On the 21st, the Ministry of Health and Welfare said it ruled three of eight clinical studies "appropriate" at the "11th Advanced Regenerative Medicine and Advanced Biopharmaceuticals Review Committee for 2025." 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 stem cell therapy studies.
Once administered, CAR-T cells proliferate in the body and continue killing cancer cells, earning 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) are blood cancer treatments. Recently, following solid tumors in organs such as breast and lung cancers, there is new hope that even intractable chronic autoimmune diseases can be treated with CAR-T cells.
As treatment areas expand, the CAR-T cell market is also steadily growing. 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 is projected to surge to over 256 trillion won by 2034.
◇"Symptoms disappeared" confirms possibility of a cure
In September, a research team led by Professor Markus Neurath at Friedrich-Alexander-Universität (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 the autoimmune disease ulcerative colitis produced sustained symptom relief for 14 consecutive weeks. The patient no longer needed medication and was able to return to work.
Since 2017, when the FDA approved Kymriah from Novartis of Switzerland, through last year's Aucatzyl from Autolus of the United Kingdom, it has approved seven CAR-T Therapy products. All were authorized to treat blood cancers, in which immune cells that should protect the body become cancer cells.
CAR-T cells are T cells that carry a "chimeric antigen receptor (CAR)." Like the chimera of Greek mythology, which had features of multiple animals, immune T cells are equipped with a protein that binds to antigens on the surface of cancer cells. T cells are extracted from a patient's blood, engineered ex vivo with the CAR gene, and then readministered.
T cells encircle invaders and either summon other immune cells or eliminate them directly. T cells bearing the CAR protein are like existing soldiers now equipped with the intelligence to find the enemy. CAR-T cell therapies bind to the CD19 protein on the surface of diseased B cells 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 fall into the same category.
B cells normally secrete antibodies to trigger immune responses. Autoimmune diseases arise when B cells malfunction and antibodies fail to distinguish normal cells. The Erlangen University Hospital team treated ulcerative colitis by using CAR-T cells that bind CD19 on B cells to block antibodies that attack even healthy cells.
◇Results in autoimmune disease treatment 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, producing positive results. In 2021, a team led by Professor Georg Schett at FAU in Germany administered CAR-T cells to a 20-year-old female patient with systemic lupus erythematosus, the first such case among autoimmune disease patients.
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 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 the same therapeutic effect using donor T cells. In May, a team led by immunologist Bing Du at East China Normal University reported in Cell Research that CAR-T treatment using donated T cells successfully treated lupus patients who had not responded 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 hailed 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 and colleagues also succeeded in treating patients with myositis and systemic sclerosis using CAR-T Therapy made from donated cells.
Carl June, a pioneer of CAR-T at the University of Pennsylvania Perelman School of Medicine, reported in Science in October that he had developed a method for patients to produce CAR T cells directly inside the body. Instead of inserting genes into T cells ex vivo, the approach delivers messenger RNA (mRNA) carrying the genetic instructions directly into T cells in the body, similar to a COVID-19 vaccine.
With clinical trials succeeding in succession and advances in how therapies are made and delivered, the spread of CAR-T treatment is expected to accelerate. Neurath told the journal Nature, "In the next few years, CAR T-cell therapy will become widespread in the treatment of chronic autoimmune diseases," adding, "Given that several teams have already reported remarkable results in lupus treatment, I am confident CAR-T therapy will soon become a standard of care."
References
New England Journal of Medicine (2025), 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