CAR-T (Chimeric Antigen Receptor T-cell) therapy represents one of the most groundbreaking advancements in modern medicine. Originally developed to treat aggressive blood cancers, it is now being explored for autoimmune diseases where the immune system mistakenly attacks the body’s own tissues.
The therapy involves taking a patient’s T cells, modifying them in the laboratory to express a special receptor called a chimeric antigen receptor (CAR), and reinfusing them back into the patient. These engineered T cells can then recognize and eliminate disease-causing cells with precision.¹
The Origins of CAR-T Therapy
The roots of CAR-T therapy trace back to the late 1980s. In 1989, Dr. Zelig Eshhar and his colleagues in Israel developed the first “chimeric” receptor by fusing the binding part of an antibody with the signaling part of a T cell. This discovery combined the targeting ability of antibodies with the killing power of T cells.
Over time, researchers refined the design by adding costimulatory molecules such as CD28 and 4-1BB that made T cells more durable and effective in fighting disease. These modifications laid the foundation for the modern generation of CAR-T therapies.²
Clinical Breakthroughs in Cancer
The major clinical milestone came in the early 2010s when Dr. Carl June’s team at the University of Pennsylvania achieved remarkable results in leukemia patients treated with CD19-directed CAR-T cells. Many patients experienced complete remission after failing all other treatments.
In 2017, the U.S. FDA approved the first CAR-T therapy, tisagenlecleucel (Kymriah) by Novartis, for pediatric and young adult leukemia. This approval marked the beginning of a new era in cell therapy.
Since then, more products have followed:
● Yescarta (Kite/Gilead) for large B-cell lymphoma
● Tecartus (Juno/Gilead) for mantle cell lymphoma
● Breyanzi (J&J) for relapsed large B-cell lymphoma
● Abecma (Bristol Myers Squibb/bluebird bio) for multiple myeloma
● Carvykti (Legend Biotech/Janssen) for multiple myeloma
Today, there are six FDA-approved CAR-T therapies that target either CD19 or BCMA, effectively treating leukemias, lymphomas, and myelomas that were once considered incurable.³
How CAR-T Therapy Works
CAR-T therapy is a personalized treatment that turns the patient’s own immune system into a powerful weapon against disease.
The process involves:
1. Cell Collection: T cells are collected from the patient through leukapheresis.
2. Genetic Engineering: The cells are modified using a viral vector to insert the CAR gene.
3. Cell Expansion: The engineered cells are multiplied in the laboratory until they reach the desired dose.
4. Reinfusion: The CAR-T cells are infused back into the patient to find and destroy target cells.
Once inside the body, these CAR-T cells recognize specific markers, such as CD19, on malignant cells. When they bind to these targets, they become activated, multiply, and release immune molecules that destroy the diseased cells. This targeted attack can lead to long-term remission in many patients.⁴
Expanding Horizons: CAR-T in Autoimmune Diseases
After transforming cancer care, researchers began testing CAR-T therapy for autoimmune diseases where an overactive immune system causes tissue damage. Conditions like systemic lupus erythematosus (SLE), multiple sclerosis (MS), myasthenia gravis, and pemphigus vulgaris are among the leading candidates for CAR-T research.
These diseases often involve faulty B cells that produce harmful autoantibodies. CAR-T therapies that target CD19 can selectively remove these pathogenic B cells and effectively “reset” the immune system.
Early results are very promising. A small German study treated five patients with severe lupus using CD19-directed CAR-T therapy. All five achieved drug-free remission within three months. Autoantibodies disappeared, inflammation subsided, and symptoms resolved. Only mild side effects, such as low-grade fever, were reported.⁵
In another trial involving 15 autoimmune patients, including eight with lupus, all experienced profound improvements with no severe cytokine-release syndrome (CRS) or neurotoxicity. These results suggest CAR-T can induce a lasting immune balance with a favorable safety profile.⁶
Beyond Lupus: Multiple Sclerosis and Other Conditions
In multiple sclerosis, CAR-T cells have shown the ability to cross into the central nervous system and selectively eliminate B cells in cerebrospinal fluid. Companies like Kyverna Therapeutics are conducting larger Phase 2 studies (KYV-101) to evaluate long-term safety and efficacy.
In myasthenia gravis, CAR-T therapy targeting the acetylcholine receptor led to a 70% reduction in disease-causing antibodies in early reports, resulting in significant clinical improvement.⁷
In pemphigus vulgaris, Cabaletta Bio developed a CAR-T candidate called rese-cel that achieved nearly complete B-cell depletion and symptom relief within weeks, even without the need for chemotherapy before treatment.⁸
These trials collectively demonstrate the growing potential of CAR-T beyond oncology, offering durable remission where traditional therapies have failed.
Industry Landscape and Key Players
The CAR-T field is now expanding rapidly into autoimmune indications.
In oncology, leaders include Novartis, Gilead/Kite, Johnson & Johnson, Bristol Myers Squibb, bluebird bio, and Legend Biotech.
In autoimmunity, emerging innovators are taking the lead:
● Kyverna Therapeutics (developing CD19 CAR-T for lupus and MS)
● Cabaletta Bio (pioneered CAR-T in pemphigus vulgaris)
● Autolus Therapeutics (investigating CAR-T for lupus nephritis)
● Academic partnerships from University of Pennsylvania, Stanford, and Dana-Farber Cancer Institute
Efforts are also underway to create allogeneic or “off-the-shelf” CAR-T therapies, which could be mass-produced rather than customized for each patient, significantly reducing cost and wait times.⁹
Challenges and Safety Considerations
While CAR-T therapy has shown remarkable promise, several challenges remain:
● Complex Manufacturing: Each CAR-T dose is made from a patient’s own cells, requiring weeks of production under strict quality control.
● High Cost: The first approved CAR-T therapy, Kymriah, costs approximately $475,000 per treatment, reflecting the resource-intensive process.
● Safety Concerns: Side effects such as cytokine-release syndrome (CRS) and immune effector cell-associated neurotoxicity (ICANS) can occur, though these are typically mild in autoimmune patients.
● Regulatory Pathways: Most current autoimmune CAR-T data come from small case studies. Larger controlled trials are essential before regulatory approvals.
Despite these challenges, safety results so far have been encouraging, with only mild and manageable adverse effects observed in autoimmune trials.
Market Outlook and Future Potential
The global CAR-T market was valued at $4.65 billion in 2024 and is projected to reach $15.97 billion by 2030, growing at an estimated 22% CAGR.¹º
This growth is driven by expanding indications, improved technologies, and ongoing partnerships between major pharmaceutical and biotech companies. As CAR-T moves beyond oncology into chronic immune diseases, the potential patient pool will grow exponentially.
If ongoing trials confirm its effectiveness, CAR-T could redefine treatment for diseases like lupus, MS, and myasthenia gravis, offering long-term remission instead of lifelong symptom control.
Conclusion
CAR-T therapy has evolved from a revolutionary cancer treatment into a promising solution for autoimmune diseases once thought untreatable. Early studies show that CAR-T can “reset” the immune system and achieve long-lasting remission with manageable side effects.
The road ahead includes challenges such as high costs and complex manufacturing, but innovation in automation, off-the-shelf models, and regulatory support may help overcome these barriers. With a projected global market exceeding $15 billion by 2030, CAR-T is poised to become a cornerstone therapy in both oncology and immunology.
CAR-T therapies, originally developed for blood cancers, are now showing exceptional promise in autoimmune diseases such as lupus and multiple sclerosis. Early studies demonstrate drug-free remissions and a favorable safety profile. Although challenges such as cost and scalability remain, ongoing investment and regulatory progress suggest CAR-T could soon transform immune-based medicine worldwide.
Frequently Asked Questions (FAQ)
1. What is CAR-T therapy?
CAR-T therapy is a personalized immunotherapy in which a patient’s T cells are engineered to recognize and destroy disease-causing cells.
2. How does CAR-T work in cancer?
The modified T cells detect specific antigens such as CD19 on cancer cells and activate an immune attack that destroys them, often leading to remission.
3. Which CAR-T products are FDA-approved?
As of 2024, six therapies are approved: Kymriah, Yescarta, Tecartus, Breyanzi, Abecma, and Carvykti.
4. Why is CAR-T being tested in autoimmune diseases?
Because many autoimmune diseases are driven by faulty B cells, CAR-T can remove these cells and restore immune balance.
5. Is CAR-T safe for autoimmune patients?
Yes, early trials show only mild side effects such as fever or mild inflammation, with no major neurotoxicity.
6. Who is leading CAR-T development in autoimmunity?
Kyverna Therapeutics, Cabaletta Bio, and Autolus Therapeutics are key biotech players, supported by major academic institutions.
7. Why is CAR-T therapy expensive?
Each treatment is customized using a patient’s own cells, requiring advanced facilities, technology, and time.
8. Can CAR-T therapy be repeated?
Possibly. Some patients in early studies achieved long-lasting remission after one dose, but retreatment strategies are still under evaluation.
9. What are the most common side effects?
Cytokine-release syndrome and mild immune suppression are most common but are typically manageable.
References
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2. Patel KK, Tariveranmoshabad M, Kadu S, Shobaki N, June C. From concept to cure: The evolution of CAR-T cell therapy. Molecular Therapy. 2025;33(5). doi:https://doi.org/10.1016/j.ymthe.2025.03.005
3. Ong MZ, Kimberly SA, Lee WH, et al. FDA-approved CAR T-cell Therapy: A Decade of Progress and Challenges. Current pharmaceutical biotechnology. 2024;25(11):1377-1393. doi:https://doi.org/10.2174/0113892010257212231001082741
4. Neagu M, Constantin C. CAR-T Cells – Main Steps for Obtaining a Proper “Live Drug” Adoptive Therapy. South East European Journal of Immunology. 2024;7:13-20. doi:https://doi.org/10.3889/seejim.2024.6063
5. Mackensen A, Müller F, Mougiakakos D, et al. Anti-CD19 CAR T Cell Therapy for Refractory Systemic Lupus Erythematosus. Nature Medicine. 2022;28(10):1-9. doi:https://doi.org/10.1038/s41591-022-02017-5
6. Müller F, Taubmann J, Bucci L, et al. CD19 CAR T-Cell Therapy in Autoimmune Disease — A Case Series with Follow-up. The New England Journal of Medicine. 2024;390(8):687-700. doi:https://doi.org/10.1056/nejmoa2308917
7. Chinas NA, Alexopoulos H. CAR T-cells meet autoimmune neurological diseases: a new dawn for therapy. Frontiers in immunology. 2025;16:1604174. doi:https://doi.org/10.3389/fimmu.2025.1604174
8. Georg Schett, Müller F, Taubmann J, et al. Advancements and challenges in CAR T cell therapy in autoimmune diseases. Nature Reviews Rheumatology. 2024;20. doi:https://doi.org/10.1038/s41584-024-01139-z
9. Barrett D, Digaudio D. The landscape of cell and gene therapy today. Molecular Therapy. 2025;33(6):2316-2323. doi:https://doi.org/10.1016/j.ymthe.2025.05.006
10. CAR T-cell Therapy Market Size And Share [2023 Report]. www.grandviewresearch.com. https://www.grandviewresearch.com/industry-analysis/car-t-cell-therapy-market-report
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