Gene Therapy: Cure or Poison? The Proper Role of the FDA in the Bubble Boy Disease Question

Abstract

On January 14, 2003, the Food and Drug Administration “suspended 27 gene therapy trials involving several hundred patients after learning that a second child treated in France had developed a condition resembling leukemia.†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. “Some of the trials being halted are intended to treat AIDS and cancer.†The “second child treated in France,†who caused the alarm, had received gene therapy treatment not for AIDS or cancer, but for the less common ailment “bubble boy disease,†a severe and fatal immunodeficiency disorder. Bubble boy disease attacks newborn boys, who while in the womb, relied on their mother’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?