Finding New Treatments for Restrictive Cardiomyopathy
hiPSC Modeling of Restrictive Cardiomyopathy for Drug Testing
['FUNDING_R01'] · STANFORD UNIVERSITY · NIH-11137666
This project uses special heart cells grown from patients to better understand Restrictive Cardiomyopathy and discover new treatment options.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | STANFORD UNIVERSITY (nih funded) |
| Locations | 1 site (STANFORD, UNITED STATES) |
| Trial ID | NIH-11137666 on ClinicalTrials.gov |
What this research studies
Restrictive Cardiomyopathy (RCM) is a serious heart condition, particularly in children, with very few treatment options beyond heart transplantation. This project creates miniature heart models in the lab using special stem cells derived from RCM patients. By studying these patient-specific heart cells, researchers aim to understand the disease's unique features and how different genetic changes contribute to it. The team will also test two promising new drugs to see if they can improve heart function in these RCM models, offering hope for future therapies.
Who could benefit from this research
Good fit: This research focuses on understanding Restrictive Cardiomyopathy, particularly in patients with specific genetic mutations like those in cardiac Troponin-T (TNNT2).
Not a fit: Patients with other forms of cardiomyopathy or heart conditions not related to Restrictive Cardiomyopathy may not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could lead to new medications that improve heart function and quality of life for patients with Restrictive Cardiomyopathy, potentially reducing the need for heart transplantation.
How similar studies have performed: While hiPSC models have been used for other heart conditions, this is a novel approach for studying Restrictive Cardiomyopathy and testing potential treatments.
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.
Conditions: Cardiac Diseases, Cardiac Disorders