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High surface-area lyophilized compositions comprising arsenic for oral administration in patients

Resumen de: AU2025259861A1

The present invention relates to treating malignancies such as tumors or cancers by orally administering lyophilized compositions comprising arsenic to a subject in such need. Malignancies include various hematological malignancies, such as acute myeloid leukemia (AML) including acute promyelocytic leukemia (APL), myelodysplastic syndrome (MDS), multiple myeloma (MM) and lymphomas and solid tumors including glioblastoma multiforme and breast cancer. Arsenic treatment has shown great promise in the treatment of several cancers but requires daily intravenous (IV) administration. This invention relates to a novel formulation comprising a lyophilized compositions comprising arsenic. As a result, the formulation facilitates a systemic bioavailability comparable to that of intravenous (IV) administration of arsenic trioxide currently practiced. The present invention also relates to a method for lyophilizing the arsenic trioxide, preparing the oral formulation comprising lyophilized compositions comprising arsenic, and a method for treating a subject with malignancies using the oral formulation. The present invention relates to treating malignancies such as tumors or cancers by orally administering lyophilized compositions comprising arsenic to a subject in such need. Malignancies include various hematological malignancies, such as acute myeloid leukemia (AML) including acute promyelocytic leukemia (APL), myelodysplastic syndrome (MDS), multiple myeloma (MM) and lymphomas and solid

CD70 and venetoclax, a BCL-2 inhibitor, combination therapy for treating acute myeloid leukemia

Resumen de: AU2025259981A1

The invention relates to combination therapies, particularly combination therapies for the treatment of myeloid malignancy. The combination therapies are particularly useful in methods for the treatment of acute myeloid leukemia (AML). The combination therapies 5 include an antibody or antigen binding fragment thereof that binds to CD70 and a BCL-2 inhibitor, preferably venetoclax or a pharmaceutically acceptable salt thereof. 10 15 20 25 The invention relates to combination therapies, particularly combination therapies for the treatment of myeloid malignancy. The combination therapies are particularly useful in 5 methods for the treatment of acute myeloid leukemia (AML). The combination therapies include an antibody or antigen binding fragment thereof that binds to CD70 and a BCL-2 inhibitor, preferably venetoclax or a pharmaceutically acceptable salt thereof. ct c t

FUSED PYRIMIDINE COMPOUNDS AS INHIBITORS OF MENIN

Resumen de: US2025353854A1

Disclosed herein are heterocyclic compounds that inhibit the binding of menin and MLL or MLL fusion proteins. Also described are specific covalent inhibitors of a menin or menin-MLL interaction. Also disclosed are pharmaceutical compositions that include the compounds described herein. Methods of using the menin or menin-MLL irreversible inhibitors are disclosed, alone or in combination with other therapeutic agents, for the treatment of autoimmune diseases or conditions, heteroimmune diseases or conditions, cancer, including lymphoma, leukemia and other diseases or conditions dependent on menin or menin-MLL interaction.

A DOSING REGIMEN

Resumen de: AU2024243824A1

The present disclosure relates to combination dosing regimen comprising an anti-KMA antibody, an IMiD/CELMoD and a steroid. Such combinations may be used for the treatment of multiple myeloma.

COMPOSITIONS AND METHODS FOR TARGETING CD13 AND TIM-3 WITH CAR T CELLS TO TREAT ACUTE MYELOID LEUKEMIA

Resumen de: US2025352582A1

The present invention includes compositions and methods for treating AML utilizing bispecific CARs. In certain aspects, the invention includes a bispecific split CAR which binds CD13 and TIM-3 on AML cells.

IDENTIFICATION, DESIGN AND VALIDATION OF PUBLIC NEOANTIGENS FOR THE TREATMENT OF DOWN SYNDROME-ASSOCIATED LEUKEMIA

Resumen de: WO2025236094A1

Identification, Design and Validation of Public Neoantigens and Tumor-associated Antigen vaccines for the Prevention and Treatment of Down Syndrome-associated Leukemia.

COMBINATION OF VDAC2 MODULATORS AND BH3 MIMETICS FOR TREATING CANCER

Resumen de: WO2025237908A1

The present invention relates to the field of the treatment of cancer. In this study, the inventors found that VDAC2 is heterogeneously expressed in MM cells. VDAC2 protein expression correlated with BAK but not with BAX protein levels. Transient silencing of VDAC2, but not VDAC1 or VDAC3, sensitized MM cells to intrinsic mitochondrial apoptosis signals, alongside with the induction of pre-activated BAK and the increase of global, MCL1 and BCL2 mitochondrial priming. They also found a that a VDAC2 compound, efesevin recapitulated the sensitization effect of VDAC2 knock-down on BH3 mimetics apoptotic response. This novel VDAC2 modulator sensitized MM cells to BH3 mimetics targeting MCL1 (S63845) or BCL2 (Venetoclax) without modifying BAK or BAX protein expression. The efficiency of the VDAC2 modulator was directly correlated with the levels of VDAC2 protein. To better understand the VDAC2/BAK interplay, The inventors generated VDAC2 KO myeloma cells. VDAC2 KO cells exhibited an important decrease of BAK protein expression while BAX remained unchanged. Accordingly, VDAC2 KO cells completely lost their mitochondrial priming. Interestingly, they also found that BAK KO myeloma cells displayed decreased levels of VDAC2. The reciprocal regulation between VDAC2 and BAK was dependent on both the proteasome and lysosome degradation pathways. Thus, the present invention relates to a combination of a VDAC2 modulator and a BH3- mimetics compound for use in the treatment of a cancer in a s

THERAPEUTIC COMPOSITIONS AND METHODS THEREOF

Resumen de: WO2025240852A1

In one aspect, the disclosure relates to composition and methods for proteolysis-targeting chimeric molecules (PROTACs). In some aspects, the disclosed compounds are useful for modulating LCK tyrosine kinase activity through targeted degradation. In some aspects, the disclosed compounds are orally bioavailable. In further aspects, the present disclosure relates to methods of making the disclosed compounds, pharmaceutical compositions comprising the disclosed compounds, and methods of treating various clinical conditions and disorders using the same, such as T-cell acute lymphoblastic leukemia. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present disclosure.

METHODS FOR DETECTING ACUTE MYELOID LEUKEMIA

Nº publicación: US2025354218A1 20/11/2025

Solicitante:

CORNELL UNIV [US]
Cornell University

Resumen de: US2025354218A1

The present technology relates to methods for predicting the risk of acute myeloid leukemia (AML) in a subject prior to the onset of AML symptoms, and whether such a subject will benefit from treatment with an AML therapy. The methods disclosed herein are based on detecting the presence of mutations in the nucleic acid sequences of IDH1/2, TP53, DNMT3A, TET2, and spliceosome genes. Kits for use in practicing the methods are also provided.