Tissue-Agnostic Activity of BRAF plus MEK Inhibitor in BRAF V600-Mutant Tumors

被引:31
作者
Adashek, Jacob J. [1 ]
Menta, Arjun K. [2 ]
Reddy, Neha K. [3 ]
Desai, Aakash P. [4 ]
Roszik, Jason [5 ]
Subbiah, Vivek [5 ,6 ,7 ]
机构
[1] Univ S Florida, H Lee Moffitt Canc Ctr & Res Inst, Dept Internal Med, Tampa, FL 33620 USA
[2] Johns Hopkins Sch Med, Baltimore, MD USA
[3] Univ Texas Austin, Dept Internal Med, Sch Med, Austin, TX 78712 USA
[4] Mayo Clin, Div Med Oncol, Dept Med, Rochester, MN USA
[5] Univ Texas MD Anderson Canc Ctr, Dept Invest Canc Therapeut, Div Canc Med, Houston, TX 77030 USA
[6] Univ Texas MD Anderson Canc Ctr, Div Pediat, Houston, TX 77030 USA
[7] Univ Texas MD Anderson Canc Ctr, MD Anderson Canc Network, Houston, TX 77030 USA
关键词
ERDHEIM-CHESTER DISEASE; CELL LUNG-CANCER; ANAPLASTIC THYROID-CARCINOMA; OPEN-LABEL; SINGLE-ARM; B-RAF; DABRAFENIB; VEMURAFENIB; TRAMETINIB; PHASE-2;
D O I
10.1158/1535-7163.MCT-21-0950
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
BRAF plus MEK inhibitor combinations are currently FDAapproved for melanoma, non-small cell lung cancer, and anaplastic thyroid cancer. The lack of clinical benefit with BRAF inhibition in BRAF V600-mutated colorectal cancer has prevented its tissueagnostic drug development. We reviewed the AACR GENIE database for the prevalence of BRAF V600 mutations across tumor types. We reviewed the literature for case reports of clinical responses, outcomes in patients with BRAF V600 mutation-positive nonmelanoma malignancies who received BRAF inhibitor therapy, and data from published adult and pediatric trials. BRAF V600 mutations are prevalent across multiple nonmelanoma malig-nancies (>40 different tumor types), lead to oncogene addiction, and are clinically actionable in a broad range of adult and pediatric nonmelanoma rare malignancies. Continued tissue-agnostic drug development is warranted beyond the current BRAF plus MEK approved cancers.
引用
收藏
页码:871 / 878
页数:8
相关论文
共 61 条
[11]   Activated B-RAF is an Hsp90 client protein that is targeted by the anticancer drug 17-allylamino-17-demethoxygeldanamycin [J].
Dias, SD ;
Friedlos, F ;
Light, Y ;
Springer, C ;
Workman, P ;
Marais, R .
CANCER RESEARCH, 2005, 65 (23) :10686-10691
[12]   BRAF Inhibitor Dabrafenib in Patients with Metastatic BRAF-Mutant Thyroid Cancer [J].
Falchook, Gerald S. ;
Millward, Michael ;
Hong, David ;
Naing, Aung ;
Piha-Paul, Sarina ;
Waguespack, Steven G. ;
Cabanillas, Maria E. ;
Sherman, Steven I. ;
Ma, Bo ;
Curtis, Martin ;
Goodman, Vicki ;
Kurzrock, Razelle .
THYROID, 2015, 25 (01) :71-77
[13]   Salutary Response to Targeted Therapy in Anaplastic Thyroid Cancer [J].
Fazeli, Sasan ;
Paal, Edina ;
Maxwell, Jessica H. ;
Burman, Kenneth D. ;
Nylen, Eric S. ;
Khosla, Shikha G. .
JOURNAL OF INVESTIGATIVE MEDICINE HIGH IMPACT CASE REPORTS, 2019, 7
[14]   Improved Survival with MEK Inhibition in BRAF-Mutated Melanoma [J].
Flaherty, Keith T. ;
Robert, Caroline ;
Hersey, Peter ;
Nathan, Paul ;
Garbe, Claus ;
Milhem, Mohammed ;
Demidov, Lev V. ;
Hassel, Jessica C. ;
Rutkowski, Piotr ;
Mohr, Peter ;
Dummer, Reinhard ;
Trefzer, Uwe ;
Larkin, James M. G. ;
Utikal, Jochen ;
Dreno, Brigitte ;
Nyakas, Marta ;
Middleton, Mark R. ;
Becker, Juergen C. ;
Casey, Michelle ;
Sherman, Laurie J. ;
Wu, Frank S. ;
Ouellet, Daniele ;
Martin, Anne-Marie ;
Patel, Kiran ;
Schadendorf, Dirk .
NEW ENGLAND JOURNAL OF MEDICINE, 2012, 367 (02) :107-114
[15]   Efficacy and Safety of Dabrafenib in Pediatric Patients with BRAF V600 Mutation-Positive Relapsed or Refractory Low-Grade Glioma: Results from a Phase I/IIa Study [J].
Hargrave, Darren R. ;
Bouffet, Eric ;
Tabori, Uri ;
Broniscer, Alberto ;
Cohen, Kenneth J. ;
Hansford, Jordan R. ;
Geoerger, Birgit ;
Hingorani, Pooja ;
Dunkel, Ira J. ;
Russo, Mark W. ;
Tseng, Lillian ;
Dasgupta, Kohinoor ;
Gasal, Eduard ;
Whitlock, James A. ;
Kieran, Mark W. .
CLINICAL CANCER RESEARCH, 2019, 25 (24) :7303-7311
[16]   Reproducible and Sustained Efficacy of Targeted Therapy With Vemurafenib in Patients With BRAFV600E-Mutated Erdheim-Chester Disease [J].
Haroche, Julien ;
Cohen-Aubart, Fleur ;
Emile, Jean-Francois ;
Maksud, Philippe ;
Drier, Aurelie ;
Toledano, Dan ;
Barete, Stephane ;
Charlotte, Frederic ;
Cluzel, Philippe ;
Donadieu, Jean ;
Benameur, Neila ;
Grenier, Philippe A. ;
Besnard, Sophie ;
Ory, Jean-Paul ;
Lifermann, Francois ;
Idbaih, Ahmed ;
Granel, Brigitte ;
Graffin, Bruno ;
Hervier, Baptiste ;
Arnaud, Laurent ;
Amoura, Zahir .
JOURNAL OF CLINICAL ONCOLOGY, 2015, 33 (05) :411-U52
[17]  
Huang Laura C, 2018, Am J Ophthalmol Case Rep, V11, P158, DOI 10.1016/j.ajoc.2018.07.005
[18]   Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations [J].
Hyman, David M. ;
Puzanov, Igor ;
Subbiah, Vivek ;
Faris, Jason E. ;
Chau, Ian ;
Blay, Jean-Yves ;
Wolf, Juergen ;
Raje, Noopur S. ;
Diamond, Eli L. ;
Hollebecque, Antoine ;
Gervais, Radj ;
Elena Elez-Fernandez, Maria ;
Italiano, Antoine ;
Hofheinz, Ralf-Dieter ;
Hidalgo, Manuel ;
Chan, Emily ;
Schuler, Martin ;
Lasserre, Susan Frances ;
Makrutzki, Martina ;
Sirzen, Florin ;
Veronese, Maria Luisa ;
Tabernero, Josep ;
Baselga, Jose .
NEW ENGLAND JOURNAL OF MEDICINE, 2015, 373 (08) :726-736
[19]   BRAF Inhibition in BRAFV600-Mutant Gliomas: Results From the VE-BASKET Study [J].
Kaley, Thomas ;
Touat, Mehdi ;
Subbiah, Vivek ;
Hollebecque, Antoine ;
Rodon, Jordi ;
Lockhart, A. Craig ;
Keedy, Vicki ;
Bielle, Franck ;
Hofheinz, Ralf-Dieter ;
Joly, Florence ;
Blay, Jean-Yves ;
Chau, Ian ;
Puzanov, Igor ;
Raje, Noopur S. ;
Wolf, Jurgen ;
DeAngelis, Lisa M. ;
Makrutzki, Martina ;
Riehl, Todd ;
Pitcher, Bethany ;
Baselga, Jose ;
Hyman, David M. .
JOURNAL OF CLINICAL ONCOLOGY, 2018, 36 (35) :3477-+
[20]   Concomitant MEK and Cyclin Gene Alterations: Implications for Response to Targeted Therapeutics [J].
Kato, Shumei ;
Adashek, Jacob J. ;
Shaya, Justin ;
Okamura, Ryosuke ;
Jimenez, Rebecca E. ;
Lee, Suzanna ;
Sicklick, Jason K. ;
Kurzrock, Razelle .
CLINICAL CANCER RESEARCH, 2021, 27 (10) :2792-2797