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ENZyt'lE REPIJACEI,IENT
TIIERAPY FOR GAUCHERDISEASE
TO BE DISCUSSED AT !{AiIOR UEETING OF CLINICAL RESEARCHERS
LA JOLLA' CALIF. NIay 2, 199L -medical
benefits
disease
will
of
a
more
be discussed
and Research Foundation
Recent research
cost-effective
indicating
therapy
for
Gaucher
by Andrea c. Kay, M.D. of scripps
at the May 4 CLinical
Meetings
the
in
clinic
Seattle,
Washington.
The Clinical
researchers
who belong
the American
Federation
Meetings
Society
for
are an annual event attended
to
for
Clinical
the
Clinical
part
of
crinic's
a
patients
team
Department
A lipid
disease
with
benign
is
to
death
that
who have severe
by
degrade
is
and the American
clinical
in
It
the
Beutler,
M.D.,
diagnosed
complex
varies
quite
head
and Experirnentar
characterized
first
trials
by the
molecules
in
Jewish
from a rapidly
year
with
Gaucher disease.
common among the
as weL1.
occurringr
it
Ernest
disease
particularly
in others,
on successful
of Molecurar
storage
enzyme required
found
Led
Investigations,
r r E n z y m eR e p r a c e m e n t T h e r a p y i n T y p e
titred
I Gaucher Disease, rr reports
Clinic
of Arnerican Physicians,
Research.
Kayts presentation,
Scripps
Association
by clinical
of
by accident.
life,
six
Kay was
of
scripps
Medicine.
deficiency
celIs,
of
Gaucher
popuration,
fatal
to
but
disorder,
a disease
Treatment
an
so
has been
PAGE
2
SCRIPPS
CIJINIC
unsatisfactory,
with
the
only
cure
achieved
by
bone
marrow
transplantation.
In the
with
to
enzyme replacement
the
shortage
yearsr
in
l-970s, American
of
enzyme to
scripps
smaller
Beutler
and
smaller
doses,
Kay
enzyme.
Within
that
the
cost
to
recent
administer
the
and the
patients
cost
for
efficacy
responded
treatment
the
a year.
$rso,ooo
investigated
the
due
and found to be effective
would be about
study
largely
doses of the enzyme on a more frequent
note
The Scripps
Clinic
one-fourth
of the
was administered
included
a decrease
of
basis.
weII
to
was decreased
the
by more
began in
in
January
L99O with
patients
used.
The enzyme
enzyme dose traditionally
the
days or three
size
improved
of
liver
enlarged
function
times
a week.
livers
Results
and spleensr
peripheral
tests,
€ts
blood
and enzyme levels.
In addition
P. Garver,
carried
which
study
on alternate
as gradually
counts,
is
In
in
funded
her
children
patients
General
by the
National
Institutes
that
or
from
benefit
small
would
least
approach
for
the
of
studies
Research
Health.
May 4 Clinical
5rOOO patients
the
cost
$r birlion
I,TORE
with
rrEven if
frorn treatment.
adults,
C1inical
A. Saven,
were
Center,
Additional
Sam and Rose Stein.
remarks
at
included
A. Dawson and B. Rosenbloom.
C1inic's
prepared
could
and Kay, researchers
Scripps
was received
estimate
the U.S.
to Beutler
D. Thurston,
out
funding
will
the disease
75 percent.
given
well
However,
clinic
to treat
but were unsuccessful
was manufactured
Gaucher disease.
administering
sought
a commercially-available
a L54-pound patient
The
than
therapy,
dD enzyme product
treaLing
researchers
of
Meetings,
Kay
Gaucher disease
many of these
enzyme alone
per year. . . r at
the
for
in
are
these
current
--_-l
PAGE 3
SCRIPPS
therapeutic
CIJINIC
doses recommended by the
She will
also
note
that
Itprovides
Gaucher
patients
the
time.
It
first
strategies
ef f ective.
that
will
is
the
with
availability
a safe
incumbent
be
enzyme manufacturer.
rl
###
enzyme therapy
and effective
upon us to
economically
of
therapy
determine
feasibre
as
for
treatment
welr
as