Poster Presentation 30th Lorne Cancer Conference 2018

A double targeted strategy for effective drug delivery to brain metastases (#262)

Joanna Macdonald 1 , Justin Henri 1 , Sarah Shigdar 1
  1. Deakin University, Waurn Ponds, Vic, Australia

Brain metastases occur in up to a quarter of all cancer patients following primary malignancy treatment, and the prognosis for these patients is very poor. The treatment of these metastatic tumours is greatly hindered by the presence of the blood brain barrier (BBB) which restricts the overwhelming majority of small molecules from entering the brain. A novel approach to overcome this is to target receptor mediated transport mechanisms present on the endothelial cell membranes, in particular the transferrin receptor. Given their specificity, safety profile and stability, nucleic acid based therapeutics are ideal for this purpose. An aptamer targeting the transferrin receptor was fused with an aptamer that binds to a cell surface marker on breast cancer cells, the epithelial cell adhesion molecule (EpCAM), enhancing binding affinity of both aptamers while maintaining specificity, and confirmed through flow cytometry and confocal microscopy. Using an in vitro BBB model, the aptamer transcytosed the barrier and targeted only EpCAM positive cells in a co-culture of EpCAM positive and negative cell. This aptamer also specifically delivered doxorubicin across the in vitro BBB in a timely manner. In vivo, we confirmed the aptamer’s ability to transcytose the BBB in a healthy mouse model following a single i.v. injection (40 nmol/kg)1, and in an animal model of breast cancer brain metastases. These promising results demonstrate that through the fusion of two aptamer sequences, a bi-functional aptamer can be generated which has the potential to be developed for the specific treatment of EpCAM positive brain metastases. 

  1. 1 J Macdonald, J Henri, L Goodman, D Xiang, W Duan, S Shigdar. Development of a Bifunctional Aptamer Targeting the Transferrin Receptor and Epithelial Cell Adhesion Molecule (EpCAM) for the Treatment of Brain Cancer Metastases. ACS Chemical Neuroscience 2017 8 (4), 777-784.