Gene therapy trials: Parents in UK say include CF kids
Gene therapy trials: Parents in UK say include CF kids
Clinical trials involving gene therapy are considered to be of great enough real and potential risk that they are not attempted in children before they have been conducted with adults. But a survey conducted by doctors at Britain's second-largest hospital specializing in cystic fibrosis treatment indicates parents are inclined to favor clinical trials for their children.
Cystic fibrosis is a life-limiting illness with high morbidity, with a median life expectancy of just more than 24 years. Gene therapy is considered a promising potential treatment for genetic diseases like cystic fibrosis, and results, experts say, might be better and more easily measured in children than in adults.
In the Journal of Medical Ethics, researchers question which is more ethically sound — to subject children to experimental genetic therapy that has not been tested in adults, or to deny children access to therapy from which they might gain even greater clinical benefits than adult patients would.
One difference, the authors say, is that the goal in the treatment of cystic fibrosis is to get to it as early a possible; therefore, the benefits of gene therapy in early childhood could be substantial.
The authors of the study, based at Great Ormond Street Hospital and the Institute of Child Health in London, presented a questionnaire to the parents of children with cystic fibrosis treated at their unit. They asked parents whether, considering the potential risks and benefits of gene therapy for cystic fibrosis, they would be in favor of their children receiving the trial therapy.
Overall, 82% of the parents considered gene therapy to be the most important area of research in cystic fibrosis, and of those, 56% hoped it would result in a cure, 31% sought alleviation of symptoms, and 10% said they hoped for both.
Almost all — 99% — of the parents surveyed said they felt it was ethically sound for children to be given the opportunity to participate in gene therapy trial, provided appropriate safeguards were in place. Slightly fewer parents (91%) said they would consider consenting to their own children participating in clinical trials of gene therapy.
The authors suggest their study findings might help in the design of future clinical trials, but warn that children with cystic fibrosis and their parents should be protected from unnecessary harm.
The authors point out that their study was based on a small sample, and the questionnaire did not explicitly describe all possible risks of gene therapy.
Cystic fibrosis is caused by a single defective gene on chromosome 7, resulting in the defective production of a protein that regulates cellular ion transport. Researchers say gene therapy, the insertion of a normal gene into deficient host cells, is a potential treatment or cure for diseases caused by single-gene defects.
The researchers' findings are found in: Jaffe A, Prasad SA, Larcher V, et. al. Gene therapy for children with cystic fibrosis — Who has the right to choose? J Med Ethics 2006; 32:361-364.
Clinical trials involving gene therapy are considered to be of great enough real and potential risk that they are not attempted in children before they have been conducted with adults.Subscribe Now for Access
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