Oral mucositis is a major complication of radiotherapy for the treatment of head and neck cancer and a major dose-limiting side effect. Our work has shown that intra-esophageal administration of plasmidlliposomes containing the transgene expressing human superoxide dismutase 2 (SOD2) (JVRSOD) protects the esophagus from radiation-induced esophagitis (a form of mucositis) in mice. The overall purpose of the Phase I section of this fast-track contract proposal is to complete the IND-enabling studies for the use of JVRSOD as a prophylactic mouthwash to reduce andlor prevent oral mucositis in head and neck cancer patients undergoing radiation treatment. The Phase I Proposal specific aims are (1) dose optimization of JVRSOD in a mouse model for protection from radiation-induced oral mucositis;(2) dosing schedule optimization of JVRSOD in the same mouse model for protection from radiation-induced oral mucositis;and (3) identification and qualification of a GMP manufacturing site. Under the Phase II Contract Proposal in this Fast-Track submission, an IND application for JVRSOD will be submitted to the FDA, cGMP JVRSOD will be manufactured, and it will be tested in humans for safety I efficacy in a Phase 1111 clinical trial with head and neck cancer patients undergoing standard of care radiation treatment

Agency
National Institute of Health (NIH)
Type
Small Business Innovation Research – Phase I (N43)
Project #
261201300014C-0-0-1
Application #
8740763
Study Section
Project Start
2013-09-12
Project End
2014-06-11
Budget Start
Budget End
Support Year
Fiscal Year
2013
Total Cost
$249,997
Indirect Cost
Name
Colby Pharmaceutical Company
Department
Type
DUNS #
611495974
City
San Jose
State
CA
Country
United States
Zip Code
95138