Targeting the misfolding of light chain amyloidosis proteins to protect organs
Official title: Probing the Proteinopathy Component of Light Chain Amyloidosis Pharmacologically
Funded at Scripps Research Institute, the · NIH-11364960 · Data updated 2026-11-12
A patient-facing overview of a project that tests a small molecule to keep light chain proteins from misfolding and causing organ damage in people with 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 | 2026 |
| Award (this year) | $732,475 |
| NIH project number | 5R01HL157566-06 |
| Project ID | NIH-11364960 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with AL amyloidosis, especially those with cardiac involvement or high risk from protein misfolding, who could benefit from a stabilizing therapy.
✕Less likely to help
Patients without AL amyloidosis or those unlikely to tolerate the stabilizer therapy may not benefit.
What this research studies
This project focuses on a deadly blood cancer called AL amyloidosis, where abnormal light chain proteins can misfold and damage organs. Scientists are developing a molecule that helps these proteins stay properly folded, potentially slowing or preventing organ deterioration. The work builds on prior findings and aims to translate a stabilizer into a therapy that targets the protein problem, alongside existing cancer treatments. If successful, it could offer a new option for patients whose heart or other organs are at risk.
Why it matters
Potential benefit: If successful, it could slow organ damage and improve outcomes for people with light chain amyloidosis.
How similar studies have gone: Previous work showed that stabilizing the light-chain protein can reduce misfolding, suggesting potential benefit, though this specific small-molecule approach is ongoing.
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
Other NIH-funded projects on the same topics.
-
Understanding how kidney-like blood flow may cause amyloid buildup
A research project uses tiny blood-flow devices to study how certain proteins misfold and form amyloid deposits in organs like the kidney and heart.
-
Blood test to detect ATTR amyloidosis earlier using a smart detection kit
A new blood test kit aims to detect ATTR amyloidosis early using a biomarker that appears in patients but not in healthy people.
-
Detection and measurement of amyloid in people carrying the V122I TTR gene variant
This research will look for early signs of heart-related amyloidosis in people with the V122I TTR gene variant using blood tests and a special imaging scan.
-
Identifying Hidden Cardiac Amyloidosis in Black V122I TTR Carriers
This project looks for early signs of heart damage in Black individuals who carry the V122I TTR variant and may benefit from new treatments if caught early.
-
Chip phosphorylation boosts breakdown of mutant TTR to help heart amyloidosis
A scientist-led project aims to help people with a heart disease caused by a mutant protein called transthyretin (TTR) by boosting the body’s protein-cleanup systems to remove the harmful TTR deposits.
-
Finding transthyretin heart amyloid from lumbar spine surgery tissue
This project looks for transthyretin (TTR) amyloid in tissue removed during lumbar spine surgery to find people who may later develop TTR cardiac amyloidosis.
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.