Bellat Vanessa, Lee Hyun Hee, Vahdat Linda, Law Benedict
Molecular Imaging Innovations Institute, Department of Radiology, Weill Cornell Medicine , 413 East 69th Street, New York, New York 10021, United States.
Department of Medicine, Weill Cornell Medicine , 425 East 61st Street, New York, New York 10065, United States.
Biomacromolecules. 2016 Jun 13;17(6):2040-9. doi: 10.1021/acs.biomac.6b00227. Epub 2016 May 24.
We previously reported a high aspect ratio peptide nanofiber that could be effectively delivered to tumors with minimal nonspecific uptake by other organs. The peptidic nature offers the design flexibility of smart formulation with unique responsiveness. Two new formulations that behave congruously as nanotransformers (NTFs) are reported herein. NTF1 and NTF2 could biomechanically remodel upon enzyme activation to generate a degradable and an aggregable effect, respectively, within the lysosomal compartment. These NTFs were further evaluated as carriers of mertansine (DM1), a microtubule inhibitor. DM1-loaded NTF1 could be degraded by cathepsin B (CathB) to release the same active metabolite, as previously described in the lysosomal degradation of antibody-DM1 conjugate. In contrast, CathB only partially digested DM1-loaded NTF2 and induced aggregate formation to become a storage reservoir with slow payload release property. The DM1-loaded NTF1 exhibited a comparable cytotoxicity to the free drug and was more effective than the NTF2 formulation in eradicating triple negative breast cancer. Our data suggested that biological transformers with distinct enzyme-induced structural changes and payload release profiles could be designed for the intracellular delivery of cytotoxic and imaging agents.
我们之前报道了一种高纵横比的肽纳米纤维,它能够以极少的非特异性摄取被有效递送至肿瘤,而其他器官对其摄取极少。肽的性质为智能制剂的设计提供了灵活性,使其具有独特的响应性。本文报道了两种表现为纳米变压器(NTF)的新型制剂。NTF1和NTF2在酶激活后可进行生物力学重塑,分别在溶酶体区室中产生可降解和可聚集的效应。这些NTF被进一步评估作为美登素(DM1,一种微管抑制剂)的载体。负载DM1的NTF1可被组织蛋白酶B(CathB)降解,以释放相同的活性代谢物,这与之前报道的抗体-DM1偶联物在溶酶体中的降解情况相同。相比之下,CathB仅部分消化负载DM1的NTF2并诱导聚集形成,使其成为具有缓慢释放载药特性的储存库。负载DM1的NTF1表现出与游离药物相当的细胞毒性,并且在根除三阴性乳腺癌方面比NTF2制剂更有效。我们的数据表明,可以设计具有不同酶诱导结构变化和载药释放曲线的生物变压器,用于细胞毒性药物和成像剂的细胞内递送。