Development of novel therapies for pediatric cancer: Successes and challenges

Development of novel therapies for pediatric cancer: Successes and challenges Peter J. Houghton from Greehey Children’s Cancer Research Institute and Mary-Ann Bjornsti from the University of Alabama discuss some of the key challenges in the development of therapies for pediatric cancer care. Despite pediatric cancer being a rare disease, constituting less than 1% of cancers overall, research into developing new effective and less genotoxic therapy is of critical importance. ‘Significance’ in terms of clinical research, for many, is determined by the number of patients afflicted with a particular cancer type. For example, in the US, there are approximately 150,000 new diagnoses for non-small cell lung cancer annually or about ten times the number of all pediatric cancers diagnosed. However, viewing pediatric cancer from the perspective of life-years saved, one can see that giving a child sixty plus years of good quality life is not only ‘significant’ but is actually cost-effective from the perspective of societal impact. Further, the burden on families following a cancer diagnosis for their child is enormous, both emotionally and financially. That stated, developing novel, effective new agents for the treatment of childhood cancer poses challenges. For three decades, most therapies for pediatric cancer have relied on chemotherapy, with or without external beam radiation. These treatments are toxic and genotoxic and often lead to secondary malignancies or life-threatening long-term sequelae.