A research team led by Assistant Professor Hiroshi Kotani at the Cancer Research Institute of Kanazawa University has successfully developed an innovative treatment for Burkitt's lymphoma. By combining CAR-T cell therapy (*1) with a novel approach that inhibits the activity of the MYC (*2) gene, the team has achieved a dramatic improvement in cure rates for this aggressive and rare form of blood cancer.
Burkitt's lymphoma is a rare and highly aggressive form of malignant lymphoma, a type of blood cancer, characterized by translocation of the MYC gene. It primarily affects children and young adults. In recent years, CAR-T cell therapy has been approved for certain types of blood cancers and has gained attention as a promising treatment option. However, its effectiveness against Burkitt's lymphoma has been reported to be limited. Moreover, despite MYC being central to the disease pathology, the development of drugs that directly target MYC has seen little progress over the years. As a result, there is a strong need for new therapeutic approaches to treat Burkitt's lymphoma.
In this study, the research team focused on a recently discovered epigenetic drug (*3) —identified by Assistant Professor Hiroshi Kotani and colleagues—that inhibits the SUMOylation (*4) pathway. This drug has been shown to suppress MYC activity (Reference 1) and enhance immune responses (Reference 2). By applying this approach to Burkitt's lymphoma and combining it with CAR-T cell therapy, the team demonstrated a dramatic improvement in cure rates.
This achievement highlights the potential of a novel therapeutic approach that combines immunotherapy with epigenetic drugs for the treatment of rare cancers with limited treatment options. It is expected that this strategy will contribute to improved treatment outcomes and enhanced quality of life for patients in the future. Furthermore, these findings are anticipated to play a key role in the future clinical development of a curative therapy for Burkitt's lymphoma.
The results of this research were published online in the international journal Signal Transduction and Targeted Therapy on October 3, 2025 at 1:00 a.m. Central European Time.
Figure 1: New curative therapy for Burkitt's lymphoma
【Glossary】
*1：CAR-T cell therapy
CAR-T cell therapy stands for chimeric antigen receptor T cell therapy. It is an advanced form of immunotherapy in which a patient’s own T cells are genetically engineered to produce synthetic receptors called CARs (chimeric antigen receptors) that can recognize and bind to specific molecules on the surface of cancer cells. Currently, CAR-T cells are created by collecting T cells from the patient, modifying them in the laboratory, and then reintroducing them into the patient’s body to target and destroy cancer cells.
*2: MYC
MYC is a transcription factor that regulates the expression of genes involved in processes such as cell proliferation and differentiation. Abnormal activation of the MYC gene plays a critical role in the development and progression of cancer.
*3：Epigenetic drugs
These drugs target the epigenome, which plays a key role in regulating gene expression. By modifying the epigenome—often described as a “switch” that turns genes on or off—they are expected to offer promising new avenues for cancer treatment.
*4: SUMOylation
SUMOylation is one of the post-translational modifications of proteins, in which proteins called small ubiquitin-like modifiers (SUMOs) bind to other proteins.
【References】
１）Dual inhibition of SUMOylation and MEK conquers MYC-expressing KRAS-mutant cancers by accumulating DNA damage.
Kotani H, Oshima H, Boucher JC, Yamano T, Sakaguchi H, Sato S, Fukuda K, Nishiyama A, Yamashita K, Ohtsubo K, Takeuchi S, Nishiuchi T, Oshima M, Davila ML, Yano S. J Biomed Sci. 2024;31(1):68.
Click here to see the press release【Japanese only】
Journal：Signal Transduction and Targeted Therapy
Researcher's Information：Hiroshi Kotani
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