THE SCRIPPS RESEARCH INSTITUTE
I0666 NORTH TORREY PINES ROAD
LA JOLLA, CALIFORNIA 920]7
6t9 455-9t00
EI,IBARGOEDBy THE NEW EI{GLAI{D iIOURNAL OF MEDICINE:
6 pn Dec. 2, L992 (Eastern Standard
Media
Electronic
Dec. 3, L992
Print
t'tedia -- Thursday,
For
(6L9)
Sue Pondrom, News Bureau Manager,
Information:
Tine)
554-8290
RESEARCII REPORTED IN NEW ENGIJAND iTOURNAIJ OF UEDICTNE INDICATES
LESS EXPENSMIJY
GAUCEER DISEASE cAN BE TREATED lloRE EFFECTMITY,
CALIFORNfA
LA JOLLA,
Scripps
Research
reported
in
Medicine
that
Gaucher
and less
effectively
disease,
liver
and spleen,
whieh
is
by both
Treatment
for
Experimental
expensj-ve
news for
that
of
causes
the
WaIl
of
Journal
for
Medicine,
the
of
of
This
the
drug,
Boston,
most expensive
and the
one year
Times.
New York
at
the
headed by Ernest
Department
provides
data
dose
our health
and insurance
of Molecular
supporting
Beutler,
and
a low-dose,
rrln an era when we need to
schedule.
use of
Gaucher
$380r000.
investigators
of
for
enlargement
rrthe worldts
Street
more
thought.
treatment
by Genzyme Corporation
director
patients
previously
and bone pain.
bleeding
by Genzlrme costs
t,reatment
more judicious
a form
of
Journal
can be treated
The
have
California
New Enqland
than
is
at
Investigators
La Jolla,
the
a 1-55 pound patient
Now, a group
TSRf's
in
has been designated
medicinert
M.D.,
anemia,
L992
patients
disease
disorder
manufactured
reconmended
of
expensively
a hereditary
Massachusetts,
issue
(Ceredase)
Alglucerase
2,
(TSRI)
Institute
week's
this
Dec.
care
resources,
this
companies, rr Beutler
IIORE
is
said.
less
make
good
PAGE 2 --
per
That's
patients
frequency
schedule
Center
Scripps
of
that
noted
and tissues
within
a day.
of
proof
is
rrthe
that
administration
Now, there
60
with
very
that
in
in
that
the
new low-dose,
the
the
General
and Research
Ctrinic
from
with
Foundation.
New England
obtained
Clinical
Research
Their
Journal,
when high
high-
response
was
doses were given
two weeks.
rrAnd, the
Beutler
than
a quarter
as much, rl
Academy of
Sciences,
Beutler
less
said.
National
the
physician/scientist
internationally-known
awarded
the
Foundationrr
molecular
rrAnnual Award
deal
treatment
that
credit
for
availability
to
to
has been seriously
an
Roscoe Brady
at
who was recently
National
low-dose
Gaucher
Ceredase
Disease
the
and of
team headed by Dr.
Health
and Genzyme Corporation
making
this
patients
sufferers
the
is
disease.
frboth the
of
available
of
Gaucher
Institutes
of
from
studies
of
biology
National
a great
his
for
He stated
abroad
costs
treatment
A member of
its
Beutler
more frequent
Ceredase
of
indistinguishable
the
blood
were treated
ds reported
treatmentr
every
the
be treated
works. rf
strategy
Eleven
two weeks,
be more effective.
doses would
this
from
disappears
why we believed
small
to
once every
kilogram
medication
patients
Genzyme recommends that
Although
units
RESEARCH INSTITUTE
TIIE SCRIPPS
with
very
Gaucher disease.
from this
compromised
IIORE
effective
disease
by its
both
high
deserve
new
However,
here
cost. rt
and
PAGE 3 --
THE SCRIPPS RESEARCH INSTITUTE
rrWhat is
at
the
administer
hours
rrThe results
for
patients
the
treatment
of
for
the
dollars
schedule
of
rtTheir
that
to
to
promote
with
in
first
full
the
to
year,
will
that
low-dose
it
is)
(and there
the
program
always
have shown to
be fully
same scaling
could
the
whose
dose/low
high
probably
suggests
every
rr Beutler
were
diminish
as a
that
very
than
six
the
down of
for
the
data
highthe
cost
of
program
that
we
Thus,
the
rrEven
months.
are no published
be achieved.
be much higher
ef fective.It
of
millions
Ceredase.rl
Genzyme currently
effective
health
The dosage
afflicted.
of
care
Newsweek magazine
recommend can be halved
is
the
to
good
is
health
the
Gaucher disease,
use of
more rational
rrlt
Genzyme Corporation
according
will
treatment
patients
the
access
rl
which
strategy
frequency,
continued
and for
save hundreds
sales,
they
indicate
for
good news
are
how inadequate
needs of
resources.
added that
dosage
recognize
bad news is
this
Beutler
this
care
schedule
of
companies
meet the
have
$100 nillion
if
two weeksr t'
every
cost, rr he added.
high
its
We all
health
representatives
high
only
who were denied
disease
we recommend will
rrThe only
frequency
of
insurance
are to
that
dollars
the
from
because
as a whole.
system
result
24
body within
the
we have performed
studies
the
suffering
too,
said.
given
if
the
that
said.
Beutler
care
unlikely
gone from
is
way to
most effective
is
effective
be optimally
will
news,
which
a drug
of
the
and it
treatment,
administration
is
here
issue
PAGE ,l --
the
Although
advocated
more frequent
is
by Beutler
every-two-weeks
by a nurse,
our
receive
Other
members of
now assistant
of
Beutler
with
Thurston,
Scripps
Clinic
Medical
at
home
how most of
is
at
analysisr'
Terri
PauI Garver,
the
Ph.D.,
M.D.
with
head of
Gelbart,
radiologic
findingsl
Figueroa,
Andrea Kay, M.D.,
Oncology/Irnmunology
TSRI;
document the
Michael
Knoll
the
who assisted
and Dennis
neasurements
and Barry
at
Rosenbloom,
Los Angeles.
Beutler
noted
that
rrthe very
performed
were made possible
sponsored
General
Stein
California;
James Koziol,
who performed
to
safely
and this
team included
Redding,
Biostatistics
data
drug
Genzymets once
can be given
patient,
Beutler
Corporation;
U.D.,
I t { .D . , o f
in
than
the
drug, rr he said.
the
director,
Pharmaceutical
convenient
rrthe drug
the
now practicing
of
adninistration
member or the
family
Division
less
treatrnent,
patients
M.D.,
RESEARCH INSTITUTE
THE SCRIPPS
Foundation.
Clinical
costly
by the
resources
Research.Center
rl
###
studies
of
that
the
and the
were
NIH-
Sam and Rose