Emerging drugs for the treatment of gastrointestinal stromal tumors

References

• Demetri GD, Von Mehren M, Antonescu CR, et al. NCCN task force report: update on the management of patients with gastrointestinal stromal tumors. J Natl Compr Canc Netw. 2010 Apr;8(Suppl 2):S1-41;quiz S42-4.
   Google Scholar
• Ducimetiére F, Lurkin A, Ranchére-Vince D, et al. Incidence of sarcoma histotypes and molecular subtypes in a prospective epidemiological study with central pathology review and molecular testing. PLoS ONE. 2011;6(8):e20294.
   PubMed Web of Science ®Google Scholar
• Goettsch WG, Bos SD, Breekveldt-Postma N, et al. Incidence of gastrointestinal stromal tumours is underestimated: results of a nation-wide study. Eur J Cancer. 2005 Dec 1; 41(18):2868–2872.
   PubMed Web of Science ®Google Scholar
• Fletcher CDM, Berman JJ, Corless C, et al. Diagnosis of gastrointestinal stromal tumors: a consensus approach. Hum Pathol. 2002 May 1; 33(5):459–465.
   PubMed Web of Science ®Google Scholar
• Sommer G, Agosti V, Ehlers I, et al. Gastrointestinal stromal tumors in a mouse model by targeted mutation of the Kit receptor tyrosine kinase. Proc Natl Acad Sci U S A. 2003 May 27; 100(11):6706–6711.
   PubMed Web of Science ®Google Scholar
• Rubin BP, Antonescu CR, Scott-Browne JP, et al. A knock-in mouse model of gastrointestinal stromal tumor harboring kit K641E. Cancer Res. 2005 Aug 1; 65(15):6631–6639.
   PubMed Web of Science ®Google Scholar
• Hirota HS. Gain-of-function mutations of c-kit in human gastrointestinal stromal tumors. Science. 1998;279(5350):577–580.
   PubMed Web of Science ®Google Scholar
• Heinrich MC, Corless CL, Duensing A, et al. PDGFRA activating mutations in gastrointestinal stromal tumors. Science. 2003;299(5607):708–710.
   PubMed Web of Science ®Google Scholar
• Hanks SK, Quinn AM, Hunter T. The protein kinase family: conserved features and deduced phylogeny of the catalytic domains. Science. 1988 Jul 1; 241(4861):42–52.
   PubMed Web of Science ®Google Scholar
• Mol CD, Dougan DR, Schneider TR, et al. Structural basis for the autoinhibition and STI-571 inhibition of c-Kit tyrosine kinase. J Biol Chem. 2004;279(30):31655–31663.
   PubMed Web of Science ®Google Scholar
• Corless CL, McGreevey L, Town A, et al. KIT gene deletions at the intron 10-exon 11 boundary in GI stromal tumors. J Mol Diagn. 2004;6(4):366–370.
   PubMed Web of Science ®Google Scholar
• Yuzawa S, Opatowsky Y, Zhang Z, et al. Structural basis for activation of the receptor tyrosine kinase KIT by stem cell factor. Cell. 2007;130(2):323–334.
   PubMed Web of Science ®Google Scholar
• Lasota J, Corless CL, Heinrich MC, et al. Clinicopathologic profile of gastrointestinal stromal tumors (GISTs) with primary KIT exon 13 or exon 17 mutations: a multicenter study on 54 cases. Mod Pathol. 2008;21(4):476–484.
   PubMed Web of Science ®Google Scholar
• Corless CL, Schroeder A, Griffith D, et al. PDGFRA mutations in gastrointestinal stromal tumors: frequency, spectrum and in vitro sensitivity to imatinib. J Clin Oncol. 2005;23(23):5357–5364.
   PubMed Web of Science ®Google Scholar
• Hirota S, Ohashi A, Nishida T, et al. Gain-of-function mutations of platelet-derived growth factor receptor α gene in gastrointestinal stromal tumors. Gastroenterology. 2003 Sep 1; 125(3):660–667.
   PubMed Web of Science ®Google Scholar
• Rubin BP, Singer S, Tsao C, et al. KIT activation is a ubiquitous feature of gastrointestinal stromal tumors. Cancer Res. 2001;61(22):8118–8121.
   PubMed Web of Science ®Google Scholar
• Duensing A, Medeiros F, McConarty B, et al. Mechanisms of oncogenic KIT signal transduction in primary gastrointestinal stromal tumors (GISTs). Oncogene. 2004;23(22):3999–4006.
   PubMed Web of Science ®Google Scholar
• Zhu MJ, Ou WB, Fletcher CDM, et al. KIT oncoprotein interactions in gastrointestinal stromal tumors: therapeutic relevance. Oncogene. 2007;26(44):6386–6395.
   PubMed Web of Science ®Google Scholar
• Bosbach B, Rossi F, Yozgat Y, et al. Direct engagement of the PI3K pathway by mutant KIT dominates oncogenic signaling in gastrointestinal stromal tumor. Proceedings of the National Academy of Sciences. 2017;201711449.
   Google Scholar
• Chi P, Chen Y, Zhang L, et al. ETV1 is a lineage survival factor that cooperates with KIT in gastrointestinal stromal tumours. Nature. 2010;467(7317):849–853.
   PubMed Web of Science ®Google Scholar
• Li F, Hung H, Li X, et al. FGFR-mediated reactivation of MAPK signaling attenuates antitumor effects of imatinib in gastrointestinal stromal tumors. Cancer Discov. 2015;5(4):438–451.
   PubMed Web of Science ®Google Scholar
• Serrano C, Wang Y, Mariño-Enríquez A, et al. KRAS and KIT gatekeeper mutations confer polyclonal primary imatinib resistance in GI stromal tumors: relevance of concomitant phosphatidylinositol 3-kinase/AKT dysregulation. J Clin Oncol. 2015 Aug 1; 33(22):e93–6.
   PubMed Web of Science ®Google Scholar
• Brems H, Beert E, De RT, et al. Mechanisms in the pathogenesis of malignant tumours in neurofi bromatosis type 1. Lancet Oncol. 2009;10(5):508–515.
   PubMed Web of Science ®Google Scholar
• Bauer S, Duensing A, Demetri GD, et al. KIT oncogenic signaling mechanisms in imatinib-resistant gastrointestinal stromal tumor: PI3-kinase/AKT is a crucial survival pathway. Oncogene. 2007 Nov 29; 26(54):7560–7568.
   PubMed Web of Science ®Google Scholar
• Demetri GD, Von Mehren M, Blanke CD, et al. Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors. N Engl J Med. 2002 Aug 15; 347(7):472–480.
   PubMed Web of Science ®Google Scholar
• Liegl B, Kepten I, Le C, et al. Heterogeneity of kinase inhibitor resistance mechanisms in GIST. J Pathol. 2008; 216(1): 64–74.
   PubMed Web of Science ®Google Scholar
• Serrano C, George S, Valverde C, et al. Novel insights into the treatment of imatinib-resistant gastrointestinal stromal tumors. Target Oncol. 2017;12(3):277–288.
   PubMed Web of Science ®Google Scholar
• Serrano C, Fletcher JA. Overcoming heterogenity in imatinib-resistant gastrointestinal stromal tumor. Oncotarget. 2019;10(59):6286–6287.
   PubMedGoogle Scholar
• Demetri GD, Van Oosterom AT, Garrett CR, et al. Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet. 2006 Oct 14; 368(9544):1329–1338.
   PubMed Web of Science ®Google Scholar
• Demetri GD, Reichardt P, Kang Y-K, et al. Efficacy and safety of regorafenib for advanced gastrointestinal stromal tumours after failure of imatinib and sunitinib (GRID): an international, multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2013 Jan 26; 381(9863):295–302.
   PubMed Web of Science ®Google Scholar
• Corless CL, Barnett CM, Heinrich MC. Gastrointestinal stromal tumours: origin and molecular oncology. Nat Rev Cancer. 2011;11(12):865–878.
   PubMed Web of Science ®Google Scholar
• Serrano C, Mariño-Enríquez A, Tao DL, et al. Complementary activity of tyrosine kinase inhibitors against secondary kit mutations in imatinib-resistant gastrointestinal stromal tumours. Br J Cancer. 2019; 120(6): 612–620.
   PubMed Web of Science ®Google Scholar
• Hemming ML, Heinrich MC, Bauer S, et al. Translational insights into gastrointestinal stromal tumor and current clinical advances. Ann Oncol. 2018 01; 29(10):2037–2045.
   PubMed Web of Science ®Google Scholar
• Call JW, Wang Y, Montoya D, et al. Survival in advanced GIST has improved over time and correlates with increased access to post-imatinib tyrosine kinase inhibitors: results from life raft group registry. Clin Sarcoma Res. 2019 Dec; 9(1):4.
   PubMedGoogle Scholar
• Blanke CD, Rankin C, Demetri GD, et al. Phase III randomized, intergroup trial assessing imatinib mesylate at two dose levels in patients with unresectable or metastatic gastrointestinal stromal tumors expressing the kit receptor tyrosine kinase: S0033. J Clin Oncol. 2008;26(4):626–632.
   PubMed Web of Science ®Google Scholar
• Verweij J, Casali PG, Zalcberg J, et al. Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: randomised trial. Lancet. 2004 Oct 25; 364(9440):1127–1134.
   PubMed Web of Science ®Google Scholar
• Blanke CD, Demetri GD, Von Mehren M, et al. Long-term results from a randomized phase II trial of standard- versus higher-dose imatinib mesylate for patients with unresectable or metastatic gastrointestinal stromal tumors expressing KIT. J Clin Oncol. 2008 Feb 1; 26(4):620–625.
   PubMed Web of Science ®Google Scholar
• Casali PG, Zalcberg J, Le Cesne A, et al. Ten-year progression-free and overall survival in patients with unresectable or metastatic gi stromal tumors: long-term analysis of the European organisation for research and treatment of cancer, Italian sarcoma group, and Australasian gastrointestinal trials group intergroup phase III randomized trial on imatinib at two dose levels. JCO. 2017 May 20; 35(15):1713–1720.
   PubMed Web of Science ®Google Scholar
• Serrano C, García‐del‐muro X, Valverde C, et al. Clinicopathological and molecular characterization of metastatic gastrointestinal stromal tumors with prolonged benefit to frontline imatinib. Oncologist. 2019 May;24(5):680–687.
   PubMed Web of Science ®Google Scholar
• Joensuu H, Eriksson M, Sundby Hall K, et al. Survival outcomes associated with 3 years vs 1 year of adjuvant imatinib for patients with high-risk gastrointestinal stromal tumors: an analysis of a randomized clinical trial after 10-year follow-up. JAMA Oncol. 2020 Aug 1; 6(8):1241–1246.
   PubMed Web of Science ®Google Scholar
• George S, Blay JY, Casali PG, et al. Clinical evaluation of continuous daily dosing of sunitinib malate in patients with advanced gastrointestinal stromal tumour after imatinib failure. Eur J Cancer. 2009 Jul;45(11):1959–1968.
   PubMed Web of Science ®Google Scholar
• Kang Y-K, Yoo C, Ryoo B-Y, et al. Phase II study of dovitinib in patients with metastatic and/or unresectable gastrointestinal stromal tumours after failure of imatinib and sunitinib. Br J Cancer. 2013 Oct;109(9):2309–2315.
   PubMed Web of Science ®Google Scholar
• Joensuu H, Blay J-Y, Comandone A, et al. Dovitinib in patients with gastrointestinal stromal tumour refractory and/or intolerant to imatinib. Br J Cancer. 2017 Oct;117(9):1278–1285.
   PubMed Web of Science ®Google Scholar
• Adenis A, Blay J-Y, Bui-Nguyen B, et al. Masitinib in advanced gastrointestinal stromal tumor (GIST) after failure of imatinib: a randomized controlled open-label trial. Ann Oncol. 2014 Sep;25(9):1762–1769.
   PubMed Web of Science ®Google Scholar
• Park SH, Ryu MH, Ryoo BY, et al. Sorafenib in patients with metastatic gastrointestinal stromal tumors who failed two or more prior tyrosine kinase inhibitors: a phase II study of Korean gastrointestinal stromal tumors study group. Invest New Drugs. 2012 Dec;30(6):2377–2383.
   PubMed Web of Science ®Google Scholar
• Montemurro M, Schöffski P, Reichardt P, et al. Nilotinib in the treatment of advanced gastrointestinal stromal tumours resistant to both imatinib and sunitinib. Eur J Cancer. 2009 Sep;45(13):2293–2297.
   PubMed Web of Science ®Google Scholar
• Dewaele B, Wasag B, Cools J, et al. Activity of dasatinib, a dual SRC/ABL kinase inhibitor, and IPI-504, a heat shock protein 90 inhibitor, against gastrointestinal stromal tumor-associated PDGFRAD842V mutation. Clin Cancer Res. 2008 Sep 15; 14(18):5749–5758.
   PubMed Web of Science ®Google Scholar
• Ganjoo KN, Villalobos VM, Kamaya A, et al. A multicenter phase II study of pazopanib in patients with advanced gastrointestinal stromal tumors (GIST) following failure of at least imatinib and sunitinib. Ann Oncol. 2014 Jan;25(1):236–240.
   PubMed Web of Science ®Google Scholar
• Mir O, Cropet C, Toulmonde M, et al. Pazopanib plus best supportive care versus best supportive care alone in advanced gastrointestinal stromal tumours resistant to imatinib and sunitinib (PAZOGIST): a randomised, multicentre, open-label phase 2 trial. Lancet Oncol. 2016 May;17(5):632–641.
   PubMed Web of Science ®Google Scholar
• Demetri GD, Antonescu CR, Bjerkehagen B, et al. Diagnosis and management of tropomyosin receptor kinase (TRK) fusion sarcomas: expert recommendations from the World Sarcoma Network. Ann Oncol. 2020 Nov;31(11):1506–1517.
   PubMed Web of Science ®Google Scholar
• Smith BD, Kaufman MD, Lu W-P, et al. Ripretinib (DCC-2618) is a switch control kinase inhibitor of a broad spectrum of oncogenic and drug-resistant KIT and PDGFRA variants. Cancer Cell. 2019 May;35(5):738–751.e9.
   PubMed Web of Science ®Google Scholar
• Janku F, Abdul Razak AR, Chi P, et al. Switch control inhibition of KIT and PDGFRA in patients with advanced gastrointestinal stromal tumor: a phase I study of ripretinib. JCO. 2020 Aug 17; 38(28):3294–3303.
   PubMed Web of Science ®Google Scholar
• Blay J-Y, Serrano C, Heinrich MC, et al. Ripretinib in patients with advanced gastrointestinal stromal tumours (INVICTUS): a double-blind, randomised, placebo-controlled, phase 3 trial. Lancet Oncol. 2020 Jul;21(7):923–934.
   PubMed Web of Science ®Google Scholar
• Evans EK, Gardino AK, Kim JL, et al. A precision therapy against cancers driven by KIT/PDGFRA mutations. Sci Transl Med. 2017 Nov 1; 9(414):414.
   Web of Science ®Google Scholar
• Serrano C, George S. Gastrointestinal stromal tumor: challenges and opportunities for a New Decade. Clin Cancer Res. 2020 Oct 1; 26(19):5078–5085.
   PubMed Web of Science ®Google Scholar
• Gebreyohannes YK, Wozniak A, Zhai M-E, et al. Robust activity of avapritinib, potent and highly selective inhibitor of mutated KIT, in patient-derived xenograft models of gastrointestinal stromal tumors. Clin Cancer Res. 2019 15; 25(2): 609–618.
   PubMed Web of Science ®Google Scholar
• Heinrich MC, Jones RL, Von Mehren M, et al. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020; 21(7): 935–946.
   PubMed Web of Science ®Google Scholar
• Serrano C, Leal A, Kuang Y, et al. Phase I study of rapid alternation of sunitinib and regorafenib for the treatment of tyrosine-kinase inhibitor refractory gastrointestinal stromal tumors. Clin Cancer Res. 2019 Aug; 25(24):7287–7293. clincanres.2150.2019
   Web of Science ®Google Scholar
• Nemunaitis J, Bauer S, Blay J-Y, et al. Intrigue: phase III study of ripretinib versus sunitinib in advanced gastrointestinal stromal tumor after imatinib. Future Oncol. 2020 Jan;16(1):4251–4264.
   PubMed Web of Science ®Google Scholar
• Heinrich MC, Maki RG, Corless CL, et al. Primary and secondary kinase genotypes correlate with the biological and clinical activity of sunitinib in imatinib-resistant gastrointestinal stromal tumor. J clin oncol. 2008 Nov;26(33):5352–5359.
   PubMed Web of Science ®Google Scholar
• Serrano C, Vivancos A, López-Pousa A, et al. Clinical value of next generation sequencing of plasma cell-free DNA in gastrointestinal stromal tumors. BMC Cancer. 2020 Feb 5; 20(1):99.
   PubMed Web of Science ®Google Scholar
• Mühlenberg T, Ketzer J, Heinrich MC, et al. KIT-dependent and KIT-independent genomic heterogeneity of resistance in gastrointestinal stromal tumors - TORC1/2 inhibition as salvage strategy. Mol Cancer Ther. 2019;18(11):1985–1996.
   PubMed Web of Science ®Google Scholar
• Grunewald S, Klug LR, Mühlenberg T, et al. Resistance to avapritinib in PDGFRA-driven GIST is caused by secondary mutations in the PDGFRA kinase domain.. Cancer Discov. 2020 Sep 24; Online ahead of print. 10.1158/2159-8290.CD-20-0487
   PubMed Web of Science ®Google Scholar
• Casali PG, Abecassis N, Aro HT, et al. Gastrointestinal stromal tumours: ESMO-EURACAN clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2018 01; 29(Suppl 4): iv267.
   PubMed Web of Science ®Google Scholar