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New pill that targets IDH gene changes works in lab tests and in mice

Original title: LY3410738, a Covalent Inhibitor of Mutant IDH1/2, is Effective in Acute Myeloid Leukemia Preclinical Models.

How far along is this research?

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

This was only done in lab cells and in mice, not in people.

The short version

This is very early work in cells and in mice, not in people.

What was studied. Researchers tested a new drug called LY3410738. It is a pill made to block the changed IDH1 and IDH2 proteins found in some leukemias and some brain tumors. They tested it on human leukemia and 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 cells in the lab, and in mice given human leukemia cells.

What they found. The drug locked onto the changed IDH proteins and lowered the harmful substance those proteins make. In mice with human leukemia cells, it slowed the disease and helped the blood cells grow up the way they should. It also still worked on cancer cells that had stopped responding to two IDH drugs already in use, ivosidenib and enasidenib. When it was added to standard leukemia treatments, it worked well together with them and the mice handled it.

What this means, and what it doesn't

What it could mean: Changed IDH genes are found in about 20% of this leukemia and about 80% of secondary gliomas. So a drug that blocks both IDH1 and IDH2 could matter to a lot of people. This one is a pill, and the researchers think it may be able to reach the brain. That is the reason it is of interest for brain tumors.

What it doesn't mean: This is not a cure, and it is not a treatment you can get. No people were given this drug in this study. Everything here happened in cells in a dish and in mice. Many drugs that work in mice do not work in people. It would need years of testing in people first. Nothing about your care changes because of this.

Source: PubMed, July 23, 2026 · Read the original

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