BRAF-mediated brain tumors in adults and children: A review and the Australian and New Zealand experience

被引:3
|
作者
Trinder, Sarah M. [1 ]
McKay, Campbell [2 ]
Power, Phoebe [3 ,4 ]
Topp, Monique [5 ]
Chan, Bosco [6 ]
Valvi, Santosh [1 ]
McCowage, Geoffrey [7 ,8 ]
Govender, Dinisha [7 ]
Kirby, Maria [6 ]
Ziegler, David S. [3 ,9 ,10 ]
Manoharan, Neevika [3 ,10 ]
Hassall, Tim [11 ]
Kellie, Stewart [12 ]
Heath, John [13 ]
Alvaro, Frank [14 ]
Wood, Paul [15 ]
Laughton, Stephen [16 ]
Tsui, Karen [16 ]
Dodgshun, Andrew [17 ]
Eisenstat, David D. [2 ,18 ,19 ]
Endersby, Raelene [20 ,21 ]
Luen, Stephen J. [22 ]
Koh, Eng-Siew [23 ,24 ,25 ]
Sim, Hao-Wen [26 ,27 ,28 ,29 ]
Kong, Benjamin [26 ,30 ]
Gottardo, Nicholas G. [1 ,20 ]
Whittle, James R. [31 ]
Khuong-Quang, Dong-Anh [2 ]
Hansford, Jordan R. [6 ,32 ,33 ]
机构
[1] Perth Childrens Hosp, Dept Paediat & Adolescent Oncol Haematol, Nedlands, WA, Australia
[2] Royal Childrens Hosp, Childrens Canc Ctr, Melbourne, Vic, Australia
[3] Univ New South Wales, Sydney Childrens Hosp, Childrens Canc Inst, Randwick, NSW, Australia
[4] Univ New South Wales, Sch Womens & Childrens Hlth, Randwick, NSW, Australia
[5] Peter MacCallum Canc Ctr, Dept Med Oncol, Melbourne, Vic, Australia
[6] Womens & Childrens Hosp, Michael Rice Canc Ctr, North Adelaide, SA, Australia
[7] Childrens Hosp Westmead, Dept Oncol, Sydney, NSW, Australia
[8] Australasian Childrens Canc Trials, Clayton, Vic, Australia
[9] Univ New South Wales UNSW Sydney, Childrens Canc Inst, Lowy Canc Res Ctr, Sydney, NSW, Australia
[10] Univ New South Wales UNSW Sydney, Univ New South Wales UNSW Med & Hlth, Sch Clin Med, Sydney, NSW, Australia
[11] Univ Queensland, Queensland Childrens Hosp, Brisbane, Qld, Australia
[12] Univ Sydney, Westmead Childrens Hosp, Westmead, NSW, Australia
[13] Royal Hobart Hosp, Dept Pediat Oncol, Hobart, Tas, Australia
[14] John Hunter Childrens Hosp, Dept Pediat Oncol, Newcastle, NSW, Australia
[15] Peter MacCallum Canc Ctr, Monash Med Ctr, Melbourne, Vic, Australia
[16] Starship Childrens Hosp, Starship Blood & Canc Ctr, Auckland, New Zealand
[17] Christchurch Hosp, Childrens Haematol Oncol Ctr, Christchurch, New Zealand
[18] Murdoch Childrens Res Inst, Melbourne, Vic, Australia
[19] Univ Melbourne, Dept Paediat, Melbourne, Vic, Australia
[20] Telethon Kids Inst, Telethon Kids Canc Ctr, Brain Tumour Res Program, Nedlands, WA, Australia
[21] Univ Western Australia, Ctr Child Hlth Res, Perth, WA, Australia
[22] Univ Melbourne, Sir Peter MacCallum Dept Oncol, Melbourne, Vic, Australia
[23] Liverpool & Macarther Canc Therapy Ctr, Dept Radiat Oncol, Liverpool, NSW, Australia
[24] Univ New South Wales, Dept Med, Sydney, NSW, Australia
[25] Ingham Inst Appl Med Res, Liverpool, NSW, Australia
[26] Univ Sydney, Natl Hlth & Med Res Council NHMRC, Clin Trials Ctr, Sydney, NSW, Australia
[27] Univ New South Wales, Fac Med & Hlth, Sch Clin Med, Sydney, NSW, Australia
[28] Kinghorn Canc Ctr, Dept Med Oncol, Sydney, NSW, Australia
[29] Chris OBrien Lifehouse, Dept Med Oncol, Sydney, NSW, Australia
[30] Royal North Shore Hosp, Dept Med Oncol, St Leonards, NSW, Australia
[31] Walter & Eliza Hall Inst Med Res, Personalised Oncol Div, Parkville, Vic, Australia
[32] South Australian Hlth & Med Res Inst South Austral, Adelaide, SA, Australia
[33] Univ Adelaide, South Australia ImmunoGEN Canc Inst, Adelaide, SA, Australia
来源
FRONTIERS IN ONCOLOGY | 2023年 / 13卷
关键词
glioma; gliomagenesis; MAPK signaling; BRAF; BRAF inhibitors; access; LOW-GRADE GLIOMA; MAPK PATHWAY ACTIVATION; CENTRAL-NERVOUS-SYSTEM; PEDIATRIC HIGH-GRADE; LONG-TERM; PILOCYTIC ASTROCYTOMA; SIGNALING PATHWAY; OPTIC PATHWAY; FOLLOW-UP; PHASE-II;
D O I
10.3389/fonc.2023.1154246
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
The mitogen-activated protein kinase (MAPK) pathway signaling pathway is one of the most commonly mutated pathways in human cancers. In particular, BRAF alterations result in constitutive activation of the rapidly accelerating fibrosarcoma-extracellular signal-regulated kinase-MAPK significant pathway, leading to cellular proliferation, survival, and dedifferentiation. The role of BRAF mutations in oncogenesis and tumorigenesis has spurred the development of targeted agents, which have been successful in treating many adult cancers. Despite advances in other cancer types, the morbidity and survival outcomes of patients with glioma have remained relatively stagnant. Recently, there has been recognition that MAPK dysregulation is almost universally present in paediatric and adult gliomas. These findings, accompanying broad molecular characterization of gliomas, has aided prognostication and offered opportunities for clinical trials testing targeted agents. The use of targeted therapies in this disease represents a paradigm shift, although the biochemical complexities has resulted in unexpected challenges in the development of effective BRAF inhibitors. Despite these challenges, there are promising data to support the use of BRAF inhibitors alone and in combination with MEK inhibitors for patients with both low-grade and high-grade glioma across age groups. Safety and efficacy data demonstrate that many of the toxicities of these targeted agents are tolerable while offering objective responses. Newer clinical trials will examine the use of these therapies in the upfront setting. Appropriate duration of therapy and durability of response remains unclear in the glioma patient cohort. Longitudinal efficacy and toxicity data are needed. Furthermore, access to these medications remains challenging outside of clinical trials in Australia and New Zealand. Compassionate access is limited, and advocacy for mechanism of action-based drug approval is ongoing.
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页数:25
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