New panel of cell-based tests may help screen potential treatments

14 September 2024

Illustration of scientist in a lab.

Ncardia has launched a panel of ready-to-use cell-based assays to simplify the screening and selection of treatment candidates for neurodegenerative diseases, including Parkinson’s disease.

The assays are based on human-derived induced pluripotent stem cells (iPSCs), a type of stem cell that can generate nearly all types of cells. The process involves collecting cells from healthy donors or patients, and reprogramming them into a stem-cell-like state in the lab.

Then, using specific chemical or biological cues, iPSCs can be differentiated into different cell types, including dopaminergic neurons, or the damaged and lost nerve cells in Parkinson’s, which can then be used to assess new drugs.

This may enhance the success of new therapies in later stages of clinical development, according to the company.

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‘Major step forward in accelerating drug discovery’

“Our new panel of assays represents a major step forward in accelerating drug discovery,” Jeroen de Groot, PhD, divisional CEO at Ncardia, said in a press release. “By offering assays that are already standardized, we eliminate much of the preliminary work, giving researchers more time to focus on what matters most — progressing fast and confidently with their most promising candidates.”

The new panel allows high throughput, or large-scale, screening at any stage of drug discovery, according to the company. In addition, it can be applied to therapies as diverse as small molecules, gene or RNA therapies, and biologics, which is a type of treatment that uses substances made from living organisms. The company says only minimal optimization is needed to configure the tests to match the needs of each project.

Available readouts for Parkinson’s disease include evaluations of alpha-synuclein aggregation, which is a hallmark feature of Parkinson’s, and phosphorylation (which accelerates the formation of aggregates), as well as neurite growth. Neurites are projections needed for normal nerve cell function and communication.

Other measures include assessing neuronal activity, the activity of the proteasome, a cellular structure where misfolded proteins are degraded, and the release of cytokines, which are molecules that mediate immune and inflammatory responses.

Besides the dopaminergic neurons model, the company also provides a model called Ncyte Neural Mix, where neurons and astrocytes (the brain cells that play a supportive role) are simultaneously generated from iPSCs to form neuronal networks.

“Our shared commitment here is to lead the integration of human iPSC technologies into the drug discovery process,” said Shushant Jain, PhD, Ncardia’s director of discovery technology. “A critical part of that is to give our clients timely, relevant efficacy and toxicity data on their drug candidates. That’s why the new panels include readouts for gene and protein expression [activity], protein aggregation … neuronal health and function, and the release of neurofilament light chain (NF-L), which is an important biomarker for neurodegeneration.”

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