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Towards Clinical Applications

Posted by Ares Wednesday, December 1, 2010

The efficacy of gene therapy will ultimately need to be assessed and demonstrated relative to conventional therapeutics. While it is widely believed that many inborn errors of metabolism may come to be best treated by gene therapy, these will also be significant advances in the development of conventional pharmaceutical products, biological products such as enzyme replacement, and transplantation, including the potential for inducing tolerance to allogeneic or xenogeneic grafts.
The question “Why gene therapy?”
must be answered by showing that proposed gene therapies are more effective and safer than other therapies,
provide adequate compliance, and are cost-effective relative to other therapies.

The present focus of gene therapy is on demonstrating that gene replacement is feasible and can be effective. Extensive work remains to be done to demonstrate the safety and practicality of gene therapy. This requires increased attention to the pharmacology,toxicity, manufacture, and economics of gene therapy in addition to the biology.
It is already apparent that the economics of gene therapy of inherited metabolic diseases need to be considered in detail in developing a gene therapy. While gene therapy has been proposed for many inborn errors of metabolism, such therapies require the development of products which are clinically validated and manufactured for clinical use. Moreover, once gene therapy is commenced, there will be an ethical imperative to make these products available on a continuous basis to treat current and future patients. Academic centers which are currently involved in gene therapy research may have a limited capacity for production and a limited commitment to do so indefinitely once such therapies move beyond the research phase. The “orphan drug” law in the United States was designed to encourage industrial interest in rare diseases. Even this incentive is unlikely to encourage the development of gene therapies for more than a handful of the hundreds of genetic disorders potentially amenable to gene therapy. It is unlikely that gene therapies will become available on a commercial or ongoing basis for most of the rare inborn errors of metabolism for which it has been proposed.

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