Home > E. Pathology by systems > Digestive system > PDGFRA-mutated GIST


Monday 28 November 2011

The therapy for gastrointestinal stromal tumors (GISTs) has been revolutionized by tyrosin kinase inhibitors.

Clinicopathologic studies have been conducted to assess therapeutical responses in cases with KIT and platelet-derived growth factor receptor α (PDGFRA) gene mutations.

Cell culture data suggest that Akt/mammalian target of rapamycin (mTOR) kinase signaling may be important in GIST. (21326036)

The activation of the mTOR pathway is not a general hallmark of GIST with KIT mutations. However, mTOR signaling seems to be activated in PDGFRA mutants and in wild-type cases, which suggests that mTOR or upstream mTOR inhibitors may be therapeutically useful in primary resistant GISTs and confirms earlier data that mTOR is a crucial survival pathway in resistant GISTs. (21326036)


- The activated targets of mTOR signaling pathway are characteristic for PDGFRA mutant and wild-type rather than KIT mutant GISTs. Sápi Z, Füle T, Hajdu M, Matolcsy A, Moskovszky L, Márk A, Sebestyén A, Bodoky G. Diagn Mol Pathol. 2011 Mar;20(1):22-33. PMID: 21326036