Funded by the Dick Vitale Pediatric Cancer Research Fund
Special white blood cells can be engineered to fight cancer. They are engineered with a molecule call CAR. This molecule helps them kill cancer cells. The special white blood cells include T and NK cells. These cells can cure some blood cancers. They show great promise for children with solid tumors. CAR T cells often get exhausted in solid tumors. CAR T cells are currently made outside the body. This can further weaken them.Our research aims to overcome these challenges. We will target Glypican-3 (GPC3). It is a molecule found on many childhood solid tumors. We propose a new way to engineer CAR T cells inside the body (in-body). We will use special virus-like particles (LVPs). These LVPs will precisely reprogram the children’s own immune cells to fight GPC3-positive cancer. We will test “armoring” strategies of CAR T cells. This is to make these in-body generated CAR T/NK cells even more powerful and long-lasting.Our hypothesis is that in-body engineered GPC3-CAR T cells will be highly effective against cancer cells. We will first maximize the effectiveness of our LVP delivery system. Next, we will compare the different armoring strategies. We will study how they boost the survival of in-body generated CAR T cells. Finally, we will select the most potent armoring strategy. Ultimately, this research aims to bring safer, more effective CAR T-cell therapy to children with solid tumors. The findings may be applicable to other cancers in the future.
