caa a22 4500 445330899 CHVBK 20180317142737.0 cr unu---uuuuu 170323e20110901xx s 000 0 eng 10.1007/s10689-011-9471-9 doi (NATIONALLICENCE)springer-10.1007/s10689-011-9471-9 A rationale for mTOR inhibitors as chemoprevention agents in Peutz-Jeghers syndrome [Elektronische Daten] [Scott Kuwada, Randall Burt] Peutz-Jeghers patients frequently develop clinically significant complications, namely hemorrhage and bowel obstruction, from small intestinal hamartomatous polyps that frequently require surgery. In addition, many PJS patients develop epithelial malignancies in a variety of organs. The vast majority of PJS is due to germline alterations in the STK11 gene that encodes a protein that modulates PI3-kinase signaling, a key regulator of cell survival and growth. One of the major downstream mediators of PI3-kinase signaling is mTOR, the mammalian target of rapamycin. Several drugs that inhibit the PI3-kinase signal transduction pathway are in development and one, RAD001 (everolimus), an mTOR inhibitor, was recently approved for the treatment of renal cell carcinoma. Effective chemoprevention of intestinal polyps would be a first step in simplifying and improving the management of PJS patients. We present here, the rationale for the first human PJS chemoprevention trial using an mTOR inhibitor. Springer Science+Business Media B.V., 2011 Peutz-Jeghers syndrome nationallicence Hamartomatous polyps nationallicence Chemoprevention nationallicence mTOR nationallicence Clinical trial nationallicence Kuwada Scott University of Hawaii, John A. Burns School of Medicine, Honolulu, HI, USA aut Burt Randall University of Utah School of Medicine, Salt Lake City, UT, USA aut Familial Cancer Springer Netherlands 10/3(2011-09-01), 469-472 1389-9600 10:3<469 2011 10 10689 https://doi.org/10.1007/s10689-011-9471-9 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/s10689-011-9471-9 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Kuwada Scott University of Hawaii, John A. Burns School of Medicine, Honolulu, HI, USA aut NATIONALLICENCE 700 1- Burt Randall University of Utah School of Medicine, Salt Lake City, UT, USA aut NATIONALLICENCE 773 0- Familial Cancer Springer Netherlands 10/3(2011-09-01), 469-472 1389-9600 10:3<469 2011 10 10689 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer