The path from a promising drug candidate to an approved therapy is long, expensive, and uncertain. A significant proportion of drug candidates that show promise in early testing fail when they reach human clinical trials, often because the models used to evaluate them in the lab did not accurately reflect human biology. Improving the predictive accuracy of preclinical testing is one of the most important opportunities in modern drug development, and advances in cell models are increasingly rising to meet it.
Why Cell Models Are Essential for Drug Development
Before a drug candidate can be tested in humans, researchers need to understand how it behaves in living biological systems; whether it reaches its target, whether it has the intended effect, and whether it causes harm. Cell models provide a controllable, reproducible environment for answering these questions at scale, making them indispensable tools across the drug discovery pipeline.
Animal models have historically played a central role in preclinical testing, but they have well-documented limitations. Species differences in metabolism, receptor biology, and immune function mean that results in animals often do not translate reliably to human outcomes. The financial, logistical, and ethical costs of animal-based research are also significant. These limitations have driven growing demand for cell-based preclinical disease models that better reflect human physiology, and that can be used earlier and more efficiently in the development process.
What Are In Vitro Models?
In vitro models are cell-based systems studied outside a living organism, typically in culture dishes, flasks, or multi-well plates under controlled laboratory conditions. They allow researchers to observe how cells respond to drug candidates, toxins, or disease-relevant conditions in a reproducible and scalable way.
Traditional in vitro models rely on two-dimensional (2D) cell culture, in which cells grow as a flat monolayer on a treated surface. 2D culture has significant practical advantages: it is straightforward to establish, cost-effective, and well-suited to high-throughput screening. However, it has recognized limitations as a model of human tissue. Cells grown in a monolayer lack the three-dimensional architecture, cell-cell interactions, and extracellular matrix environment that characterize real tissue, factors that can significantly influence how cells respond to drugs and how diseases progress. The gap between 2D in vitro results and clinical outcomes remains a persistent challenge in drug development, and has motivated the development of more sophisticated in vitro model systems.
The Rise of Human Cell Models
A parallel and related challenge has been the use of non-human and non-disease-relevant cell lines in preclinical research. Many widely used cell lines are derived from cancer cells or non-human species, which introduces additional layers of biological distance between the model and the human patient.
The shift toward human cell models, particularly those derived from human induced pluripotent stem cells (iPSCs), has addressed many of these limitations. iPSC-derived human cell models can be generated from human donors, including patients with the specific disease being studied, making them genetically and physiologically relevant in a way that animal-derived or cancer cell line models are not. They also offer practical advantages: unlike primary human cells, which are limited by donor availability and batch-to-batch variability, iPSC-derived cell lines can be produced at scale with consistent quality. For researchers working on iPSC disease models, this combination of human relevance and scalability represents a significant advance over earlier approaches.
Advances in 3D Cell Culture and 3D Cell Models
Alongside the move toward human cell models, the field has seen rapid development in three-dimensional cell culture systems. 3D cell models better replicate the architecture and microenvironment of real tissue than their 2D counterparts, capturing the cell-cell and cell-matrix interactions that influence how cells behave in vivo.
The most widely used 3D cell model formats include spheroids, self-aggregating clusters of cells that form a roughly spherical structure, and organoids, which are more complex self-organizing structures that can replicate aspects of organ-level architecture. Scaffold-based systems, in which cells are seeded into a three-dimensional matrix material, offer another approach for applications where structural support is important.
The predictive value of 3D cell culture for drug testing is increasingly well established. 3D models show improved recapitulation of drug penetration, cell viability gradients, and resistance mechanisms compared to 2D systems, making them particularly valuable for oncology research and, increasingly, for metabolic and endocrine disease modeling. As manufacturing processes for 3D cell models continue to mature, their use in both drug discovery and toxicity screening is expected to grow substantially.
Human Cell Models in Diabetes and Pancreatic Disease Research
Diabetes research has historically faced particular challenges in the cell model space. The gold standard for in vitro pancreatic research has long been cadaver-derived islet cells, clusters of insulin-producing and hormone-regulating cells isolated from donor pancreases. While valuable, cadaver-derived islets are constrained by significant practical limitations: donor availability is unpredictable, cell quality varies between donors, and costs are high. These factors make it difficult to conduct the large-scale, reproducible studies that drug discovery programs require.
Animal-derived pancreatic models present their own limitations. Rodent islets, while widely used, differ from human islets in important ways, including cellular composition, glucose sensitivity, and insulin secretion dynamics. This reduces their translational relevance for human diabetes research.
iPSC-derived islet cell models offer a compelling solution to both sets of limitations. By generating pancreatic islet-like cells from human iPSC lines, researchers gain access to a scalable, consistent, and human-relevant cell source for iPSC drug discovery applications, including drug candidate screening, efficacy testing, and toxicity assessment. The ability to derive cells from patients with type 1 or type 2 diabetes also opens the door to disease-specific modeling that was not previously possible at scale.
RMS’s Human Cell Models for Diabetes Research
Regenerative Medical Solutions (RMS) has developed proprietary iPSC-derived cell technology specifically designed to address the limitations of existing pancreatic cell models. RMS’s Islet-Like Clusters (ILCs) are produced from human iPSC lines and are morphologically and functionally similar to human pancreatic islets, containing insulin-producing beta cells alongside glucagon-producing alpha cells and somatostatin-producing delta cells, mirroring the cellular composition of the native islet.
RMS is a privately held biotech company that offers its ILC technology through two research products. The ILC Cell Kit provides ready-to-use single-cell ILCs intended for in vitro cell studies and drug development applications including drug discovery, efficacy testing, and toxicity screening. The ProgenMix™ media formulation is designed to enable researchers to cultivate pancreatic cells more efficiently, supporting laboratories that wish to culture their own ILC populations.
Because RMS’s ILCs are produced from human iPSC lines, they offer scalable production capacity, consistent cell quality, and on-demand supply, addressing the donor variability, limited availability, and high cost that constrain cadaver-derived islet models. For pharmaceutical companies and research institutions working in diabetes and pancreatic disease, these products provide a more reliable and human-relevant platform for preclinical research.
RMS’s cell model technology is underpinned by the same iPSC research program that is driving the company’s prospective therapeutic approach to diabetes, backed by over two decades of foundational research from Dr. Jon Odorico’s laboratory at the University of Wisconsin, a strong patent portfolio, and NIH grant funding.
To learn more about RMS’s ILC Cell Kit and ProgenMix™ media formulation, or to discuss research collaboration and partnership opportunities, contact our team.
Frequently Asked Questions About Cell Models in Drug Discovery
What is a cell model in drug discovery? A cell model is an in vitro model, a biological system using living cells grown in a laboratory, to study disease mechanisms, test drug candidates, and assess toxicity. Cell models allow researchers to observe how potential therapies interact with human-relevant biology before progressing to clinical trials.
What is the difference between 2D and 3D cell culture? In 2D cell culture, cells grow as a flat monolayer on a treated surface. In 3D cell culture, cells are grown in three-dimensional structures, such as spheroids or organoids, that better replicate the architecture and microenvironment of real tissue. 3D cell models generally offer improved physiological relevance and predictive value for drug testing.
Why are human cell models important for drug development? Human cell models more accurately reflect the biology of human patients than animal-derived or cancer cell line models, reducing the species translation gap that contributes to high failure rates in clinical trials. iPSC-derived human cell models offer the additional advantage of being scalable and consistent, unlike primary human cells which are limited by donor availability.
What are the limitations of cadaver-derived islet cells for diabetes research? Cadaver-derived islet cells are constrained by unpredictable donor availability, significant batch-to-batch variability in cell quality, and high procurement costs. These factors make it difficult to conduct the large-scale, reproducible studies that drug discovery programs require.
How are iPSC-derived cell models used in diabetes research? iPSC-derived islet cell models provide a scalable, consistent, and human-relevant source of pancreatic cells for drug discovery, efficacy testing, and toxicity screening. They can also be derived from patients with type 1 or type 2 diabetes, enabling disease-specific modeling that was not previously achievable at scale.
What cell model products does RMS offer? RMS offers the ILC Cell Kit, containing iPSC-derived Islet-Like Clusters for in vitro and drug development applications, and ProgenMix™, a media formulation designed to support more efficient cultivation of pancreatic cells. Both products are intended for research use in diabetes and pancreatic disease.