Gene Therapy: Cure or Poison? The Proper Role of the FDA in the Bubble Boy Disease Question
Woodruff, Michele R.
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CitationGene Therapy: Cure or Poison? The Proper Role of the FDA in the Bubble Boy Disease Question (2004 Third Year Paper)
AbstractOn January 14, 2003, the Food and Drug Administration â€œsuspended 27 gene therapy trials involving several hundred patients after learning that a second child treated in France had developed a condition resembling leukemia.â€ Gene therapy treats diseases caused by defective genes by introducing healthy genes into the body. For patients who are battling life-threatening diseases, the only chance of survival may be the hope of gene therapy, perhaps because there is no known cure, no available matched transplant donor, or no cures without even worse side effects. â€œSome of the trials being halted are intended to treat AIDS and cancer.â€ The â€œsecond child treated in France,â€ who caused the alarm, had received gene therapy treatment not for AIDS or cancer, but for the less common ailment â€œbubble boy disease,â€ a severe and fatal immunodeficiency disorder. Bubble boy disease attacks newborn boys, who while in the womb, relied on their motherâ€™s immunity but, once born, are left helpless in fighting other diseases that attack their infant bodies. With the trials halted, parents are left helpless to seek gene therapy for their children. Is it right to eliminate alternate, but risky remedies?
Citable link to this pagehttp://nrs.harvard.edu/urn-3:HUL.InstRepos:8852213
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