Grant Award Details
To enable an FDA pre-IND meeting for an autologous iNKT TCR gene-modified hematopoietic stem cell therapeutic to treat cancer
Grant Application Details
- Stem Cell-Based iNKT Cell Therapy for Cancer
Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs)
Area of Impact
The targeted area of impact for the candidate is cancer therapy, in particular cancers that are lacking existing effective treatments.
Mechanism of Action
The proposed candidate will generate therapeutic levels of invariant natural killer T (iNKT) cells in cancer patients, helping them to battle their deadly diseases. These iNKT cells can both directly kill tumor cells, and activate other immune cells like natural killer (NK) cells and cytotoxic T cells (CTLs) to eradicate tumor.
Unmet Medical Need
Despite the existing therapies, cancer patients still suffer from the ineffectiveness of these treatments, their toxicities, and the risk of relapse. Our proposed Stem Cell-Based iNKT Cell Therapy represents a novel therapy for cancer that can potentially help many cancer patients.
Major Proposed Activities
- Conduction of Preclinical Studies
- Development of a Clinical Trial Protocol
- Preparation for and Conduction of a Pre-IND Meeting with the FDA
Cancer is a leading threat to public health in the United States and in the State of California. In 2015, it is estimated that over 160,000 Californians can be diagnosed with cancer. Cancer is the second leading cause of death in California, and also brings devastating economic impacts to the State. Our proposed Stem Cell-Based iNKT Cell Therapy, if successful, has the potential to save the lives of Californians and reduce the economic burden for cancer treatment.
- Cell Stem Cell (2019) Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer. (PubMed: 31495780)
- Stem Cells Dev (2016) Propagating Humanized BLT Mice for the Study of Human Immunology and Immunotherapy. (PubMed: 27608727)