Emerging and investigational therapies for neuroblastoma.
Emerging and investigational therapies for neuroblastoma.
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DOI：
10.1080/21678707.2017.1304212
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发表时间：
2017
影响因子：
0.8
通讯作者：
Cohn SL
中科院分区：
文献类型：
--
作者：
Applebaum MA;Desai AV;Glade Bender JL;Cohn SL
患有临床侵袭性高危神经母细胞瘤的儿童的治疗仍然具有挑战性。不到 50% 的高危神经母细胞瘤患者在目前的治疗下能够长期生存，幸存者面临着与治疗相关的严重后期毒性的风险。在这里，我们回顾了新的和不断发展的治疗方法，这些治疗方法可能最终改善高危神经母细胞瘤儿童的预后，并降低晚期不良事件的可能性。综述了治疗高危神经母细胞瘤的新策略，包括：放射疗法、靶向细胞毒素、生物制剂、免疫疗法和分子靶向药物。最近完成和正在进行的测试这些新疗法的神经母细胞瘤临床试验得到了强调。此外，我们还讨论了正在进行的临床试验，旨在评估针对可操作的体细胞突变和致癌细胞途径的精准医学方法。基因组医学和分子生物学的进步促进了早期研究的发展，测试针对异常激活的细胞途径的生物学合理疗法。由于这些药物中的许多药物比标准化疗药物具有更广泛的治疗指数，因此这些治疗可能比目前的策略更有效且毒性更小。然而，为了有效整合这些有针对性的策略，必须开发强大的预测生物标志物，以识别将从这些方法中受益的患者，并在诊断时快速将治疗与患者相匹配。
Treatment for children with clinically aggressive, high-risk neuroblastoma remains challenging. Less than 50% of patients with high-risk neuroblastoma will survive long-term with current therapies, and survivors are at risk for serious treatment-related late toxicities. Here, we review new and evolving treatments that may ultimately improve outcome for children with high-risk neuroblastoma with decreased potential for late adverse events. New strategies for treating high-risk neuroblastoma are reviewed including: radiotherapy, targeted cytotoxics, biologics, immunotherapy, and molecularly targeted agents. Recently completed and ongoing neuroblastoma clinical trials testing these novel treatments are highlighted. In addition, we discuss ongoing clinical trials designed to evaluate precision medicine approaches that target actionable somatic mutations and oncogenic cellular pathways. Advances in genomic medicine and molecular biology have led to the development of early phase studies testing biologically rational therapies targeting aberrantly activated cellular pathways. Because many of these drugs have a wider therapeutic index than standard chemotherapeutic agents, these treatments may be more effective and less toxic than current strategies. However, to effectively integrate these targeted strategies, robust predictive biomarkers must be developed that will identify patients who will benefit from these approaches and rapidly match treatments to patients at diagnosis.
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复发的神经母细胞瘤显示出频繁的RAS-MAPK途径突变。
影响因子：
30.8
作者：
Eleveld TF;Oldridge DA;Bernard V;Koster J;Colmet Daage L;Diskin SJ;Schild L;Bentahar NB;Bellini A;Chicard M;Lapouble E;Combaret V;Legoix-Né P;Michon J;Pugh TJ;Hart LS;Rader J;Attiyeh EF;Wei JS;Zhang S;Naranjo A;Gastier-Foster JM;Hogarty MD;Asgharzadeh S;Smith MA;Guidry Auvil JM;Watkins TB;Zwijnenburg DA;Ebus ME;van Sluis P;Hakkert A;van Wezel E;van der Schoot CE;Westerhout EM;Schulte JH;Tytgat GA;Dolman ME;Janoueix-Lerosey I;Gerhard DS;Caron HN;Delattre O;Khan J;Versteeg R;Schleiermacher G;Molenaar JJ;Maris JM
通讯作者：
Maris JM
DOI：
10.1158/1078-0432.ccr-08-1815
发表时间：
2009-05-15
期刊：
Clinical cancer research : an official journal of the American Association for Cancer Research
影响因子：
--
作者：
Brodeur GM;Minturn JE;Ho R;Simpson AM;Iyer R;Varela CR;Light JE;Kolla V;Evans AE
通讯作者：
Evans AE
影响因子：
11.5
作者：
Desai, N;Trieu, V;Soon-Shiong, P
通讯作者：
Soon-Shiong, P
影响因子：
11.2
作者：
Ara T;Song L;Shimada H;Keshelava N;Russell HV;Metelitsa LS;Groshen SG;Seeger RC;DeClerck YA
通讯作者：
DeClerck YA
DOI：
10.1056/nejmoa1001527
发表时间：
2010-09-30
期刊：
The New England journal of medicine
影响因子：
--
作者：
Baker DL;Schmidt ML;Cohn SL;Maris JM;London WB;Buxton A;Stram D;Castleberry RP;Shimada H;Sandler A;Shamberger RC;Look AT;Reynolds CP;Seeger RC;Matthay KK;Children’s Oncology Group
通讯作者：
Children’s Oncology Group