“Your child has brain cancer.” Thousands of parents hear those terrible words every year. For some, when it is Diffuse Intrinsic Pontine Glioma (DIPG), the diagnosis is especially devastating. DIPG is a particularly aggressive and inoperable brain tumor that chiefly strikes children. It attacks the brainstem- the control center of the brain. This region is needed for very basic functions, like breathing, or the child’s heartbeat. As this cancer’s name suggests, a DIPG tumor diffusely infiltrates the brain stem, making it impossible to remove it surgically. Radiation may slow the growth of the tumor, but there is no real treatment. No cure. The survival rate is near zero.

However, federally funded researchers at Stanford University are now rewriting the future of DIPG.

Dr. Michelle Monje and her team have spent years studying how DIPG tumors grow and how they blend themselves into healthy brainstem tissue, becoming entwined with the very cells that help a child breathe, swallow and live. One of the most important discoveries of Monje’s team was that the tumor cells weren’t just blending in: they were directly communicating with neuronal cells in the brain.

This discovery opened up a daring new line of attack to treat DIPG. If the communication is important for the cancer growth, then, if they could interrupt the cellular “conversation”, it might be possible to treat the cancer. Work to disrupt these key interactions is ongoing and yielding very promising results. Realizing that the cancer cells were communicating with regular brainstem cells through points of electrical contact called synapses inspired a new kind of approach to treat the cancer – using the immune system to disrupt the integration, and thus remove DIPG cancerous cell by cancerous cell.

Monje’s team turned to CAR T-cell therapy, a cutting-edge treatment that trains a patient’s own immune cells to recognize and destroy cancer cells. It had never been used for DIPG, and no one knew if it would work. Lab studies had given Monje’s team hope that this therapy could reach and eliminate tumor cells deeply entwined with healthy brain tissue. But whether or not it would work in patients remained to be seen.

What happened next in clinical trials exceeded every expectation.

One patient was completely cured. Several others had tumors that shrank significantly in size.

It is important to note that not all patients in the trial responded to CAR-T cell therapy- some saw no measurable improvement, highlighting the promise of the therapy but also the urgent need for continued work.

This was the first time such dramatic responses were seen in DIPG patients. The results mark a turning point in the decades-long search for a treatment. Some patients finally have hope. Rapid progress to find cures for childhood brain cancers like DIPG heavily rely on federal funding from the National Institute of Health. Government grants helped Dr. Monje, and others explore long-term research that was high risk but high reward and that wouldn’t happen otherwise. Continued investment is essential for finding treatments for childhood brain cancers like DIPG.