Table Of ContentS. HrG. 106-447
GENE THERAPY: IS THERE OVERSIGHT FOR
PATIENT SAFETY?
HEARING
BEFORE THE
SUBCOMMITTEE ON PUBLIC HEALTH
OF THE .
COMMITTEE ON HEALTH, EDUCATION,
LABOR, AND PENSIONS
UNITED STATES SENATE
ONE HUNDRED SIXTH CONGRESS
SECOND SESSION
ON
EXAMINING CERTAIN ISSUES REGARDING PATIENT SAFETY IN GENE
THERAPY CLINICAL TRIALS, FOCUSING ON FETDANETR ALT OOVUEDR SIGHT PRO-
CEDURES AND GUIDELINES FOR INFORMING PATIENTS AND THEIR
FAMILIES OF POTENTIAL RISKS AND BENEFITS OF GENE THERAPY
FEBRUARY 2, 2000
Printed for the use of the Committee on Health, Education, Labor, and Pensions
oe
U.S. GOVERNMENT, PRINTING OFFICE
62-393 CC WASHINGTON : 2000
For sale by the U.S. Government Printing Office
Superintendent of Documents, Congressional Sales Office, Washington, DC 20402
ISBN 0-16-060615-2
COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS
JAMES M. JEFFORDS, Vermont, Chairman
JUDD GREGG, New Hampshire EDWARD M. KENNEDY, Massachusetts
BILL FRIST, Tennessee CHRISTOPHER J. DODD, Connecticut
MIKE DeWINE, Ohio TOM HARKIN, Iowa
MICHAEL B. ENZI, Wyoming BARBARA A. MIKULSKI, Maryland
TIM HUTCHINSON, Arkansas JEFF BINGAMAN, New Mexico
SUSAN M. COLLINS, Maine PAUL D. WELLSTONE, Minnesota
SAM BROWNBACK, Kansas PATTY MURRAY, Washington
CHUCK HAGEL, Nebraska JACK REED, Rhode Island
JEFF SESSIONS, Alabama
MARK E. POWDEN, Staff Director
SUSAN K. HATTAN, Deputy Staff Director
J. MICHAEL MYERS, Minority Staff Director and Chief Counsel
SUBCOMMITTEE ON PUBLIC HEALTH
BILL FRIST, Tennessee, Chairman
JUDD GREGG, New Hampshire EDWARD M. KENNEDY, Massachusetts
MICHAEL B. ENZI, Wyoming TOM HARKIN, Iowa
SUSAN M. COLLINS, Maine BARBARA A. MIKULSKI, Maryland
SAM BROWNBACK, Kansas JEFF BINGAMAN, New Mexico
JEFF SESSIONS, Alabama JACK REED, Rhode Island
JAMES M. JEFFORDS, Vermont
(Ex Officio)
ANNE E. PHELPS, Staff Director
DAVID NEXON, Minority Staff Director
(II)
WELLCOME
LIBRARY
iGce neral Collections
a. EA LIT
Ne PYIKG
S
2501827592
‘ Denice
TSH Oe Geeeaceaar
STATEMENTS
WEDNESDAY, FEBRUARY 2, 2000
Frist, Hon. Bill, chairman of the subcommittee, opening statement ..................
Kennedy, Hon. Edward M., a U.S. Senator from the State of Massachusetts,
Opening statement <3 ss. 2 6S Fc. Peery h APE reo. Be week ahaa ono rensorys et ersieens:
Hutchinson, Hon. Tim, a U.S. Senator from the State of Arkansas, opening
SERCO TMENS Utena cet seo doves Seek opceaetieaaseroa tice natn>bccus otepace sce tects siege ossise banker meee ee mee Te
Bingaman, Hon. Jeff, a U.S. Senator from the State of New Mexico, opening
BeR ORNCT N Ga nag acy ANN AG oo i tig Sect enna csenastbarsccenagerantosapunets=surnee s
Gelsinger, Paul, Tucson, AZ, father of Jesse Gelsinger, participant in gene
therapy; and Eric Kast, Norman, OK, on behalf of the Cystic Fibrosis
phGae P OL Nene 8 he PAE Ae, Se gc ae Ree Oy me Pe ee = fee,
Prepared statements of:
BES Ea a 81202 a ec AE AN ae OE ER a ale ep HE RR oR VO EYo ra AR as APNE TAN wal
Pee eee eo eee deer ease resus eee sess ESOS HESS ETOR OSES EL OS ESESES OS ESEEEDE SOOO ESSE STEED BESS ES EEEEEEE EE ESS
RIESEC US UEC IDEN isp oon cos costae Ae ede aon heats Lsecvaneadatase cit ERE. AOE
Patterson, Dr. Amy, Director, Office of Biotechnology Activities, National
Institutes of Health, Bethesda, MD; and Dr. Jay P. Siegel, Director, Office
of Therapeutic Research and Review, Food and Drug Administration, Rock-
VL Sree teeta cee ee eee ee OE, Nena cloak oot s Pec svsdaevencbessseedersesnansesots
Prepared statements of:
Dee a GLO SR OT aie sot OF cca ened > cca saree sso es sans tewennasde <entsoncaht damian cesonins
AB Re NTS eS, AR SR es eet Darras Ritts be ier ap atria an eps ion Me dh cp ee eth
Verma, Inder, professor of molecular biology, Laboratory of Genetics, the
Salk Institute, La Jolla, CA; Leroy Walters, director, Kennedy Institute
of Ethics, Georgetown University, Washington, DC; and H. Stewart Parker,
president and CEO, Targeted Genetics Corporation, Seattle, WA , on behalf
of the Biotechnology Industry Organization ..0................eeesceceecececeeeeeecceeeseeeseeees
Prepared statements of: .
Introgen Therapeutics, Inc., prepared statement ...0.............-cceseeccecesenenseceeeeeeseeees
Citizens For Responsible Care & Research, Vera Hassner Sharav, president
& cofounder and Adil E. Shamoo, Ph.D., vice president & cofounder, pre-
PIAELU SUHLCTINETIG: Soo ccuoterccccarneeigar tens cee Tecan cree Pte ns sb Tides sd a Nb
ADDITIONAL MATERIAL
Articles, publications, letters, etc.:
Frist, Hon. William, chairman, Subcommittee on Public Health, from
Judith Rodin, president, University of Pennsylvania, dated January
Bie 8 Sp ee eres ety> teen demote cet Tenth RN atte te Meter eee fe 25
Gene Therapy in the United States: A Five-Year Status Report, by Gail
Ross, Robert Erickson, Debra Knorr, Arno G. Motulsky, Robertson
Parkman, Jude Samulski, Stephen E. Straus, and Brian Smith, from
Human Gene Therapy, September 10, 1996 ...........eccccccccesessseeeceeseseceesees
A Look At . . . Informed Consent, A Lot of Rules, Too Many Exceptions,
by Abbey SaMeyers: 10. marcia io eli ine l, bene n-gececran
Subcommittee on Public Health, from Bill Freese, dated February 8,
AMIEL 2 det cnes velc eeg ard en ae UReS ones anctae vont at rereeateee eeates eet ee ae ese eee
Frist, Hon. William, chairman, Subcommittee on Public Health, from
William N. Kelley, M.D., ceo, University of Pennsylvania Medical Cen-
ter and Health System, dated February 4, 2000 .............eccecccecceesseeeteeeees
(1)
aTMaMatare mas | Pee ene
\
¥ ‘ a +? A i Veit -
; 000? ig ranges wacaanasw ala hy ly ae lee
gt‘ et (aibgesr| toapienaenpm bta: ot atatae | 2 gianlotq mo oSait iiishttaemneseedi iohEee1 3a nd9 10 near; ed i 20H nita
“a ; ,
£ te , stera et “*eyB aoneirosh A3 AttSoo ornh t?s m tovL} Sw iyeen eBe iOeF p et« a n; i? #t. ooH ammao! ain ¢ ir-i ots ¥ iaf
py ata or. a -ee shes : t Rba oiAerLna e,g aii c R++»e haaJxlOeA ,Mg eaKehSti M.lawMe y,}. naJ iMEesaBbiaEEtesOedeRd G aE:s .eoY , snVseiArhm mepspeoer ntetA3i°et IC gO+EceaA Rea SR4Fra % .d . gi0i2)eM0 0,s7el1 i' taWfcaroaDg edndtdl meerWseSnmryeeedatn diaseeet)gtl,ae y n it ~ea ) - =ee' h e- e*"
Ray 3 ae oe iat 3 PAL D. WREATR
S#MEL Aa ei. : PATTY MURRAY, ¥
aa Pe eneratnbep el of orld uoti d BN.
My Daa; s s SatBnioc oOaats s arGooytrzwiae nRdse edirt 2ttt:S)s i. ivinoAntiesa acnn aa cb iipotg ecaoDit‘iRieianGlr n ntrbaiaoan nndaoecit i ts OOjFai EeTeaR E( sOvENEOLi MOe sK O R Olsh whes eiaDeeesrnw) odn w e sealoA g i l aeomHs lt1 o exoieprisihesn al <0 2
pobmipb hin Rboeataen n tke tee | P08 iFPO sENs we ode Onhh Oae blet N ees aMug l i “=j
ohd
7 ‘2a$a m 9 R8e ) ¢R‘ aOefiSW esuMaNine itEee is ‘ teAee si Cc sdKur]: e neh“ov idbaes) etr wgeneas Oa wajooe sS !Jew eioob o pathenisn eaaeaes a.tgs 2srneodn Xlir7vv-w aree ,r ataeesnos ttgonuijOJg ;Ue1 PE a0E tmSe2} euG ohPat) y sn)eS ttHVeSoAeemTeGt ArO"o NitAaaEl ll7 o l: YO eoFnJl otnNoiiuensiet biaseeilm naep arsC: afee}dr ise oB e. ~nS nyw taa e
A Ts; ae . 4P urmsateasa S, matnri —P* e Ma ss ev eyietehede Lothar’‘ oo vOinEe st ORD E OO eee OO P ewee earnotaict a aSMb fi
AAO NO en yee eeeb ee yt seu tee bolew ee w POE ne Ohne bee Om kae e settee T 3M . ; ae,
feeb vei ie E
Reda esaBuOL t P.iid eoe lke oa i . : niliek 7 ott #L00>A w8ide R geeS ee EbeneN xam ee “ AKOTTIGTA ee ruea s
mow, ‘sitet sikdud. nb setsieumosdne
- caatinit Eiteky ta se tre ‘bSo enmovialt
H« ¥e ys yf ata, eae Vad iFe opaiqaoanld He i cexoiMs t DPtoeén ee VmiCALw ibtire n eAiwa odceal byl ee 2
7 pinl ornehompedandesn oni
mot heh amiga pieo ma cre rete
orth bbnbes' senna tote
lootayy seal) FA a aI on 3A.5g s
7 Mawceae s ts Asbelte hae3 oe .
a wa sh
9
GENE THERAPY: IS THERE OVERSIGHT FOR
PATIENT SAFETY?
WEDNESDAY, FEBRUARY 2, 2000
U.S. SENATE,
SUBCOMMITTEE ON PUBLIC HEALTH, OF THE COMMITTEE ON
HEALTH, EDUCATION, LABOR, AND PENSIONS,
Washington, DC.
The subcommittee met, pursuant to notice, at 9:35 a.m., in room
SD-—430, Dirksen Senate Office Building, Senator Frist (chairman
of the subcommittee) presiding.
Present: Senators Frist, Jeffords, Hutchinson, Kennedy, Binga-
man, and Dodd.
OPENING STATEMENT OF SENATOR FRIST
Senator FRIST. Good morning and welcome, everyone, to today’s
hearing on what are truly critical issues regarding the subject of
patient safety in gene therapy clinical trials.
The events of the past few months involving gene therapy re-
search have given all of us, our Government and our society, rea-
son to pause and have prompted the subcommittee today to under-
take and initiate a thorough review of this truly revolutionary re-
search field and the systems that surround it.
Today’s hearing is a very important one. We will be talking about
death, we will talking about life. We will be talking about promis-
ing new research that could not have even possibly been imagined
30 years ago. We will be talking about the tough ethical issues
such as informed consent and what conflict of interests exist. with
the research community. We will be talking about the nature of ex-
perimentation in human beings—inherently risky, fundamentally
profound. We will be talking about gene therapy, what is gene
therapy, how does it differ from other therapy, and what promise
does it hold for each of us and for our children.
Gene therapy is an experimental procedure whose effectiveness
has not yet been demonstrated, but it does offer real hope and real
promise for those people who are suffering from genetic and non-
genetic diseases. It holds great promise, but because it is experi-
mental, there is an absolute necessity that we in Government and
in the private sector provide appropriate and vigilant oversight.
If we ask patients to participate in clinical trials to more promis-
ing science forward, we must assure them that their safety is first
and foremost.
I hope that today’s hearing will be a thoughtful discussion and
will provide a thorough review of the oversight mechanisms that
(1)
2
are in place, both in place on paper as well as giving us the oppor-
tunity to see whether what is on paper is actually being carried
out.
Doctors and scientists do have what I regard as a truly noble
journey to pass along promising research and to explore that re-
search in an appropriate way in this new scientific field. But in
that journey, there is absolutely no place for mistakes that com-
promise patient safety.
The tragic death of Jesse Gelsinger, who was the first patient we
learned about who died from gene therapy, has sombered us all.
Jesse, as all of you know, was 18 years old. He had a rare liver
disease. He unselfishly volunteered for a gene therapy clinical trial
which was designed not to cure him but to develop a treatment
that others, children and babies, could benefit from in the future.
Mr. Paul Gelsinger, Jesse’s father, has generously agreed to be
with us here today, and we offer our deepest condolences to the
Gelsinger family and our true gratitude that he comes before us at
a painful time in his own life to share with us his experience and
to help us learn how we can make the big picture better and make
the system better. As we will hear Mr. Gelsinger graphically de-
scribe today, we absolutely must chart a new path that ensures
that no family will have to endure what he and his family have
had to endure over the last several months.
Yet I predict that in the future, we are going to continue to learn
more about adverse events that have occurred in gene therapy
trials. Just this week, we have learned about more unreported
deaths. The National Institutes of Health, who is with us today,
following Jesse Gelsinger’s death put out a far-reaching call for all
investigators conducting gene therapy research to remind them
that they must report serious adverse events to NIH and to the
FDA. The fact that NIH received 652 previously unreported serious
adverse events in response to this request is really inexcusable.
Clearly, our oversight system is failing.
Our hearing today is the first step in the congressional examina-
tion of the oversight procedures and guidelines in place at the NIH
and the FDA to determine whether we have adequate systems to
approve and then monitor these revolutionary new gene therapy
trials. If we learn that the appropriate systems and guidelines are
in place, on paper, we must then ask are they working in reality.
I suspect they are not. The deaths of several patients reported over
the last several days and the 652 previously unreported adverse
events suggest the system is not working.
All of these events trigger serious questions regarding Federal
oversight of these gene therapy trials as well as adherence to the
Federal guidelines that are out there on the part of the individual
investigators themselves and the research community.
So much of science today, at the pace at which it is moving, is
introducing new ethical questions that we as policymakers must
address—ethical questions regarding the risk to patients in human
experimentation in these trials and the high financial interests
iat are at stake as increasingly the private sector is funding such
trials.
We must examine our process of informing patients—is it time
for us to go back and look at what truly informed consent means
3
in this new era of rapidly advancing science, and what constitutes
truthful and complete information to patients. We must ask if
there is an atmosphere of unrealistic expectations with regard to
gene therapy, and we will talk about that with our third panel.
What does gene therapy truly hold as a promise for cures for dis-
eases, and how quickly can we realize those promises? Are patients
given full information regarding past adverse events as they enter
these clinical trials?
My objective as we go forward is to keep the hearings balanced
and to make sure that our discussion is careful. Nobody is on trial
here—not the Government, not universities, not research scientists.
It is clearly to everyone’s advantage to put everything on the table
in as quick a fashion as we can. It is tough, because every day, new
information is coming out, but I am very hopeful that over the
course of this morning, we can achieve a balanced and careful ex-
amination and discussion.
We know that we can learn from previous mistakes. But we do
need to know what those mistakes are if we are going to learn from
them and if we are going to improve the system and if we are going
to move forward in this field of gene therapy.
The tragedy of Jesse Gelsinger should not result in another trag-
edy, and that other tragedy would be to bring a halt to this promis-
ing research. That is not the objective, and in fact, I feel strongly
that we need to develop a system in which we allow this promising
research to continue and to flourish. I believe it does have the po-
tential of treating cancer and AIDS and cystic fibrosis and genetic
and nongenetic diseases alike. Yet throughout, we must remain
steadfast in our commitment to ensure that patients are safe, that
there are adequate human protections in place, and that the re-
search is meaningful and substantial.
With that as an overview and before turning to Senator Ken-
nedy, let me just tell people how I would like the hearing to go for-
ward today. We will have opening statements, then we will have
brief opening remarks from our witnesses. We have three panels
today, and witnesses are asked to keep their remarks to around 5
to 6 minutes in length so we can carry on a dialogue among mem-
bers and witnesses. Following the witness presentations, each of
the Members will be allowed to ask questions. The hearing record
will remain open for individuals who would like to submit no more
than 10 double-spaced pages of written testimony until February 9,
at which time the hearing record will be closed.
Senator Kennedy.
OPENING STATEMENT OF SENATOR KENNEDY
Senator KENNEDY. Thank you, Senator Frist, and thank you very
much for having these hearings.
As you have mentioned, this issue is of critical importance to the
Nation. No other medical technology offers greater potential to pro-
vide needed cures for so many diseases, and at the same time, no
other medical technology has illustrated more clearly the need to
protect patient safety and to provide the opportunity for informed
consent.
The tragic death of Jesse Gelsinger reminds us of the hope felt
by those who look to gene therapy for a cure of their illnesses and
4
the need for constant watchfulness in protecting the safety of pa-
tients involved in medical research.
In many ways, Jesse Gelsinger was a typical teenager. He loved
motorcycles and professional wrestling; he spent time with friends
and worked at the local grocery store. Yet in one respect, he was
very different from all of his friends—not because he had a rare ge-
netic disorder, but because he had the courage to put himself at
risk to test what he deeply believed to be the best hope of a cure
for those with genetic disorders. Tragically, Jesse’s courage led to
his death.
We must do all we can to see that Jesse’s sacrifice was not in
vain. His hope was that someday, gene therapy would provide
cures to the tens of thousands of babies born with genetic disorders
every year and for the millions more affected by cancer, heart dis-
ease, AIDS and many other serious illnesses.
The promise of gene therapy is shared by thousands of patients
and is daily being brought closer to reality by talented researchers
across the country. Today we will hear about some of the ways in
which gene researchers are making gene therapy safer and more
effective than it has ever been before.
Our challenge is to see that those who volunteer to participate
in gene therapy trials are fully protected from unnecessary risk.
Oversight of this field should be as comprehensive as possible. In
particular, when tragic events do occur, they must be reported in
a timely way to the appropriate oversight agencies. We must also
see that those who volunteer to serve as research subjects are fully
informed of the possible risks of the treatment they are about to
receive so that the consent they give is well-informed.
I look forward to the testimony of our witnesses and to working
with the members of the committee to assess the adequacy of the
current rules governing this extraordinary area of medical re-
search. We are particularly indebted to Mr. Paul Gelsinger for
being here today and thank him for his own courage. It is never
easy to talk about this kind of extraordinary human tragedy, and
we are very, very grateful to him for being willing to appear before
the committee today.
Thank you, Mr. Chairman.
Senator FRIST. Thank you, Senator Kennedy.
Senator Hutchinson.
OPENING STATEMENT OF SENATOR HUTCHINSON
Senator HUTCHINSON. Thank you, Mr. Chairman.
I think Senator Frist and Senator Kennedy have outlined the
issues very clearly and very well.
I just want to say, Mr. Gelsinger, that I was deeply moved as I
read your testimony, and I look forward to hearing your testimony
and your story.
I noted in your written testimony you say that one of the leaders
in the field called your son’s death “a pot-hole in the race to gene
therapy,” and you observe that the concern should not be on get-
ting to the finish line first but on making sure that no unnecessary
risks are taken and no lives filled with potential and promise are
lost forever.
5
How true, and I want to join Senator Kennedy in saying that in
fact your son’s death was not in vain, and I think that already it
has called the Nation’s attention to some very great dangers. So I
appreciate your courage and your willingness to come forward
today.
I thank you for the hearing, Mr. Chairman.
Senator FRIST. Thank you.
Senator Bingaman.
OPENING STATEMENT OF SENATOR BINGAMAN
Senator BINGAMAN. Mr. Chairman, you and Senator Kennedy
and Senator Hutchinson have outlined the issues. I thank you for
having the hearing, and [I thank Mr. Gelsinger and the other wit-
nesses for being here, and I look forward to the testimony.
Senator FRIST. Thank you.
Senator FRIST. Our first panel will discuss patient safety con-
cerns and informed consent issues in clinical research.
Mr. Paul Gelsinger of Tucson, AZ is the father of Jesse
Gelsinger, who tragically passed away last September during par-
ticipation in a gene therapy trial at the University of Pennsylvania.
Mr. Eric Kast of Norman, OK—welcome, Mr. Kast—is a cystic fi-
brosis patient who has participated in a number of clinical trials,
including a gene therapy trial, in order to contribute to the search
for a cure for cystic fibrosis. He currently works for Blue Cross/
Blue Shield of South Carolina, where he educates medical equip-
ment suppliers about Medicare claims. He received his bachelor’s
and master’s degree in journalism and public relations from the
University of Oklahoma. He is testifying on behalf of the Cystic Fi-
brosis Foundation.
Mr. Kast, again, thank you for being with us today and for your
dedication in combatting a disease that we have made tremendous
progress on over the last 15 or 20 years.
We will begin with Mr. Gelsinger, who will be followed by Mr.
st.
STATEMENTS OF PAUL GELSINGER, TUCSON, AZ, FATHER OF
JESSE GELSINGER, PARTICIPANT IN GENE THERAPY TRIAL;
AND ERIC KAST, NORMAN, OK, ON BEHALF OF THE CYSTIC
FIBROSIS FOUNDATION.
Mr. GELSINGER. Thank you, Senator Frist, and thank you mem-
Dens of this panel for allowing me to contribute to this hearing
today.
I am addressing this committee in the hope of bringing to light
some very serious concerns that I have as a result of my son’s
death. My son, Jesse Gelsinger, was participating in a first phase
clinical trial conducted by the Institute for Human Gene Therapy
at the University of Pennsylvania in Philadelphia.
Jesse was recruited to participate in the trial after being told
that his efforts would benefit newborns and other children with
this specific disorder—ornithine transcarbamylase deficiency syn-
drome—and for a myriad of other liver disorders.
Jesse did not need to participate in this clinical trial; indeed, he
was told that the trial would not benefit him in the least. At the
time, Jesse was doing exceptionally well on his medications, and
6
nothine should have prevented him from living a full and happy
ife. :
He believed after discussions with the representatives from Penn
that the worst that could happen in the trial would be that he
would have flu-like symptoms for a week. He was also told that the
most dangerous part of the procedure was to be the catheterization
procedure by which the gene therapy would be introduced to his
liver and the liver biopsy that was to be done a week later.
With the knowledge I had at that time, I was comfortable enough
to send my son, just 18 on June 18, 1999, to Philadelphia alone on
September 9th to participate in this clinical trial. I was scheduled
to fly to Philadelphia on September 18th to be present for the liver
biopsy and to bring Jesse home.
Jesse put his personal life aside, took an unpaid leave of absence
from his job and accommodated everyone else’s schedule to do what
he did. He was excited to help. In addition, Jesse relied on my
judgment in participating in this clinical trial, and I trusted this
to be a well-controlled and purely ethical effort.
Less than 24 hours after they injected Jesse with the vector in
an amount only one other person had ever been given, Jesse’s en-
tire body began reacting adversely. He went into a coma before I
could get to Philadelphia to see him and died 2 days after my arriv-
al, directly as a result of that gene therapy experiment. While his
death has been a devastating blow to us, his example has sustained
us through it all.
As you can imagine, my family and I have many concerns over
what happened to Jesse. Jesse and I were told in late July 1999
that a prior patient—the patient before him—had shown a clinical
improvement of 50 percent in her ability to eliminate ammonia
from her system following gene therapy. At the Recombinant DNA
Advisory Committee meeting in December, I discovered that no ef-
ficacy was achieved at all in this patient. We were also unaware
of the severity of liver injury incurred by several patients prior to
Jesse. I learned after Jesse’s death that Penn had removed from
the information they gave Jesse and me any reference to deaths of
monkeys which had previously appeared in their documents. At the
RAC meeting in December, I also learned that at least one other
monkey died in:a related study using the same adenoviral vector
used on Jesse.
I learned that Penn neglected to follow its own and FDA’s proto-
cols when it found that Jesse had ammonia levels above the per-
missible limits—a clear danger sign—and yet went forward with
the procedure anyway.
I learned that a pharmaceutical company had conducted experi-
ments similar to the one Jesse was in and had obtained adverse
results which, if disclosed, would have fully informed Jesse and me
of the real risks in this procedure.
I had very close contact with the doctors involved until December
10, 1999, immediately following the RAC meeting. Looking back, I
can see that I was very naive to have been as trusting as I was.
As serious as my concerns are with the Penn trial as Jesse’s fa-
ther, I have equally great concerns regarding the Federal oversight
of gene therapy as an American citizen. As a result of Jesse’s
death, many important issues regarding gene therapy have come to
