KMT2A mutation

Among these components, MLL mutations frequently co-occur with CHD1 mutations in various cancers, suggesting a shared pathway in cancer development. These findings underscore the importance of chromatin remodeler condensation as a regulatory hub in various cellular processes and tumor suppression.

Multivariate analysis identified older age, a high white blood cell count, KMT2A::MLLT4, and KRAS mutations as independent adverse prognostic factors for both EFS and OS. These age-specific mutation profiles suggest distinct disease mechanisms across age groups and may help refine risk stratification and treatment strategies for pediatric KMT2A-r AML.

P1/2, N=66, Active, not recruiting, Institut de Recherches Internationales Servier (I.R.I.S.) | Recruiting --> Active, not recruiting | N=266 --> 66

This study described the clinical features, mutation landscape and TCR rearrangement profile in a relatively larger PRCA cohort, which may contribute to the clear perception of PRCA and the development of more potent treatment approaches.

The present study systematically reviewed biological functions of the menin protein and its application in targeted therapy for specific AML subtypes. Notably, menin inhibitors have demonstrated potential in KMT2A/NPM1-mutant leukemia, however, the off-target effects, resistance mechanisms and a lack of biomarkers due to the extensive nature of their binding interface remains to be elucidated.

P1, N=420, Recruiting, Kura Oncology, Inc. | N=212 --> 420 | Trial completion date: May 2027 --> Apr 2030 | Trial primary completion date: May 2026 --> Apr 2030

P3, N=415, Not yet recruiting, Australasian Leukaemia and Lymphoma Group

These findings justify the integration of menin inhibitors into the AML therapeutic landscape, and support ongoing randomized trials to confirm their benefit in both frontline and relapse or refractory settings.

P1/2, N=266, Recruiting, BioNova Pharmaceuticals (Shanghai) LTD. | N=90 --> 266

The innovative mutation detection system is appropriate to small-to large-sized ITDs and other pathogenic tandem duplication mutations, expected to save 96.3% of the workload. This offers significant potential for accurate clinical assessment of ITD mutations and subsequent prognosis in AML patients.

In older adults newly diagnosed with NPM1m or KMT2Ar AML, the combination of azacitidine, venetoclax, and revumenib was able to be safely administered with high rates of CR and clinical activity.

Finally, we show that nucleoporin and KMT2A fusion proteins form condensates that are biophysically indistinguishable from NPM1c condensates. Together, these data define a new condensate underlying leukemias that we term coordinating bodies (C-bodies), and propose C-bodies as a therapeutic vulnerability.