HUMAN G E N E T H E R A P Y 2:115-122 (1991) Mary A n n Liebert, Inc., Publishers Human Gene Therapy: Ethics and Public Policy* LEROY W A L T E R S ABSTRACT The first t h r e e h u m a n gene t r a n s f e r / t h e r a p y clinical p r o t o c o l s a r e n o w u n d e r w a y after h a v i n g b e e n s u b j e c t e d t o a n extensive review process b y t h e Recombinant D N A A d v i s o r y Committee (RAC) a n d i t s H u m a n Gene Therapy S u b c o m m i t t e e . T h e "Points t o C o n s i d e r " d o c u m e n t d e v e l o p e d b y t h e R A C established t h e f r a m e w o r k for e v a l u a t i n g genetic i n t e r v e n t i o n protocols. T h i s r e v i e w process is t a k i n g place in a b r o a d e r social c o n t e x t . Public attitude surveys in t h i s country have indicated a general lack of k n o w l e d g e in t h e area of genetic engineering b u t a n a c c e p t a n c e o f s o m a t i c - c e l l g e n e t h e r a p y a s t r e a t m e n t f o r d i s e a s e . I n t e r n a t i o n a l l y , n u m e r o u s policy s t a t e m e n t s o n h u m a n genetic i n t e r v e n t i o n h a v e b e e n p u b l i s h e d , a l l o f w h i c h s u p p o r t t h e m o r a l legitimacy of s o m a t i c - c e l l g e n e t h e r a p y f o r t h e c u r e o f d i s e a s e . T h e d e b a t e o v e r t h e e t h i c a l i s s u e s r e l a t e d t o s o m a t i c - c e l l g e n e therapy has e v o l v e d over a t e n - y e a r - p e r i o d . T h e time h a s n o w come to b e g i n a f o r m a l public process f o r the ethical a s s e s s m e n t o f g e r m - l i n e g e n e t i c i n t e r v e n t i o n . OVERVIEW SUMMARY human patients s i n c e 1 9 8 0 , a n d the f i r s t p u b l i c l y r e v i e w e d a n d approved h u m a n trials e v e r . An extensive discussion of the ethical issues relating to M y essay will f o c u s first o n the s o c i a l a n d ethical q u e s t i o n s somatic-cell g e n e t h e r a p y h a s b e e n t a k i n g p l a c e f o r a n u m - raised b y these t h r e e p r o p o s a l s a n d b y the r e v i e w process t h a t ber o f y e a r s . A c o n s e n s u s n o w a p p e a r s t o e x i s t t h a t s o m a t i c - was employed to evaluate them. Next, I w i l l consider the cell g e n e therapy for the treatment of serious disease is broader s o c i a l c o n t e x t o f r e s e a r c h o n g e n e t h e r a p y , both in the morally correct. W a l t e r s , f r o m his position as a leading United S t a t e s a n d a b r o a d . F i n a l l y , I will d i s c u s s p o s s i b l e future ethicist w h o has f o l l o w e d t h e field o f g e n e t h e r a p y t h r o u g h - developments, e s p e c i a l l y g e n e t i c i n t e r v e n t i o n s t h a t w o u l d a f f e c t out i t s h i s t o r y , n o w proposes t h a t a f o r m a l p u b l i c d i s c u s s i o n future g e n e r a t i o n s . I n t h i s c o n n e c t i o n , I w i l l r e c o m m e n d that a should b e g i n o n g e r m - l i n e g e n e t h e r a p y . formal public process for t h e ethical a s s e s s m e n t of g e r m - l i n e genetic i n t e r v e n t i o n b e g i n n o w . THE E T H I C A L E V A L U A T I O N O F GENE THERAPY IN T H E U N I T E D S T A T E S , 1 9 6 9 - 1 9 9 0 INTRODUCTION Public discussion of the ethics of human gene therapy goes THREE IMPORTANT MILESTONES in the history of medicine back in t h i s c o u n t r y at l e a s t t o t h e 1 9 6 9 A m e r i c a n Association were p a s s e d b e t w e e n M a y 1989 a n d J a n u a r y 1 9 9 1 . In t h o s e for t h e A d v a n c e m e n t o f S c i e n c e ( A A A S ) m e e t i n g h e l d i n B o s t o n 20 months, t e a m s of researchers at t h e National Institutes o f in l a t e D e c e m b e r . I n a s y m p o s i u m at t h a t m e e t i n g B e r n a r d D a v i s Health ( N I H ) i n i t i a t e d o n e h u m a n gene transfer s t u d y a n d two presented a p a p e r entitled "Threat and Promise in G e n e t i c human g e n e t h e r a p y p r o t o c o l s . T h e s e a r e t h e first s u c h s t u d i e s i n Engineering." T h i s p a p e r w a s l a t e r p u b l i s h e d i n S c i e n c e w i t h t h e Director, Center f o r B i o e t h i c s , K e n n e d y Institute o f E t h i c s , and A s s o c i a t e P r o f e s s o r o f P h i l o s o p h y , G e o r g e t o w n U n i v e r s i t y .