Using stem cells to model a heart condition for drug testing
hiPSC Modeling of Restrictive Cardiomyopathy for Drug Testing
This study is looking at Restrictive Cardiomyopathy, a heart condition that makes it hard for the heart to fill with blood, and it's using special cells from patients to learn more about the disease and test two new medications that could help, especially for kids who don't have many treatment choices.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-10845660 on NIH RePORTER |
What this research studies
This research focuses on Restrictive Cardiomyopathy (RCM), a serious heart condition that primarily affects the heart's ability to relax and fill with blood. By utilizing human induced pluripotent stem cells (hiPSCs) derived from patients, the researchers aim to create models that mimic the disease, allowing them to study its underlying mechanisms and test new drug therapies. The goal is to better understand how specific genetic mutations contribute to RCM and to evaluate the effectiveness of two promising new medications that may improve heart function. This approach is particularly important for pediatric patients, who often face limited treatment options.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with Restrictive Cardiomyopathy, particularly those with a genetic predisposition to the condition.
Not a fit: Patients with other forms of cardiomyopathy or those without a genetic basis for their heart condition may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatment options for patients with Restrictive Cardiomyopathy, potentially improving their quality of life and outcomes.
How similar studies have performed: While this specific approach using hiPSC models for RCM is novel, similar methodologies have shown promise in other cardiac conditions, indicating potential for success.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Mercola, Mark — Stanford University
- Study coordinator: Mercola, Mark
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.