Aims This paper will examine the slow progress of drug development to treat paediatric cancers beginning in the 1940s with Sidney Farber’s promising development of aminopterin and published 1947 study. Nearly seventy years later there have been very few new drugs to treat either blood or solid tumours in children since the 1980s. The study will include the financial and political issues of this slow progress and will conclude with an overview of the most recent drug launches in the United States in leukemias and neuroblastoma.

Methods The sources for this paper included published articles in Paediatric Blood and Cancer, Leukaemia, Cancer: Journal of the National Cancer Institute and press releases of the Cancer Therapy Evaluation Program Paediatric Branch (National Cancer Institute–NCI). The author has also examined the archives of the NCI and primary sources in the National Institutes of Health Library and oral and written sources at the National Institutes of Health Office of History.

Results In part because of the nature of childhood cancers as rare diseases national funding for new drug development has greatly lagged behind that of development of drugs to treat breast and colon cancer and other adult cancers. Public groups in cooperation with medical groups have in the last decade put increasing pressure on the United States Congress to look into and fund some of the most recent pipeline drugs for paediatric cancers.

Conclusion Public pressure particularly from childhood cancer organisations has resulted in some new drug development, the first in nearly thirty years in the United States. Along with promising personalised and targeted therapies of very recent note these therapeutics come after a long struggle to begin to prioritise funding for children with cancer.