The Wall Street Journal - 26.11.2019

THE WALL STREET JOURNAL. Tuesday, November 26, 2019 |A

U.S. NEWS

BYPETERLOFTUS

New Sickle Cell Drugs Emerge

A microscope image shows a sickle cell, left, and normal red blood

cells. Oxbryta is the latest drugapproved for sickle cell disease.

deadly to small liberal-arts

schools by hurting their ability

to recruit students and deep-

ening their financial woes.

Many small liberal-arts col-

leges have shut down or

merged with other colleges, in

part because prospective stu-

dents have been questioning

the value of the degrees and

the heavy debt to cover tuition

in a strong job market.

The accreditor cited Hamp-

shire’s latest efforts, including

a fundraising push led by

filmmaker Ken Burns, a 1975

graduate, as the Amherst,

Mass., school tries to stabilize

its still-shaky finances. The

college was thrown into tur-

moil early this year when

school leaders, citing financial

deficits, opted not to admit a

fall class and considered part-

nering with another school.

Students and alumni didn’t

like the idea of losing indepen-

dence at the school known for

not having majors and formal

grades. Instead, the college set

about trying to right the ship

and faced a big test in the re-

cent accreditation review.

“There are people that are

watching Hampshire with

some skepticism, particularly

parents and some high-school

counselors,” said Edward Win-

genbach, who became Hamp-

shire’s new president in July,

in an interview. “I hope this

will provide them the sense of

security they need to know we

will be around in the future.”

The regional accreditor said

Hampshire made considerable

progress on factors like fi-

nances and fundraising. Still,

there is work ahead: the ac-

creditor said it would continue

to scrutinize the school’s insti-

tutional resources, which in-

cludes finances, and expects a

report in two years.

Hampshire College, one of
many small liberal-arts
schools in the U.S. trying to
stay afloat while competing
for a dwindling supply of high-
school graduates, has cleared
a key hurdle with its regional
accreditor.
The New England Commis-
sion of Higher Education said
after a review that it isn’t put-
ting Hampshire on probation,
which can lead to losing ac-
creditation needed to access
federal student loans and
grants. Such losses can prove

BYJONKAMP

Hampshire College Clears Crucial Hurdle

The U.S. Food and Drug Ad-
ministration approved a new
drug for sickle cell disease
Monday, adding to a new wave
of treatments that promise re-
lief from the life-threatening
blood disorder that largely af-
flicts African-Americans.
Oxbryta, a once-daily pill
fromGlobal Blood Therapeu-
ticsInc., blocks a process in
blood cells that can lead to
anemia and organ damage,
hallmarks of sickle cell disease.
It is the second treatment to
get FDA approval in recent
weeks, after the agency ap-
provedNovartisAG’s Adakveo
to reduce the frequency of
bouts of pain that sickle cell
patients can suffer.
Doctors say the new drugs
have the potential to ease the
complications of sickle cell dis-
ease, which afflicts about
100,000 Americans.
More new treatments are
on the horizon. Drugmakers
includingBluebird BioInc.,
Vertex PharmaceuticalsInc.
andCrispr TherapeuticsInc.,
along with government re-
searchers, are developing ex-
perimental therapies that may
have potential to cure or pro-
vide long periods of relief.
“Everybody’s been waiting
for this moment where the
flood gate of new treatments
is opening,” said Dr. Biree An-
demariam, chief medical offi-
cer of the Sickle Cell Disease
Association of America and a
sickle cell specialist at UConn
Health, in Farmington, Conn.
The therapies are expensive,
part of a growing trend of high
prices for drugs that treat rare
diseases. Global Blood Thera-
peutics priced Oxbryta at
$125,000 a year, though the
company said the drug would
cost most health plans
$96,000 a year after discounts.
Novartis’s drug lists for as
much as $113,100 a year, de-
pending on the weight of the
patient.
Drugmakers say the treat-
ments can help cut other costs,
such as hospitalization.
Sickle cell disease is caused
by an inherited mutation in an

oxygen-carrying protein known

as hemoglobin. Decades ago,

most children diagnosed with

it didn’t live into adulthood.

Life expectancy has risen sig-

nificantly since the 1980s, due

to newborn screening and

treatment advances like blood

transfusions and penicillin to

prevent severe infections.

But most patients don’t live

longer than 50 years, doctors

say, and many face debilitating

complications including

strokes and kidney failure. Few

drugs have been approved in

recent decades.

Global Blood Therapeutics,

of South San Francisco, Calif.,

designed Oxbryta to stop a

molecular process that results

in sickle-shaped red blood

cells, the feature that gives the

disease its name.

After 24 weeks of treat-

ment, about 51% of the clinical-

trial subjects who had received

a high dose of the drug had

higher levels of hemoglobin,

compared with 33% among

those getting a lower dose and

7% in the placebo group. Those

taking the drug also experi-

enced reduced anemia.

“We think what this drug is

doing is fundamental in terms

of reversing disease,” said

Global Blood Therapeutics

Chief Executive Ted Love.

Reported side effects in-

clude headaches and diarrhea.

The FDA approved the

drug’s use in patients aged 12

years and older.

Patricia Rose, 52, of Ber-

wyn, Ill., said she took Oxbryta

in a clinical trial along with

another drug called hydroxyu-

rea she has been taking since

around 2000, and the combi-

nation helped alleviate the

painful episodes resulting from

the disease more than the

older drug alone.

Novartis’s Adakveo, infused

intravenously once a month,

was approved in patients age

16 and older to reduce the fre-

quency of a painful complica-

tion of sickle cell that occurs

when blood circulation be-

comes obstructed. The pain of-

ten sends patients to the hos-

pital.

The National Institutes of

Health said last month it plans

to invest at least $100 million

in the next four years toward

finding affordable, gene-based

cures for sickle cell disease

and HIV. The Bill & Melinda

Gates Foundation said it would

invest an equal amount toward

the goal.

FROM LEFT: JANICE HANEY CARR/CDC/CELL FOUNDATION OF GEORGIA/ASSOCIATED PRESS, GLOBAL BLOOD THERAPEUTICS

The life-threatening

blood disorder

largely afflicts

African-Americans.

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