Targeted therapy for genetic cancer syndromes: Fanconi anemia, medullary thyroid cancer, tuberous sclerosis, and RASopathies.
With the advent of genomics-based treatment in recent years, the use of targeted therapies in the treatment of various malignancies has increased exponentially. Though much data is available regarding the efficacy of targeted therapies for common malignancies, genetic cancer syndromes remain a somewhat unexplored topic with comparatively less published literature. This review seeks to characterize targeted therapy options for the following genetic cancer syndromes: Fanconi anemia, inherited medullary thyroid cancer, tuberous sclerosis, and RASopathies. By understanding the pathophysiology of these conditions as well as available molecularly targeted therapies, oncologists, in collaboration with geneticists and genetic counsellors, can begin to develop effective clinical management options and therapy regimens for the patients with these genetic syndromes that they may encounter in their practice.
Department of Medicine Division of Hematology/Oncology, University of Cincinnati, Cincinnati, OH 45267, USA.
Rishi
R
Agarwal
UT Health, University of Texas - Houston, Houston, TX 77030, USA.
Sarah
S
Liebe
CollabRx Inc., San Francisco, CA 94104, USA.
Michelle L
ML
Turski
CollabRx Inc., San Francisco, CA 94104, USA.
Smruti J
SJ
Vidwans
Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Filip
F
Janku
Huntsman Cancer Institute, University of Utah, Salt Lake City, UT 84112, USA.
Ignacio
I
Garrido-Laguna
Banner MD Anderson Cancer Center, Gilbert, AZ 85234, USA.
Javier
J
Munoz
Center for Personalized Cancer Therapy, Moores Cancer Center, University of California, San Diego, CA 92093, USA.
Richard
R
Schwab
Vall d'Hebron Institut d'Oncologia and Universitat Autonoma of Barcelona, Barcelona, 08035, Spain.
Jordi
J
Rodon
Center for Personalized Cancer Therapy, Moores Cancer Center, University of California, San Diego, CA 92093, USA.
Razelle
R
Kurzrock
Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Vivek
V
Subbiah
D018278:Carcinoma, Neuroendocrine; D005199:Fanconi Anemia / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D058990:Molecular Targeted Therapy; D013577:Syndrome; D013964:Thyroid Neoplasms / Q000235:genetics / Q000628:therapy*; D014402:Tuberous Sclerosis / Q000235:genetics / Q000628:therapy*; D018631:ras Proteins / Q000378:metabolism*
D018631:ras Proteins