Acute Myelogenous Leukemia

P=N/A, N=99, Not yet recruiting, University Hospital, Toulouse

P2, N=82, Active, not recruiting, Beth Israel Deaconess Medical Center | Trial primary completion date: Dec 2023 --> Sep 2026

Unlike another CAR, SSR does not produce significant cytotoxicity against normal CD38+ tissues. Our study thus shows that SSR arming enhances targeting of antigenically heterogeneous cancers without compromising safety and selectivity of therapeutic T cells.

We also identify a human ILC1 subset as Lin-CD127+CD161-CRTH2-CD117- (CD161- ILC1s) that can be generated from umbilical cord blood CD34+ hematopoietic stem cells. This method could provide a reliable source of ILC1s for potential adoptive transfer therapies in AML, offering a therapeutic approach to prolong disease-free survival in AML.

Overexpression of the IRX5 gene in human MOLM13 leukemic cells suppressed cell growth both in vitro and in mouse xenografts. Public patient data confirmed expression levels of PLAG1 and SMAD1 as markers of AML status and survival, suggesting that multiomic analysis of evolving clones in a mouse model is a valuable predictive approach relevant to human AML.

P2, N=65, Completed, First Affiliated Hospital of Zhejiang University | Recruiting --> Completed | N=30 --> 65 | Trial primary completion date: Aug 2025 --> Apr 2025

P=N/A, N=30, Recruiting, Groupe Hospitalier de la Region de Mulhouse et Sud Alsace | Not yet recruiting --> Recruiting

Moreover, YL-5092 functionally impaired leukemia stem cells yet spared normal hematopoietic counterparts. Collectively, our work demonstrates the efficacy of a selective YTHDC1 inhibitor and suggests that targeting of m6A readers is a potential strategy in the treatment of hematologic cancers.

This allogeneic CAR T product demonstrated acceptable safety and preliminary anti-leukemic activity. ClinicalTrials.gov: NCT05984199.

Given the well-documented association between SF3B1 mutations and MECOM rearrangements, analysis of MECOM expression and RNA sequencing (RNA-seq) is crucial for SF3B1-mutated patients, even in the absence of elevated blast counts. Furthermore, this case underscores the need for further research into the synergistic biological role of spliceosome mutations and MECOM rearrangements in driving leukemia.

Further investigation revealed acute myeloid leukemia (AML) arising from blastic transformation of ET, with probable cutaneous involvement consistent with leukemia cutis. This case highlights the importance of maintaining a high index of suspicion when evaluating new or treatment-refractory skin lesions in patients with myeloproliferative neoplasms (MPNs), as early recognition may allow timely diagnosis of leukemic transformation and prompt initiation of appropriate therapy.

This pilot study demonstrates that imaging flow cytometry-based FISH of circulating CD34/CD45-positive cells enables real-time, blood-based surveillance for cytogenetic evolution in myelofibrosis. The ability to dynamically track clone size and hierarchy highlights its potential as an early predictor of leukemic transformation in myelofibrosis.