A novel immuno-oncology approach to treating a rare childhood brain cancer

 rare type of childhood brain tumor called diffuse intrinsic pontine glioma (DIPG) has a distinct characteristic that may prove to be its undoing—a misshapen protein displayed prominently on the surfaces of its cells. Scientists at the University of California at San Francisco have developed a therapeutic vaccine that can recognize the protein and prompt the immune system to launch an attack against the tumor.

The UCSF scientists have started a phase 1 trial in children with DIPG and related gliomas, according to a press release from the university. They also believe their preclinical discoveries should support the development of a new approach to fighting the disease, which would involve removing immune cells from patients and engineering them to recognize the errant protein.

Many cancers possess such misshapen proteins, which are dubbed "neoantigens," and several ideas for basing cancer therapies on them are being studied around the world. Problem is, neoantigens can be difficult to target because they often resemble normal proteins in the body, making it unlikely the immune system would recognize them as a threat that needs to be eradicated.

The protein in DIPG, called histone 3 variant 3, doesn’t present that challenge—it is found only on the tumor cells. Furthermore, a similarly mutated form of the protein is present in 70% of DIPG cases, and when it’s there, it’s on nearly all of the tumor cells, according to UCSF.