Human and Veterinary Cancer Therapeutic Agent Utilizing Anthrax Toxin-Based Technology

Due to the disorganized nature of blood vessels that run through tumors, chemotherapeutic agents often fail to penetrate tumors and kill cancer cells at the tumor’s center. This can lead to ineffective chemotherapeutic treatments, because tumors can quickly grow back if the entire tumor is not destroyed. NIH researchers have developed a therapeutic agent that solves this problem facing current chemotherapy treatments. By elegantly exploiting cell surface proteases present at high levels in tumors, they have developed a tumor-targeted anthrax based toxin that inactivates the blood vessels within tumors. While in some cases cancer cells are also killed by the tumor-targeted toxin, the primary mechanism of action is thought to be a decrease in blood flow to the center of tumors, causing cancer cell death and tumor necrosis. Preliminary and on-going studies have demonstrated that the targeted toxins have antitumor effects on melanomas, lung cancers and colon cancer in mouse models, and on feline and canine oral tumors. Interestingly, this therapy does not target a specific type of cancer cell, rather it targets the vasculature in and around tumors. Therefore, it has great potential to treat a wide range of solid tumors. Additionally, because few non-surgical treatments are available to treat many human and veterinary solid tumors, this technology would fill an unmet need in cancer therapy.

Potential Commercial Applications: Competitive Advantages:
Therapeutic agent for a wide range of human and veterinary solid tumors, including:
  • Melanomas
  • Lung and colon cancers
  • Oral squamous carcinomas
  • Proven effective in a variety of models, including models of important veterinary cancers
  • Agent is only active in tumor micro-environments, resulting in low toxicity to healthy tissue
  • Cancer cells are not directly targeted, so this agent can be used to treat a broad spectrum of solid tumors and resistance is unlikely to arise
  • Fills an unmet need in cancer therapy, because few non-surgical treatments exist

Development Stage:
  • Early-stage
  • In vitro data available
  • In vivo data available (animal)


Stephen Leppla (NIAID)  ➽ more inventions...

Christopher Bachran (NIAID)  ➽ more inventions...

Intellectual Property:
US Pat: 9,890,369 issued 2018-02-13
US Application No. 61/837,428 filed on 2013-06-20
PCT Application No. PCT/US2014/043131 filed on 2014-06-19

Chen, KH et al. PMID 17251181
Liu S, et al. PMID 27357689
Wein AN, et al. PMID 26584669
Peters DE, et al. PMID 24971906
Wein AN, et al. PMID 22843210
Phillips DD, et al. PMID 23393143

Collaboration Opportunity:

The National Institute of Allergy and Infectious Diseases is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize this invention. For collaboration opportunities, please contact Dr. Natalie Greco at or 301-761-7898.

Licensing Contact:
Wade Green,
Phone: 301-761-7505

OTT Reference No: E-120-2013-0
Updated: Oct 2, 2017