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Composition and Methods of Multi-Targeted siRNA Cocktail for Treatment of Glioblastoma Multiforme
Patent Number: US8541568B2

Executive Summary:
  • Invention Type: Therapeutic
  • Patent Status: Granted
  • Patent Link: https://patents.google.com/patent/US8541568B2
  • Related Patent Link: https://patents.google.com/patent/WO2017066557
  • Research Institute: Duke University
  • Disease Focus: Gliomas, including Glioblastoma
  • Basis of Invention: Suppress oncogenic genes within glioblastoma cells using small interfering RNA (siRNA)
  • How it works: siRNA can be delivered with a pharmaceutical carrier to suppress multiple oncogenic genes within glioblastoma cells. This modality may help elicit tumoricidal effects and cause glioblastoma cells to respond to conventional treatments
  • Lead Challenge Inventor: Hai Yan
  • Inventors: Darell Bigner, Patrick Lu, Hai Yan
  • Development Stage: Preclinical
  • Novelty:
    • Usage of siRNA to suppress oncogenic genes using glioblastoma cell-focused ligands
    • Targets multiple mechanisms involved in glioblastoma viability
  • Clinical Applications:
    • Targeting oncogenic proteins EGFR, VEGF, and/or MGMT in glioblastoma and non-glioma tumors in order to make the tumors vulnerable to chemotherapy
 
General Description:
​
Uses siRNA molecules to suppress multiple oncogenic genes within glioblastoma cells, including EGFR, VEGF, and MGMT. siRNA can be administered with a pharmacologic carrier and targeted to glioblastoma cells specifically using distinct ligands. Targeting a broad array of molecules may lead to increased likelihood of therapeutic success.
 
Scientific Progress:
Successful in mouse models.
 
Future Directions:
  • Clinical validation
 
Strengths:
  • Targets multiple mechanisms leading to glioblastoma validity: proliferation, angiogenesis, and chemotherapeutic resistance
  • Focuses on molecular heterogeneity of glioblastoma tumors
 
Weaknesses:
  • Not yet tested in humans
 
Patent Status:
Legal status: Granted
  • Priority date: 2008-05-24
  • Filing date: 2009-05-26
  • Publication date: 2013-09-24
  • Grant date: 2013-09-24
 
Publication PMID: 19777070, 22684456
 
Publications:
Kanu OO, Hughes B, Di C, Lin N, Fu J, Bigner DD, Yan H, Adamson C. Glioblastoma Multiforme Oncogenomics and Signaling Pathways. Clin Med Oncol. 2009 Apr 8;3:39-52.
 
Wortham M, Yan H. Apples to origins: identifying brain tumor stem cell genes by comparing transcriptomes of normal and cancer stem cells. Cancer Discov. 2012;2(6):492-4.
 
Inventor Bio: Hai Yan
https://medschool.duke.edu/about-us/our-faculty/hai-yan
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