Ethics-Governance: Article: eMJA: Gene therapy: great expectations?

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eMJA: Gene therapy: great expectations? Unrealistic expectations may overshadow genuine advances and focus attention more on failures For many years, scientists and clinicians have sought to harness the power of genes for treating disease. The potential for gene therapy to cure otherwise untreatable conditions, and to offer a completely new strategy where conventional medicine has limited efficacy, has attracted huge interest and investment of time and money from both academic and commercial biotechnology sectors. The field of gene therapy has therefore grown rapidly. However, unrealistic expectation has overshadowed genuine advances and focused attention more on clinical failures and unnecessary mistakes. Only recently, federal law enforcement officials announced a substantial settlement with the University of Pennsylvania after the death of a patient in a gene therapy trial in 1999. Consequently, gene therapy has been viewed with suspicion, and the tight regulatory control on the conduct of clinical studies has to some extent restricted progress. But is the frequently cited accusation that gene therapy has failed to deliver in the clinical arena justified, or is it another manifestation of unrealistic expectation? At the start of the 1990s, the first clinical trials of gene therapy were attempted for an inherited severe combined immunodeficiency (SCID) caused by deficiency of the intracellular enzyme adenosine deaminase (ADA).1-4 In the absence of definitive treatment, SCID of any molecular type is usually fatal within the first year of life, although patients with ADA deficiency can be supported by administration of exogenous bovine enzyme. Full Article: http://www.mja.com.au/public/issues/182_09_020505/thr10181B_fm.html


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