On the 21st, the Ministry of Health and Welfare said the 11th Advanced Regenerative Medicine and Advanced Biopharmaceuticals Review Committee of 2025 ruled three of eight clinical studies as "appropriate." One of them is a clinical trial administering CD19 CAR-T cells to pediatric and adolescent patients with refractory systemic lupus erythematosus (SLE). The others were studies on stem cell therapies.
Once administered, CAR-T cells proliferate and keep killing cancer cells, earning them the nicknames "living drug" and "serial killer" of cancer cells. All seven CAR-T cell products approved by the U.S. Food and Drug Administration (FDA) treat blood cancers. Recently, there is rising hope that CAR-T cells may treat not only solid tumors in organs such as breast and lung cancers but also hard-to-treat chronic autoimmune diseases.
As the scope of treatment expands, the CAR-T cell market continues to grow. According to global market research firm Polaris Market Research & Consulting, CAR-T Therapy formed a market of about 10 trillion won last year, is expected to exceed 15 trillion won this year, and to surge to more than 256 trillion won by 2034.
◇"Symptoms disappeared" possibility of a cure confirmed
In Sept., a research team led by Markus Neurath at Friedrich-Alexander University (FAU) Erlangen University Hospital in Germany reported in the New England Journal of Medicine (NEJM) that administering CAR-T cells to a 21-year-old female patient with ulcerative colitis, an autoimmune disease, produced symptom relief for 14 consecutive weeks. The patient no longer needed medication and was able to return to work.
Since 2017, from Kymriah by Novartis of Switzerland to Aucatzyl by Autolus of the United Kingdom last year, the FDA has approved seven CAR-T Therapy products. All were authorized for treating blood cancers in which immune cells that should protect the body turn into cancer cells.
CAR-T cells are T cells with a "chimeric antigen receptor (CAR)." Like the chimera in Greek mythology that combines features of several animals, T cells, which are immune cells, are endowed with a protein that binds to antigens on the surface of cancer cells. T cells are extracted from a patient's blood, engineered outside the body by inserting the CAR gene, and then infused back.
T cells encircle invaders and summon other immune cells or remove them directly. T cells with the CAR protein are like existing fighters now equipped with the intelligence to find the enemy. CAR-T cell therapies bind to the CD19 protein on the surface of B cells—immune cells that have become cancerous—and destroy them.
Systemic lupus erythematosus, for which the ministry approved a CAR-T clinical trial, is an autoimmune disease that occurs when immune cells mistake normal cells for invaders and attack them. Crohn's disease, ulcerative colitis, and rheumatoid arthritis are similar.
B cells normally secrete antibodies to trigger immune responses. Autoimmune diseases occur when B cells malfunction and antibodies no longer distinguish normal cells. The Erlangen University Hospital team treated ulcerative colitis by using CAR-T cells that bind to CD19 on B cells to block antibodies that attack even normal cells.
◇Progress in treating autoimmune diseases over the past 3 years
Over the past three years, scientists have conducted 12 CAR-T cell clinical trials in patients with severe autoimmune diseases such as ulcerative colitis, rheumatoid arthritis, and lupus, yielding positive results. In 2021, a research team led by Georg Schett at FAU in Germany administered CAR-T cells to a 20-year-old female patient with systemic lupus erythematosus, the first time in an autoimmune disease patient.
Lupus is Latin for wolf. The disease was named because its hallmark skin rash looks like a bite mark from a wolf. Since then, CAR-T therapy has entered phase 1–2 clinical trials in patients with systemic sclerosis, myositis, and rheumatoid arthritis. Phase 3 trials are also underway for lupus and myasthenia gravis.
CAR-T technology is also advancing. Until now, CAR-T therapy used a patient's own T cells. Chinese researchers have confirmed equivalent effects using donor T cells. In May, an immunology team led by Bing Du at East China Normal University reported in Cell Research that CAR-T therapy using donated T cells succeeded in treating lupus patients who did not respond to existing drugs. One of four female patients reached remission after three months and no longer needed medication. The other three received low-dose steroids as maintenance therapy.
The scientific community assessed the Chinese team's achievement as akin to finding a generic for CAR-T therapy. If CAR-T cells are manufactured in advance from donated cells, patients can receive treatment immediately without hospitalization for cell collection, gene insertion, and cell culture. Last year, Du also succeeded in treating patients with myositis and systemic sclerosis using CAR-T Therapy made from donated cells.
Carl June of the Perelman School of Medicine at the University of Pennsylvania, a pioneer of CAR-T, reported in Oct. in the journal Science that his team developed a method for patients to produce CAR T cells directly in the body. Instead of inserting genes into T cells outside the body, the approach delivers messenger RNA (mRNA) carrying the genetic instructions directly to T cells in the body, similar to a COVID-19 vaccine.
With clinical trials succeeding in succession and methods of producing and delivering therapies advancing, the spread of CAR-T therapy is expected to accelerate. Neurath told the journal Nature, "In the coming years, CAR T cell therapy will become standard in the treatment of chronic autoimmune diseases," adding, "Given the remarkable results already reported by several groups in lupus treatment, I am confident CAR-T therapy will soon become a standard of care."
References
New England Journal of Medicine (2021), DOI: https://doi.org/10.1056/NEJMc2508023
Cell Research (2025), DOI: https://doi.org/10.1038/s41422-025-01128-1
Science (2025), DOI: https://doi.org/10.1126/science.ads8473
Cell (2024), DOI: https://doi.org/10.1016/j.cell.2024.06.027
New England Journal of Medicine (2021), DOI: https://doi.org/10.1056/NEJMc2107725