Download The Misuse of Gene Therapy - Michigan State University

The goal of medicine has always been to cure human disease and suffering. Mythologies
from many cultures have described the need for such a cure-all therapy. The fairy-tailed fountain of
youth is nothing short of a symbolic manifestation of the need to live forever in perfect health.
As technology progresses, the understanding of nature becomes more and more clear. The once
unattainable goal of harnessing a method of curing all human disease is becoming more and more
feasible. The technology that seems to be catching the eye of such "fountain of youth" seeking folks
is that of gene therapy. This technique has people predicting great leaps in medical science. Claims
of curing cancer and genetic disorders have become a popular topic among many (Jaroff, 2003).
Many, including the father of gene therapy, question its ability to do good as well as bad. Few
among the scientific community push for gene therapy blindly without giving thought to the power
in which could be released. All scientists agree on one thing. Gene therapy will become a
powerhouse in cutting edge technology. Whether that power will go to good or bad, is up to those
who harness its potential. Designer babies, genetically modified athletes, and a super race are but
few of the concerns facing the future of man-kind. Gene therapy technologies should be regulated
and slowed in its progress due to its catastrophic potential misuse.
A physician named W. French Anderson first made gene therapy popular in the early
1980's. Though the idea of gene therapy was not new, the technology in which to achieve it was
beginning to emerge. His idea was simple; if a patient were ill due to a defective gene, why not
insert a functioning one to make up for the genetic deficiency? His research was not the first of its
kind. But his timing and success grabbed the media’s attention. Anderson became known as the
father of gene therapy (Jaroff, 2003).
From that point on, gene therapy became a field with great expectation. The public
embraced gene therapy as a godsend. Such excitement was shown through the media (Angier,
1990). Reports of successful gene therapy on animals became commonplace. What the media really
craved was a successful human treatment using gene therapy. In 2002 the Washington Post
reported that people with a specific heart condition were receiving gene therapy and taking to it
very well. Another story broke out reporting the successful treatment of children with a disease
known as SCID (Severe Combined Immunodeficiency). This was all very exciting to the scientific
community and to the public as well.
An article published by the Washington Post in 2005 reported a disturbing turn of events.
Gene therapy research in France had been performed on ten different children with SCID. The
treatments were successful. However, three years after the treatment, children began developing
cancer. Three have fallen ill so far. One of the three children succumbed to his illness and died
(Weiss, 2005). The research done in France was lead by Dr. Alain Fischer. "We want to continue,
but of course there is a safety issue," Dr. Fischer admitted. His research was and is focused on the
treatment of SCID in pediatric patients. This disorder can be linked back to a faulty gene on the X
chromosome. Fischer and his team masked the faulty gene with a functioning synthetic gene. MLV
(murine leukemia virus) derived vectors were used in Fischer’s research. Two forms of SCID have
been successfully treated with MLV-derived vectors. The technique of infecting hematopoietic
progenitor cells in vitro has been successful. However, avoiding damaging gene insertion during
this procedure is nearly impossible. The effects of mutagenic gene insertion is not obvious during
the early stages after treatment (Fischer, 2005). Therefore the treatment initially seemed to be very
successful. Within weeks the children were thriving (Weiss, 2005). However, with the inability to
regulate the synthetic genes placed within the bodies of Fischer’s patients, problems began arising.
The children began falling ill with leukemia. In sum, the synthetic gene was causing the production
of too many white blood cells in the body (Amalfitano, 2005). Treatments using gene therapy on
SCID patients have been extensive throughout Europe. The effective outcome of the treatments is
hardly astounding. Out of several dozen treatments, only twelve children have been cured of SCID.
Out of the twelve cured patients three of them have developed cancer. The first successful
treatment was performed on the boy who was the first to die of leukemia. Physicians fear that the
remaining nine will develop leukemia and succumb to its effects. In lieu of these findings, the FDA
has halted gene therapy research throughout the United States (Weiss, 2005).
This was not the first time that a death had resulted from gene therapy. In 2000, an 18-yearold patient at the University of Pennsylvania was killed in gene therapy trials conducted by Dr.
James Wilson. Wilson was president of the Institute for Human Gene Therapy and a golden boy of
sorts in the field of gene therapy. The university president had this to say about Dr.Wilson’s
research failures, “We think that Dr. Wilson was overloaded with regard to responsibility”. The
FDA found many discrepancies within Wilson’s studies during the course of the investigation
(Stolberg, 2000). One such oddity was found between Wilson’s research and his connections to a
biotechnology company in which he founded called Genovo. Genovo was contributing one fifth of
its $25 million dollar budget to Wilson’s research. They also granted him exclusive rights to the
commercial production of successful gene therapy serums. The FDA found this relationship
disturbing due to the high self-interest involved. Wilson had a lot of reasons to push research faster
than it should’ve been (Stolberg, 2000).
Money has a larger role to play in the image of gene therapy. Not only does the dollar drive
researchers like Wilson to overstep their bounds, but it has a large role in public perception. In
2002, the Washington Post wrote an article called “Gene-Therapy Tests Show Positive Results”.
The article praised the effects gene therapy was having on patients with severe coronary artery
disease. The lone source of information for this article was a multi-million dollar corporation
known as GenVec. GenVec is a biopharmaceutical company that funds gene therapy research to
treat heart disease, cancer, and vision loss (Unknown 2, 2005). The research they gave to the
Washington Post seems promising. However, GenVec was the only source of information for this
article. Its intentions were to put on a pretty face for the world to see. More press equals more
money. The true intentions of GenVec are reflected in its CEO and president, Dr. Paul H. Fischer.
He had this to say about a recent merger, "We are combining the key strengths, capabilities and
facilities of the two companies to form a strong, focused company with a reduced cash burn, an
efficient work force and a significant cash position". The CEO of GenVec makes over a quarter of a
million dollars a year (Unknown, 2005). He looks to make significant personal gains in the
successful production of gene therapy technologies (Unknown, 2005). The intentions of GenVec
could be corrupt by money, as it might have been with James Wilson.
Many people have concerns about the misuse of gene therapy. A general consensus runs
through the public, “If they can alter our genes to cure us of disease, what else can they do to us?”
When asked about fears relating to gene therapy, many were concerned about the person behind the
needle rather than the technology itself. A local woman had this to say, “My concerns with regard
to gene therapy would be the ethical ramifications. For instance, what kinds of maladies are
considered in need of cure? Who would decide?” Many share her fear, like Jeffery of Vernon
Michigan, “…Great good and unimagined evil are usually the outcomes of man’s attempt bend
nature...” Despite the fear of misuse, the general public seems to perceive gene therapy as a good
thing. Many perceive it as a perfectly safe, yet underdeveloped technology. When asked about the
future of gene therapy, a large majority agreed that it was the future of medicine. All of them
agreed that they would subject their children to gene therapy if need be. Peggy Friess of Corunna
Michigan had this to say, “I believe I would strongly consider this option. I would have to take into
consideration, success rate, quality of life and moral concerns. This is a very interesting idea and
the ramifications for good and bad are plentiful. As a mother, I would do anything to save my
child.” This shows a disturbing willingness to use this technology. People have their doubts in gene
therapy, yet condone its use. Those who wish to propagate gene therapy could easily exploit this
primal instinct.
Popular magazines and short news clips seem to be where the majority of people get their
information. This is a very narrow channel of communication. However, in recent years, the
Internet has been used more by the public. Websites from all over the world have been created for
this purpose. A good example of such a web site is GMWatch.org. This web site focuses its energy
on informing the public about topics such as designer babies, gene doping, and inbreeding effects
due to gene manipulation (Matthews, 2005). A topic recently posted on the web site is that of
designer babies. It shows an interview with Dr. W. French Anderson, the founder and father of
gene therapy. Anderson is considered to be one of the largest advocates of gene therapy (Jaroff,
2003). The posting shows a very reassuring side to Anderson. He too shares the fears of gene
therapy misuse. During an interview with CNN, Anderson had this to say about gene therapy, “The
primary concern is the damage we could do while trying to manipulate, while trying to improve our
genes. We have to be very careful that this powerful technology is used only for the treatment of
disease and not in attempting to create designer babies or any other non-medical purpose.” He
further claims that arguments brought against gene therapy are points he raised in the mid 1980’s.
He seems to have a healthy respect for the power of gene manipulation and that comforts those
fearful of gene therapy. Many scientists and researchers share Anderson’s feelings (Jaroff, 2003).
“When it comes to research, there is always a risk associated with it.” said Dr. Andrea
Amalfitano of Michigan State University. He is a medical geneticist who studies the Glycogen
storage disorder known as Pompe’s Disease. He believes strongly in the research associated with
gene therapy and that patient death is a natural consequence of this cutting edge technology. Dr.
Amalfitano believes that a little bad justifies a greater good. Many researchers feel the same way.
This type of trade off has been ubiquitous in pharmaceutical development since its incarnation
(Amalfitano, 2005). When Dr. Amalfitano was asked what he feared of gene manipulation, he said
that he doesn’t fear its implications any time in the near future, but that its potential should be
closely monitored to avoid misuse.
So it seems there are two primary positions on gene therapy. One position wishes to watch
it closely and regulate it, the other wishes to push the technology to its limits, unnecessarily risking
lives for the sake of a profit. The media has been publishing stories for years on gene therapy, some
good, and some bad. It seems that biopharmaceutical companies have some influence on the stories
published, making popular news media a tool. This may have given the public a false sense of
security. The general public is aware of gene therapy and its objective. They are unaware of the
corruption driving the development of this technology. Parents would do anything to save their
children, including the solicitation of profit-hungry scientists. The large majority of scientists are
not looking for profit or personal gain. Scientists like Drs. Amalfitano and Anderson expect gene
therapy to revolutionize medicine, but they are conscientious of possible misuse.
What can be done to keep gene therapy in check and under control? The answer lies with
the public. A well-informed community is the most effective weapon against the misuse of this
powerful technology. With an informed public, unnecessary gene manipulation would not be
tolerated and corrupt scientists looking for money and personal gain would be under the critical eye
of the people. This amazing technology is only in its youth; its full potential is far from being
realized. To keep this research safe and ethically sound the people must have the power of
knowledge. French Anderson said it best, “…The only protection is an informed society. It is
imperative that we remain alert to insure that gene technology is not misused by anyone…”
Works Cited
Amalfitano, Andrea. Personal Interview. 3 November 2005.
Angier, Natalie. “FDA Approves Radical Gene Therapy for Advanced Cancer.” New York Times.
14 Nov. 1990; A23
Barbaro, Michael. “Gene-Therapy Tests Show Positive Results, GenVec Says.” Washington Post.
21 Nov. 2002; E05
“Dr. French Anderson on the pros and cons of gene manipulation.” CNN. 2000. 26 Jun. 2000
<EDThttp://www.cnn.com/chat/transcripts/2000/6/26/genome.
anderson/index.html>
Fischer, Alain. “Integration of Retroviruses: A Fine Balance between Efficiency and Danger.” PloS
Med. 2(1): e10
Foland, Barbara. Personal Interview. 6 November 2005.
Foland, Jeffery. Personal Interview. 6 November 2005.
Friess, Peggy. Personal Interview. 5 November 2005.
Jaroff, Leon. “The Pioneers of Molecular Biology: W.French Anderson.” Time Magazine. 17
Feb.2003; A03
Matthews, Jonathan. “Grim tally from gene therapy / More on French Anderson.” GMWatch. 2005.
5 Nov. 2005 <http://www.gmwatch.org/archive2.asp?arcid=52>.
Norris, Marie. Personal Interview. 5 November 2005.
Stolberg, Sheryl. “Institute Restricted After Gene Therapy Death.” New York Times. 25 May 2000
Unknown. “Compensation for Paul H. Fischer, GENVEC, CEO President and Director GNVC Pay
Salary.” Salary.com. 2005. <http://swz.salary.com/execcomp/layouthtmls
/excl_execreport_122601.html>
Unknown 2. “GenVec”. GenVec. 2005. <http//:www.genvec.com/>
Weiss, Richard. “Boy’s Cancer Promts FDA to Halt Gene Therapy.” Washington Post.
4 Mar. 2005: A02
The Misuse
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Gene Therapy
Walker Foland
A32967279
LBS 492
Dr. Luckie