Abstract: A novel approach to discover new drugs against MS and other autoimmune diseases is disclosed. The p40 family of cytokines has four members which include interleukin-12 (IL-12), the p40 monomer (p40), the p40 homodimer (p402), and the IL-23. To facilitate the studies on p402 and p40, neutralizing monoclonal antibodies (mAb) against mouse p402 and p40 were generated for the first time. MS and other autoimmune disease drug therapies including recombinant p40 and/or monoclonal antibody against p402 (mAb-p402 a3-1d) are disclosed.

Abstract: One aspect of the invention provides a method for treating a cancer including administering to a subject in need of such treatment a composition including a therapeutically effective amount of an antibody directed against p40 monomer or an immunologically active fragment thereof. In various embodiments, the antibody is a polyclonal, monoclonal, human, humanized, and chimeric antibody; a single chain antibody or an epitope-binding antibody fragment. In other embodiments, the cancer is, for ex ample, prostate cancer, breast cancer or liver cancer.

Abstract: This disclosure relates to pharmaceutical compositions useful for inhibiting the progression of urea cycle disorders and neurodegenerative disorders, such as Parkinson's disease, Alzheimer's disease, and multiple sclerosis. The pharmaceutical compositions may include glyceryl tribenzoate and glyceryl dibenzoate. The pharmaceutical compositions may be orally administered to the patient one time per day.

Abstract: One aspect of the invention provides a method for treatment of a lysosomal storage disorder. The method may include administering to a subject in need of such treatment a composition including a therapeutically effective amount of an agent that mediates upregulation of Transcription Factor EB. In one embodiment, the composition includes a fibrate, such as gemfibrozil or fenofibrate. In another embodiment, the composition also includes all-trans retinoic acid or vitamin A.

Abstract: Provided herein are methods for treatment of a neurodegenerative disease, such as neuronal ceroid lipofuscinosis including administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent that mediates upregulation of TPP1.

Abstract: Provided herein are methods for treatment of a neurodegenerative disease, such as neuronal ceroid lipofuscinosis including administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent that mediates upregulation of TPP 1.

Abstract: This disclosure relates to pharmaceutical compositions useful for inhibiting the progression of urea cycle disorders and neurodegenerative disorders, such as Parkinson's disease, Alzheimer's disease, and multiple sclerosis. The pharmaceutical compositions may include glyceryl tribenzoate and glyceryl dibenzoate. The pharmaceutical compositions may be orally administered to the patient one time per day.

Abstract: Alzheimer's disease (AD) is the most common human neurodegenerative disease of the CNS resulting in progressive neuronal death and memory loss. Despite intense investigations, no effective therapy is available to stop its onset or halt its progression. It was discovered that antisense oligonucleotide against neutral sphingomyelinase and GW4869, a chemical inhibitor of neutral sphingomyelinase, inhibit activation of glial cells and protect neurons in AD cell culture and animal models. These results suggest the following new treatment options for AD patients: Antisense oligonucleotide against neutral sphingomyelinase and GW4869.

Abstract: Provided herein are methods for treatment of a neurodegenerative disease, such as neuronal ceroid lipofuscinosis including administering to a subject in need of such treatment a composition comprising a therapeutically effective amount of an agent that mediates upregulation of TPP 1.

Abstract: Alzheimer's disease (AD) is the most common human neurodegenerative disease of the CNS resisting in progressive neuronal death and memory loss. Despite intense investigations, no effective therapy is available to stop its onset or halt its progression. It was discovered that antisense oligonucleotide against neutral sphingomyelinase and GW4889, a chemical inhibitor of neutral sphingomyelinase, inhibit activation of glial cells and protect neurons in AD cell culture and animal models. These results suggest the following new treatment options for AD patients: Antisense oligonucleotide against neutral sphingomyelinase and GW4889.

Abstract: Alzheimer's disease (AD) is the most common human neurodegenerative disease of the CNS resulting in progressive neuronal death and memory loss. Despite intense investigations, no effective therapy is available to stop its onset or halt its progression. It was discovered that antisense oligonucleotide against neutral sphingomyelinase and GW4869, a chemical inhibitor of neutral sphingomyelinase, inhibit activation of glial cells and protect neurons in AD cell culture and animal models. These results suggest the following new treatment options for AD patients: Antisense oligonucleotide against neutral sphingomyelinase and GW4869.

Abstract: A novel approach to discover new drugs against MS and other autoimmune diseases is disclosed. The p40 family of cytokines has four members which include interleukin-12 (IL-12), the p40 monomer (p40), the p40 homodimer (p402), and the IL-23. To facilitate the studies on p402 and p40, neutralizing monoclonal antibodies (mAb) against mouse p402 and p40 were generated for the first time. MS and other autoimmune disease drug therapies including recombinant p40 and/or monoclonal antibody against p402 (mAb-p402 a3-1d) are disclosed.

Abstract: Alzheimer's disease (AD) is the most common human neurodegenerative disease of the CNS resulting in progressive neuronal death and memory loss. Despite intense investigations, no effective therapy is available to stop its onset or halt its progression. It was discovered that antisense oligonucleotide against neutral sphingomyelinase and GW4869, a chemical inhibitor of neutral sphingomyelinase, inhibit activation of glial cells and protect neurons in AD cell culture and animal models. These results suggest the following new treatment options for AD patients: Antisense oligonucleotide against neutral sphingomyelinase and GW4869.

Abstract: Provided herein are methods and materials for treating neurodegenerative disorders. The methods may use inhibitors of small G-proteins, such as p21rac, p21ras, or the combination thereof.