Gene Therapy

Thesis Outline

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In order to fully understand the field of gene therapy and its ethical implications it is important to examine its original purpose and application. With this understanding, we would be ready to investigate the evidence which clearly supports the idea that gene therapy should not be regulated or slowed because of its direct applicability and life-saving potential. “Though gene therapy is in its early stages, the promise it holds for future medical advances is vast” (Maese, 2005).

After watching family members suffer from muscle atrophy due to aging, Dr. H. Lee Sweeney began researching genetic therapy as a way to restore atrophied muscles in the elderly and people affected by diseases such as Muscular Dystrophy. Dr. Sweeney discovered that by injecting mice with a small amount of genetic material contained in a harmless delivery truck called Adeno-Associated virus (AAV) that he could enhance muscle growth in mice and decrease the rate of atrophy caused by sedentary behavior (Sweeney, 2004). This therapy is revolutionary in its applicability and potential for saving lives. “As soon as his (Sweeney’s) research hit medical journals, his e-mail inbox started to fill up. The requests came from grandparents suffering from muscular dystrophy. They came from the wealthy, hoping for as long a life as possible. And they came from athletes and coaches, thinking they might have found the magic pill… The subject line of the e-mails were all similar: I'll be your guinea pig.” (Maese, 2005). Although gene therapy has its advantages, it is not without its ethical disagreements.

It is clear that Gene therapy is a very controversial topic in general. Most people are unaware of the medical implications of genetic therapy. Some think it is playing god because we are altering our genetic makeup while others see it as the God-given miracle that it is. “Gene Therapy, experts say, could change everything” (Maese, 2005).

When some people discuss gene therapy there is a sense of fear, fear of the uncertain, fear of what they do not understand. We need to focus on the positive aspects of gene therapy and illustrate that we must recognize the unstoppable progress that will occur, “Either way, many experts think a fundamental change is unavoidable” (Maese, 2005). Since gene therapy is a reality, it must be pressed forward in an attempt to use it for the good of the people. “If scientists can develop ways to strengthen muscles, gene therapy can change the life of someone suffering from a degenerative disease, such as muscular dystrophy” (Maese, 2005).

In many people’s minds, there is a pressure to regulate this new technology in terms of limiting its funding or requiring advisory committees to approve a study or treatment before it is carried out. I agree that all options need to be explored in treating a genetic illness and sometimes gene therapy might not be the best fit. I support the idea of informing the patient of all risks and benefits before he or she decides on a treatment option. I do not support limiting patients’ treatment options through HMO bureaucracy or funding cuts. This regulations being considered are going to leave us with a patient who is running out of options.

Take for instance the Genetic Therapy Advisory Committee (GTAC) in the United Kingdom. “The primary concern of the GTAC is whether each research proposal meets accepted ethical criteria for research on human subjects. This includes therapeutic and non-therapeutic research” (GTAC website). Why is the UK appointing a group of experts to decide someone’s fate based on pre-formed partisan guidelines? If I remember correctly we are all free-willed creatures with the ability to live our lives and decide our own fate. If the GTAC begins regulating treatments that a patient and their doctor have determined necessary, where does the regulation end? If a treatment option does not fit the committee’s standard ethical handbook it is not approved. What will be the next step? Will they be telling patients that X-rays cause cancer and therefore will not be approved? In the future will they limit access to risky operations that are commonplace today? Power and authority are dangerous bedfellows. These are what need regulation. Deciding who receives what treatment and when is inherent to the doctor and the patient. There are no third party considerations that should be present to influence