The Ethics Of Human Gene Therapy

The periodical of Leroy Walters not only talks about the ethical issues of Human Gene Therapy but also the "political" and economical issues as well. It explains how the therapy can help only a certain categories genetic disorders, those that have "the simplest kinds of genetic defects". So far, only lab animals are being used to check if these treatments actually work, and even with these animals, the modification is still inefficient. No human genetic alterations have even been contemplated on, moreover any actual introduction of such treatment on humans, been made.

It goes on to talk about the therapy's relationship of possible benefits and harms, dubbed as "risk assessment". How many people actually have the disease, are at risk to have the disease, how many generations it can affect, and lastly, how many people actually die from it. The process assesses the present medical therapies, diets, alternative treatments that render the patient control over his/her condition. It involves laboratory experiments in animals and tissue samples to further determine and evaluate the safety and of course the effectiveness of the new technique. Using lab animals however, poses an exclusive problem; there are limited animals that have single gene defects that cause problems in humans and there are none that have the human disease of enzyme deficiency. So in short the resources for experimentation are very limited, time consuming and have to be induced to the animal by inserting a new gene into the animal cell and monitor the gene and even by doing this, it still will not be able to demonstrate a clinical "cure" in a diseased laboratory animal.

A benefit of gene therapy thou is that the affected cells are treated outside the animal's and introduced in vitro. So if anything goes wrong with the cells, they are not returned back into the animal's body. The downside of this procedure is that it is the new gene will be introduced back into the body via the stem cells and in order to do this they have to create retroviruses where in a lot of the original genetic material has been removed. These new flawed retro viruses will be used as vectors for the new gene. Now the complication of this is that there is a possibility that the retroviral vectors will recombine with unnoticed viruses or endogenous DNA sequences in cells. Should this happen they the retroviral vectors will become infectious. Another possibility is that they can trigger previously dormant protoncogenes or disrupt essential, properly functioning genes. After all this said, there still no apparent proof if this will generate high levels of expressions in the lab animals to offer reasonable hope of any clinical benefit.

Other ethical issues involved in germ line therapy are those that other "controversial" (dialysis, heart transplants, etc) treatments before used to face. One of them and probably the most difficult is that of selecting which patients are to receive the treatment. Even with a specialized committee created to screen the candidates it is and will always be very difficult to avoid making decisions about the comparative social worth when not all patients can be offered treatment. Another issue is about the informed consent of the patient or the patients parents/legal guardian. The awareness not only should provide all the necessary information about the procedure, the expense, the possible outcome, but also it should emphasize that they are embarking on essentially uncharted territory. It should also strengthen their hopes and make them realize that should the treatment become successful, they are participating in a major therapeutic break through. The issue of privacy may also come about. The pioneers of heart transplants' names were very public. Unlike that of the newborn receiving a baboon heart in 1984, where her name was only revealed upon her death. There should be a sensible balance between the personal privacy of the patient and the need of the public and media to know. It would be the researchers' primary accountability to ensure this. Infants and young children who do not have the ability to express their own views about publicity should be given special protection.

The article also emphasizes special guidelines and ethical codes that should be followed. The Declaration of Helsinki is an international "standard" used in human experimentation. The revised edition was adopted by the World Medical Association and it includes ethical guidelines on research design, risk-benefit analysis, informed consent, privacy and accuracy in reporting research results. In most nations that have "ethical committees", this is a standard guideline that they follow in accordance to their country's individual laws. An example of such process is that done in the United States where in a framework called "Points to Consider in the Design and Submission of Human Somatic-Cell Gene Therapy Protocols" was not only scrutinized by an ethical committee, but public national review as well. Primary areas discussed in the document were:

1. Objectives and rationale of the proposed research

2. Research design, anticipated risks and benefits

a. Structure and characteristics of the biological system

b. Preclinical studies

c. Clinical Procedures, including monitoring the patient

d. Public health considerations

e. Qualifications of investigators and adequacy facilities

3. Selection of patients

4. Informed consent

5. Privacy and confidentiality

The Working Group on Human Gene Therapy, a subcommittee of the NIH Recombinant DNA Advisory Committee (RAC) is composed of: three laboratory scientists, three clinicians, three ethicists, three attorneys, two public policy specialists, and a lay member. They released public nation review on each gene therapy proposal and all interested persons will therefore have ready access to information about gene therapy proposals, to the review process itself and to its outcome.

Using genetic diagnosis has again its pros and cons. The pros are of course, early diagnosis of the gene abnormality in a fetus; or if the child will have the tendency to develop a particular disease like that of atherosclerosis. Will the child have a late onset of a disorder such as that of Huntington's disease? The cons are then of course ethical questions that go with the benefits of such "early notice". Questions like, which children should be tested and what should