PDGFRA (Platelet Derived Growth Factor Receptor Alpha)

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.

Intranasal IL-4 delivery may represent a promising therapeutic strategy for enhancing white matter integrity in leukodystrophies and MS. Abbreviations: MS, multiple sclerosis; IL-4, Interleukin 4; CNS, central nervous system; OPCs, oligodendrocytes precursor cells; IL-4R, IL-4 receptor; i.n., intranasal; LPC, lysophosphatidylcholine; PVL, periventricular leukomalacia; EAE, experimental autoimmune encephalomyelitis; dpi, days post immunization; dpl, days post lesion; Nkx2.2, NK2 homeobox 2; Olig2, oligodendrocyte transcription factor 2; OLs, oligodendrocytes; SOX10, SRY-Box Transcription Factor 10; PDGFRα, platelet derived growth factor receptor alpha; CNPase, 2',3'-Cyclic Nucleotide 3' Phosphodiesterase; APC, adenomatous polyposis coli; ASPA, aspartoacylase; GFAP, glial fibrillary acidic protein; Iba1, ionized calcium binding adaptor molecule 1; MBP, myelin basic protein; TEM, transmission electron microscopy; CC, corpus callosum; GCC, genu of the corpus callosum; CG, cingulum; CTX, cortex; STR, striatum; EC, external capsule; Pre-OLs, pre-myelinating oligodendrocytes; STAT6, signal transducer and activator of transcription 6; Pio, pioglitazone; T3, triiodothyronine; CNTF, ciliary neurotrophic factor; PPARγ, peroxisome proliferator-activated receptor γ.

One algorithm, Arriba, detected all driver fusions. These findings will inform algorithm selection in clinical settings.

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

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

Investigation of patient samples identifies analogous cell states and developmental trajectories. This study uncovers a fibroblastic origin for DRTP sarcoma and provides a faithful mouse model for future mechanistic and translational investigation.

ALK+ squamous and adenosquamous NSCLCs are rare, but biologically distinct, with inferior outcomes on 1L ALK TKI, highlighting the need for further research to develop effective treatment strategies.

In this study, targeted next-generation sequencing using the AmpliSeq for Illumina Cancer HotSpot Panel identified pathogenic mutations in canonical cancer driver genes: tumor protein p53 (TP53) NM_000546.6:c.730G>T (p.Gly244Cys) and platelet-derived growth factor receptor alpha (PDGFRA) NM_006206.6:c.2525A>T (p.Asp842Val) mutations in renal leiomyosarcoma and NRAS proto-oncogene, GTPase (NRAS) NM_002524.5:c.35G>T (p.Gly12Val) mutation in the ovarian tumor. These findings suggest a potential benefit of personalized therapies for XP patients with internal malignancies.

Finally, we demonstrate that multiscale spatial analysis provides a quantitative readout of stromal-immune-epithelial remodeling following therapy. These findings establish a simplified, translationally relevant CAF framework and highlight spatially resolved stromal dynamics as measurable indicators of therapeutic response in CRC.

The presence of CDK4, CCND2, PDGFRA, PIK3R1, MYCN, and EGFR alterations result in an intermediate patient survival in IDH-mutant astrocytoma. Adding these molecular alterations should be considered in future diagnostic classification systems to improve stratification of high-risk patients.