Technology ID

Functions and Targets of Therapeutic MicroRNAs to Treat and Diagnose Cancer

Lead Inventor
Van Waes, Carter (NIDCD)
Saleh, Anthony (miRecule, Inc)
Cheng, Hui (NIDCD)
Chen, Zhong (NIDCD)
Research Materials
Therapeutic Areas
Development Stages
Pre-clinical (in vivo)
Lead IC

This technology includes a method to identify potentially therapeutic microRNAs in cancer, particularly squamous cell carcinoma of the head and neck (HNSCC). This approach first utilizes a large and publicly available expression dataset, which is then validated by a smaller independent dataset to determine deregulated microRNAs expression. These results are then intersected with in vitro functional anti-proliferative screening data to select for microRNAs that play a functional tumor suppressive role and likely serve as therapeutic targets. Utilizing the recently published data from 279 tumor specimens, this analysis strategy identified 9 potentially therapeutic microRNAs, four of which were members of the miR-30-5p family. To further validate its role in tumor suppression, several classical oncogenes centering on Receptor tyrosine kinase (RTK) signaling as novel targets of miR-30 and display regulation both in vitro and in vivo were identified. Finally, its validity as a therapeutic target is supported by showing strong tumor growth delay for a synthetic miR-30a-5p mimic in a xenograft tumor model of HNSCC.

Commercial Applications
Agents to be used in the treatment of cancer, either alone or in combination with chemotherapy/radiation, in addition to a companion diagnostic test.

Competitive Advantages
The greatest advantage of such a therapeutic would be the ability to target and repress multiple driving oncogenic pathways simultaneously, reducing the chances of resistance.
Licensing Contact:
Shmilovich, Michael