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Drug Repurposing to Treat Merkel Cell Carcinoma (MCC)

Investigators at the National Cancer Institute (NCI) and National Center for Advancing Translational Sciences (NCATS) have identified novel methods to treat the orphan disease, Merkel Cell Carcinoma (MCC) by repurposing existing drugs that were selected from a comprehensive compound screen. NCI researchers seek co-development partners and/or licensees for this invention.
NIH Reference Number
Product Type
  • Orphan Disease, Merkel Cell Carcinoma, MCC, Skin, Drug Repurposing, Aurora Kinase (AURK) Inhibitors, Histone Deacetylases (HDAC) Inhibitors, and Bcl-2 Family Member Inhibitors, National Center for Advancing Translational Sciences, NCATS, Brownell
Collaboration Opportunity
This invention is available for licensing and co-development.
Description of Technology

Merkel Cell Carcinoma (MCC) is a rare and aggressive neuroendocrine tumor of the skin.  It is most commonly found on areas of the skin with higher ultraviolet (UV) exposure, and in Caucasian patients of advanced age.  Interestingly, approximately 80% of MCC tumors are caused by integration of the Merkel cell polyomavirus (MCPyV) into the host genome (VP-MCC).  The remaining 20% are virus negative (VN-MCC) and caused by somatic mutations induced by UV exposure.  

Current therapeutic strategies include surgical resection and radiotherapy for localized, and chemotherapy for metastatic, disease. However, recurrence is common and is associated with high mortality. In May 2017, avelumab, a checkpoint inhibitor targeting the PD-1/PD-L1 pathway became the first FDA-approved treatment for MCC. Though an important step, a majority of patients treated with avelumab treatment do not achieve durable responses. Further, some patients are not eligible for treatment if they have received solid organ transplants or are severely immunocompromised.  Therefore, development of additional therapeutic strategies is still needed.

The technology developed by the National Institutes of Health (NIH) describes novel uses for known compounds to treat MCC.  NCI collaborated with the National Center for Advancing Translational Sciences (NCATS) to identify existing treatments for repurposing. Together, they screened the NCATS Chemical Genomics Center (NCGC) Pharmaceutical Collection (NPC, ~2400 compounds) and Mechanism Interrogation PlatE (MIPE, ~1900 compounds) library against MCC lines (VP-MCCs & VN-MCCs) and control cell lines. Several drug classes were identified as potential “hits” for MCC – including aurora kinase (AURK) inhibitors, histone deacetylases (HDAC) inhibitors, and Bcl-2 family member inhibitors. Currently, the inventors have validated the efficacy of these three inhibitors in vivo using mouse xenograft models of MCC. 

The inventors seek licensing opportunities and/or collaboration opportunities, such as clinical trial agreements, to further test the therapeutic effects of these compounds.  

Potential Commercial Applications
  • Method for treating Merkel Cell Carcinoma (MCC)
Competitive Advantages
  • Identification of novel therapeutic methods for treating orphan disease indication
  • ‘Drug Repurposing’ existing drugs for new orphan disease indications
  • in vitro and in vivo validation data

Isaac Brownell (NIAMS, NCI), Tara Gelb (NIAMS, NCI), Matthew Hall (NCATS), Daniel Urban (NCATS), Min Shen (NCATS)

Development Stage
Patent Status
  • U.S. Provisional: U.S. Provisional Patent Application Number 62/657,398 , Filed 13 Apr 2018
Therapeutic Area
Thursday, August 16, 2018