KIT (KIT proto-oncogene, receptor tyrosine kinase)

P2, N=28, Recruiting, Asan Medical Center | Not yet recruiting --> Recruiting

Resistance to drugs in sarcoma is multifactorial and changeable. Nevertheless, fusing mechanistic knowledge with biomarker - guided combination/sequential therapies offers a clear way to improve patient situations.

Since the introduction of imatinib in the early 21st century, the management of metastatic GIST has shifted from solely surgical intervention to a systemic, chronic disease management model centered on tyrosine kinase inhibitors (TKIs)...These challenges underscore the necessity of this review, which discusses current standard drug treatment strategies for advanced GIST, including sequential TKIs therapy and investigations into mechanisms of drug resistance. Finally, the review explores precise and actionable future directions for GIST drug development and clinical management, including mutation-stratified therapeutic sequencing, rational TKI-based combination regimens, and circulating tumor DNA (ctDNA)-guided real-time treatment monitoring and resistance surveillance.

P2, N=40, Completed, Asan Medical Center | Recruiting --> Completed

P1/2, N=48, Recruiting, Asan Medical Center | Not yet recruiting --> Recruiting

BRCA1-deficient tumors in short survivors have evidence of immunosuppressive c-kit signaling and EMT. Our findings confirm that outcome is not determined by BRCA status alone, but rather a combination of co-occurring genomic alterations, the extent of DNA repair deficiency, and the tumor-immune microenvironment.

Moreover, E5-LNP@siAE significantly enhanced the therapeutic efficacy of doxorubicin (DOX) in the AML mice, evidenced by the reduced AML cells in peripheral blood, bone marrow and multiple organs, meanwhile, did not bring negative impacts on the counts of red blood cells and platelets. Consequently, the combination regimen achieved the much better result of survival prolongation than E5-LNP@siAE monotherapy, and the other control groups did not show therapeutic effects on the survival of the AML mice.

Collectively, these findings establish a modular platform for producing allogeneic CAR T cells using mRNA technology, offering a practical approach for rapid, on-demand CAR T-cell therapy.

Furthermore, the addition of CD33xCD28 IgG4-scFv2 showed faster time to cell attachment, increased lytic events, and improved specificity towards double-target expressing cells. In summary, the data indicate that combining co-stimulation via a second tumor-associated antigen to CD3-TCEs enhances T-cell lytic activity and simultaneously increases specificity against double-target expressing AML cells.

Histopathology consistent with low-grade combined type GIST with immunochemistry positive for CD117 (c-KIT). This case highlights an unusual presentation of small bowel bleeding originating from an aberrant artery in continuity with a hypervascular jejunal GIST.

This case expands the morphologic and immunophenotypic spectrum of PFM and indicates the possible diagnostic utility and biological significance of D2-40 expression. Although molecular confirmation of MALAT1::GLI1 fusion is definitive for the diagnosis of PFM, the findings of the present case may aid diagnosis in challenging cases that mimic GIST.