A 2017 fellowship recipient focuses on protein- and peptide-based siRNA delivery systems.
A recipient of the 2017 AAPS Foundation Graduate Student Fellowship, Jain is a graduate student at University of Missouri Kansas City School of Pharmacy, working under the guidance of Kun Cheng, Ph.D. With the support of the AAPS Foundation, Akshay can continue passionately researching the “discovery and development of protein- and peptide-based siRNA delivery system for liver fibrosis and cancer therapy.”
My research is mainly focused on the development of delivery systems for macromolecules such as siRNA, peptides and proteins. siRNA is arguably one of the biggest inventions to date in the field of medical science. Despite its exceptional potency, siRNA-based therapy showed drastic side effects during clinical trials, leading to deaths of a few patients and resulting in withdrawal from the clinical trials. After so many years of research in the field, very few effective therapies have been approved by the Food and Drug Administration for the treatment of fatal diseases like cancer, liver fibrosis, etc. There is an urgency to develop a potent therapeutic modality for amelioration of liver fibrosis. Therefore, we discovered PCBP2 siRNA that inhibits the collagen I protein production, which results in the resolution of the liver fibrosis. To exploit PCBP2 siRNA’s therapeutic potential, we developed a novel neutravidin-based delivery system that forms a nanocomplex with biotin-ylated siRNA and targets the nanocomplex to the target site with the help of biotinylated IGFIIR peptide.
I am also working in the selection and identification of cancer stem-cell targeting and prostate cancer-targeting ligands. A major concern with cancer therapy is the relapse of the cancer after a chemotherapy regimen. To extend patients’ life expectancy and permanently eradicate the cancer, we decided to specifically focus on cancer stem cells; they can regenerate the cancer progenitor cells even after regression of mature cancer cells. Our research group has discovered IKK asiRNA, which has been shown to potentially inhibit prostate cancer invasion and metastasis. I am developing a delivery system to codeliver IKK asiRNA and a novel small molecule anticancer drug to target castration-resistant prostate cancer cells and cancer stem cells to achieve complete eradication of cancer.
The biggest obstacle between siRNA and its translational success is its safe and efficient delivery. Neutravidin-based siRNA nanocomplex is a versatile platform that can be used not only in the delivery of PCBP2 siRNA to liver, but it also has potential application in the delivery of molecules to the relatively more complex targets such as solid tumors and across the blood brain barrier.
I strongly believe that my research will help improve current knowledge and lead to a better understanding of designing and developing a delivery system for treating the world’s most fatal diseases like cancer and liver fibrosis/cirrhosis.
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