Korean researchers prove stem cell therapy’s effectiveness for hereditary cerebellar ataxia in animal models

A team of Korean researchers has successfully verified the therapeutic efficacy of human-derived stem cells in treating hereditary cerebellar ataxia using an animal model.

The Korea Health Industry Development Institute (KHIDI) said that the joint research team, led by Professors Kim Sang-ryong and Suk Kyoung-ho of the Brain Science and Engineering Institute from Kyungpook National University, Lee Ho-won of the Department of Neurology at Kyungpook National University Chilgok Hospital, and Corestem ChemOn, achieved notable results in improving motor functions and protecting neural cells through the administration of human mesenchymal stem cells (hMSCs).

Cerebellar ataxia, akin to dementia and Parkinson’s disease, is a neurodegenerative brain disorder affecting 2.63 million people worldwide. Its exact cause and progression remain largely unknown, making it one of the rare diseases for which no definitive cure exists. Diagnosis is challenging due to the wide variability of symptoms, and treatment options are currently limited.

The researchers used human-derived mesenchymal stem cells known for their anti-inflammatory properties and ability to secrete neuroprotective factors. These stem cells were administered into the subarachnoid space between the cerebellum and medulla of hereditary cerebellar ataxia animal models. The study aimed to explore their potential in suppressing brain inflammation and protecting neural cells.

Results showed a significant increase in neurotrophic factors within the cerebellum of treated models compared to untreated ones.

Notably, untreated models exhibited a nearly 50 percent reduction in neurotrophic factor levels compared to normal models, while treated models demonstrated levels comparable to those of normal subjects. These findings underscore the stem cells’ ability to preserve cerebellar function and protect neural cells.

The researchers also observed an increase in the expression of FSTL1 protein in the cerebellum, which contributed to anti-inflammatory effects and enhanced neural cell preservation.

As a result, treated mice showed restored motor function, returning to normal levels, indicating recovery from neurological damage.

“This study demonstrates the potential of human mesenchymal stem cells as a crucial therapeutic option for cerebellar ataxia, a disease with no effective treatment,” Professor Kim said. “It also provides a foundation for expanding clinical research related to its treatment.”

The findings were published in Stem Cell Research & Therapy.