New cell therapy for treating IPEX syndrome
Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome [revised IND and clinical protocol to be submitted to FDA by 10/11/2021]
This study is testing a new treatment for IPEX syndrome that uses your own modified immune cells to help improve your immune system and reduce symptoms, and it's designed for people with this rare autoimmune disease.
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-10893400 on NIH RePORTER |
What this research studies
This research investigates a novel therapy using a patient's own modified T cells to treat IPEX syndrome, a rare autoimmune disease caused by genetic mutations. The therapy involves taking CD4+ T cells from the patient, modifying them in the lab to express a healthy version of the FOXP3 gene, and then reintroducing these cells back into the patient. The goal is to restore proper immune function and reduce the autoimmune symptoms associated with IPEX syndrome. This Phase 1 trial will assess the safety and feasibility of this approach in a small group of participants.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with IPEX syndrome who have not responded to existing treatments.
Not a fit: Patients with IPEX syndrome who are not eligible for the trial due to specific health conditions or those who have already undergone other treatments may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new, effective treatment option for patients with IPEX syndrome, potentially improving their quality of life and health outcomes.
How similar studies have performed: While this approach is novel, similar cell therapies have shown promise in treating other autoimmune diseases, indicating potential for success.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Bacchetta, Rosa — Stanford University
- Study coordinator: Bacchetta, Rosa
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.