Novel Targeted Agents in Neuroblastoma

The still unsatisfactory long-term survival rates in children with high risk neuroblastoma and the high long-term sequelae like hearing loss in the surviving patients require urgently new therapeutic approaches. Increasing knowledge of tumor-specific molecular characteristics and the availability of an array of new small molecules evoke much of hope.

The translation into the clinical reality has been slow and is discussed for the genes MYCN, ALK, PLK-1, the proteasome, and the ganglioside GD2 as potential targets. These examples demonstrate that experimental evidence is a useful starting point, but insufficient to predict clinical success. The effects in the patients proved to be much more complex than the terms „targeted therapies“ and „individualized medicine“ suggest. The high cost for the production of a new agent, the administrative burden and the many levels of main and side effects in the patients make it mandatory to treat patients within the frame of clinical trials only. Cooperative efforts as in the frame of ITCC are preferable to produce reliable answers to burning clinical needs within a justifiable time frame.