Seid Christopher A, Jones Kathryn M, Pollet Jeroen, Keegan Brian, Hudspeth Elissa, Hammond Molly, Wei Junfei, McAtee C Patrick, Versteeg Leroy, Gutierrez Amanda, Liu Zhuyun, Zhan Bin, Respress Jonathan L, Strych Ulrich, Bottazzi Maria Elena, Hotez Peter J
a Sabin Vaccine Institute and Texas Children's Hospital Center for Vaccine Development , Houston , TX , USA.
b Departments of Pediatrics and Molecular Virology and Microbiology , National School of Tropical Medicine, Baylor College of Medicine , Houston , TX , USA.
Hum Vaccin Immunother. 2017 Mar 4;13(3):621-633. doi: 10.1080/21645515.2016.1242540. Epub 2016 Oct 13.
A therapeutic vaccine for human Chagas disease is under development by the Sabin Vaccine Institute Product Development Partnership. The aim of the vaccine is to significantly reduce the parasite burden of Trypanosoma cruzi in humans, either as a standalone product or in combination with conventional chemotherapy. Vaccination of mice with Tc24 formulated with monophosphoryl-lipid A (MPLA) adjuvant results in a Th1 skewed immune response with elevated IgG2a and IFNγ levels and a statistically significant decrease in parasitemia following T. cruzi challenge. Tc24 was therefore selected for scale-up and further evaluation. During scale up and downstream process development, significant protein aggregation was observed due to intermolecular disulfide bond formation. To prevent protein aggregation, cysteine codons were replaced with serine codons which resulted in the production of a non-aggregated and soluble recombinant protein, Tc24-C4. No changes to the secondary structure of the modified molecule were detected by circular dichroism. Immunization of mice with wild-type Tc24 or Tc24-C4, formulated with E6020 (TLR4 agonist analog to MPLA) emulsified in a squalene-oil-in-water emulsion, resulted in IgG2a and antigen specific IFNγ production levels from splenocytes that were not significantly different, indicating that eliminating putative intermolecular disulfide bonds had no significant impact on the immunogenicity of the molecule. In addition, vaccination with either formulated wild type Tc24 or Tc24-C4 antigen also significantly increased survival and reduced cardiac parasite burden in mice. Investigations are now underway to examine the efficacy of Tc24-C4 formulated with other adjuvants to reduce parasite burden and increase survival in pre-clinical studies.
赛宾疫苗研究所产品开发合作组织正在研发一种用于人类恰加斯病的治疗性疫苗。该疫苗的目标是显著降低人体中克氏锥虫的寄生虫负荷,既可以作为单一产品,也可以与传统化疗联合使用。用单磷酰脂质A(MPLA)佐剂配制的Tc24对小鼠进行疫苗接种,会导致Th1型免疫反应偏向,IgG2a和IFNγ水平升高,并且在克氏锥虫攻击后寄生虫血症有统计学上的显著降低。因此,选择了Tc24进行扩大生产和进一步评估。在扩大生产和下游工艺开发过程中,由于分子间二硫键的形成,观察到大量蛋白质聚集。为了防止蛋白质聚集,将半胱氨酸密码子替换为丝氨酸密码子,从而产生了一种不聚集且可溶的重组蛋白Tc24-C4。通过圆二色性未检测到修饰分子二级结构的变化。用在角鲨烯水包油乳液中乳化的E6020(MPLA的TLR4激动剂类似物)配制的野生型Tc24或Tc24-C4对小鼠进行免疫,脾细胞产生的IgG2a和抗原特异性IFNγ水平没有显著差异,这表明消除假定的分子间二硫键对该分子的免疫原性没有显著影响。此外,用配制好的野生型Tc24或Tc24-C4抗原进行疫苗接种也显著提高了小鼠的存活率并降低了心脏寄生虫负荷。目前正在进行研究,以检验在临床前研究中用其他佐剂配制的Tc24-C4降低寄生虫负荷和提高存活率的功效。