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Combination Chemotherapeutics for the Treatment of Chordoma

Posted Mar 11 2013 8:00pm

Description of Invention:
Utilizing high-throughput screening methodology, NIH scientists have identified two classes of clinically-available drugs, proteasome inhibitors and topoisomerase inhibitors, that synergize to promote chordoma cell death. Moreover, use of the two-part chemotherapeutic regimen in animal models effectively suppressed the growth of chordoma cells and resulted in significant tumor regression. Currently, no chemotherapeutic agents have been approved for the treatment of chordoma. Using FDA approved drugs in a combination therapeutic regimen will help expedite the availability of a therapeutic for chordoma.

Chordoma is a rare form of bone cancer that arises within the skull, sacrum or bony spine. Surgical resection and radiation therapy are the current standards-of-care; however, post-treatment complications remain significant and neither modality is effective for the control of metastatic tumors.

  • Chemotherapeutic regimen for the treatment of inoperable chordomas.
  • Therapy for the treatment of recurrent or metastatic chordomas.
  • Therapeutic kit combining an FDA-approved proteasome inhibitor with a topoisomerase inhibitor.

  • Therapy utilizes FDA-approved drugs with known pharmacokinetics and safety profiles.
  • Reduced drug dosing from combination therapy may result in fewer patient side effects.
  • Combination therapy inhibits multiple molecular targets, enhancing disease response.

Development Status:
  • Pre-clinical
  • In vitro data available
  • In vivo data available (animal)

Menghang Xia (NCATS)
Ruili Huang (NCATS)
Christopher P Austin (NCATS)

Patent Status:
HHS, Reference No. E-156-2012/0
US, Application No. 61/692,560 filed 23 Aug 2012

Collaborative Research Opportunity:
The National Center for Advancing Translational Sciences, Division of Pre-Clinical Innovation, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize Combination Chemotherapeutics for the Treatment of Chordoma. For collaboration opportunities, please contact Lili M. Portilla, MPA at .

For Licensing Information Please Contact:
Sabarni Chatterjee Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5587
Fax: 301-402-0220

Ref No: 2544

Updated: 03/2013

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