Cancer Progress in Embattled Gene Therapy Field

The field of gene therapy - the premise of providing therapeutic benefit by introducing genes into a patient - has been beleagured by a lack of promising clinical results, coupled with a history of erratic clinical attempts, with the most prominent example being that of patient Jesse Gelsinger, who was improperly managed as a patient in a gene therapy trial, leading to his death.  A lack of truly informed consent regarding conflicts of interest held by the researchers running the trial meant that the Gelsinger family was underinformed about the risks and biases that attended to the clinical trial. This episode of mismanagement of the patient- researcher relationship has served as a cautionary tale for other biotechnology therapies which are studied by those with a financial stake in a (positive) outcome. Beyond this individual case, gene therapy trials are still subject to the regulatory review of the NIH Recombinant DNA Advisory Committee (RAC). Against this backdrop, the recent report of a significant remission achieved using genetically altered T cells from leukemia patients illustrates again the promise of gene therapy, coupled with the theoretical assumption that the patient's immune system can be harnessed to fight cancer cells. Article in New England Journal of Medicine.