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A protein treatment did not shrink nerve tumors in mice

Original title: A Multicenter Confirmatory Randomized-Controlled Study of rhNRGβ1 Protein Replacement Therapy in a Murine Model of NF2-related Schwannomatosis

How far along is this research?

This is a preprint. Other scientists have not checked it yet, so treat it as an early signal rather than an answer.

This was only done in mice, and the treatment did not work.

The short version

A drug that looked promising for NF2 nerve tumors did not work when tested carefully in mice.

What was studied. Researchers tested a lab made protein called rhNRG1 in 216 mice bred to grow nerve tumors like the ones in NF2-related schwannomatosis. The mice were split at random at three separate research labs, and got either the protein or an inactive liquid for 13 weeks.

What they found. The nerves of the treated mice were not any thinner than the nerves of the untreated mice. Tests on the tumor tissue showed no change in the cell markers the drug was supposed to change. The researchers found no sign that the drug was reaching and acting on its target in the body at the dose they used.

What this means, and what it doesn't

What it could mean: For now, this protein is not a promising treatment for these nerve tumors. Careful testing like this helps rule out dead ends early, so time and money can go to ideas that are more likely to help people. The researchers say the drug might still be worth testing at a different dose, given a different way, or on a different schedule.

What it doesn't mean: This does not mean a treatment is coming soon. This was an animal study in mice, not a study in people. Results in mice often do not carry over to humans. It does not change any care you can get today, and it is not a promise of a cure. It also does not prove the underlying idea is wrong, only that this drug at this dose did not work here.

Source: bioRxiv (preprint), August 4, 2026 · Read the original

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