Clinical and Translational Oncology Group, Clínica del Country, Calle 116 No. 9-72, c. 318, Bogotá, Colombia.
Molecular Oncology and Biology Systems Research Group (FOX-G), Universidad el Bosque, Bogotá, Colombia.
Ther Adv Respir Dis. 2020 Jan-Dec;14:1753466620938553. doi: 10.1177/1753466620938553.
Precision oncology is the field that places emphasis on the diagnosis and treatment of tumors that harbor specific genomic alterations susceptible to inhibition or modulation. Although most alterations are only present in a minority of patients, a substantial effect on survival can be observed in this subgroup. Mass genome sequencing has led to the identification of a specific driver in the translocations of the tropomyosin receptor kinase family (NTRK) in a subset of rare tumors both in children and in adults, and to the development and investigation of Larotrectinib. This medication was granted approval by the US Food and Drug Administration for NTRK-positive tumors, regardless of histology or age group, as such, larotrectinib was the first in its kind to be approved under the premise that molecular pattern is more important than histology in terms of therapeutic approach. It yielded significant results in disease control with good tolerability across a wide range of diseases including rare pediatric tumors, salivary gland tumors, gliomas, soft-tissue sarcomas, and thyroid carcinomas. In addition, and by taking different approaches in clinical trial design and conducting allocation based on biomarkers, the effects of target therapies can be isolated and quantified. Moreover, and considering developing nations and resource-limited settings, precision oncology could offer a tool to reduce cancer-related disability and hospital costs. In addition, developing nations also present patients with rare tumors that lack a chance of treatment, outside of clinical trials. This, in turn, offers the possibility for international collaboration, and contributes to employment, education, and health service provisions.
精准肿瘤学是一门注重诊断和治疗具有特定基因组改变的肿瘤的学科，这些改变容易受到抑制或调节。虽然大多数改变只存在于少数患者中，但在这个亚组中可以观察到对生存有显著影响。大规模基因组测序已经确定了儿童和成人罕见肿瘤中 Tropomyosin 受体激酶家族（NTRK）易位的特定驱动因素，并开发和研究了 Larotrectinib。这种药物已被美国食品和药物管理局批准用于 NTRK 阳性肿瘤，无论组织学或年龄组如何，因此，larotrectinib 是第一个在分子模式比组织学在治疗方法上更重要的前提下获得批准的药物。它在广泛的疾病中表现出良好的耐受性和显著的疾病控制效果，包括罕见的儿科肿瘤、唾液腺癌、神经胶质瘤、软组织肉瘤和甲状腺癌。此外，通过在临床试验设计中采取不同的方法，并根据生物标志物进行分配，可以分离和量化靶向治疗的效果。此外，考虑到发展中国家和资源有限的地区，精准肿瘤学可以提供一种工具来减少癌症相关的残疾和医院成本。此外，发展中国家也为那些除临床试验外缺乏治疗机会的罕见肿瘤患者提供了可能性。这反过来又为国际合作提供了可能性，并有助于就业、教育和卫生服务的提供。
