Background: Despite impressive improvements of treatment results in children suffering from hepatoblastoma (HB), advanced tumor stages still produce unsolved problems for treating physicians. HB tumors are commonly treated following trial regimens, which have been set up by multicenter study groups based on an ongoing treatment evaluation. Recently, treatment outcomes have reached a phase of stagnation and currently alternative treatment options are under investigation. Gene-directed anti-HB treatment approaches have been newly identified as promising option in this regard.
Objective: To optimize and extend gene directed treatment strategies in order to improve treatment results of advanced stage HB.
Methods: A review of the literature on genetic alterations in HB and of the current state of experimental gene-directed treatment approaches against HB.
Results/conclusion: HB seems to be a suitable target for gene-directed treatment approaches. Further development of gene transfer systems and HB-specific strategies will lead to new alternatives for the treatment of this malignancy.