Emerging treatments

COX-2

The enzyme COX-2 has been shown to be overexpressed in both the intestinal hamartomas and cancers associated with PJS.[12][13] Limited evidence has demonstrated a benefit from the use of celecoxib, a COX-2 inhibitor, in decreasing polyp burden in experimental animals and humans with PJS and appears to be a possible target for chemoprevention research.[54] To date, there is insufficient evidence to recommend pharmacologic agents as a treatment or for chemoprevention in PJS.[17][18]

mTOR inhibitors

LKB1, the serine-threonine kinase encoded by STK11, modulates PI3-kinase signaling, which is a key regulator of cell growth and survival. The mammalian target of rapamycin, mTOR, is an important downstream regulator of PI3-kinase signaling. Studies of Lkb1 ± mice have shown that treatment with rapamycin reduced the number of intestinal hamartomatous polyps.[55][56] Rapamycin and everolimus are being studied, but no conclusions can be drawn.[57][58]

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