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精准治疗中2个相互依存的元素:精准医学和纳米医药
张付雷,李威*
0
(第二军医大学国际合作肿瘤研究所, 上海 200433
*通信作者)
摘要:
以基因组测序、生物信息和大数据库为基础,寻找到个体疾病原因、治疗靶点和精确分类的精准医学(precision medicine)为肿瘤治疗带来了新的希望,但真正的精准治疗(precision therapy)还包括如何将所筛药物精准递送并按剂量适时释放到治疗靶点,这也是临床上实现抗肿瘤精准治疗时面临的系列问题,具体包括如何突破体内生物屏障将药物安全递送到病灶、如何增加药物的瘤内富集、如何促进靶细胞的内吞,以及如何将药物精准递送到细胞内的作用靶点等。基于纳米技术的纳米医药(nanomedicine)能有效解决这些问题:通过精细设计和纳米载体裁剪,实现将治疗药物分子精确高效地递送到靶器官、靶组织、靶细胞和靶细胞器,并在病灶部位的刺激下按需控制药物剂量。精准医学和纳米医药的结合能够实现真正意义上的精准治疗,即从肿瘤分子分型、药物筛选、药物精准投递、控制释放到治疗。因此,基于精准医学对疾病进行精准诊断和药物靶点筛选,其可被形象地称为肿瘤精准治疗的“先遣部队和侦察兵”;而通过纳米医药可实现将筛选的抗肿瘤药物精确递送和释放到治疗靶点,可将其比喻为肿瘤精准治疗的“精准战略轰炸”,精准医学和纳米医药是精准治疗中2个相互依存且必不可少的元素。
关键词:  肿瘤  精准治疗  精准医学  纳米药物  靶向递送
DOI:10.16781/j.0258-879x.2017.06.0689
投稿时间:2017-05-15修订日期:2017-06-02
基金项目:国家自然科学基金(31470964,81171450)
Precision medicine and nanomedicine: two essential and interdependent elements in precision therapy
ZHANG Fu-lei,LI Wei*
(International Joint Cancer Institute, Second Military Medical University, Shanghai 200433, China
*Corresponding author)
Abstract:
Precision medicine has shown a new hope for cancer treatment because it can provide clear individual disease causes, precision therapeutic targets and accurate classification based on the genome sequencing, bioinformatics, and large database. However, the effective precision therapy should also include how to deliver and release drugs with controlled dosage at the precision therapeutic targets. A challenge in clinical anti-tumor precision therapy is how to achieve precision delivery and regulate the dosage of screened drugs, including how to overcome the biological barrier, how to safely deliver the drugs to nidus, how to enhance intratumoral accumulation of drugs, how to promote endocytosis of target cells, and how to accurately deliver drugs to intracellular therapeutic targets. Nanomedicine can effectively solve the problems as mentioned above. Through fine design and nanocarrier tailoring, nano-products will accurately and efficiently deliver the screened drug molecules to target organs, target tissues, target cells or intracellular target organelles, and precisely release the drug to a desirable dosage under the stimulation of lesions. The combination of precision medicine and nanomedicine can efficiently achieve precision therapy: from the tumor molecular classification, drug screening, drug precision delivery, controlled drug release to the treatment. Consequently, precision medicine mainly refers to accurate diagnosis and accurate screening of drug targets, which is the so called "advance troops and scouts" of precision treatment of cancer. Nanomedicine mainly focuss on the accurate delivery and controlled release of drugs to the therapeutic target, which can be called "accurate strategic bombing". Both precision medicine and nanomedicine are essential and interdependent elements in precision therapy.
Key words:  neoplasms  precision therapy  precision medicine  nanomedicine  targeted delivery