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	Presentation on Gene Therapy 3/26/03 Implementing Gene Therapy Karin and Steve   Treatment of CF Patients: In several clinical studies using various vectors, patients have shown temporary improvement Viral vector regarded as foreign by body The Case of Jesse Gelsinger: Condition: OTC, a metabolic disorder in which the liver is unable to metabolize ammonia GT Vector: Adenovirus (similar to that which causes a common cold) Experienced massive organ failure and died within three days of first treatment Outcome: other research facilities “came clean” on other adverse events, even deaths associated with gene therapy clinical trials Questions For Discussion: Does gene therapy represent an unacceptable level of “tinkering” with nature? If so, what are the alternatives to pursuing gene therapy treatments? If not, what minimum precautions or standards should be followed in carrying out GT clinical trials? Informed Consent: Which results from previous studies (animal trials, in vitro studies) are relevant? Two Perspectives: Disclosure of “adverse events” out of context or not rigorously tied to treatments could cause undue alarm. Even remotely possible adverse responses to the treatment should be communicated fully. Does efficacious research imply no risk? How much risk is acceptable? A Perspective… …”the privatization of science, combined with patients’ ardent desire for a cure, conspire to prevent meaningful protections for participants in all kinds of studies. Trade secrets, financial conflicts of interest and overloaded review committees obstruct informed consent by keeping news about ongoing studies beyond the reach of patients and researchers alike.” Arthur Caplan, Univ. of Penn Bioethics Reporting An “Adverse Event”: 1. Events must be formally reported immediately, whether or not they are thought to be directly associated with treatment 2. Reports must be public and are not proprietary 3. Patient privacy must be respected…no individual identities 4. The information will be analyzed by a committee at the NIH and communicated to the public in an “interpreted” form Response From ASGT (American Society of Gene Therapy): While the Society strongly favors public disclosure of adverse events, we believe adverse events must be reported in the clinical context in which they occurred. Patient protection is not served if the data presented is preliminary or out of context and therefore prone to misinterpretation. There is precedence where anecdotal reports, performed without appropriate controls, provided animal data which was misleading. Public disclosure of the initial information without the proper context could have resulted in major harm to the field. Successful Treatment of X-SCID: Severe Combined Immunodeficiency Disorder (“Bubble Boy” Disease) successfully treated in a young boy in England   Many treated individuals still thriving, but… Two infants who had received the largest “doses” developed leukemia in a French trial This trial, and others deemed closely related, were temporarily halted Might this be unfair to the children and parents who were experiencing a vast improvement in the quality of life as a result of the treatment? Who has the ultimate say in whether treatments should continue? Should desperate patients be able to “demand” risky treatments? A Final Question: Is there a tension between the “big picture”, long-term perspective which expects a certain amount of “collateral damage”, and the more focused, immediate perspective which acknowledges the value of every individual? Does this apply to our efforts to develop an effective gene therapy? “Jesse knew he wouldn’t be cured, but he wanted to be a hero.” --- Jesse's Dad   References:    ..
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 Updated 4/1/03 by thatcher@sonoma.edu