing on the pur-
chase of a house in the prosper-
ous suburb of Southlake. Joseph
indeed had Batten disease—one of
the rarest forms, known as CLN7.
As the disease progressed, he would
lose not only his sight but also his
ability to walk and speak. He’d be
lucky to live into his teenage years.
Minassian had come to Dallas
for the specific purpose of trying
to cure patients very much like Jo-
seph. For twenty years, the native
of Lebanon had happily worked at
Toronto’s Hospital for Sick Chil-
dren. But when UT Southwestern
asked him to become its new headGina Hann reads
to Joseph as
he undergoes
an EEG test in
April.COURTESY^ OF^ UT^ SOUTHWESTERNMEDICALCENTER56 TEXAS MONTHLY
MEDICINE