人源腫瘤異種移植（PDX）模型是一種將人源腫瘤組織或患者來(lái)源的原代細(xì)胞植入免疫缺陷鼠體內(nèi)，經(jīng)傳代培養(yǎng)后形成的體內(nèi)腫瘤模型。這一模型來(lái)源于臨床患者，且能與鼠體內(nèi)微環(huán)境互相作用，作為動(dòng)物模型可以較大程度地還原人類腫瘤的特征，因而其在臨床前抗腫瘤藥物的藥效學(xué)評(píng)價(jià)、個(gè)性化治療，以及腫瘤分布及轉(zhuǎn)移機(jī)制研究等方面廣受青睞。PDX模型構(gòu)建成功與否與腫瘤的惡性程度、腫瘤組織的新鮮程度、以及小鼠的免疫缺陷程度均有較大的關(guān)聯(lián)性。全球每年只有極少數(shù)抗癌新藥通過(guò)監(jiān)管部門審批，其原因是缺乏規(guī)范的臨床前藥效學(xué)研究模型和評(píng)價(jià)技術(shù)體系，進(jìn)而導(dǎo)致抗腫瘤藥物的研發(fā)進(jìn)程被嚴(yán)重限制。因此，構(gòu)建合理、可靠的臨床前抗腫瘤藥物的藥效學(xué)評(píng)價(jià)模型和建立科學(xué)的評(píng)價(jià)技術(shù)體系勢(shì)在必行。為更好地了解PDX模型，從PDX模型的構(gòu)建方法和實(shí)際應(yīng)用進(jìn)行綜述，以期為我國(guó)臨床前抗腫瘤藥物藥效學(xué)評(píng)價(jià)的有效新模型構(gòu)建及應(yīng)用提供參考信息。

Patient-derived tumor xenograft (PDX) model is an in vivo tumor model implanting human tumor tissues or patientderived primary cells into immunodeficient mice, and then culturing the cells in passaging. PDX model is derived from clinical patients and can interact with microenvironment in mice. As an animal model, PDX model can largely restore the characteristics of human tumors, thus it is widely used in preclinical pharmacodynamic evaluation of anti-tumor drugs, personalized medical treatment, as well as research on the distribution and metastatic mechanism of tumors, etc. The success of PDX model establishment is highly correlated with tumors' malignancy and freshness, as well as immunodeficiency in mice. It's a fact that only few new anticancer drugs are approved by the regulatory authorities each year. Due to the lack of standardized preclinical evaluation models and system, the research and development process of antitumor drugs in China has been seriously limited, thus it is imperative to establish reasonable and reliable preclinical efficacy evaluation models and a scientific evaluation technology system for anti-tumor drugs. This paper will review the establishment and application of PDX model, with a view to providing reference for establishing and applicating effective new models for the preclinical evaluation of anti-tumor drugs in China.

R965.1

中國(guó)食品藥品檢定研究院關(guān)鍵技術(shù)研究基金項(xiàng)目（GJJS-2022-6-5）