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Experimental Drug MLi-2 Reverses Parkinson’s-Related Brain Damage

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Parkinson’s disease is currently considered incurable, with modern treatments only alleviating symptoms. However, it may soon be possible to halt the progression of Parkinson’s and restore brain function. Research from Stanford University has shown that the experimental drug MLi-2 successfully restored Parkinson’s-damaged brains in mice. The drug even saved nerve cells that do not reproduce.

The trial was conducted on mice with a genetic form of Parkinson’s disease. This condition damages small, non-motile, hair-like structures on the surface of cells, which are responsible for receiving and transmitting information. Their damage disrupts the connections between nerve cells and hinders the control of movement.

Scientists administered the drug, MLi-2, to mice for three months. MLi-2 blocks LRRK2 kinase, an enzyme that causes the breakdown of these hair-like structures during the disease. The treatment nearly completely restored their number and length, and the damaged nerve cells began to produce neurotrophic factors again. These include GDNF and Neurturin, which help dopaminergic cells survive.

The dopaminergic fiber network was also restored. These fibers play a crucial role in movement speed and coordination. In Parkinson’s disease, this network degrades, leading to impaired movement regulation. As a result of the treatment, the density of dopaminergic fibers almost doubled, which is a significant indicator of restored motor function.

Researchers state that these are the most powerful and comprehensive results obtained so far. The fact that the drug saved even non-reproducing cells was particularly significant. This finding suggests that if treatment is initiated early, it may be possible to restore lost brain functions even in a disease currently considered incurable.

References:

Pfeffer, S. R., Jaimon, E., Lin, Y.-E., Tonelli, F., Antico, O., & Alessi, D. R. (2024). Restoration of striatal neuroprotective pathways by kinase inhibitor treatment of Parkinson’s linked-LRRK2 mutant mice. bioRxiv.

Stanford Medicine. (2025, July 10). Experimental drug reverses Parkinson’s-related damage in mice.

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