Bloomberg Businessweek - USA (2019-11-11)

B U S I N E S S

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18

Edited by

James E. Ellis

● A costly targeted gene

therapy shows the promise and

pitfalls of medical advances

A $1.8 Million Drug

To Treat Sicily’s Curse

On a sweltering summer morning, Daniela Miccichè

headed from her home in the center of Sicily to a hos-

pital in the ancient city of Palermo on the coast. It’s a

trip she repeats every month. A travel agent from the

town of Caltanissetta, Miccichè has no choice but to

make the two-hour journey to the Franco and Piera

Cutino clinic. Scores of others pass through its front

doors, all with the same disease. She’s among an

estimated 7,000 Italians, many of them Sicilian, who

dependontransfusionstotreatbetathalassemia,a

geneticdisorderthathampersredbloodcells’ability

to carry oxygen.

Miccichè has waited a long time for the day she

can be free from these procedures, a constant in

her life since she was 3 years old. “I grew up think-

ing that one day we would reach this goal,” she says.

PatientssuchasMiccichèareclosetohaving

theirwishgranted,butit’sgoingtocosttheItalian

medicalsystema hugesum.A novelgenetherapy

from U.S.-based Bluebird Bio Inc., approved in

EuropeinJune,issettoberolledoutnextyear

following reimbursement negotiations with

government health systems. The treatment, called

Zynteglo, has a sophisticated mechanism and a

ILLUSTRATION BY REBEKKA DUNLAP

Bloomberg Businessweek November 11, 2019