Developing powerful immunotherapy treatments to fight a brain tumor that’s currently a deadly diagnosis
About DIPG
Diffuse intrinsic pontine glioma (DIPG) is one of the most devastating diagnoses a family can receive. This aggressive brain tumor grows deep in the brainstem, making it impossible to remove with surgery. Radiation is the only standard treatment — but even that only gives patients a few additional months of life. DIPG remains universally fatal, with most children living less than a year from diagnosis.
For families, this terminal diagnosis is one that leaves them with very little hope – if any at all.
With your support, our researchers have developed not one, but two forms of new promising immunotherapy treatment options:
Their strategy? Build on the progress of two approaches already funded by your donations: logic-gated CAR-T cell therapy and a novel anti-CD99 monoclonal antibody — both designed to more precisely and powerfully target DIPG cells when paired with radiation.
1. Smarter CAR-T cell therapy in harmony with radiation
First, our researchers have developed a new form of (CAR-T) therapy that safely and effectively targets the DIPG tumor cells while leaving normal cells unharmed. They do so by logic-gating these CAR-T cells to hyper focus them on two molecules highly expressed on the tumor cells (CD99 and B7-H3) but not present on in healthy tissue. This leads them safely and effectively kill solely the cancerous cells.
Now, with funding from The Morgan Adams Foundation, the research team is testing how pairing radiation with these logic-gated CAR-T cells can best be used to further boost their effectiveness.
2. Monoclonal antibodies add another powerful tool to the fight
Second, they have also developed a novel monoclonal antibody (a lab-made antibody that mimics the body’s own immune defenses) that also targets the CD99 molecule. This novel antibody does what most can’t – it successfully crosses the blood-brain barrier to bind with the tumor cells and kill them. Just like with CAR-T therapy, it works even better when combined with radiation.
In animal models, this powerful combination has already shown remarkably promising results! The team is now working on an antibody suitable for children and performing rigorous safety testing to prepare for a future Phase I clinical trial in kids with DIPG!
Why this matters
Investing in both approaches allow our researchers to attack DIPG from multiple angles. While still preclinical, they hold real potential to finally make progress against this deadly disease.
Want to learn more about the science behind this and other studies you’re helping to fund?
Meet just a few of the young lives impacted by the devasting diagnosis of a DIPG brain tumor
Want to learn more about the science behind this and other studies you’re helping to fund?