Desmoglein‑3–targeting T‑cell therapy for mucosal pemphigus vulgaris
Immunomodulatory effects of desmoglein 3 chimeric autoantibody receptor T cells (DSG3-CAART) in mucosal pemphigus vulgaris
This work uses a patient's own modified T cells that target desmoglein‑3 to remove the B cells making the harmful antibodies in people with mucosal pemphigus vulgaris.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11289341 on NIH RePORTER |
What this research studies
You would have blood taken so your T cells can be collected and genetically modified to carry a desmoglein‑3 chimeric autoantibody receptor (DSG3‑CAAR). The modified cells are grown in the lab and then infused back into you to seek and kill the B cells that produce the disease‑causing antibodies. The project is an open‑label, dose‑escalation first‑in‑human trial focused on safety, tolerability, antibody levels, and mucosal healing. The aim is to remove only the autoantigen‑specific B cells and spare the rest of the immune system.
Who could benefit from this research
Good fit: Adults with mucosal‑dominant pemphigus vulgaris who have desmoglein‑3 autoantibodies and can undergo apheresis and follow‑up visits are the most likely candidates.
Not a fit: People who lack desmoglein‑3–specific autoantibodies, have only skin‑predominant disease without mucosal involvement, or cannot tolerate cell collection or required monitoring may not benefit.
Why it matters
Potential benefit: It could provide a one‑time, targeted treatment that eliminates the specific B cells causing disease and may lead to durable remission.
How similar studies have performed: CAR T‑cell therapies have cured some B‑cell cancers, CAAR T cells showed strong proof‑of‑concept in preclinical pemphigus models, and this approach is now entering early human testing.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Payne, Aimee S — Columbia University Health Sciences
- Study coordinator: Payne, Aimee S
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.