Autophagy Modulators For Use in Treating Cancer

Cancer cells can upregulate autophagy – cell destruction – as a response to chemotherapy. Investigators in Dr. Melvin DePamphilis’ laboratory at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) have shown that compounds identified by screening a library of compounds blocks autophagy in some cancer cells (e.g., melanoma) but are not toxic to normal cells. Cancer cells with mutations in the BRAF oncogene are especially dependent on autophagy. Treatment of cancer cells with the BRAF mutation can increase the efficacy of chemotherapy. Proof of concept studies in xenograft mice showed reduction of melanoma tumor size upon treatment with WX8, a lead compound described in the patent application cited below. The technology is available for licensing and/or co-development under a collaborative research agreement.  

Potential Commercial Applications: Competitive Advantages:
  • Cancer therapeutic
  • Administration of the compounds that inhibit autophagy can be used to sensitize cancer cells to chemotherapeutic agents
  • The compounds inhibiting autophagy are selective for cancer cells and are not toxic to normal cells 

Development Stage:
Pre-clinical (in vivo)

Related Invention(s):


Melvin DePamphilis (NICHD)  ➽ more inventions...

Gaurav Sharma (NICHD)  ➽ more inventions...

Juan Marugan (NCATS)  ➽ more inventions...

Marc Ferrer-Alegre (NICHD)  ➽ more inventions...

Ajit Roy (NICHD)  ➽ more inventions...

Intellectual Property:
US Pat: - issued -

Sharma et al. A family of PIKFYVE inhibitors with therapeutic potential against autophagy-dependent cancer cells disrupt multiple events in lysosome homeostasis.    PMID 30806145

Collaboration Opportunity:

Licensing and research collaboration

Licensing Contact:
John Hewes, Ph.D.
Phone: 240-276-5515

OTT Reference No: E-003-2018
Updated: Aug 6, 2020