mds

Pipobroman (Vercyte) is an anti-cancer drug that probably acts as an alkylating agent. Pipobroman has well documented clinical activity in polycythemia vera (PV) and essential thrombocythemia (ET). Pipobroman allows, within 3 months, to attain a response in more than 90% of patients, without clinically relevant toxicities. The 10-years risk of thrombosis of patients treated with Pipobroman is about 15%, The anti-proliferative activity of Pipobroman on bone marrow megakaryocytes seems of particular value in lowering the occurrence of post-PV and post-ET MMM, whose risk is the lowest registered with available treatments. The 10-year risk of acute leukemia with Pipobroman is 5% in PVand 3% in ET, which is only slightly higher than that expected as a natural evolution of the disease. In conclusion, the use of Pipobroman is a definite alternative to hydroxyurea in patients with PV and ET at high risk of thrombosis.

Ezatiostat hydrochloride (TLK199 HCl) is an effective inhibitor of glutathione S-transferase. Ezatiostat hydrochloride is a novel glutathione analog and the potential treatment of cytopenias. In addition, Ezatiostat hydrochloride has been revealed to selectively bind to and thus inhibit GSTP1-1.

Ezatiostat (TER199(free base)) is a tripeptide analog of glutathione that can selectively inhibit GSTP1-1 catalytic activity.

Ilorasertib (ABT-348) (ABT-348) is an ATP-competitive multitargeted kinase inhibitor, which inhibits Aurora A/Aurora B/Aurora C (IC50s: 120 nM/7 nM/1 nM). It also suppresses RET tyrosine kinase, PDGFRβ, and Flt1 (IC50s: 7 nM, 3 nM, and 32 nM).

Calcium D-saccharate tetrahydrate (Calcium D-saccharate tetrahydrate) is an oxidized non-cyclic hexose sugar. It may inhibit carcinogenesis and proliferation of cancer cells.

SX-682 is an orally available allosteric inhibitor of CXCR1 and CXCR2 that blocks tumor myeloid suppressor cell recruitment and enhances T cell activation and anti-tumor immunity, with the potential to treat castration-resistant prostate cancer.

Calcium D-saccharate is a reagent used in biochemical reactions.

Pevonedistat (MLN4924) is a potent and selective NEDD8 activating enzyme (NAE) inhibitor (IC50=4.7 nM) with therapeutic potential for myelodysplastic syndromes (MDS) and antitumor activity.

Laniquidar (R101933) is a third generation non-competitive inhibitor of P-glycoprotein (P-gp) (IC50: 0.51 μM) with limited oral bioavailability. It can also be used to modulate multidrug resistance transporters.

Pevonedistat hydrochloride (MLN4924 hydrochloride) is a selective and potent NEDD8 activating enzyme inhibitor (IC50: 4.7 nM) that induces apoptosis and cell cycle inhibition in cancer cells, and is used in the study of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Pelubiprofen has been used in the study of AML and MDS.)

Olutasidenib (FT-2102) is under the study in the treatment of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Olutasidenib is a highly effective, brain penetrant, and selective inhibitor of the mutant Isocitrate dehydrogenase 1 (IDH1) (IC50: 21.2 nM and 114 nM for IDH1- R132H and IDH1- R132C, respectively).

IRAK4-IN-31 is a crystalline inhibitor of IRAK4. It is applicable in research related to myelodysplastic syndromes (MDS).

MAO-B-IN-51 (Compound 8f) is an orally active and selective MAO-B inhibitor that can cross the blood-brain barrier, with an IC50 value of 48.9 nM (MDS). It is known to enhance long-term memory.

Menin-MLL-IN-36 (compound 398) is an inhibitor of the Menin/MLL protein interaction, with an IC50 value of 0.043 μM for the expression of MEIS1 mRNA. This compound is applicable to research on cancer, myelodysplastic syndrome (MDS), myeloproliferative neoplasms (MPN), and diabetes.

Menin-MLL-IN-35 (compound 286) is an inhibitor of the Menin/MLL protein interaction, with an IC50 value of 0.096 μM for MEIS1 mRNA expression. It is applicable in research related to cancer, myelodysplastic syndrome (MDS), myeloproliferative neoplasm (MPN), and diabetes.

Laniquidar TFA (R101933) is a novel orally available non-competitive P-glycoprotein (P-gp) inhibitor with an IC50 value of 0.51 μM.Laniquidar has limited bioavailability and can be used to modulate the multidrug resistance transporter.Laniquidar can be used for the study of AML and myelodysplastic syndrome (MDS). Laniquidar is used to study acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Asunercept (APG101; CAN008), a soluble CD95-Fc fusion protein, selectively targets and binds to CD95L, effectively disrupting CD95/CD95L signaling. It is utilized in research for treating glioblastoma multiforme (GBM), myelodysplastic syndrome (MDS), and graft-versus-host disease (GvHD) [1] [2] [3].

Tomaralimab (OPN-305) is a humanized IgG4 monoclonal antibody targeting TLR2, used to study myelodysplastic syndromes (MDS).

UC-764864 is a selective UBE2N inhibitor that blocks E1-UBE2N thioester transfer, inhibits innate immune signaling and induces cytotoxic effects in MDS/AML cell lines and primary cells. In addition, UC-764864 reduces the ubiquitination status of UBE2N and can be used to study leukemia.

MGTA-117 is a humanized monoclonal antibody that targets CD117. It carries an amanitin payload and is used to synthesize antibody-drug conjugates (ADC). MGTA-117 demonstrates potent antitumor activity, enhancing survival in three acute myeloid leukemia (AML) xenograft models [Kasumi-1, AML PDX 1, and AML PDX 2]. It is applicable for AML, myelodysplastic syndromes with excess blasts (MDS-EB), and preconditioning for hematopoietic stem cell transplantation (HSCT) in gene therapy.