Abstract: The invention provides methods of preventing, treating or ameliorating autoimmune diseases, such as diabetes and celiac disease, by decreasing the binding of MHC class II molecules to antigenic peptides or fragments of antigenic peptides of the autoimmune disease by the administration of small organic compounds. The invention also provides pharmaceutical compositions comprising the therapeutically effective small organic compounds and methods of using the same.

Abstract: The invention features compositions and methods that are useful for the treatment of neoplasia (e.g., breast cancer) by increasing DNA damage and reducing base excision repair (BER).

Abstract: A method of treating cancer in a subject comprising the step of administering to the subject in need thereof an effective amount of a combination of a compound that binds a nuclear export signal (NES inhibitor) on topoisomerase II? and a topoisomerase inhibitor. Twenty small molecule inhibitors (SMI) that bind to the two nuclear export sequences (NES) topo II? have been identified from the NCI database using computer-generated molecular modeling. These SMI will improve the effectiveness of topo II directed therapeutics, particularly in the treatment of diseases such as multiple myeloma (MM). In vitro apoptosis assays indicate that these drugs may be effective as single agents or in combination with currently used cancer drugs that target topo II.

Abstract: The invention relates to cyclin dependent kinase inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions for treating cell proliferative disorders, especially cancer.

Abstract: Topoisomerase II alpha (topo II?) is exported from the cell nucleus in human myeloma cells by a chromosome-maintenance protein-1 (CRM1)-dependent mechanism, resulting in topo II inhibitor resistance. The nuclear export signal (NES) of topo II? is unique, making it a potential target for small molecule inhibitors. Small molecules NES inhibitors were identified, which inhibited binding of topo II? to the export receptor CRM1. Inhibition was specific to topo II? as p53 trafficking was unaffected along with topo II? protein expression and function (decatenation). These topo II?-specific nuclear export inhibitors may potentially lead to a new approach in circumventing drug resistance in multiple myeloma. The compounds provide a protocol for treating multiple myeloma or an oncogenic disease. Further, the topoisomerase II nuclear export signal inhibitor may be combined with a topoisomerase II inhibitor.

Abstract: The subject application provides small compounds that are able to suppress autophagy in various cells. These compounds are useful in augmenting the existing treatments of various cancers and microbial/parasitic infections. Thus, the subject application also provides methods of treating various types of cancers and microbial/parasitic infections. Also provided by the subject application are methods of suppressing the expansion of autophagosomes within cells or individuals and inhibiting the lipidation of autophagy-related protein 8 (Atg8).

Abstract: The present invention relates to compounds as novel thymidylate synthase inhibitors, novel strategies to treat or prevent neoplasia in a subject, methods and compositions thereof.

Abstract: The subject invention pertains to uses of PKC-iota inhibitors for treatment of breast cancer. In one embodiment, the subject invention provides novel uses of 1H-imidazole-4-carboxamide, 5-amino-1-[2,3 -dihydroxy-4-[(phosphonooxy) methyl]cyclopentyl]-,[1R-(1?, 2?, 3?, 4?)] (ICA-1) and related compounds for treatment of breast cancer. The compounds of the subject invention have potent anti-proliferative effects against human breast cancer cells. The compounds of the subject invention also inhibit the phosphorylation of IKK-?/IKK-?, induce chromatin condensation, and/or induce DNA fragmentation in cancer cells.

Abstract: The invention relates to methods of treating or preventing acquired or congenital mitochondrial diseases or disorders, including treating cell proliferation related disorders or diseases, such as cancer. The invention further relates to pharmaceutical compositions for treating such disorders or diseases.

Abstract: The invention relates to methods of treating cardiovascular and cardiopulmonary diseases and associated conditions, including hypertension. The invention further relates to pharmaceutical compositions for treating cardiovascular and cardiopulmonary diseases, especially hypertension, and lung injury.

Abstract: PKCiota inhibitor 1H-imidazole-4-carboxamide, 5-amino-1-[2,3-dihydroxy-4-[(phosphonooxy)methyl]cyclopentyl]-,[1R-(1?,2?,3?,4?)], (ICA-1), targets a unique sequence (amino acid residues 469-475) in the catalytic domain of PKCiota and inhibits PKCiota activity. The data shows suprising and unexpected ability of ICA-1 to selectively inhibit the proliferation of cells that overexpress PKCiota.

Abstract: The invention features compositions and methods that are useful for treating or preventing AAT deficiency and associated conditions. In addition, the invention provides methods for identifying compounds useful for treatment of AAT deficiency and associated conditions.

Abstract: The invention relates to protein binding inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions and methods for treating a variety of diseases and disorders, including cell proliferative disorders, especially cancer.

Abstract: The invention relates to phosphorylation inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions and methods for treating cell proliferative disorders, especially cancer.

Abstract: The invention provides methods of preventing, treating or ameliorating autoimmune diseases such as diabetes by modulating the binding of MHC class II molecules to antigenic peptides or fragments of antigenic peptides of the autoimmune disease by the administration of small organic compounds. The invention also provides pharmaceutical compositions comprising the therapeutically effective small organic compounds and methods of using the same.

Abstract: The present invention relates to compounds as novel thymidylate synthase inhibitors, novel strategies to treat or prevent neoplasia in a subject, methods and compositions thereof.

Abstract: The invention relates to methods of treating cardiovascular and cardiopulmonary diseases and associated conditions, including hypertension. The invention further relates to pharmaceutical compositions for treating cardiovascular and cardiopulmonary diseases, especially hypertension, and lung injury.

Abstract: The invention provides compounds capable of treating a subject suffering from or being susceptible to a cell proliferative disorder (especially, cancer), methods of identifying and using the compounds, pharmaceutical compositions and kits thereof.

Abstract: The invention relates to protein binding inhibitor compounds and methods of identifying and using them. The invention further relates to pharmaceutical compositions and methods for treating a variety of diseases and disorders, including cell proliferative disorders, especially cancer.

Abstract: This invention relates to therapeutic methods for treating or preventing an osteoclast-related disease or disorder in a subject identified as in need of a treatment of bone diseases, compounds for such uses, and compositions thereof.