title: Targeted therapy for hereditary cancer syndromes: neurofibromatosis type 1, neurofibromatosis type 2, and Gorlin syndrome.
abstract: Hereditary cancer syndromes are well known in the oncology community, typically affecting children, adolescents, and young adults and thereby resulting in great cumulative morbidity and mortality. These syndromes often lag behind their de novo counterparts in the development of approved novel treatment options due to their rarity in the general population. Recent work has allowed the identification of molecular aberrations and associated targeted therapies that may effectively treat these conditions. In this review, we seek to characterize some of the involved aberrations and associated targeted therapies for several germline malignancies, including neurofibromatosis types 1 and 2, and Gorlin syndrome. Though patients with hereditary cancer syndromes may be too rare to effectively include in large clinical trials, by understanding the pathophysiology of these diseases, clinicians can attain insights into the use of targeted therapies in their own practice when treating affected individuals.
authors.affiliation: Department of Medicine Division of Hematology/Oncology, University of Cincinnati, Cincinnati, OH 45267, USA.
authors.forename: Rishi
authors.initials: R
authors.lastname: Agarwal
authors.affiliation: UT Health, University of Texas - Houston, Houston, TX 77030, USA.
authors.forename: Sarah
authors.initials: S
authors.lastname: Liebe
authors.affiliation: CollabRx Inc., San Francisco, CA 94104, USA.
authors.forename: Michelle L
authors.initials: ML
authors.lastname: Turski
authors.affiliation: CollabRx Inc., San Francisco, CA 94104, USA.
authors.forename: Smruti J
authors.initials: SJ
authors.lastname: Vidwans
authors.affiliation: Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Filip
authors.initials: F
authors.lastname: Janku
authors.affiliation: Huntsman Cancer Institute, University of Utah, Salt Lake City, UT 84112, USA.
authors.forename: Ignacio
authors.initials: I
authors.lastname: Garrido-Laguna
authors.affiliation: Banner MD Anderson Cancer Center, Gilbert, AZ 85234, USA.
authors.forename: Javier
authors.initials: J
authors.lastname: Munoz
authors.affiliation: Center for Personalized Cancer Therapy, Moores Cancer Center, University of California, San Diego, CA 92093, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Schwab
authors.affiliation: Vall d'Hebron Institut d'Oncologia and Universitat Autonoma of Barcelona, Barcelona, 08035, Spain.
authors.forename: Jordi
authors.initials: J
authors.lastname: Rodon
authors.affiliation: Center for Personalized Cancer Therapy, Moores Cancer Center, University of California, San Diego, CA 92093, USA.
authors.forename: Razelle
authors.initials: R
authors.lastname: Kurzrock
authors.affiliation: Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Vivek
authors.initials: V
authors.lastname: Subbiah
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D001478:Basal Cell Nevus Syndrome / Q000188:drug therapy* / Q000235:genetics; D006801:Humans; D009456:Neurofibromatosis 1 / Q000188:drug therapy* / Q000235:genetics; D016518:Neurofibromatosis 2 / Q000188:drug therapy* / Q000235:genetics
chemical_list: D000970:Antineoplastic Agents