CAR T-Cell Therapy for Autoimmune Diseases

Autoimmune diseases remain an area of unmet medical need. Most autoimmune diseases lack targeted therapies and rely on broad immunosuppressants or monoclonal antibodies, which carry risks like infection, organ toxicity, and long-term side effects.

As the landscape of autoimmune and inflammatory diseases evolves, so does the urgency to deliver targeted transformative therapies. One of the most promising frontiers CAR T-cell therapy − a modality once confined to oncology is now making strides in conditions like systemic lupus erythematosus (SLE), rheumatoid arthritis, and myositis.

How CAR T Works: Resetting the Immune System

Autoimmunity occurs when autoantibodies and autoreactive cytotoxic T cells mistakenly attack self-antigens, leading to tissue damage, inflammation, and organ damage. CAR T-cell therapies offer a more precise approach by targeting and eliminating pathogenic B cells (e.g., anti-CD19 CAR T) or modulating T-cell activity by potentially resetting the immune system in conditions like SLE and rheumatoid arthritis. These therapies commonly target to remove dysfunctional B cells responsible for autoantibody production.

CAR T therapy offers a novel approach to reset the immune system by targeting pathogenic B cells and autoreactive T cells. With over 1500 non-oncologic CAR T trials globally, the field is rapidly expanding. This shift presents both opportunities and challenges for biotech innovators, particularly in navigating the operational and regulatory complexities of early-phase trials.

Operational challenges and considerations in CAR T trials

Medical science and technology have spurred development of these unique therapies, to address the unique demands of these studies, several core capabilities are essential:

1. Operational Expertise: Multidisciplinary team with CAR-T and autoimmune experience to manage complex site infrastructure.
2. Regulatory Strategy: Tailored site-level submission planning, including IRB/IBC coordination and CMC readiness.
3. Risk Management: Proactive planning for sites, external vendors involved, study materials required, autologous CAR-T product pathway, and other factors for risk mitigation.
4. Site Selection: Accommodating the complexity of the CAR-T processes, having access to the required SLE population, working seamlessly, and having interdisciplinary capabilities (rheumatology, hematology, apheresis).
5. Flexibility: Ability to adapt to protocol amendments—average of seven per early-phase CGT study.
6. Logistics: Specialized support for time-sensitive apheresis and CAR-T product delivery, including trained logistics teams and vendor partnerships.
7. Data Quality: CGT-specific tools to ensure clean, real-time data during dose escalation and safety reviews.

What makes a site suitable for CAR T trials?

Selecting well-resourced sites is key to successful CAR T studies. Study sites must be capable of accommodating the complexity of the CAR T processes, have access to the required study population, and be able to work seamlessly with an interdisciplinary, interdepartmental approach within a high operational burden.

Read more about Fortrea’s Autoimmune Therapy solutions

References

• 1. Khan SH, Choi Y, Veena M, Lee JK, Shin DS. Advances in CAR T cell therapy: antigen selection, modifications, and current trials for solid tumors. Front Immunol. 2025 Jan 6;15:1489827.
• 2. Cao LY, Zhao Y, Chen Y, Ma P, Xie JC, Pan XM, Zhang X, Chen YC, Wang Q, Xie LL. CAR-T cell therapy clinical trials: global progress, challenges, and future directions from ClinicalTrials.gov insights. Front Immunol. 2025 May 20;16:1583116.
• 3. Citeline Trialtrove database. Active non-oncologic CAR-T clinical trials as of January 2025. Trialtrove; accessed June 2025.