New Medicines for Light Chain Amyloidosis
Official title: Probing the Proteinopathy Component of Light Chain Amyloidosis Pharmacologically
Funded at Scripps Research Institute, the · NIH-11132076 · Data updated 2026-11-12
This work looks for new medicines to help stop the harmful protein buildup in people with light chain amyloidosis.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scripps Research Institute, the NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | July 1, 2021 |
| Project end | May 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $747,423 |
| NIH project number | 2R01HL157566-05 |
| Project ID | NIH-11132076 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with light chain amyloidosis, particularly those with kinetically unstable light chains or cardiac involvement, are the focus of this research.
✕Less likely to help
Patients without light chain amyloidosis or those whose condition is not related to light chain misfolding would not directly benefit from this specific approach.
What this research studies
Light chain amyloidosis is a serious condition where abnormal proteins build up and damage organs. Current treatments often don't fully stop the problem, especially for those with heart issues. This project focuses on developing a new type of medicine that directly targets these misfolding proteins. The goal is to stabilize the proteins, preventing them from clumping together and causing further damage. Researchers have already identified a promising new drug candidate in earlier work.
Why it matters
Potential benefit: If successful, this new medicine could offer a different way to treat light chain amyloidosis, potentially improving outcomes for patients who don't respond well to current chemotherapy.
How similar studies have gone: The first phase of this grant successfully identified a clinical candidate, indicating promising initial results for this novel approach.
Where this research is happening
-
Scripps Research Institute, the
La Jolla, United States Active
Researchers
- Principal investigator: Kelly, Jeffery W — Scripps Research Institute, the
- Study coordinator: Kelly, Jeffery W
Related research
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-
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Finding transthyretin heart amyloid from lumbar spine surgery tissue
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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.