Monoclonal antibody therapeutic targeting GPR49 expressed on human cancer stem cells

Funding Type: 
Early Translational III
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Despite advances in molecular medicine and technology, the majority of cancer patients with late-stage disease do not survive five years after diagnosis. A growing body of scientific and clinical research indicates that a subset of stem cells within a tumor, called cancer stem cells, play a critical role not only in tumor initiation but also in chemoresistance, tumor relapse and metastasis that eventually leads to cancer death. Scientists believe that selectively targeting these cancer stem cells is the key to significantly increasing survival for patients with late-stage and metastatic tumors. Our group has developed highly effective methods to identify drugs that specifically inhibit cancer stem cell growth, and generated a large panel of candidate antibody drugs targeting a cancer stem cell protein essential for the growth of colorectal and many other cancers. The research in this grant will focus on selecting a lead antibody drug to develop and prepare for human clinical trials in advanced cancer patients within 3 years. We will optimize anti-tumor activity of our drug, manufacture sufficient high quality drug material for these studies, and evaluate safety and potential toxicity. Finally, to reduce the time, costs and high risk of failure traditionally associated with cancer drug development, we will identify markers of drug activity and clinical efficacy, as well as patient subpopulations most likely to respond to our drug, prior to initiating human studies.

Statement of Benefit to California: 

This project will benefit the citizens of California in three distinct ways. First, we are developing a novel drug that will target a group of cancers that significantly reduce the quality and duration of life for thousands of Californians each year. Significant improvement in cancer survival will benefit not only our citizens, and will also benefit the State through increased worker productivity and decreased financial burden on the health care system. Secondly, we will support the novel approach of targeting cancer stem cells in the clinical setting. This will benefit the California biotechnology industry by helping it maintain its standing as the pre-imminent state for developing novel therapeutics. By validating the concept of treating cancer by attacking cancer stem cells, our research may also lead to additional drugs in this category or in other indications in the future. Finally, this project will benefit the state of California through job creation. Continued advancement and development of our drug candidates will directly lead to increased jobs at our company.