ADA gene therapy enters the competition

Article Abstract:

Perhaps one fourth of the children in the world who suffer from a deadly form of immune deficiency lack the gene for adenosine deaminase (ADA, an enzyme), and could be treated by the insertion of a healthy ADA gene into their cells. A proposal by NIH (National Institutes of Health) scientists R. Michael Blaese and W. French Anderson has been approved to do just that. Patients will be infused with T cells (a type of white blood cell active in immune response) that carry healthy ADA genes. Proposals for gene therapy must show that alternative therapy does not exist and that the researchers have data that show how the gene therapy should work. One new drug for treating the immune deficiency, PEG-ADA (ADA coated with polyethylene glycol so that it remains in the body long enough to perform a metabolic role), is not a cure and costs $60,000 per year, but it threatened the gene therapy project, since it could have been considered an alternative therapy. After considerable debate during the NIH review process, it was decided that the children treated with gene therapy will also take PEG-ADA. Some reviewers feared that taking the drug away would be unethical. Other concerns regarding the safety of injecting the retroviral DNA carrier for the ADA gene into children were raised. However, these were answered satisfactorily. Finally, will ADA gene therapy ultimately be better than PEG-ADA for these patients? Experiments in animals indicate that gene therapy can, in effect, give animals an immune system, while administration of ADA does not have this effect. Experiments to carry out the proposed studies may begin in October 1990. (Consumer Summary produced by Reliance Medical Information, Inc.)

Usage, Immunological deficiency syndromes, Immunologic deficiency syndromes

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One step closer for gene therapy

Article Abstract:

True gene therapy, in which a gene is inserted into a patient to correct severe disease, will most likely be attempted in 1990. The way for such an approach was cleared when a subcommittee of the National Institutes of Health (NIH) approved a proposal by the National Cancer Institute to insert the ADA (adenosine deaminase) gene into a young child with ADA deficiency. This rare condition, affecting fewer than 50 children worldwide, leaves patients unable to fight off infection, causing death during the first decade of life. Bone marrow transplantation and PEG-ADA (a drug) can help, but cannot be used by all patients. Nor is PEG-ADA a cure. Four or five children with ADA deficiency will undergo the procedure. The subcommittee's earlier review of the gene therapy proposal did not encourage researchers to hope for approval. However, changes in the proposal itself, as well as developments in animal models that indicate the experiment should be effective, may have affected the group's evaluation. Final approval is expected on July 30, when the acting director of NIH and the Food and Drug Administration will make their decision. (Consumer Summary produced by Reliance Medical Information, Inc.)

Genetic disorders

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