COURTESY ST. JUDE. DATA: THE JOURNAL OF GENE
MEDICINE
Bloomberg Businessweek | Sooner Than You Think June 10, 2019Manufacturing facilities
like the one at St. Jude
can spend months
growing, purifying, and
testing therapiesthoughttooffera solutionsincethe1980s,
whenanMITresearcherfirstmodifiedone
todelivera healthygeneintoa humancell.
In 1990 theNationalInstitutesofHealthused
sucha treatmenttosave4-year-oldAshanthi
DeSilvafromanimmunedisorderthatwould
otherwisehavekilledherin a matterofyears.
A handfulofearlydisasterstemporarily
haltedprogressongenetherapies.In 1999
anoverwhelmingimmuneresponsetoone
treatmentkilledan18-year-oldfromArizona,
turninghiseyesyellowandspikinghisfever
above104F.Researchersabandoneda SCID
trialinFrancein 2002 afteroneofthe 10 bub-
ble boys developed leukemia; eventually, four
of them did. Still, scientific work continued,
and as researchers created better tech-
nology, they started delivering remarkable
results. In 2007 patients with a genetic form
of blindness saw their vision improve. Blood
cancer patients given only weeks to live in
2012 went into remission after their immune
systems were reprogrammed to attack malig-
nant cells. Hemophiliacs started producing
clotting proteins. The past five years have
been revolutionary, says Lindsey George, a
hematologist who leads gene therapy trials at
Children’s Hospital of Philadelphia. “This is a
transformative time,” she says. “It isn’t just a
hope and a whim. It’s supported by data and
significantly so.”
George cautions that it’s tough to iden-
tify rare side effects with samples as
small as most of the studies so far have
used. Part of the problem is the supply of
the therapies themselves, which requiremonthsofgrowing,purifying,andtestingat
manufacturingfacilitiessuchastheoneat
St.Jude.InMemphis, 200 1-millilitervialsof
themastercelllineusedtotargetSCIDsitin
a stainless-steeltankofliquidnitrogen 4 feet
in diameter, kept at a temperature of -256F.
After thawing one of the tubes, technicians
covered in sterilized clothes feed and nur-
ture the cells for weeks in enclosed cabinets,
moving the treatments from vials to flasks to
stacked trays as they grow. Eventually, tril-
lionsofparticles,testedformonthstoensure
theirpotencyandsafety,canbedistilledinto
treatmentsforasmanyas 600 infants with
the most severe form of SCID.
St. Jude spent millions of dollars to
develop a bank of cells so it can treat new
patients in a little more than a week at a cost
of tens of thousands of dollars per case.
For less practiced facilities, the manufac-
turing process for each treatment can cost
$500,000 or more.R
esearchersanddoctorswanttocure
diseasesthataffectthemostpeople,
and the industry is focused on develop-
ingthosetreatmentstosell.Novartissays
Zolgensmadosesareworthmorethandou-
blethe$2.1million the company is charging.
Spark Therapeutics Inc. has set the price
for Luxturna, its treatment for an inherited
form of blindness, at $425,000 an eye. “We
really tried to focus on what is sight worth
for a young child or an adult,” says Spark
Chief Executive Officer Jeff Marrazzo, “as
well as what we needed to charge to be able
to reinvest.”
For some patients, gene therapy would
be cheaper than current lifelong treatments.
Take Tim Sullivan, a 63-year-old computer pro-
grammer born with hemophilia. Sullivan’s ear-
liest memories involve lying in a hospital bed,
looking up at a bottle of blood slowly dripping
essential clotting proteins into his veins. Even
though medical advances eventually made it
easiertoextracttheproteinsfromdonated
blood,suchtreatmentsleftSullivaninfected
withHIVandhepatitisC,thediseases that
killed his two younger brothers, also hemo-
philiacs. He suffered almost every day with
pain that came with even minor bumps and
bruises and with inflammation from his weak-
ened joints. “It’s been my life,” he says. “Pain
1989 2019 was my constant companion.”2001000Number of gene
therapy clinical trials
approved worldwide51