Background:

• A year ago, Jesse Gelsinger, and 18-year-old from Arizona died while undergoing gene therapy for his inborn metabolic disorder. Recently, Gelsinger's parents filed a lawsuit against Arthur Caplan, the bioethicist who advised that the therapy be attempted.
• Gelsinger's disease, a one-gene disorder, caused him to lack one protein in a certain genetic pathway. While the disease was not immediately life threatening, it was also one of the easiest types of diseases to correct with gene therapy.
• The therapy attempted to use an adenovirus as vector to deliver the corrected gene into target cells. Gelsinger probably died because his immune system reacted negatively to these adenovirus vectors.
• SCIDS, an immunodeficiency disease had been treated in France using a protocol similar to the one used on Gelsinger.
• One year later, Gelsinger's parents have filed lawsuits against Arthur Caplan and the University of Pennsylvania's Genome Group, which conducted the therapy.

• Originally the plan was for the gene therapy to be performed on newborns who would die anyway. These newborns were not capable of making their own decisions, however, while parents of these infants would be emotionally stressed, and could not make objective decisions or give informed consent. Consequently, Caplan advised that the therapy be attempted on consenting adults instead.

• The family's claim against Caplan is indirect cause of death. Had newborns been used in the trials (an action which Caplan advised against), Gelsinger would not have been involved in the trials, and would not have died.

Additional Thoughts/Reactions/Speculations/Opinions:

• Fault in this suit will be depend on how the court construes consent. Did the family really give consent towhat happened?
• Caplan only gave unofficial advice to the family. It's surprising that the family chose to sue him.
• A research setting is different from a medical setting when obtaining informed consent. Also, much of the actual cause of death depends on how the research was administered (dosages, etc.).
• Is it ever ethical to use gene therapy to cure non-lethal diseases?
• If we choose to continue with gene therapy, there will be many environmental factors and not-understood interactions that could lead to more unforeseen tragedies.
• Caplan had a financial stake in the company that was attempting the gene therapy. Was there a conflict of interests?
• Because so much research is now provided by the private sector, financial interests are tempering the direction of research. Also, restrictions on funding cannot be used to regulate research in the private sector.
• In other words, it's hard to tell science NOT to do something. Science will find a way to proceed.
• Which should come first: the interests of the patient or the researcher? When there's a dispute, who should we protect with legislation?
• Since Gelsinger's death, the federal government has frozen all gene therapy trials, and proposing that all NIH-funded researchers receive ethical training. Interesting idea… but the federal government will not provide funding for this training.
• We can use one death to make a difference in gene therapy. It will arouse public interest and awareness, and make everyone realize we should seriously consider how to proceed, if at all.