clinical applications and approved medicines, which cancers can cell therapy treat today?
Acute lymphoblastic leukemia (ALL) was one of the first cancers in which CAR-T therapy achieved a major breakthrough. The disease often affects children and young adults, and in relapsed or refractory cases, traditional treatments may offer limited effectiveness.
One of the first CAR-T therapies approved globally is Kymriah (tisagenlecleucel), developed by Novartis. The therapy was approved in 2017 by the U.S. Food and Drug Administration for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia.
Clinical studies have shown that some pediatric and young adult patients can achieve high complete remission rates with this therapy. In certain cases, patients have experienced durable remission after CAR-T treatment, making it a major milestone in leukemia therapy.
In China, domestically developed CAR-T therapies have also made progress. For example, Relma‑cel (relmacabtagene autoleucel)developed by JW Therapeutics has been approved by the National Medical Products Administration for the treatment of certain hematologic malignancies.
Diffuse large B-cell lymphoma is the most common type of non-Hodgkin lymphoma. While many patients respond to first-line therapy, a significant proportion may relapse and require additional treatment options.
In recent years, several CAR-T therapies have been approved for relapsed or refractory DLBCL, including:
● Yescarta (axicabtagene ciloleucel) — developed by Gilead Sciences through its subsidiary Kite Pharma
● Breyanzi (lisocabtagene maraleucel) — developed by Bristol Myers Squibb
These CAR-T therapies target the CD19 antigen expressed on lymphoma cells, directing engineered T cells to specifically attack malignant B cells. In clinical studies, some patients have achieved long-term remission following CAR-T treatment, making it an important option for relapsed lymphoma.
Multiple myeloma is a malignant plasma cell disorder in which patients often experience relapse after multiple lines of therapy. As a result, new treatment strategies remain a major focus of research.
In recent years, CAR-T therapies targeting BCMA (B-cell maturation antigen) have demonstrated significant progress in treating multiple myeloma. Examples include:
● Abecma (idecabtagene vicleucel)
● Carvykti (ciltacabtagene autoleucel)
Among them, Carvykti has shown deep response rates in clinical studies, with some patients achieving stringent complete remission. These results have made BCMA-targeted CAR-T therapy a major innovation in the treatment of multiple myeloma.
Mantle cell lymphoma is a relatively rare but aggressive type of B-cell lymphoma. For patients with relapsed or refractory disease, CAR-T therapy has also become an emerging treatment option.
For example: Tecartus (brexucabtagene autoleucel)
This therapy also targets the CD19 antigen and works by engineering T cells to recognize and eliminate malignant B cells. Clinical studies have demonstrated high objective response rates in some patients with relapsed or refractory disease.
Although CAR-T therapy has achieved remarkable success in hematologic cancers, treating solid tumors remains more challenging. Several factors contribute to these difficulties, including:
● Immunosuppressive tumor microenvironments
● Heterogeneous tumor antigen expression
● Limited infiltration of immune cells into tumor tissues
To address these challenges, researchers are exploring several next-generation cell therapy approaches, such as:
1. TCR-T therapy (T-cell receptor engineered therapy)
2. NK cell therapy (natural killer cell therapy)
3. Allogeneic CAR-T (off-the-shelf CAR-T therapies)
Clinical studies involving these technologies are currently being conducted in cancers such as melanoma, lung cancer, and gastric cancer, potentially expanding the future indications of cell therapy.
It is important to note that many cell therapies are closely linked to genetic engineering technologies.
For example, CAR-T therapy typically requires viral vectors or other gene-editing techniques to introduce specific genes into T cells, enabling them to recognize tumor antigens. For this reason, CAR-T therapy is often considered part of the broader field of cell and gene therapy (CGT).
If you would like to learn more about the differences between cell therapy and gene therapy, as well as CGT drugs currently approved in China, you can read our related article:
“Cell Therapy vs Gene Therapy in China: Key Differences and Approved CGT Drugs.”
Overall, cell therapy is providing new treatment options for a growing number of cancer patients.
From acute lymphoblastic leukemia and diffuse large B-cell lymphoma to multiple myeloma, immune cell therapies such as CAR-T have already demonstrated strong clinical potential in hematologic malignancies. At the same time, researchers continue to explore the use of cell therapy for solid tumors.
As innovative therapies continue to emerge, the role of global pharmaceutical supply chains is becoming increasingly important for research, manufacturing, and international access to advanced treatments.
HongKong DengYue Medicine, an international pharmaceutical distribution platform, connects healthcare resources across regions through its global medicine supply network, supporting compliant access and international circulation of innovative therapies.
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