6.2 Gene Therapy
Julie Samuël, LLB. LLM and Jacques P. Tremblay, PhD
- To understand the ethical and legal issues relating to the inclusion of children in clinical studies on gene therapy.
- To identify allowable limits regarding the inclusion of children in these clinical studies.
Twelve-year-old Felix has Duchenne muscular dystrophy (DMD). Because of the progression of the disease and the weakness of his muscles, Felix must use a wheelchair. Given that the diagnosis has been confirmed by molecular therapy techniques, which have identified the mutation in the dystrophin gene, Felix's physician, Dr. Williams, is certain that Felix's muscles will continue to degenerate rapidly. Although there are certain steroid-based medicines that can help children suffering from DMD to save their muscular strength for a certain amount of time, there is no treatment that can fight the disease.
Dr. Williams, who is also a researcher, has prepared a research project on an ex vivo somatic gene therapy to treat DMD. This therapy consists of removing myoblasts from children with the disease, modifying them genetically in a laboratory to insert the normal dystrophin gene, and re-injecting them into the child with a large number of injections to the muscle. This is still an experimental therapy with various risks for the health of participating children, such as a potential immune system reaction against the dystrophin. There is also a potential risk of tumour formation by the genetically modified cells, even if samples of the genetically corrected cells are pre-tested by transplantation in immunodeficient mice. Despite these risks, this therapy offers hope to improve the quality of life of children suffering from DMD.
- What special challenges does gene therapy research pose?
- Is it ever acceptable to perform gene therapy research involving children?
- What are the benefits and harms of such research? Are they reasonable?
- Does a child need to assent to his/her participation in the gene therapy research? If a child dissents, should his/her decision be respected?
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