Progress in cancer gene therapy

The "First International Symposium on Genetic Anticancer Agents," which too k place in Amsterdam on March 8-9, 2000, served as a forum to review the re sults of preclinical and clinical gene therapy studies for cancer endeavore d to date. Despite the fact that gene therapy was initially conceptualized as an appro ach for inherited genetic disease, it is currently finding its widest emplo y for treating neoplastic disorders. In this regard, more than 70% of patie nts treated to date in human clinical gene therapy protocols have been in t he context of anticancer regimens.' Of note, the application of gene therap y for cancer has proceeded from the same rational basis as was originally c onceptualized for inherited genetic disorders. Specifically, the molecular basis of those disorders is increasingly being understood, therapeutic gene s are available, and alternative therapies are often lacking. Most recently , the field of gene therapy has enjoyed the realization of the first incont rovertible evidence of clinical benefit, for hemophilia and cardiovascular disease, in its first 15 years of human application.(2) This recent recogni tion of the potential power of gene therapy, and the current lack of realiz ing such ends for neoplastic disease, has led to a reassessment of the fiel d. Such a critical analysis is a necessary step in defining the means to pr ogress the technology toward achieving the potential benefits of gene thera py for cancer.