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A drug that blocks a DNA repair switch helped radiation work better in a hard-to-treat childhood brain tumor

Original title: H3K27M enhances basal ATM signaling but is not required for radiosensitization of diffuse midline glioma via ATM inhibition with AZD1390.

How far along is this research?

This was done in animals, not people. Most findings at this stage never become treatments.

This was done in lab cells and mice, and testing in people is only just starting.

The short version

In lab and mouse tests, a drug called AZD1390 made radiation work better against a childhood brain tumor called diffuse midline glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary.

What was studied. Researchers looked at tumor samples from patients, at tumor cells grown in the lab, and at mice with diffuse midline glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary. They tested a drug called AZD1390, which blocks a protein named ATM that helps tumor cells repair damage from radiation.

What they found. Tumors with the H3K27M change made more ATM than tumors without it. These cells had more DNA repair activity going on, even without radiation. Adding AZD1390 made radiation work better against these tumors in mice, and the mice had few bad effects. Removing the H3K27M change did not stop the drug from working.

What this means, and what it doesn't

What it could mean: Radiation is the usual treatment for this tumor, but the tumor almost always comes back. This work supports the idea of adding an ATM blocking drug to radiation. A study of AZD1390 with radiation in newly diagnosed children has recently started.

What it doesn't mean: This does not mean a new treatment is available. Almost all of this work was done in cells and in mice, not in people. The drug is not approved for this tumor. The human study has only just begun, so we do not yet know if it helps patients or is safe for them. This is not a cure, and it is a long way from everyday care.

Source: PubMed, July 14, 2026 · Read the original

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