BloombergBusinessweek |SoonerThanYouThink June 10, 2019O
marionJordanspentalmostallofhisfirstyear
oflifeinhospitalisolationrooms.Thenightmare
beganwithwhatlookedatfirstlikediaperrash,a string
ofredmarksthatquicklyspreadacrosshisbodywhen
hewasjustshyof3 monthsold.Creamsandointments
failed,asdidtheeczemashampootreatmentanemer-
gencyroomdoctorprescribed.LastJuly,hoursafter
Omarion’spediatricianinjectedhisthree-monthvac-
cinesintohisthighs,theboy’sscalpbeganweeping
a greenpusthathardenedandpeeledoff,takinghis
wispybrowncurlswithit.Hisheadkeptcrustingover,
cracking,andbleeding,andhismom,KristinSimpson,
startedto panic.“Hiscriessounded terrible,”she
recalls.“I thoughtI wasgoingtolosehim.”
ShetookhersonbacktotheERtwonightsina row,
onlytohavethedoctorssendthemhomeeachtime
totheirapartmentinKendallville,Ind.“Theythought
I wassomeantivaccinationperson,”shesays.“They
lookedatmelikeI hada foilhatonmyhead.”Thenext
day,however,theboy’spediatriciandiagnosedpneu-
moniaandsentthembacktothehospitalfora third
time,atwhichpointtheygotmoreattention.A battery
oftestsrevealeda raregeneticdisordercalledsevere
combinedimmunodeficiencysyndrome(SCID),bet-
terknownasthe“bubbleboy”disease,whichmakes
40 to 100 Americannewbornseachyearextremelyvul-
nerabletoinfections,likeJohnTravoltaintheoldTV
movie.Omarion,transferredtoanOhiohospitalthree
hoursaway,wasconfinedtoanisolationroomwith
specialairfilters.
Leftuntreated,SCIDkillsmostchildrenbeforethey
turn2.Simpsonspentfivemonthswaitingfora bone
marrowtransplantforherson,theonlyconventional
treatmentandonethatthedoctorstoldhercarried
seriousrisks.Thentheytoldheraboutanalternative:
anexperimentalgenetherapythatjustmightcure
Omarionoutright.“Itwaskindoflikea leapoffaith,”
shesays,butshefiguredthatif it didn’twork,they
couldgobacktoprayingforthetransplant.
It worked.InApril,Omarionwasreleasedfromthe
St.JudeChildren’sResearchHospitalinMemphis,
wherea teamofresearchershadtakenstemcellsfrom
hisbonemarrow,bathedthemintrillionsofviralparti-
clesengineeredtocarrythegenemissingfromSCID
patients,andreimplantedthemintheboytobegin
replicating,repairingtheerrorsencodedinhiscells.
A preservativeinthecelltreatmentlefthimsmell-
inglikecreamedcornfordaysafterward,Simpson
says,buthisimmunesystemhasbegunworkingnor-
mally,hiswhitebloodcellcountrisinglikethoseofthe
otherninekidsinhisstudy.BloombergBusinessweek
watchedSimpsonandOmarionventureforthefirst
timeoutsideoftheirSt.Jude housing facility to play.
Inside what’s known as the Target House, they’d spentmonthsina filtered,isolatedapartmentforchildren
with compromised immune systems. “He’s just a
healthy baby now,” Simpson says in the house’s Amy
Grant Music Room, sponsored by and lined with photos
of the Christian pop singer. “It’s definitely a miracle.”
This is the tantalizing promise of gene therapies, the
potentialcuresfordozensofonce-incurableillnesses.
TheU.S.FoodandDrugAdministrationissueditsfirst
approvalofasystemicgenetherapy,a NovartisAGtreat-
mentforspinalmuscularatrophy,onMay 24 andsaysit
expectstoapprove 10 to 20 therapiesa yearstartingin
2025.Therearemorethan 800 trials under way, targeting
diseases including rare metabolic disorders, sickle cell
anemia, hemophilia, and Parkinson’s. As the list grows,
such treatments have the potential to fundamentally
remakethehealth-caresystemateverylevel.
Therearetwobigcaveats. First, moststudies
haven’t run longer than a few years, so it’s impossible
to know yet whether the therapies will remain effective
for life, help everyone the same, or yield side effects
decades in the future. Only about 150 children have
received the Novartis muscle treatment, Zolgensma,
and at least two have died, though the therapy doesn’t
appear to have been to blame.
The other problem is cost: These treatments
are expectedtorunseveralmilliondollarsa pop.
Zolgensmais themostexpensivedrugeverapproved
intheU.S.,witha pricetagof$2.1million for a one-
time infusion.
“The cures are coming,” says Alexis Thompson,
a gene therapy pioneer who heads the hematology
department at the Ann & Robert H. Lurie Children’s
HospitalofChicago,“buttherearestilla lotofcon-
siderations.”Safety,efficacy,fairness,andlong-term
follow-up care top her list. “Even if someone under-
goes gene therapy and is cured of a disease, we need
toensurethattheyhaveaccesstoa health-caresys-
temthatwillallowustofollowthemforconceivably
10 to 15 years,” she says. Then there are the more
ghoulish concerns about returns on investment that
tend to come with pricey research and development
projects. “While this proposition carries tremendous
value for patients and society, it could represent a
challenge for genome medicine developers looking for
sustainedcashflow,”GoldmanSachsanalystSalveen
Richterwrotelastyearina notetoclientstitled“Is
CuringPatientsa SustainableBusiness Model?”E
very human has about 20,000 genes, half each
from Mom and Dad, that make proteins to break
downfood,maintaincellhealth,provideenergy,pass
signalstothebrain,andsoon.A defectin a singlegene
cancauseanyoneofabout7,000potentiallydevas-
tating or life-threatening diseases. Viruses have been50