Gene Therapy for Pediatric Cancer: State of the Art and Future Perspectives

[摘要] While modern treatments have led to a dramatic improvement insurvival for pediatric malignancy, toxicities are high and asignificant proportion of patients remain resistant. Genetransfer offers the prospect of highly specific therapies forchildhood cancer. “Corrective” genes may be transferred toovercome the genetic abnormalities present in the precancerouscell. Alternatively, genes can be introduced to render themalignant cell sensitive to therapeutic drugs. The tumor can alsobe attacked by decreasing its blood supply with genes thatinhibit vascular growth. Another possible approach is to modifynormal tissues with genes that make them more resistant toconventional drugs and/or radiation, thereby increasing thetherapeutic index. Finally, it may be possible to attack thetumor indirectly by using genes that modify the behavior of theimmune system, either by making the tumor more immunogenic, or byrendering host effector cells more efficient. Several genetherapy applications have already been reported for pediatriccancer patients in preliminary Phase 1 studies. Although no majorclinical success has yet been achieved, improvements in genedelivery technologies and a better understanding of mechanisms oftumor progression and immune escape have opened new perspectivesfor the cure of pediatric cancer by combining gene therapy withstandard therapeutic available treatments.

[学科分类] 基础医学