Research And Grants

Engineering Suppression-Resistant CAR-T Cells for the Treatment of Pediatric DIPG by Overcoming the Tumor Microenvironment

Diffuse Intrinsic Pontine Glioma (DIPG) is an aggressive and fatal brain tumor that affects young children. Because it grows in the brainstem, it cannot be removed with surgery, and radiation offers only temporary relief. Chemotherapy and other standard treatments have not been effective. Most children with DIPG survive less than a year, highlighting the urgent need for new therapies.

One promising approach is CAR-T cell therapy, which uses engineered mmune cells, reprogrammed to recognize and kill cancer. However, DIPG has proven especially difficult to treat with CAR-T cells because the tumor creates a highly immunosuppressive environment. It actively shuts down immune cells and hides from them, preventing a sustained attack.

Our project uses advanced gene editing tools to protect CAR-T cells against these suppressive signals. By knocking out a specific combination of genes inside the T cells using a technique called base editing, we make them more resistant to suppression and cancer evasion. This allows the cells to survive longer, stay active, and more effectively eliminate tumor cells.

This research could lead to a new generation of immunotherapies that overcome the unique defenses of DIPG and offer real hope for children diagnosed with this devastating disease.