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Targeting a Key Enzyme Could Reverse Early Parkinson's Effects

  • Stanford Medicine published on July 1, 2025, a study showing that feeding a kinase inhibitor to mice reversed effects of overactive LRRK2 in Parkinson's disease.
  • The research examined a specific mutation that leads to excessive activity of the LRRK2 enzyme, which interferes with neuron signaling by causing striatal cells to lose their primary cilia.
  • Mice consuming the inhibitor for three months restored primary cilia in affected neurons, normalizing hedgehog signaling and initial dopamine nerve ending recovery.
  • Suzanne Pfeffer described the results as "astounding" and said findings suggest treatment might improve, not just stabilize, symptoms if started early in patients.
  • Multiple clinical trials of LRRK2 inhibitors are underway, and the team plans to test if this treatment helps other Parkinson's types and neurodegenerative diseases.
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Medical Xpress broke the news in on Tuesday, July 1, 2025.
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