title: Proof-of-concept rare cancers in drug development: the case for rhabdomyosarcoma.
abstract: Rare diseases typically affect fewer than 200,000 patients annually, yet because thousands of rare diseases exist, the cumulative impact is millions of patients worldwide. Every form of childhood cancer qualifies as a rare disease-including the childhood muscle cancer, rhabdomyosarcoma (RMS). The next few years promise to be an exceptionally good era of opportunity for public-private collaboration for rare and childhood cancers. Not only do certain governmental regulation advantages exist, but these advantages are being made permanent with special incentives for pediatric orphan drug-product development. Coupled with a growing understanding of sarcoma tumor biology, synergy with pharmaceutical muscle disease drug-development programs, and emerging publically available preclinical and clinical tools, the outlook for academic-community-industry partnerships in RMS drug development looks promising.
authors.affiliation: Department of Student Affairs, Oregon State University, Corvallis, OR, USA.
authors.forename: E
authors.initials: E
authors.lastname: Sokolowski
authors.affiliation: 1] Department of Student Affairs, Oregon State University, Corvallis, OR, USA [2] Pediatric Cancer Biology Program, Department of Pediatrics, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR, USA.
authors.forename: C B
authors.initials: CB
authors.lastname: Turina
authors.affiliation: Pediatric Cancer Biology Program, Department of Pediatrics, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR, USA.
authors.forename: K
authors.initials: K
authors.lastname: Kikuchi
authors.affiliation: 1] Division of Molecular Pathology and Cancer Center, Massachusetts General Hospital, Boston, MA, USA [2] Harvard Medical School and Harvard Stem Cell Institute, Boston, MA, USA.
authors.forename: D M
authors.initials: DM
authors.lastname: Langenau
authors.affiliation: Pediatric Cancer Biology Program, Department of Pediatrics, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR, USA.
authors.forename: C
authors.initials: C
authors.lastname: Keller
mesh_terms: D002648:Child; D055808:Drug Discovery; D006801:Humans; D035583:Rare Diseases; D012208:Rhabdomyosarcoma