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New England
Journal
Embargo: After
6 p.m. (Eastern
Feb. 20, L99L
Tirne)
NEW TREATIIENT FOR GENETIC DISEASE CITED IDI NEW ENGI,AND 'IOURNAI,
Scripps
Clinic
Pbysician
is lrtenber of Researeh
Team
LA JOLLA,
Journal
CALIF.
of
Medicine
international
study
treatment
of
disorder
Feb.
reports
of
Scripps
studying
some 250 to
C1inic
successful
in
boys,
Prognosis
has previously
treatment,
some children
a 13-center
disease
a
(cGD),
the
genetic
was John T. curnutte,
also
who has specialized
some girls,
in
in
ccD usuarly
the
first
but
with
agressive
into
their
been poor,
M.D.,
19 years.
for
within
are
of
garnma for
Foundation,
but
apparent
results
New England
hurnan interferon
investigators
CGD patients
seen prinariry
week's
4OO Americans.
and Research
and treating
becomes crinically
the
This
aranulomatous
one of the principar
of
L99L
recombinant
chronic
affecting
20,
now living
two
years
of
life.
antibiotic
early
twenties
or thirties.
The one-year
England
times
a
Journal
found
week
reduced
prolonged
infections,
effective
in
the
study
reported
that
25 were seen at
Feb.
of
frequency
interval
and well-tolerated
study,
the
injections
the
the
in
2L issue
interferon
and
severity
between infections,
therapy
Scripps
for
CGD.
Clinic.
Of the
of
the
New
garnma three
of
serious
and was an
128 patients
PAEE 2 --
IIEW ENGIJAND i'OURNAIJ STUDY
rrThe disease
frequent,
Iiver
or
disease
infections
kidneysr
rr Curnutte
can lead
in
rrf
in
a
within
the
fungar
pneumonia.
first
weeks of
the
notice
severe
who
girl
as the
lungs,
from
two years
first
infections
rn
just
are
cranky
the
of
life
that
are
with
an
ear
about big time pneumonia
r
saw a baby
different
times
rwho,
continued,
in the newborn nursery,
December,
three
Alabama, rf he
from
from
within
developed
ohio
the
who had
first
six
life.rl
her
resident
baby during
take
his
my handrtr
his
Rosa Garcia
year
first
who couldn't
noticed
of
temperaturer'
r
something
was wrong
life.
seemed to have a very
could
high
feer
fever.
r didntt
how hot
his
even
head was
she remernbers.
When he was 18 months old,
child
fight
rl
liver.
baby
rrRamon arways
with
such
to
rfComplications
within
few days of life
Chula Vista
need to
organs
rrWe're talking
said.
pneumonia
developed
with
children
the
recall
inability
and children.
arentt
abscesses
vital
an
explained.
physicians
and
by
death.It
rr Curnutte
infection,
to
diagnosed
babies
rrThese
or
to
usually
parents
unusual
characterized
serious
CGD is
when
is
diagnose
had a serious
she took
the cause.
lung
hirn to a series
One physician
infection.
within
of doctors
discovered
a short
time,
that
the
the
lung
take
out
collapsed.
[A doctor
his
lungr tr Garcia
one diagnosed
Two years
antibiotic
other
told
me he had cancer
said.
I
took
hirn to
have to
other
doctors
and
CGD.rl
old
treatment,
serious
rrl,uckily
and theytd
by
this
which
infections
time,
Ramon hras irnrnediately
saved his
over
the
lung
years.
and helped
hin
put
on
cope with
PAGE 3 --
NEI9 EIIGIJAIID {'OURNAIJ STUDY
In
1988,
portion
of
nearly
nine-year-old
the
Iives
qanma studies
interferon
infection-free,
Ramon entered
his
as any normal
child,
mother
while
the
Scripps
and since
says.
then
He attends
continuing
on his
Clinic
has
been
school
and
ganma
interferon
therapy.
rrRamon and the
to
Scripps
taught
Clinic
them
or
other
for
24 children
we saw during
a week while
parents
their
we verified
how to
inject
the
the
the
study
came
diagnosis
and
drugrfr
curnutte
said.
ganma needs to
The interferon
three
times
the drug
a week,
be injected
thigh
rrwetre not, certain
indefinitely.
can be stopped
in the
without
loosing
its
beneficial
or arm
when or
effects,
if
tt he
added.
Now that
plans
the
further
13-center
studies
of
test
of
the
drug
has
garnma with
interferon
ended,
Curnutte
an additional
25 to
3O patients.
side
the
rrwetll
follow
effects,
if
drug's
biochemistry
cells
to
of
saw his
doctors
a
look
diseases
in
he continued
researcher,
he
ganma works,
that
over
his
was
long-term
will
benefit.rl
L972 while
the
research
physician,
a
the
the mechanism of
how interferon
university.
As
at
arso study
ccD patient
Harvard
f was an intern
I had developed
were
Since
as
to
he
he was a
years,
as he
on the
blood
specialized
awarded
an
in
additional
biocheuristry.
rrBy the time
in Boston,
at
patients.
and
in
trwetlr
additional
first
become a doctor,
ccD
months
we can discover
identify
major
pediatrics,
doctorate
If
to
curnutte
six
anyrt' he said.
action.
we may be able
studied
them every
sending
L979, hets
their
at Massachusetts
a reputation
cases
seen close
to
for
General
work with
Hospitat
CGD patients;
merr he said.
to 60 patients
from throughout
the
PAGE 4 --
united
NE]r ENGIJAIID iTOI'R}IAIJ STUDY
states,
with
trccD is
longer
a
lives
most of
chronic
of
given
the
have
infections,
and within
of
CGD so
at
scripps
condition,
patients
for
antibiotics
aware
those
us
that
but
crinic
two
factors
disease, rr he
the
abirity
recent
years
children
are
to
since
have
some very
more physicians
getting
red
to
trrmproved
said.
treat
1986.
have
antibiotic
nasty
become
treatment
sooner.ll
ganma,
Interferon
appears
to
he
underlying
defect.
At
for
with
clinical
antibiotics,
improvement
therers
no
reversar
now
of
CGD
of
the
rl
Clinic,
Clinical
along
rHowever,
Curnutte
and Experimental
General
funded
therapy
continued.
Scripps
Molecular
rfbest
be the
patientsrrr
administered
a member of
Medicine
Research
by the National
is
Center,
Institutes
the
and associat,e
one
of
Department
director
78 centers
of Health
to provide
of
of
the
nationwide
experimental
therapies.
In
Young
June,
Investigator
Inflammation
Last
use
L99o,
of
therapy
for
Curnutte
Award
his
December,
rrappears to
research
the
marketed
the
the
body,
by Genentech,
with
the
International
on chronic
form
the
of
white
granulomatous
rnc.,
for
cerrsr
ganma is
san Francisco,
technology.
###
on
disease.
approved
CGD, noting
that
the
the
effectiveness.,
irnmune-enhancing
interferon
Outstanding
Congress
Food and Drug Adninistration
boost
A bioengineered
in
from
gallma as a treatment
interferon
naturarly
was presented
substance
deveroped,
using
produced
produced
recombinant
and
DNA