Piro Lito: Publications

Lito P. KRAS oncoprotein signaling in cancer. N Engl J Med. 2025 Jan 16;392(3):296-298. doi: 10.1056/NEJMcibr2408099. PMID: 39813652

Cuevas-Navarro A, Pourfarjam Y, Hu F, Rodriguez DJ, Vides A, Sang B, Golgur Y, de Stanchina E, and Lito P. Pharmacologic restoration of GTP hydrolysis by mutant RAS. Nature. 2025 Jan;637(8044):224-229. doi: 10.1038/s41586-024-08283-2. Epub 2024 Oct 30. PMID: 39476862

Carcamo S, Cruz-Rodriguez N, Sirenko M, Maiti A, Olszewska M, Ulukaya G, Wang T, Sango J, Olivier E, Jaud M, Chaligne R, Mansour H, Demircioglu D, Landau D, Papaemmanuil E, DiNardo C, Hasson D, Konopleva M, Lito P., Tomalin L, Chung S, Kroger B, Eisfeld A, Wagenblast E, Nadorp B, Silverman L and Papapetrou E. RAS-mutant AML LSCs originate from GMPs and drive clinical resistance to BH3 mimetics. Nature. 2024 Oct 30. doi: 10.1038/s41586-024-08137-x. PMID: 39478230

Holderfield M, Lee B, Jiang J, Tomlinson A, Seamon K, Mira A, Patrucco E, Goodhart G, Dilly J, Gindin Y, Dinglasan N, Wang Y, Lai LP, Cai S, Jiang L, Yang Y, Nasholm N, Evans J, Montazer N, Lai O, Shi J, Ahler E, Chang S, Salvador A, Marquez A, Cregg J, Liu Y, Milin A, Chen A, Ziv TB, Parsons D, Knox J, Roth J, Rees M, Ronan M, Cuevas-Navarro A, Hu F, Lito P., Santamaria D, Aguirre A, Waters A, Der C, Ambrogio C, Wang Z, Gill A, Koltun E, Smith J, Wildes D and Singh M. Concurrent inhibition of oncogenic and wild-type RAS-GTP for cancer therapy. Nature. 2024 May;629(8013):919-926. doi: 10.1038/s41586-024-07205-6. PMID: 38589574

Kim D, Herdeis L, Rudolph D, Zhao Y, Böttcher J, Vides A, Ayala-Santos C, Pourfarjam Y, Mantoulidis A, Wolkerstorfer B, Kroatisch KG, Sang B, Li C, McConnell DB, Kraut N and Lito P. Pan KRAS inhibitor selectively inactivates oncogenic signaling and tumor growth. Nature. 2023 Jul;619(7968):160-166 31. doi: 10.1038/s41586-023-06123-3. PMID: 37258666

Schulze C, Seamon KJ, Zhao Y, Yang YC, Cregg J, Kim DS, Tomlinson A, Choy TS, Wang Z, Sang B, Pourfarjam Y, Lucas J, Cuevas Navarro A, Ayala Santos C, Vides A, Marquez A, Zhong M, Vemulapalli V, Weller C, Gould A, Whalen D, Salvador A, Saldajeno-Concar M, Dinglasan N, Chen A, Evans J, Knox JE, Koltun ES, Li C, Singh M, Nichols R, Wildes D, Gill AL, Smith JAM and Lito P. Chemical remodeling of a cellular chaperone to target the active state of mutant KRAS. Science. 2023 Aug 18;381(6659):794-799. doi: 10.1126/science.adg9652. Epub 2023 Aug 17. PMID: 37590355

Dy GK, Govindan R, Velcheti V, Falchook GS, Italiano A, Wolf J, Sacher AG, Takahashi T, Ramalingam SS, Dooms C, Kim DW, Addeo A, Desai J, Schuler M, Tomasini P, Hong DS, Lito P., Tran Q, Jones S, Anderson A, Hindoyan A, Snyder W, Skoulidis F and Li BT. Long-term outcomes and molecular correlates of sotorasib efficacy in patients with pretreated KRAS G12C mutated non–small-cell lung cancer: 2-year analysis of CodeBreaK 100. J Clin Oncol. 2023 Apr 25:JCO2202524.

Li C, Vides A, Kim D, Xue J, Zhao Y and Lito P. The G-protein signaling regulator RGS3 enhances the GTPase activity of KRAS. Science. 2021 Oct 8;374(6564):197-201. PMID: 34618566

Zhao Y, Murciano-Goroff Y, Xue Y, Ang A, Lucas J, Da Cruz Paula A, Mai T, Sisk A, Arora K, Roy R, Kim D, Li C, Lim L, Li M, Bahr A, Loomis B, de Stanchina E, Reis-Filho J, Weigelt B, Berger M, Riely G, Arbour K, Lipford R, Li B and Lito P. Diverse genetic alterations associated with resistance to KRAS(G12C) inhibition. Nature. 2021 Nov 10;599(7886):679-683. doi: 10.1038/s41586-021-04065-2. PMID: 34759319

Murciano-Goroff YR, Pak T, Mondaca S, Flynn JR, Montecalvo J, Rekhtman N, Halpenny D, Plodkowski AJ, Wu SL, Kris MG, Paik PK, Riely GJ, Yu HA, Rudin CM, Hellmann MD, Land JD, Buie LW, Heller G, Lito P., Yaeger R, Drilon A, Liu D, Li BT, Offin M. Immune biomarkers and response to checkpoint inhibition of BRAF V600 and BRAF non-V600 altered lung cancers. Br J Cancer. 2021 Dec 28. doi: 10.1038/s41416-021-01679-1. PMID: 34963703

Arbour KC, Manchado E, Bott MJ, Ahn L, Tobi Y, Ni AA, Yu HA, Shannon A, Ladanyi M, Perron V, Ginsberg MS, Johnson A, Holodny A, Kris MG, Rudin CM, Lito P., Rosen N, Lowe S, Riely GJ. Phase 1 Clinical Trial of Trametinib and Ponatinib in Patients With NSCLC Harboring KRAS Mutations. J Thorac Oncol Clin Res Rep. 2021 Nov 23;3(1):100256. PMID: 34984405

Caldas C, Rescigno M, Turajlic S, Madabhushi A, Zhang Z, Lito P., Brown CE, Pantel K, Haanen J, Duma N. With one eye on the future. Cancer Cell. 2022 Mar 14;40(3):221-225. doi: 10.1016/j.ccell.2022.02.019. PMID: 35290781

Arbour KC and Lito P. Expanding the arsenal of clinically active KRAS G12C inhibitors. J Clin Oncol. 2022 Aug 10;40(23):2609-2611. doi: 10.1200/JCO.22.00562. Epub 2022 Jun 28. PMID: 35763705

Murciano-Goroff YR and Lito P. The KRYSTAL-1 study of adagrasib - a new trial for KRAS G12C-mutated non-small-cell lung cancer. Nat Rev Clin Oncol. 2022 Aug 23. doi: 10.1038/s41571-022-00676-4. PMID: 35999291

Jee J, Lebow ES, Yeh R, Das JP, Namakydoust A, Paik PK, Chaft JE, Jayakumaran G, Rose Brannon A, Benayed R, Zehir A, Donoghue M, Schultz N, Chakravarty D, Kundra R, Madupuri R, Murciano-Goroff YR, Tu HY, Xu CR, Martinez A, Wilhelm C, Galle J, Daly B, Yu HA, Offin M, Hellmann MD, Lito P., Arbour KC, Zauderer MG, Kris MG, Ng KK, Eng J, Preeshagul I, Victoria Lai W, Fiore JJ, Iqbal A, Molena D, Rocco G, Park BJ, Lim LP, Li M, Tong-Li C, De Silva M, Chan DL, Diakos CI, Itchins M, Clarke S, Pavlakis N, Lee A, Rekhtman N, Chang J, Travis WD, Riely GJ, Solit DB, Gonen M, Rusch VW, Rimner A, Gomez D, Drilon A, Scher HI, Shah SP, Berger MF, Arcila ME, Ladanyi M, Levine RL, Shen R, Razavi P, Reis-Filho JS, Jones DR, Rudin CM, Isbell JM, Li BT. Overall survival with circulating tumor DNA-guided therapy in advanced non-small-cell lung cancer. Nat Med. 2022 Nov 10. doi: 10.1038/s41591-022-02047-z. Online ahead of print. PMID: 36357680

Kim D, Xue J and Lito P. Targeting KRAS G12C: from inhibitory mechanism to modulation of antitumor effect in patients. Cell. 2020 Oct 14:S0092-8674(20)31241-1. PMID: 33065029

Hong DS*, Fakih MG, Strickler JH, Desai J, Durm JA, Shapiro GI, Falchook GS, Price TJ, Denlinger CS, Bang YJ, Dy GK, Krauss JC, Kuboki Y, Kuo JC, Coveler AL, Park K, Kim TW, Barlesi F, Munster PN, Burns TF, Meric-Bernstam F, Henary H, Ngang J, Ngarmchamnanrith G, Kim J, Houk BE, Canon J, Lipford JR, Lito P.*, Govindan R*, Li BT*. KRAS G12C inhibition in advanced solid tumors. N Engl J Med. 2020 Sep 24;383(13):1207-1217. PMID: 32955176

Xue J and Lito P. Quest for clinically effective RAF dimer inhibitors. J Clin Oncol. 2020 May 20:JCO2000401. PMID: 32432945

Xue JY, Zhao Y, Aronowitz J, Mai TT, Vides A, Qeriqi B, Kim D, Li C, de Stanchina E, Mazutis L, Risso D, Lito P. Rapid non-uniform adaptation to conformation-specific KRAS(G12C) inhibition. Nature. 2020;577(7790):421-5. doi: 10.1038/s41586-019-1884-x. PMID: 31915379

Hallin J, Engstrom LD, Hargis L, Calinisan A, Aranda R, Briere DM, Sudhakar N, Bowcut V, Baer BR, Ballard JA, Burkard MR, Fell JB, Fischer JP, Vigers GP, Xue Y, Gatto S, Fernandez-Banet J, Pavlicek A, Velastagui K, Chao RC, Barton J, Pierobon M, Baldelli E, Patricoin EF, 3rd, Cassidy DP, Marx MA, Rybkin, II, Johnson ML, Ou SI, Lito P., Papadopoulos KP, Janne PA, Olson P, Christensen JG. The KRAS(G12C) Inhibitor MRTX849 Provides Insight toward Therapeutic Susceptibility of KRAS-Mutant Cancers in Mouse Models and Patients. Cancer Discov. 2020;10(1):54-71. doi: 10.1158/2159-8290.CD-19-1167. PMID: 31658955

Wang VE, Xue JY, Frederick DT, Cao Y, Lin E, Wilson C, Urisman A, Carbone DP, Flaherty KT, Bernards R, Lito P., Settleman J, McCormick F. Adaptive Resistance to Dual BRAF/MEK Inhibition in BRAF-Driven Tumors through Autocrine FGFR Pathway Activation. Clin Cancer Res. 2019;25(23):7202-17. doi: 10.1158/1078-0432.CCR-18-2779. PMID: 31515463

Mai TT, Lito P. A treatment strategy for KRAS-driven tumors. Nat Med. 2018;24(7):902-4. doi: 10.1038/s41591-018-0111-x. PMID: 29988139

Arbour KC, Jordan E, Kim HR, Dienstag J, Yu HA, Sanchez-Vega F, Lito P., Berger M, Solit DB, Hellmann M, Kris MG, Rudin CM, Ni A, Arcila M, Ladanyi M, Riely GJ. Effects of Co-occurring Genomic Alterations on Outcomes in Patients with KRAS-Mutant Non-Small Cell Lung Cancer. Clin Cancer Res. 2018;24(2):334-40. doi: 10.1158/1078-0432.CCR-17-1841. PMID: 29089357

Xue Y, Martelotto L, Baslan T, Vides A, Solomon M, Mai TT, Chaudhary N, Riely GJ, Li BT, Scott K, Cechhi F, Stierner U, Chadalavada K, de Stanchina E, Schwartz S, Hembrough T, Nanjangud G, Berger MF, Nilsson J, Lowe SW, Reis-Filho JS, Rosen N, Lito P. An approach to suppress the evolution of resistance in BRAF(V600E)-mutant cancer. Nat Med. 2017;23(8):929-37. doi: 10.1038/nm.4369. PMID: 28714990

Xue Y, Lito P. Predicting MEK Inhibitor Response in Lung Cancer: A Proper Signature Is Required. Clin Cancer Res. 2017;23(6):1365-7. doi: 10.1158/1078-0432.CCR-16-2576. PMID: 27923842

Naidoo J, Santos-Zabala ML, Iyriboz T, Woo KM, Sima CS, Fiore JJ, Kris MG, Riely GJ, Lito P., Iqbal A, Veach S, Smith-Marrone S, Sarkaria IS, Krug LM, Rudin CM, Travis WD, Rekhtman N, Pietanza MC. Large Cell Neuroendocrine Carcinoma of the Lung: Clinico-Pathologic Features, Treatment, and Outcomes. Clin Lung Cancer. 2016. doi: 10.1016/j.cllc.2016.01.003. PMID: 26898325

Lito P., Solomon M, Li LS, Hansen R, Rosen N. Allele-specific inhibitors inactivate mutant KRAS G12C by a trapping mechanism. Science. 2016;351(6273):604-8. doi: 10.1126/science.aad6204. PMID: 26841430

Lito P., Saborowski A, Yue J, Solomon M, Joseph E, Gadal S, Saborowski M, Kastenhuber E, Fellmann C, Ohara K, Morikami K, Miura T, Lukacs C, Ishii N, Lowe S, Rosen N. Disruption of CRAF-mediated MEK activation is required for effective MEK inhibition in KRAS mutant tumors. Cancer Cell. 2014;25(5):697-710. doi: 10.1016/j.ccr.2014.03.011. PMID: 24746704

Chung SS, Kim E, Park JH, Chung YR, Lito P., Teruya-Feldstein J, Hu W, Beguelin W, Monette S, Duy C, Rampal R, Telis L, Patel M, Kim MK, Huberman K, Bouvier N, Berger MF, Melnick AM, Rosen N, Tallman MS, Park CY, Abdel-Wahab O. Hematopoietic stem cell origin of BRAFV600E mutations in hairy cell leukemia. Sci Transl Med. 2014;6(238):238ra71. doi: 10.1126/scitranslmed.3008004. PMID: 24871132

Lito P., Rosen N, Solit DB. Tumor adaptation and resistance to RAF inhibitors. Nat Med. 2013;19(11):1401-9. doi: 10.1038/nm.3392. PMID: 24202393

Lito P., Pratilas CA, Joseph EW, Tadi M, Halilovic E, Zubrowski M, Huang A, Wong WL, Callahan MK, Merghoub T, Wolchok JD, de Stanchina E, Chandarlapaty S, Poulikakos PI, Fagin JA, Rosen N. Relief of profound feedback inhibition of mitogenic signaling by RAF inhibitors attenuates their activity in BRAFV600E melanomas. Cancer Cell. 2012;22(5):668-82. doi: 10.1016/j.ccr.2012.10.009. PMID: 23153539

Lito P., Pantanowitz L, Marotti J, Aboulafia DM, Campbell V, Bower M, Dezube BJ. Gastroenteropancreatic neuroendocrine tumors in patients with HIV infection: a trans-Atlantic series. Am J Med Sci. 2009;337(1):1-4. doi: 10.1097/MAJ.0b013e31817d1cb7. PMID: 19155751

Lito P., Mets BD, Appledorn DM, Maher VM, McCormick JJ. Sprouty 2 regulates DNA damage-induced apoptosis in Ras-transformed human fibroblasts. J Biol Chem. 2009;284(2):848-54. doi: 10.1074/jbc.M808045200. PMID: 19008219

Lito P., Mets BD, Kleff S, O’Reilly S, Maher VM, McCormick JJ. Evidence that sprouty 2 is necessary for sarcoma formation by H-Ras oncogene-transformed human fibroblasts. J Biol Chem. 2008;283(4):2002-9. doi: 10.1074/jbc.M709046200. PMID: 18048363