Abstract: Provided herein is an amorphous form of a pharmaceutically acceptable salt of ezatiostat, for example, ezatiostat hydrochloride, compositions, uses and methods of preparation thereof.

Abstract: The present invention provides compounds having formula (I): and additionally provides methods for the synthesis thereof and methods for the use thereof in the treatment of cancer, wherein R1-R7, X1, X2, R, Q, and n are as defined herein.

Abstract: Provided are methods for treating MLL-rearranged ALL by administering to a patient an HDAC inhibitor alone or in combination with a DNA demethylating agent. Also provided are methods of treating MLL-rearranged infant ALL. Methods of treating cells by these agents are also provided. Additionally, disclosed is a method for screening for compounds capable to treat MLL-rearranged ALL, in particular, MLL-rearranged infant ALL. In one embodiment, the HDAC inhibitor is romidepsin.

Abstract: In alternative embodiments, the invention provides compositions and methods for inhibiting or ablating cancer stem cells. In alternative embodiments, the invention provides compositions and methods for inhibiting the action of double-stranded RNA-specific adenosine deaminases, or ADAR, enzymes. In alternative embodiments, the invention provides compositions and methods for treating, ameliorating or preventing diseases and conditions responsive to the inhibition of cell differentiation and/or self-renewal of dysfunctional cells, cancer cells, leukemia cells, hematopoietic stem cells or cancer stem cells, e.g., leukemia or Chronic Myeloid Leukemia (CML). In alternative embodiments, the invention provides compositions and methods for inhibiting a Sonic Hedgehog (Shh) pathway, e.g., by using a Smoothened (SMO) protein inhibitor.

Abstract: N-acyldipeptide derivatives are described. Compositions comprising the N-acyldipeptide derivatives are therapeutically effective for topical or systemic administration to alleviate or improve conditions, disorders, diseases, symptoms or syndromes associated with a tumor, cancer, immune, nervous, vascular, musculoskeletal or cutaneous system, or other tissue or system in a subject.

Abstract: Base-labile crosslinkers, base-labile conjugates comprising such crosslinkers, methods of their synthesis and use are disclosed.

Abstract: MOTS3 is a novel polypeptide. Methods of treating diseases such as diabetes, obesity, fatty liver, and cancer using MOTS3 and pharmaceutical compositions thereof are disclosed herein.

Abstract: The invention relates to tumor therapy. In one aspect, the present invention relates to conjugates of an amatoxin and a target-binding moiety, e.g. an antibody, connected by certain linkages, which are useful in the treatment of cancer. In a further aspect the invention relates to pharmaceutical compositions comprising such conjugates.

Abstract: Disclosed are immunomodulatory polypeptides that elicit an unusual induced cytokine profile, compositions comprising such polypeptides, compositions comprising antibodies that specifically bind to such polypeptides, and methods of using the same, including in cancer treatment, in the treatment of autoimmune diseases, in organ transplantation and for reducing graft rejection, for promoting fertility, and for identifying a neutrophil subset and/or other cellular subset including by flow cytometry. Pharmaceutical compositions and kits, and treatment methods are also disclosed.

Abstract: Synthesis of compounds having varying degrees of conformational rigidity is obtained via a low cost, high yield and efficient synthetic reactions. The library of compounds is structurally diverse having at least one or more chiral centers and providing large numbers of compounds having building block diversity and substantial scaffold diversity. The compounds further provide a novel method for obtaining candidate therapeutic agents for prevention, treatment or diagnosis of diseases.

Abstract: An inducing agent or enhancing agent, for the expression of HM1.24 antigen in hematopoietic tumor cells, comprising interferon ?, interferon ?, or the IRF-2 protein as an active ingredient, as well as an antitumor agent for hematopoietic tumors which comprises a combination of said inducing agent or enhancing agent and an antibody against HM1.24.

Abstract: A method of using a Smac mimetic and pharmaceutical compositions thereof.

Abstract: The invention provides compositions and methods to treat cancer with an agent that increases reactive oxygen species (ROS) levels in cancer cell mitochondria (“an XTPP agent”) or a pharmaceutically acceptable salt thereof, an inhibitor of hydroperoxide metabolism and a pharmaceutically acceptable diluent or carrier.

Abstract: The invention relates to compounds of Formula I: and pharmaceutically acceptable salts and prodrugs thereof, wherein R1, R2, and R3 are defined as set forth in the specification. The compounds are agonists of neurotrophin (such as nerve growth factor) receptors.

Abstract: Compositions and methods for the treatment of leukemia by inhibiting FOXO signaling are described.

Abstract: It is provided PACE4 inhibitors and their uses for treating infection, cancer. Particularly, it is provided a method or use for the treatment of a cancer in a subject, comprising administering to the subject a therapeutically effective amount of the PACE4 inhibitors or the composition disclosed.

Abstract: Disclosed are: a peptide comprising an amino acid sequence composed of contiguous nine amino acid residues derived from a WT1 protein, wherein an amino acid residue at position 2 in the amino acid sequence is selected from the group consisting of Ala, Ile, Leu, Val, Phe, Tyr, Ser and Asp and an amino acid residue at position 9 in the amino acid sequence is Arg; a polynucleotide encoding the peptide; a pharmaceutical composition comprising the peptide; and a method of treating cancer using the peptide.

Abstract: Disclosed are: a peptide comprising an amino acid sequence composed of contiguous nine amino acid residues derived from a WT1 protein, wherein an amino acid residue at position 2 in the amino acid sequence is selected from the group consisting of Ala, Ile, Leu, Val, Phe, Tyr, Ser and Asp and an amino acid residue at position 9 in the amino acid sequence is Arg; a polynucleotide encoding the peptide; a pharmaceutical composition comprising the peptide; and others.

Abstract: The present invention provides methods and compositions for treatment, screening, diagnosis and prognosis of acute myeloid leukemia (AML), B-cell chronic lymphocytic leukemia, breast cancer, colorectal cancer, kidney cancer, head and neck cancer, lung cancer, ovarian cancer or pancreatic cancer, for monitoring the effectiveness of acute myeloid leukemia (AML), B-cell chronic lymphocytic leukemia, breast cancer, colorectal cancer, kidney cancer, head and neck cancer, lung cancer, ovarian cancer or pancreatic cancer treatment, and for drug development. The present invention also provides methods and compositions for depletion of immune cells to treat inflammatory diseases.

Abstract: Compositions and methods are provided to destroy internal cancerous lesions selectively by the administration of a combination of a debridement protease enzyme and a denaturant of cell structural proteins and or cell adhesion proteins.

Abstract: Antitumoral compounds of Formula I, and pharmaceutically acceptable salts, derivatives, tautomers, prodrugs or stereoisomers thereof useful as antitumour agents.

Abstract: A method of diagnosing B-CLL in a subject in need thereof is provided. The method comprising determining in a biological sample of the subject a level of CD84 isoform C (SEQ ID NO: 30), wherein an increase in the level of the CD84 isoform C (SEQ ID NO: 30) beyond a predetermined threshold with respect to a level of the CD84 in a biological sample from a healthy individual is indicative of the B-CLL.

Abstract: The present invention provides the use of a peptide comprising an amino acid sequence of SEQ ID NO:3, or the encoding nucleic acid thereof, for manufacturing medicament for treatment of diseases associated with increased NF-?B activity. The peptide can interact with IKB and influence phosphorylation of IKB in the tyrosine at position 42, thereby inhibiting the signal pathway of NF-?B.

Abstract: Novel compounds are provided that bind to polo-like kinases through the polo-box domain. In certain embodiments, the novel compounds are PEGylated peptides. The PEGylated peptides in accordance with the invention demonstrate high PBD-binding affinity. In certain embodiments, the PEGylated peptides have also achieved activities in whole cell systems. The invention also provides compounds that bind polo-like kinases through the polo-box domain and possess reduced anionic charge. Further provided are methods of design and/or synthesis of the PEGylated peptides and methods of use thereof. The invention provides methods of use of the compounds and methods of synthesis of the compounds. The compounds of the invention have potential therapeutic activity in view of their binding and inhibitory activities towards Plk1. They are based on the amino acid sequence PLHSpT (phosphorylated Thr). The PEG moiety, when present, is covalently attached at the N-terminus.

Abstract: The present invention is based upon the identification of regions within the NOTCH-1 receptor that, when mutated, lead to increase receptor signaling. The mutations are associated with uncontrolled cellular growth and this growth may be arrested using agents that interfere with NOTCH-1 activity, such as inhibitors of gamma-secretase. Assays for the NOTCH-1 mutations may be used diagnostically or as part of a treatment regimen for cancer patients.

Abstract: The invention features targeted cytotoxic compounds and methods relating to their therapeutic use for the treatment of neoplasia and other conditions.

Abstract: According to the present invention, a cancer antigen protein to be specifically expressed on the surfaces of cancer cells is identified and thus the use of an antibody targeting the cancer antigen protein as an agent for treating and/or preventing a cancer is provided. Specifically, the present invention provides a pharmaceutical composition for treating and/or preventing a cancer, which comprises an antibody comprising a heavy chain variable region that comprises SEQ ID NOS: 39, 40, and 41 and a light chain variable region that comprises SEQ ID NOS: 43, 44, and 45 or a fragment thereof as an active ingredient and having immunological reactivity with a CAPRIN-1 protein.

Abstract: This disclosure features modified release pharmaceutical formulations (e.g., extended release pharmaceutical formulations; e.g., solid dosage forms, e.g., tablets) that are useful for the oral administration of oprozomib, or a pharmaceutically acceptable salt thereof, to a human or animal subject as well as methods of making and using the formulations.

Abstract: The present disclosure provides a humanized anti-CD37 small modular immunopharmaceutical (SMIP) molecule, as well as synergistic combination therapies of CD37-specific binding molecules (such as anti-CD37 SMIP proteins or antibodies) with bifunctional chemotherapeutics (such as bendamustine) that can be administered concurrently or sequentially, for use in treating or preventing B-cell related autoimmune, inflammatory, or hyperproliferative diseases.

Abstract: The present invention provides materials and methods useful to treat various sGC?1-expressing cancers. Materials include peptides which interfere with sGC?1's pro-survival functions, thereby resulting in apoptosis of sGC?1-expressing cells. In addition, the present invention provides screening assays, diagnostic assays, methods to prognose, methods to treat, and kits.

Abstract: One aspect of the invention relates to inhibitors that preferentially inhibit immunoproteasome activity over constitutive proteasome activity. In certain embodiments, the invention relates to the treatment of immune related diseases, comprising administering a compound of the invention. In certain embodiments, the invention relates to the treatment of cancer, comprising administering a compound of the invention.

Abstract: A composition comprising leukotoxin proteins isolated from a bacterium is provided. In this composition, greater than 85% of the leukotoxin proteins are chemically modified at a basic amino acid residue, and the proteins induce cell death in myeloid leukocytes, while remaining substantially non-toxic to lymphoid leukocytes, lymphocytes, and red blood cells. Also provided is a method of selectively inducing cell death in myeloid leukocytes. The method comprises contacting the myeloid leukocytes with a composition comprising leukotoxin proteins. These leukotoxin proteins may be isolated from the NJ4500 strain of Actinobacillus actinomycetemcomitans. A method of purifying leukotoxin protein from the NJ4500 strain of Actinobacillus actinomycetemcomitans is also provided, as well as an assay that allows for the rapid determination of the activity of a given drug against leukemic cells either taken from a patient or derived from a cell line. The assay is performed in the presence of whole blood or serum.

Abstract: Two vIRF4 (Kaposi's-sarcoma-associated-herpesvirus vIRF4) peptides, vif1, corresponding to aa202-216 of vIRF4, and vif2, corresponding to aa220-236 of vIRF4, are potent and selective HAUSP antagonists. The vif1 and vif2 peptides robustly suppress HAUSP DUB enzymatic activity, ultimately leading to p53-mediated anti-cancer activity. The vif1 and vif2 peptides, along with their homologues, are useful in treating ALL.

Abstract: The invention provides methods that relate to a novel therapeutic strategy for the treatment of hematological malignancies and inflammatory diseases. In particular, the method comprises administration of a compound of formula A, wherein R is H, halo, or C1-C6 alkyl; R? is C1-C6 alkyl; or a pharmaceutically acceptable salt thereof; and optionally a pharmaceutically acceptable excipient.

Abstract: The invention provides human secreted proteins (SECP) and polynucleotides which identify and encode SECP. The invention also provides expression vectors, host cells, antibodies, agonists, and antagonists. The invention also provides methods for diagnosing, treating, or preventing disorders associated with aberrant expression of SECP.

Abstract: Short peptide mimetics of adiponectin suitable for development as pharmaceutical agonists in the treatment of cellular proliferative disorders such as cancer and atherosclerosis are provided.

Abstract: Provided are a pharmaceutical composition which enhances the sensitivity of a cancer to a molecular target drug, such as gefitinib and erlotinib, wherein the cancer has resistance to the molecular target drug, and a cancer therapeutic agent effective against a cancer having resistance to a molecular target drug, such as gefitinib and erlotinib. The pharmaceutical composition comprising an HGF-MET receptor pathway inhibitor enhances the sensitivity of a cancer to a molecular target drug, such as gefitinib and erlotinib, even though the cancer has resistance to the molecular target drug. The cancer therapeutic agent comprising a molecular target drug in combination with an HGF-MET receptor pathway inhibitor is effective against a cancer having resistance to the molecular target drug.

Abstract: The present invention thus provides microbial products as anticancer agents and pharmaceutical compositions comprising isolated and purified proteins or synthetic peptides, and methods of using them for the treatment of cancer. It is very important to develop new anticancer bioactive peptides having high activity and low toxicity; given that most currently available anticancer therapies either have significant toxicity, and/or are prone to development of resistance.

Abstract: We describe preparation of compounds of an AT1 receptor antagonist(s) and Angiotensin (1-7), for example, Angiotensin-(1-7) losartanate and analogues thereof, and/or mixtures of these systems, pharmaceutical compositions thereof and use of their derivative products. Cyclodextrins and derivatives thereof may be used for the micro-encapsulation of compounds, for example, Angiotensin (1-7) losartanate, liposomes and biodegradable polymers and/or mixtures of these systems and/or derivative products for the obtainment of nano- or microparticles as controlled or sustained release devices of Ang-(1-7) losartanate and analogues and/or mixtures thereof. The compounds may be used as agents for treating or preventing hypertension, cardiovascular diseases, heart hypertrophy, heart failure, coronary diseases, such as angina pectoris, endothelial disorder or endothelial lesions, as a consequence, for example, of atherosclerosis processes or in association with diabetes mellitus.

Abstract: The present invention relates to novel regulatory T cell proteins. One protein, designated PD-L3, resembles members of the PD-L1 family, and co-stimulates ?CD3 proliferation of T cells in vitro. A second, TNF-like, protein has also been identified as being upregulated upon ?CD3/?GITR stimulation. This protein has been designated Treg-sTNF. Proteins, antibodies, activated T cells and methods for using the same are disclosed. In particular methods of using these proteins and compounds, preferably antibodies, which bind or modulate (agonize or antagonize) the activity of these proteins, as immune modulators and for the treatment of cancer, autoimmune disease, allergy, infection and inflammatory conditions, e.g.

Abstract: Compounds which are ?-halogenoacryloyl distamycin derivatives of formula (I) wherein R1 is a bromine or chlorine atom; R2 is a distamycin or distamycin-like framework as set forth in the specification; or a pharmaceutically acceptable salt thereof; are cytotoxic agents particularly effective in the treatment of tumors over expressing GSH/GSTs system and which are poorly responsive or even resistant to conventional antitumor therapies.

Abstract: Disclosed are 1-(arylmethyl)quinazoline-2,4(1H,3H)-diones thereof, represented by the Formula (I) wherein Ar, R1-R6 are defined herein. Compounds having Formula (I) are PARP inhibitors. Therefore, compounds of the invention may be used to treat clinical conditions that are responsive to the inhibition of PARP activity.

Abstract: The invention provides for methods of treating a mammal who has a BCR/ABL-mediated leukemia, including chronic myelogenous leukemia (CML), particularly the blast crisis stage of CML, Philadelphia-positive acute lymphoblastic leukemia (Ph?-ALL), and refractory leukemias. The invention also provides for compounds for the treatment of these leukemias and methods of identifying anti-leukemic agents.

Abstract: The present invention is directed to cyclodepsipeptide compounds having antineoplastic and/or antimicrobial activity, preferably Kitastatin 1. The present invention is further directed to methods of inhibiting cancer cell growth and/or microbial growth in a host inflicted therewith by administering cyclodepsipeptide compounds to the inflicted host.

Abstract: This invention describes an ICE inhibitor prodrug (I) having good bioavailability. Compound I is useful for treating IL-1 mediated diseases such as rheumatoid arthritis, inflammatory bowel disease, Crohn's disease, ulcerative colitis, inflammatory peritonitis, septic shock, pancreatitis, traumatic brain injury, organ transplant rejection, osteoarthritis, asthma, psoriasis, Alzheimer's disease, myocardial infarction, congestive heart failure, Huntington's disease, atherosclerosis, atopic dermatitis, leukemias and related disorders, myelodysplastic syndrome, uveitis or multiple myeloma.

Abstract: Disclosed are peptides for inhibiting mdm2 (mouse double minute 2) and a pharmaceutical composition comprising the same.

Abstract: The present application includes methods for using IL-8 as a biomarker for, e.g., tumor size, for example, during course of treatment with an anti-cancer agent such as an ERK inhibitor.

Abstract: A recombinant galectin 9 (rGal 9) is provided which exhibits an immune system-mediated action and a direct action on tumor cells (i.e., activity of inducing the intercellular adhesion and apoptosis of the tumor cells), thereby potent in inducing the inhibition of cancer metastasis and reduction. Moreover, the rGal 9 exerts no efficacy on non-activated lymphocytes but can induce apoptosis in activated T cells, in particular, CD4-positive T cells causing an excessive immune response.

Abstract: The present invention relates to a method of treating cancer in a human and to pharmaceutical combinations useful in such treatment. In particular, the method relates to a cancer treatment method that includes administering a proteasome inhibiting compound, and N-{(1S)-2-amino-1-[(3-fluorophenyl)methyl]ethyl}-5-chloro-4-(4-chloro-1-methyl-1H-pyrazol-5-yl)-2-thiophenecarboxamide, or a pharmaceutically acceptable salt thereof, and optional additional antineoplastic agents, to a human in need thereof.

Abstract: Described herein is a pharmaceutical composition for treating a subject having a cancer and/or myeloproliferative disorder associated with overexpression of a HOXA1 gene product where an effective amount of at least one miR-10a gene product or an isolated variant or biologically-active fragment thereof is administered to the subject sufficient to decrease expression of the HOXA1 gene product in the subject.