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Prevention of Head and Neck Cancer Using Rapamycin and Its Analogs

Posted Sep 29 2010 8:00pm

Description of Invention:
It is frequently observed in head and neck squamous cell carcinoma (HNSCC), a cancer occurring mostly in the mouth, that the Akt/mTOR pathway is abnormally activated. Therefore, inhibiting this signaling pathway may help in treating this disease. Rapamycin and its analogs are known to inhibit the activity of mTOR so in principle they could serve as therapeutics for treating HNSCC.

Researchers at the NIH have developed a method of potentially preventing or treating HNSCC through the inhibition of mTOR activity. The proof of this principle was demonstrated by rapid regression of mouth tumors in mice afflicted with Cowden syndrome with the administration of rapamycin. Like HNSCC, development of this disease is linked to over activation of the Akt/mTOR pathway. Furthermore, the therapeutic potential of rapamycin was demonstrated using mice in experiments that model chronic exposure to tobacco, which promotes the development of HNSCC. Therefore, inhibitors of mTOR have considerable potential in the prevention and treatment of HNSCC.

Preventing the development of oral cancer using mTOR inhibitors to halt progression of pre-cancerous lesions.

Development Status:
Pre-clinical proof of principle

J Silvio Gutkind (NIDCR)

Patent Status:
HHS, Reference No. E-302-2008/0
PCT, Application No. PCT/US2009/54478 filed 20 Aug 2009

Relevant Publication:
  1. Squarize CH, Castilho RM, Gutkind JS. Chemoprevention and treatment of experimental Cowden's disease by mTOR inhibition with rapamycin. Cancer Res. 2008 Sep 1;68(17):7066-7072. [ PubMed: 18757421 ]
  2. Czerninski R, Amornphimoltham P, Patel V, Molinolo AA, Gutkind JS. Targeting mTOR by rapamycin prevents tumor progression in an oral-specific chemical carcinogenesis model. Cancer Prevention Res. 2009 Jan;2(1):27-36. [ PubMed: 19139015 ]
  3. Raimondi AR, Molinolo A, Gutkind JS. Rapamycin prevents early onset of tumorigenesis in an oral-specific K-ras and p53 two-hit carcinogenesis model. Cancer Res. 2009 May 15;69(10):4159-4166. [ PubMed: 19435901 ]

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The National Institute of Dental and Craniofacial Research, Oral and Pharyngeal Cancer Branch, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize this technology. Please contact David W. Bradley, Ph.D. at for more information.

Cancer - Therapeutics

For Licensing Information Please Contact:
Whitney Hastings
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-451-7337
Fax: 301-402-0220

Ref No: 1895

Updated: 09/2010

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