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AndrewJowers
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33
FORIMMEDIATE
RELEASE
#070987-3
L A J0 L L A ,0 ct. 19 -- T h e U.S. Foodand Dr ugAdm inistr ationtoday
a n n o u nced
a p p rova lo f a h i gh ly pur ified concentr ateof Factor VIII, a
b l o o d c oa g u l ati onp rote i n u sed to tr eat hem ophilia.The pur ification
p r o c e s s ,d e ve l op e dat S cri pp s Clinic and Resear chFoundation,allows
t r e a t m en to f he mop h i l i acsw i th a gr eatly r educedr isk of AIDS,hepatitis
a n d o t h er vi ral i n fe cti on .
U n ti l no w , he mop h i l i acshavehad to r isk tr eatm entwith concentr ate s
o f F a c to r V III co n ta mi n a te with
d
vir uses pr esent in the blood of the
o r i g i n a l d o n o r - a n d th e y a l most invar iably developnon- A, non- B
h e p a t i t i s, w hi ch ca n n o tbe sc r eenedfr om the blood supply. In r ecent
y e a r s , up to 90 p e rcen tof h em ophiliacs
whor eceivedconcentr atesin the
e a r l y l 9B 0 s h a ve sho w nevi de nceof infection with the AIDSvir us, and
betweenI percent and 2 percent havedevelopedacquired immune
deficiency
syndromeor have died from the disease.
The processof purifying the Factor VIII protein from normalblood
plasmawas developedby Drs. TheodoreZimnennan
and Carol Fulcher of the
Research
I n s t i t u t e o f S c r i p p sC l i n i c b a s i c a n d c l i n i c a l r e s e a r c h
department.
(More)
# 0 7 0 9 8 7 -3
0ct. 19, 1987
Page2
C o m merci adle ve l op menand
t clinical tr ials of the nev,concentr ate,
knowncommerciallyas Monoclate,were fundedby ArmourPharmaceuticalCo.,
w h i c h w i l l un d e rtakei ts w o rldwidem ar ketingand distr ibution. Ar m ouris
a d i v i s i on o f R o rer G ro u pInc. of For t l,lashington,Penn.
is at
Thespecific activity, or units per milligram, of Monoclate
l e a s t 1 ,0 0 0 ti mes grea te r than Factor VIII concentr atespr eviously
to other com m er cial
a v a i l a bl e. T h e p u ri ty i s 99 .9 per cent, com par ed
F a c t o r V III prod u cts,w hi ch have a pur ity of less than I per cent,
a c c o r d i ngto R orer o ffi ci a l s.
dt v a r i o u si n s t i t u t i o n s
I n c l i n i c a l t r i a l s o f M o n o c l a t ec, o n d u c t e a
throughout the country, the concentratethus far has not transmitted
v i r a l i nfe cti on . Mon o cl ate ,s elf- adm inister edintr avenously,also solves
problemof crude concentrates: It does not depressthe
another common
immune
syste m.In fa ct, i n som ehem ophiliacstr eated with M onoclate,
i n c l u d i ng th o se e xp o se dto the AIDSvir us, depr essionof som easpectsof
the immunesystemwas stoppedor reversed.
" T h e p o s s i b i l i t y o f w i d e s p r e a ad p p l i c a t i o no f a p u r i f i e d F a c t o r V I I I
a p p ar entwhenwe r ealized howr elatively sim ple
c o n c e n tra teon l y be ca me
t h e p r o ce ssw as, an d th a t i t had an excellent yie' |d," said Zim m er m an.
"And, of course, the AIDSepidemicmakesvery clear the importanceof
s u c h a te ch n i qu e".
T h e meth o do f p u ri fyi ng Factor VIII involves exposingplasm aor a
F a c t o r V III-co n ta i ni ng fra cti on of plasm ato m onoclonalantibodies
a t t a c h edto a b e d o f i n e rt m ater ial. Theseantibodies bind and r etain th e
F a c t o r V I I I c o m p l e xw h i l e a l l o t h e r m a t e r i a l s , i n c l u d i n g v i r u s e s , a r e
w a s h e daw a y.P u re F acto r V III is then str ipped fr om the antibodies,
c o l l e c t ed , h e a te d , froze n a n d dr ied to ensur estability.
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#070987-3
0ct. 19, 1987
Page3
Zinunerman
and Fulcher's work, fundedin part by the National
I n s t i t u te s of H ea l th a n d A rmour ,beganin 1980as a r esear chpr oject to
c h a r a c teri ze F a cto r V III, a c oagulationfactor essential for the
f o r m a t ion of fi bri n , an i nso luble pr otein that binds platelets together
i n t o a to u g h , w o u n d -sea l i nm
g ass- a blood clot.
H e mop h i l i a,o n e o f morethan 1,400diseasesnowknownto r esult fr om
t h e i n h eri tan ce o f a si ng l e m utantgene, is a blood- clotting disor der
a f f e c t i ng o n e i n 1 0 ,0 0 0p e o plewor ldwide.About80 per centof
h e m o p hi l i acsare d e fi ci e n t i n Factor VIII, the m ost sever elyaffected
h a v i n g less tha n 1 p e rcen t o f nor m allevels in their blood.
A l t h o u g hh e mop h i l i ai s i ncur able, for the past 30 year s victim s
of Factor VIII
u n d e r g o i ngb l ee d i nge p i sod e shave taken supplem ents
e x t r a c t ed fro m n o rma lb l oo d plasm a.In m ost cases, this extr a Factor VIII
i s s u f f i c i e n t t o a l l o w b l o o dc l o t t i n g , b u t h a s c a r r i e d t h e r i s k o f v i r a l
i nfection.
Zirnnerman,
a graduateof HarvardMedica'lSchool, Boston, has been at
Sc r i p p s C l i ni c an d R e se a rchFoundationfor l7 year s. Chief of the Scr ip ps
C l i n i c C oa g u l ati onL a b o rato r y,he is headof the exper im entalhem ostas i s
h e p a r t m e n tI .n J u l y 1 9 8 5 , h e
d i v i s i o n i n t h e B a s i c a n d C l i n i c a l R e s e a r cD
w a s a p po i nte dcha i rmano f th e National Hem ophiliaFoundation' sM edical
a n d S c i e n t i f i c A d v i s o r yC o u n c i l .
F u l c h e r , a g r a d u a t eo f t h e U n i v e r s i t yo f V i r g i n i a , C h a r l o t t e s v i l l e ,
Foundationsince 1979.
h a s b e ena t S cri pp s C l i n i c and Resear ch
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