Gene Therapy Regualtion: A Proactive Approach

Ross Laframboise

Copyright 1996

Gene therapy techniques are a rapidly growing area of interest and concern. Gene therapy technologies will have great impacts on how deal with medical problems and perhaps even on how we live our lives. Yes, gene therapy is something that will probab ly impact your life in one form or another. There are many issues discussed related to this topic, one of which is regulation. Who should regulate this research and to what extent should it be regulated? The following essay addresses the later question . This essay will explain what gene therapy is, how it is regulated, and will discuss the philosophies on how it should be regulated. Who ultimately decides the uses of gene therapy, and will those making these decisions keep your interests in mind? It is in the best interests of everyone that society develop proactive means of regulating gene therapy to maximize benefits and minimize the risks.

Human gene therapy is one of the newest advances bridging science and medicine in our modern world. In the most basic sense, it involves changing the genetic composition of certain cells in the human to correct a "defect" in the genetic code that is c ausing undesirable characteristics such as a disease. A person's genetic code is responsible for coding proteins which constitutes our physical makeup. A single alteration to this code can result in a lethal disease. In gene therapy, genes with the cor rect code are inserted into the cell. The method of choice to insert desired genes into human cells is through a virus vector in which the virus inserts modified DNA to replace the DNA that is causing the genetic problem (Coutts). Once the DNA is in th e cell, it can code for the desired protein needed to make the patient well. If the cells that are treated in your body are not reproductive cells, this type of gene therapy is called somatic cell therapy. This technique means it only affects those trea ted cells and will not be a genetic trait passed on to future generations. In somatic gene therapy, treatments often have to be conducted every couple of months because cells continually are being replaced in the body. Germ-line therapy is a procedure in which reproductive cells are altered. This results in future generations that carry this same alteration.

Much debate over gene therapy has erupted as scientific advances have brought this technology to the application phase. The first use of human gene therapy illustrates its potential healing power. At age four, Ashanti DeSilva received therapy for a g enetic condition called severe combined immune deficiency (SCID). Ashanti did not have a normal immune system and thus was susceptible to basically any infection. Such infections could even be life threatening. The gene therapy she received involved mo difying her white blood cells by inserting "correcting" DNA into them (Coutts). The success of the treatment proved to be encouraging for the advancement of human gene therapy. Now over 40 somatic cell gene therapies have been approved. Germ-line gene therapy has yet to be performed. Scientific findings and public views will play key roles in the future of germ-line therapy.

Gene therapy technologies are now making it possible to provide treatments that 20 years ago were hardly dreamed possible. These new technologies will bring about a whole new era in medicine. The answers to many medical problems people thought were i ncurable are now becoming common medical procedures. Not unlike other technologies, it is important to have a system to regulate gene therapy. Regulation plays an important role in ensuring that research and applications are being done to benefit human welfare and that the rights of others are not infringed.

So who controls the use of gene therapy? Currently gene therapy is regulated by the US Food and Drug Administration (FDA) and the National Institute of Health (NIH). The FDA looks at safety and control measures associated with gene therapy. Their ap proval is required for all gene therapy protocols. The NIH also plays a large role in regulation. All gene therapy research that is supported by NIH must be approved. The NIH has a subcommittee, the Recombinant DNA Advisory Committee (RAC), that reviews gene therapy protocols. The committee is composed of professionals in the science, medical, and law fields. Because of the reputation of the standards set by the NIH, it has also been a precedent for those not supported financially by the NIH to also s eek approval in order to satisfy public opinions (Gavaghan 202).

The Recombinant DNA Advisory Committee as well as the NIH and FDA must remain active in their control over gene therapy for several reasons. First of all they must continue to look at the regulation of these types of medical advances as an awesome resp onsibility. By no means are these techniques dealing with superficial clinical procedures. Gene therapy involves changing the genetic makeup of an individual, a genetic makeup that constitutes a vast majority of who that person is. The human biological system is so complicated that it can not be fully understood by decades of research. As it is true that gene therapy can offer many benefits, even life itself, it must always be realized that there is the chance that its effects could be devastating.

Government regulation will become increasingly important as technology allows for germ-line therapy. Although this could have potentially huge benefits to truly cure diseases forever, there are several factors that need to be considered. One factor that must be addressed is that future generations will forever be affected by our decisions. Whether our genetic composition is the result of divine power or a billion years of evolution, it is exists for reasons we do not and may not ever completely und erstand. Therefore, extreme caution must be taken in implementing change that will effect all future generations.

Secondly, ethical issues must remain a consideration. The feelings of individuals must be listened to and taken into account when dealing with an issue as controversial as gene therapy. It must be kept in mind that applications, particularly germ-lin e therapy, are a consideration for society as a whole to make. Decisions that should not be made by the wealthy or knowledgeable alone.

Thirdly human gene therapy must be kept in check to prevent potential social problems. Problems like only the wealthy having access to expensive gene therapy treatments have already presented problems while problems such as genetic alterations becoming a luxury instead of a life saving treatment could become important. The problems seem but limitless when one starts thinking about certain people having access to becoming more intelligent or stronger through gene therapy. Where would the line be drawn ?

Many of those in favor of diminishing regulations on science feel that it impedes progress. It is true that regulation causes prolonged effects on getting procedures or treatments into use. But we must keep in mind the importance of safety and what i s best for all. With less regulation, there is bound to be mistakes. These are mistakes society will not stand, and without society's support, research will be in much worse position to make future advancements that benefit human welfare.

Researchers and special interests groups often want to speed up the regulation process to get their experiments to clinical trials before experiments show the technique is completely safe or effective. Steven Rosenberg, a prominent cancer researcher, is an example of someone whose research has been halted due to regulatory intervention. He wanted to place gene modified immune cells in cancer patients before experiments have showed conclusive results. To Steven Rosenberg, the research was valid and should have proceeded to this next step (Anderson 1391). Although many such concerns are legitimate, one must keep in mind that regulation is essential to minimize risks and keep the general public's rights in mind.

The simple truth is that regulation does not need to be a long, drawn out process. For example, in 1990, the gene therapy protocol for Ashanti DeSilva was reviewed, approved and implemented all with in the same year. Steps are being taken to reduce t he time needed to approve new gene therapy protocols especially when dealing with situations requiring immediate action. The RAC approved a expedited review mechanism that allows the NIH director to approve cases when dealing with emergency situations (G ershon 196). Steps like this will ensure that those with special circumstances such as life threatening diseases will receive rapid consideration.

Another proposition against government regulation is that it is the individual's decision to make such decisions concerning their lives. What must be kept in mind is that the purpose of regulation is for the safety of individuals. Patients are requir ed to make decisions on treatments that are highly technical and virtually impossible to understand with a limited scientific background. Would you feel comfortable making a potential life or death decision on something you did not completely understand? It is particularly important for regulation to oversee decisions in future germ-line therapy treatments because this therapy affects all future generations.

With all of the former concerns in mind, it is crucial that regulation be a necessary component of gene therapy research and applications. Government regulators and scientists must take a lead role in adopting a proactive approach to address these con trol issues and determining the correct procedures for dealing with them. The knowledge and expertise in the world is growing at such astonishing rates that society is not going to keep up. Steps will need to be taken to bridge this gap to keep citizens involved in the decision making process. It must be remembered that a lack of knowledge breeds ignorance and fear. These are two characteristics that can greatly damage the effectiveness of applying science to benefit human life. The scientific commun ity must do their best through regulation to ensure that science works for the people. The public must also accept responsibility by becoming educated and involved in the political processes that make these decisions. Why? Because gene therapy and othe r scientific advances will affect your lives.


Coutts, M. C. Human Gene Therapy. Obtained from the WWW 10/23/96:

Gavaghan, Helen. "Gene Therapy Approval May Be Rocky Road for Industry." Nature 374: 202.

Gershon, Diane. Nature. "RAC Defers to NIH Director on some Gene Therapy Cases." 361: 196.

Anderson, Christopher. "A Speeding Ticket for NIH's Controversial Cancer Star" Science 259: 1391.

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