At1 Research Culture And Ethics Case Study H-Gene Therapy
AT1 – Research Culture and Ethics
Case Study H – Gene Therapy
Word Count: 999
Uni ID: 211322231
Prusoth Yoga
3/14/2014
Prusoth Yoga
Ethics are moral principles that govern a researcher’s behaviour on conducting of an activity; values that embrace ethics are; Voluntas aegroti suprema lex (The will of the patient is the highest law), Salus aegroti suprema lex (The safety of the patient is the supreme law), primum non nocere (First, do no harm) and fairness and equality.1 The National Statement on Ethical
Conduct (2007) encompasses ethical guidelines the Human Research Ethics Committees
(HREC) practices to identify ethical issues that may be prevalent in research proposals.2 The
HREC’s responsibility is to make sure participants …show more content…
are fully aware of the risks/benefits involved and that their consent is well informed. Ethical guidelines are not only there to protect the participants, but also ensure the longevity of clinical trials.2 Since the 1970; influence of ethics in medicine has been increasing; via use of “Institutional Review Boards to evaluate experiments on human subjects, establishment of ethics committees, implementation of the role of clinician ethicists, and integration of ethics into many medical school curriculums”.3 Case study H centers on a gene therapy trial, where bone marrow cells of a lymphocyte defective child are reconstituted using a retrovirus carrier.
A functional gene carried by the
“vector” repairs the faulty lymphocyte gene; and then inserted back into the patient to reconstitute their bone marrow to produce specific types of white blood cells that were formerly defective. According to researchers, gene therapy seemed more promising than the conventional treatment of unrelated donor bone marrow transplant since present lymphocytes identify self-MHC on repaired cells and prevent graft rejection; a high possibility via the transplant method.4
During treatment, the patient was unable to reconstitute bone marrow strongly and a top-up of modified gene was considered. However reports came in from Europe that 3 of their …show more content…
12 patients in the trial had developed Leukaemia and 2 had died as a consequence. The cause of leukaemia was unknown, but a statement made by “HREC Chair A” suggested that a small fragment of gene had either stimulated proliferation of leukemia cells or stimulated an inhibitor that allowed to cells to reproduce4. Therefore the patient wasn’t topped up and treated with
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the conventional therapy; bone marrow transplant. Subsequently the patient died due to complications.4 Predominant ethical issues which arose from this case study include; response to an adverse effect, risk/benefit balance and consent. Risks were justified on the premise of treatment options available at the time. Risks associated with the vector were reasoned, as patients had a lower chance of survival with the conventional treatment. The ethical issue of adverse effect came in the form of patients developing leukaemia and as a result passing away; this caused the risk/benefits ratio to be increased. Risks also increased due to the limited amount of clinical data available; only 4 successful treatments resulted in Europe. Furthermore, the information regarding adverse effects resulting in leukaemia from other trials wasn’t available to the parent’s during the consent process, as it was only found out later; if informed about this earlier they may have opted for alternatives. Therefore, communicating these unknowns during consent is challenging due to the complexity of the treatment. The ethical issue to be discussed in detail is be of consent, as it’s an imperative issue since the participant is a child and would the obligation of the parents to provide free, informed consent. However; consent might be influenced through seeing their child in a desperate situation; where an element of coercion is always involved.4
The National Statement on Ethical Conduct (2007) states that consent must be obtained on the moralities that “each human being must be recognized as having inherent value, and has the right to autonomy to determine his or her own path in life”.2 Consent from a participant is attained via a consent form, after being informed of the risks/benefits involved.2
Due to the nature of medical decisions it’s often the mentally disabled and the physically incapable aren’t able to give binding consent; as seen with case H.
A child’s ability or competence to provide proper informed consent is often lacking and it’s the responsibility of the parents/legal guardians to provide the consent.2
Ethical dilemmas often arise when the judgment of the parents/guardians differ from medical professional as to what constitutes appropriate decisions "in the best interest of the child".5
Due to this in the past gene therapy trials have been restricted to only adults 6. It’s apparent
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from interviews with “HREC Chair A”; it was explained to the parents that if gene therapy wasn’t successful they could fall back to the conventional treatment; but gene therapy was a more optimal procedure.4 It was also understood the parent’s decision for electing gene therapy was acquired through information they had read on the internet; which may
have been skewed by bias reflecting just the positives of the therapy.4 Albeit both parties agreed on administering gene therapy; the question is, was consent for treatment out of coercive pressure; often witnessed out of desperation, particularly when no other optimal treatments are available.
To understand the consent giving process, a study in 2010 focused on participants in clinical trials. The study showed 26% of the participants consented before reading the form and 43% were ignorant to adverse side effects. It concluded; if participants signed consent on a different day to receiving it they better understood the risks/benefits involved. Indicating that the consent process would benefit from giving participants more time to better understand the research they are in.7
In summary, case H displayed incompetence towards ethics, due to the researcher’s reliance on data from trials in Europe which weren’t fully available at time of consent. The declaration of
Helsinki (1964) requires professionals to provide informed consent 8 which wasn’t apparent in case H. Moreover, when faced with no other successful option of treatment, consent maybe coercive and out of desperation; therefore it’s imperative that researchers ensure consent is well informed by making all possible data available, not just for the participant’s benefit but also to safeguard future clinical trials.
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References
1. Gillon, R (1994). "Medical ethics: four principles plus attention to scope". British Medical
Journal 309 (184).
2. National Health and Medical Research Council (2007). National Statement on Ethical
Conduct in Human Research. Australian Government, Canberra.
3. Lakhan SE, Hamlat E, McNamee T, Laird C (2009). "Time for a unified approach to medical ethics". Philosophy, Ethics, and Humanities in Medicine 4 (3): 13.
4. National Health and Medical Research Council (2006). Challenging Ethical Issues in
Contemporary Research on Human Beings. Australian Government, Canberra.
5. Committee on Bioethics (1995). Informed consent, parental permission, and assent in pediatric practice. Pediatrics. 95(2):314-7.
6. Christopher Baum, Boris Fehse, Klaus Kuhlcke, Olga S. Kustikova, Birgit Stable, Anke
Wahlers and Axel R. Zander, and. 2003. Dose finding with retroviral vectors: correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population. Blood journal. 102:3934-3937.
7. Josep M. Amau, Inma Fuentes, Pilar Hereu, Eulalia Perez, Pilar Sune and XavierVidal
(2010). Consent in clinical trials: What do patients know? Contemporary Clinical Trials.
31:443-446.
8. Jones J (1981). Bad Blood: The Tuskegee Syphilis Experiment. New York: Free Press.
Case Study H – Gene Therapy
Word Count: 999
Uni ID: 211322231
Prusoth Yoga
3/14/2014
Prusoth Yoga
Ethics are moral principles that govern a researcher’s behaviour on conducting of an activity; values that embrace ethics are; Voluntas aegroti suprema lex (The will of the patient is the highest law), Salus aegroti suprema lex (The safety of the patient is the supreme law), primum non nocere (First, do no harm) and fairness and equality.1 The National Statement on Ethical
Conduct (2007) encompasses ethical guidelines the Human Research Ethics Committees
(HREC) practices to identify ethical issues that may be prevalent in research proposals.2 The
HREC’s responsibility is to make sure participants …show more content…
are fully aware of the risks/benefits involved and that their consent is well informed. Ethical guidelines are not only there to protect the participants, but also ensure the longevity of clinical trials.2 Since the 1970; influence of ethics in medicine has been increasing; via use of “Institutional Review Boards to evaluate experiments on human subjects, establishment of ethics committees, implementation of the role of clinician ethicists, and integration of ethics into many medical school curriculums”.3 Case study H centers on a gene therapy trial, where bone marrow cells of a lymphocyte defective child are reconstituted using a retrovirus carrier.
A functional gene carried by the
“vector” repairs the faulty lymphocyte gene; and then inserted back into the patient to reconstitute their bone marrow to produce specific types of white blood cells that were formerly defective. According to researchers, gene therapy seemed more promising than the conventional treatment of unrelated donor bone marrow transplant since present lymphocytes identify self-MHC on repaired cells and prevent graft rejection; a high possibility via the transplant method.4
During treatment, the patient was unable to reconstitute bone marrow strongly and a top-up of modified gene was considered. However reports came in from Europe that 3 of their …show more content…
12 patients in the trial had developed Leukaemia and 2 had died as a consequence. The cause of leukaemia was unknown, but a statement made by “HREC Chair A” suggested that a small fragment of gene had either stimulated proliferation of leukemia cells or stimulated an inhibitor that allowed to cells to reproduce4. Therefore the patient wasn’t topped up and treated with
Prusoth Yoga
the conventional therapy; bone marrow transplant. Subsequently the patient died due to complications.4 Predominant ethical issues which arose from this case study include; response to an adverse effect, risk/benefit balance and consent. Risks were justified on the premise of treatment options available at the time. Risks associated with the vector were reasoned, as patients had a lower chance of survival with the conventional treatment. The ethical issue of adverse effect came in the form of patients developing leukaemia and as a result passing away; this caused the risk/benefits ratio to be increased. Risks also increased due to the limited amount of clinical data available; only 4 successful treatments resulted in Europe. Furthermore, the information regarding adverse effects resulting in leukaemia from other trials wasn’t available to the parent’s during the consent process, as it was only found out later; if informed about this earlier they may have opted for alternatives. Therefore, communicating these unknowns during consent is challenging due to the complexity of the treatment. The ethical issue to be discussed in detail is be of consent, as it’s an imperative issue since the participant is a child and would the obligation of the parents to provide free, informed consent. However; consent might be influenced through seeing their child in a desperate situation; where an element of coercion is always involved.4
The National Statement on Ethical Conduct (2007) states that consent must be obtained on the moralities that “each human being must be recognized as having inherent value, and has the right to autonomy to determine his or her own path in life”.2 Consent from a participant is attained via a consent form, after being informed of the risks/benefits involved.2
Due to the nature of medical decisions it’s often the mentally disabled and the physically incapable aren’t able to give binding consent; as seen with case H.
A child’s ability or competence to provide proper informed consent is often lacking and it’s the responsibility of the parents/legal guardians to provide the consent.2
Ethical dilemmas often arise when the judgment of the parents/guardians differ from medical professional as to what constitutes appropriate decisions "in the best interest of the child".5
Due to this in the past gene therapy trials have been restricted to only adults 6. It’s apparent
Prusoth Yoga
from interviews with “HREC Chair A”; it was explained to the parents that if gene therapy wasn’t successful they could fall back to the conventional treatment; but gene therapy was a more optimal procedure.4 It was also understood the parent’s decision for electing gene therapy was acquired through information they had read on the internet; which may
have been skewed by bias reflecting just the positives of the therapy.4 Albeit both parties agreed on administering gene therapy; the question is, was consent for treatment out of coercive pressure; often witnessed out of desperation, particularly when no other optimal treatments are available.
To understand the consent giving process, a study in 2010 focused on participants in clinical trials. The study showed 26% of the participants consented before reading the form and 43% were ignorant to adverse side effects. It concluded; if participants signed consent on a different day to receiving it they better understood the risks/benefits involved. Indicating that the consent process would benefit from giving participants more time to better understand the research they are in.7
In summary, case H displayed incompetence towards ethics, due to the researcher’s reliance on data from trials in Europe which weren’t fully available at time of consent. The declaration of
Helsinki (1964) requires professionals to provide informed consent 8 which wasn’t apparent in case H. Moreover, when faced with no other successful option of treatment, consent maybe coercive and out of desperation; therefore it’s imperative that researchers ensure consent is well informed by making all possible data available, not just for the participant’s benefit but also to safeguard future clinical trials.
Prusoth Yoga
References
1. Gillon, R (1994). "Medical ethics: four principles plus attention to scope". British Medical
Journal 309 (184).
2. National Health and Medical Research Council (2007). National Statement on Ethical
Conduct in Human Research. Australian Government, Canberra.
3. Lakhan SE, Hamlat E, McNamee T, Laird C (2009). "Time for a unified approach to medical ethics". Philosophy, Ethics, and Humanities in Medicine 4 (3): 13.
4. National Health and Medical Research Council (2006). Challenging Ethical Issues in
Contemporary Research on Human Beings. Australian Government, Canberra.
5. Committee on Bioethics (1995). Informed consent, parental permission, and assent in pediatric practice. Pediatrics. 95(2):314-7.
6. Christopher Baum, Boris Fehse, Klaus Kuhlcke, Olga S. Kustikova, Birgit Stable, Anke
Wahlers and Axel R. Zander, and. 2003. Dose finding with retroviral vectors: correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population. Blood journal. 102:3934-3937.
7. Josep M. Amau, Inma Fuentes, Pilar Hereu, Eulalia Perez, Pilar Sune and XavierVidal
(2010). Consent in clinical trials: What do patients know? Contemporary Clinical Trials.
31:443-446.
8. Jones J (1981). Bad Blood: The Tuskegee Syphilis Experiment. New York: Free Press.