Nerve Cell Lines Derived From Stem Cells of PPMI Study Donors Created
Fujifilm Cellular Dynamics reported that it has developed, using stem cells from people with Parkinson’s disease, dopaminergic neurons containing two common, disease-associated mutations for use in Parkinson’s research. Stem cell donors were patients involved in the Parkinson’s Progression Markers Initiative (PPMI) through the Michael J. Fox Foundation for Parkinson’s Research (MJFF).
Called iCell DopaNeurons, these cells can be used as tools for advancing work into potential new Parkinson’s therapies, Fujifilm stated in a press release. Mutations in the leucine-rich repeat kinase-2 (LRRK2) and glucocerebrosidase (GBA) genes have been linked to an increased risk of Parkinson’s. Fujifilm used stem cells from patients with either the G2019S mutation in the LRRK2 gene or the N370S mutation in GBA gene to generate dopamine-producing nerve cells, those mostly affected in Parkinson’s.
The company reported that these cells exhibit many of the features of patients’ cells, including altered dopamine metabolism, increased accumulation of the toxic alpha synuclein protein, and greater nerve cell loss compared with control cells harboring no mutations. These cells can be used to study disease mechanisms and screen potential therapeutics. They anticipate human stem cell-derived disease models, such as the iCell DopaNeurons for LRRK2 and GBA mutations, will become the standard for research and development of disease modifying therapeutics for Parkinson’s disease patients.
PPMI, also known as PPMI 2.0, is a longitudinal, observational study of people with and without Parkinson’s disease. At participating medical sites, clinical, genetic, imaging, and other biological data are collected with the aim of identifying biomarkers associated with Parkinson’s risk and progression. The hope is that information gained through PPMI will inform the development of better treatments. Last year, PPMI launched an online survey platform, called PPMI Online, to supplement this work.
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