Better Cell Models Are Breaking Barriers in Neurodegenerative Research

Cell & Gene Therapy
Mar 19, 2025  |  5 min read

Neurodegenerative diseases are as devastating as they are complex. These conditions gradually chip away at the structure and function of neurons, leading to declines in motor skills, memory, and cognitive abilities—changes that profoundly impact the lives of patients and their loved ones. Here’s a quick look at how better cell models are helping speed up progress. 
   

 

The Burden of Gradual Decay

Alzheimer's and Parkinson's diseases are among the most common neurodegenerative disorders, each presenting distinct challenges. Alzheimer’s slowly steals memories and cognitive abilities, while Parkinson’s is known for its hallmark motor symptoms, like tremors and stiffness. The ripple effects of these diseases are immense—not just for the millions of individuals directly affected, but also for their families and the healthcare systems that support them. 

For perspective, The Alzheimer's Association estimates that 6.7 million Americans aged 65 and older live with Alzheimer’s dementia, while Parkinson’s disease affects nearly 1 million people in the United States, according to the Parkinson’s Foundation.

 

A Need for Better Treatments

Current treatments for neurodegenerative diseases offer limited hope. Most approved medications can temporarily ease symptoms but fail to address the root causes or halt disease progression. For example, cholinesterase inhibitors used in Alzheimer’s may improve memory or judgment in the short term, but they do nothing to stop neuronal  loss. Similarly, while dopamine precursors help manage Parkinson’s motor symptoms, they cannot prevent the ongoing cellular damage that drives the disease forward.

This gap highlights the urgent need for therapies that go beyond symptom management to target the underlying mechanisms of these diseases.

 

Overcoming Barriers to Progress

Developing effective treatments for neurodegenerative diseases brings its own set of battles. One significant problem is the blood-brain barrier (BBB), which acts as a formidable gatekeeper, blocking nearly 99% of substances from reaching the brain. While the BBB is essential for protecting brain health, it also makes drug delivery to the central nervous system incredibly difficult.

Another obstacle lies in research models. Current options often fall short of mimicking human neurodegenerative diseases accurately. Primary neuronal cultures—sourced from post-mortem tissues—are limited by availability, a short lifespan, and the complexity of their handling. Immortalized cell lines, though easier to work with, often fail to replicate the behavior of actual neurons or the brain's 3D structure. Animal models frequently produce results that don’t translate well to human clinical trials, contributing to high failure rates.

To address these issues, researchers are turning to advanced cell culture systems.

 

Advanced Cell Models Offer New Hope

Cutting-edge cell models like induced pluripotent stem cells (iPSCs) and 3D cultures are transforming neurodegenerative disease research.

iPSCs allow scientists to model diseases and study drug responses in ways tailored to a patient’s unique genetic makeup. This personalized approach is invaluable for understanding how diseases evolve and how treatments might perform over time. iPSCs are also long-lasting in culture, enabling deeper investigations into disease progression and treatment efficacy.

3D cultures, including iPSC-derived brain organoids and spheroids, add another layer of sophistication. These models closely mimic the structure and function of the brain, creating a more realistic environment for studying complex diseases. Their scalability makes them particularly useful for high-throughput drug testing, accelerating the path to new therapies.

Advanced models like iPSCs help address ethical concerns too. Unlike embryonic stem cells, iPSCs are derived from adult cells, offering a more ethical and sustainable research option. By overcoming the limitations of older models, these tools are pushing the boundaries of what’s possible in neurodegenerative disease research.

 

Delivering on the Promise

While the potential of iPSCs and 3D cultures is exciting, obstacles remain. Standardizing iPSC culture protocols and scaling up production for high-throughput drug screening are critical steps. Validation studies are also essential to ensure that findings from these models translate effectively to human biology and clinical settings.

Companies like Sartorius are stepping up to meet these challenges. By providing innovative analytical solutions for advanced cell systems, we are helping researchers streamline workflows and gen erate translational insights. These technologies are bridging the gap between research and clinical outcomes, advancing the development of effective treatments—and offering new hope to patients and their families worldwide. 

 

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