The Highly Anticipated "CAR-T Therapy": Which Diseases Is It Actually Suitable For?

A patient with advanced leukemia relapsed after multiple rounds of chemotherapy, their life hanging by a thread. However, a new type of treatment turned the immune cells inside their body into "super killers," precisely eliminating cancer cells and ultimately achieving the miracle of complete tumor disappearance.

This miracle-working treatment is the recent breakthrough in the field of cancer therapy: CAR-T cell therapy. It has been hailed as the fourth major cancer treatment modality following surgery, radiotherapy and chemotherapy.

Unlike traditional treatments, CAR-T therapy does not rely on external drugs or radiation to attack cancer cells. Instead, it empowers the body's own immune system to become a "living weapon" for precise disease targeting.

01 What is CAR-T?: Installing a "GPS" in Your Body's Immune Cells

CAR-T stands for Chimeric Antigen Receptor T-cell therapy. Its core concept is "borrowing troops to defeat the enemy": T cells (a crucial type of immune cell) are extracted from the patient's blood, genetically engineered and multiplied in vitro, and then reinfused back into the patient [1].

Simply put, scientists install a "GPS navigation system" named "CAR" onto these T cells. This navigation system can precisely recognize specific proteins on the surface of cancer cells.

After being reinfused into the patient's body, the modified and expanded CAR-T cells can efficiently hunt down and kill cancer cells, achieving "precision targeting." These engineered T cells can proliferate to hundreds of millions within weeks, forming a "biological army" dedicated to eradicating cancer.

02 Applicable Diseases: Breakthroughs from Blood Cancers to Solid Tumors

CAR-T therapy initially achieved revolutionary breakthroughs primarily in the treatment of hematologic malignancies. Multiple products targeting CD19 and BCMA have been approved for treating relapsed/refractory leukemia, lymphoma and multiple myeloma.

Hematologic Malignancies: Mature and Highly Effective

B-cell lymphoma: CAR-T products targeting CD19 can achieve response rates as high as 60%-80% even in patients with relapsed/refractory diffuse large B-cell lymphoma after multiple lines of therapy. Some patients can achieve long-term survival or even clinical cure.

B-cell acute lymphoblastic leukemia (B-ALL): CAR-T therapy performs exceptionally well in treating children and young adults, with initial remission rates exceeding 90%. Inoclinalted, approved in China, reported an objective response rate of 92.8% within 3 months.

Multiple myeloma (MM): CAR-T cell therapies targeting BCMA have brought deep and durable responses for patients with multiple relapses and multi-drug resistance, significantly prolonging survival.

03 Solid Tumors: Challenges and Breakthroughs Coexist

1. Gastrointestinal Cancers

Liver cancer: CAR-T therapy targeting GPC3 has shown preliminary efficacy in clinical trials.

Gastric/esophageal cancer: Research targeting Claudin18.2 has made breakthroughs, with related clinical trials showing good safety.

Colorectal cancer: Explorations targeting GCC, EGFR and other targets are ongoing.

2. Other Solid Tumors

Breast cancer: Clinical trials targeting ROR1, HER2, etc., have been initiated.

Glioma: Explorations targeting IL13Ralpha2, EGFRvIII, etc., face challenges but hold considerable promise.

In August 2025, a research team from Shandong University also developed a novel CAR-T therapy for treating chronic kidney disease, successfully reducing multi-organ fibrosis by targeting ECM-producing cells [2]. This opens new directions for expanding CAR-T therapy into non-oncological disease areas.

04 Challenges and Prospects: The Road Ahead is Broader

Although CAR-T therapy has achieved remarkable success, it also faces challenges. It can cause some side effects, primarily Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), which may manifest as fever, hypotension, confusion, etc. However, doctors closely monitor patients during treatment to promptly identify and manage these side effects.

CAR-T therapy is relatively expensive. Nevertheless, China is exploring "pay-for-performance" models, and numerous clinical trials offer free treatment opportunities for patients.

1. Developing "off-the-shelf" or universal CAR-T [3], using T cells from healthy donors to reduce cost and preparation time.

2. Exploring multi-target CAR-T to address cancer cell escape.

3. Expanding applications into non-oncological disease areas such as autoimmune diseases and chronic infections.

With the continuous emergence of dual-target designs, universal CAR-T technology and breakthroughs in solid tumors, more patients are expected to benefit from this therapy in the next 5 years.

As of April 2024, over a thousand CAR-T clinical trials have been initiated globally, with China accounting for more than 40% of these studies. The application landscape of CAR-T therapy is also expanding rapidly. From blood cancers to solid tumors, and further to non-oncological diseases, this innovative therapy is continuously pushing the boundaries of traditional treatment.

With the discovery of more targets and technological optimizations, CAR-T therapy is expected to play a significant role in more disease areas within the next 5-10 years, bringing new hope to more patients.

References:

1. Jongsma, M.L.M., de Waard, A.A. et al. (2021). The SPPL3-Defined Glycosphingolipid Repertoire Orchestrates HLA Class I-Mediated Immune Responses. Immunity 54, 132-150.e9.

2. Zhao S, Li R, Xia Y, Wang X, et al. Targeting ECM-producing cells with CAR-T therapy alleviates fibrosis in chronic kidney disease. Cell Stem Cell. 2025 Aug 18.

3. Wu Z, Shi J, Lamao Q, Qiu Y, et al. Glycan shielding enables TCR-sufficient allogeneic CAR-T therapy. Cell. 2025 Aug 14.