| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
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| 434 | Invention E-258-2010/0 | Novel Thio-Ether Analogues as a Treatment for Huntington's Disease Lead Inventor: Juan Marugan (NCATS) Inventors: Ehud Goldin (NHGRI), Omid Motabar (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS) Ref. No.: E-258-2010/0 Abstract: This technology is a collection of small molecules screened for their ability to prevent or reduce the cytotoxic effects of the protein, Huntingtin. Huntington's disease is a neurodegenerative disorder due to a dominantly acting expansion of a CAG trinucleotide repeat in exon 1 of the Huntington (HTT) gene resulting in production of the altered (mutant) protein Huntingtin, which has a long chain of polyglutamine (poly Q) attached to the exon 1 encoded protein sequence. | ||||||||
| 433 | Invention E-257-2010/0 | Glucocerebrosidase Activators as a Treatment for Gaucher Disease Lead Inventor: Juan Marugan (NCATS) Inventors: Ehud Goldin (NHGRI), Omid Motabar (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS) Ref. No.: E-257-2010/0 Abstract: This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Publications: Discovery, Structure-Activity Relationship, and Biological Evaluation of Noninhibitory Small Molecule Chaperones of Glucocerebrosidase • Journal of Medicinal Chemistry • June 28, 2012 • Probe Development Branch, NCATS Chemical Genomics Center New Non-Iminosugar Glucocerebrosidase Chaperone Series • MedChemComm • January 2012 • Probe Development Branch, NCATS Chemical Genomics Center A High Throughput Glucocerebrosidase Assay Using the Natural Substrate Glucosylceramide • Analytical and Bioanalytical Chemistry • January 2012 • Probe Development Branch, NCATS Chemical Genomics Center Evaluation of Quinazoline Analogues as Glucocerebrosidase Inhibitors with Chaperone Activity • Journal of Medicinal Chemistry • Feb. 24, 2011 • Probe Development Branch, NCATS Chemical Genomics Center | ||||||||
| 432 | Invention E-256-2010/0 | Alpha-Glucosidase Chaperones and Inhibitors for Treatment of Pompe Disease and Type 2 Diabetes Lead Inventor: Juan Marugan (NCATS) Inventors: Ehud Goldin (NHGRI), Noel Southall (NCATS), Wei Zheng (NCATS), Jingbo Xiao (NCATS), Ellen Sidransky (NHGRI), Omid Motabar (NHGRI) Ref. No.: E-256-2010/0 Abstract: Scientists at the NIH have discovered small molecules that can act as chaperones and correct the misfolding of mutated alpha-glucosidase enzyme. Pompe disease is caused by deficiency or dysfunction of alpha-glucosidase. The only FDA-approved treatment of Pompe disease is enzyme replacement, which in this case costs approximately $300,000 per year and elicits an immune reaction in most patients that limits clinical utility. Publications: Discovery of a Novel Noniminosugar Acid α Glucosidase Chaperone Series • Journal of Medicinal Chemistry • Sept. 13, 2012 • Probe Development Branch, NCATS Chemical Genomics Center Evaluation of 2-thioxo-2,3,5,6,7,8-hexahydropyrimido[4,5-d]pyrimidin-4(1H)-one Analogues as GAA Activators • European Journal of Medicinal Chemistry • May 2010 • Probe Development Branch, NCATS Chemical Genomics Center | ||||||||
| 431 | Invention E-240-2011/0 | Small-Molecule Inhibitors of Human Galactokinase for the Treatment of Galactosemia and Cancers Lead Inventor: Matthew Boxer (NCATS) Ref. No.: E-240-2011/0 Abstract: Lactose, found in dairy products and other foods, is comprised of two simple sugars, glucose and galactose. In galactosemia, where galactose is not properly metabolized, build-up of toxic compounds, such as galactose-1-phosphate, can lead to liver disease, renal failure, cataracts, brain damage, and even death if this disorder is left untreated. Currently, the only treatment for galactosemia is elimination of lactose and galactose from the diet, but in some cases this is not sufficient to avoid long-term complications from the disorder. | ||||||||
| 430 | Invention E-230-2009/0 | Small Molecule Kinase Inhibitors for the Potential Treatment of Down's Syndrome and Alzheimer's Disease and for Studies of Alternate Gene Splicing Lead Inventor: Craig Thomas (NCATS) Ref. No.: E-230-2009/0 Abstract: NIH investigators have discovered a series of potent, selective small molecule inhibitors of cdc2-like kinases (Clk) and dual-specificity tyrosine-regulated kinase 1A (Dyrk1A) with potential as modulators of gene splicing and for the treatment of Down's syndrome and Alzheimer's disease. Publications: Potent and Selective Small Molecule Inhibitors of Specific Isoforms of Cdc2-like Kinases (Clk) and Dual Specificity Tyrosine-Phosphorylation-Regulated Kinases (Dyrk) • Bioorganic & Medicinal Chemistry Letters • May 2011 • NCATS Chemical Genomics Center Evaluation of Substituted 6-arylquinazolin-4-amines as Potent and Selective Inhibitors of Cdc2-like Kinases (Clk) • Bioorganic & Medicinal Chemistry Letters • December 2009 • NCATS Chemical Genomics Center | ||||||||
| 429 | Invention E-211-2010/1 | Methods of Treating Giardiasis Using Food and Drug Administration-Approved Compounds Lead Inventor: Wei Zheng (NCATS) Inventors: Christopher Austin (NCATS), Catherine Chen (NCATS), Andrey Galkin (University of Maryland), Osnat Herzberg (University of Maryland Biotechnology Institute), Liudmila Kulakova (University of Maryland), Juan Marugan (NCATS), Noel Southall (NCATS) Ref. No.: E-211-2010/1 Abstract: This technology includes a group of at least twenty-nine, diverse, commercially available compounds that are newly identified for activity against Giardia lamblia parasites. At least six of the candidate compounds, Bortezomib, Decitabine, Hydroxocobalamin, Amlexanox, Idarubicin, and Auranofin have pre-existing FDA approval for human use for other (non-Giardia) conditions. Another three compounds, Fumagillin, Nitarsone and Carbadox have preexisting approval for veterinary use for non-Giardia conditions. Additional active compounds identified include: Acivicin, Riboflavin butyrate, BTO-1, GW9662, Dinitroph-dfgp, Deserpidine, Tetramethylthiuram disulsulfide, Disulfiram, Mitoxantrone, Ecteinascidin 743, 17-allyaminogeldanamycin, Carboquone and Nocodazole. The anti-Giardial activity of these compounds presents a cost saving opportunity for the rapid development of new, better tolerated treatments for the most prevalent human intestinal parasite infection in the United States and the world. Publication: High-Throughput Giardia Lamblia Viability Assay Using Bioluminescent ATP Content Measurements • Antimicrobial Agents and Chemotherapy • February 2011 • Probe Development Branch, NCATS Chemical Genomics Center | ||||||||
| 428 | Invention E-162-2008/0 | Substituted Oxadiazole 2-Oxides as a Treatment of Schistosomiasis Lead Inventor: Craig Thomas (NCATS) Inventors: Ganesha Bantukallu (NCATS), David Maloney (NCATS), Ahmed Sayed (Illinois State University), Anton Simeonov (NCATS), David Williams (Illinois State University) Ref. No.: E-162-2008/0 Abstract: Available for licensing and commercial development are pharmaceutical compositions and methods for the treatment of schistosomiasis in mammals. The various compositions are based on a number of compounds derived from 1,2,5-oxadiazole that are potent inhibitors of thioredoxin glutathione reductase (TGR), a critical parasite redox protein. Publications: Structure Mechanism Insights and the Role of Nitric Oxide Donation Guide the Development of Oxadiazole-2-Oxides as Therapeutic Agents against Schistosomiasis • Journal of Medicinal Chemistry • October 2009 • NCATS Chemical Genomics Center Identification of Oxadiazoles as New Drug Leads for the Control of Schistosomiasis • Nature Medicine • April 2008 • NCATS Chemical Genomics Center | ||||||||
| 427 | Invention E-156-2012/0 | Combination Chemotherapeutics for the Treatment of Chordoma Lead Inventor: Menghang Xia (NCATS) Inventors: Christopher Austin (NCATS), Ruili Huang (NCATS) Ref. No.: E-156-2012/0 Abstract: Utilizing high-throughput screening methodology, NIH scientists have identified two classes of clinically-available drugs, proteasome inhibitors and topoisomerase inhibitors, that synergize to promote chordoma cell death. Moreover, use of the two-part chemotherapeutic regimen in animal models effectively suppressed the growth of chordoma cells and resulted in significant tumor regression. Currently, no chemotherapeutic agents have been approved for the treatment of chordoma. Using FDA approved drugs in a combination therapeutic regimen will help expedite the availability of a therapeutic for chordoma. | ||||||||
| 426 | Invention E-148-2012/0 | Novel Tocopherol and Tocopheryl Quinone Derivatives as Therapeutics for Lysosomal Storage Disorders Lead Inventor: Wei Zheng (NCATS) Inventors: Juan Marugan (NCATS), John McKew (NCATS), Jingbo Xiao (NCATS) Ref. No.: E-148-2012/0 Abstract: Novel tocopherol derivatives and tocopheryl quinone derivatives useful in the decrease of lysosomal substrate accumulation, the restoration of normal lysosomal size, and the treatment of lysosomal storage disorders (LSDs) are provided. The inventors have discovered that tocopherol and tocopheryl quinone derivatives with side chain modifications (such as terminal tri-halogenated methyl groups) exhibit improved pharmacokinetics, modulation of mitochondrial potential and restoration of some LSDs phenotypes. | ||||||||
| 425 | Invention E-144-2012/0 | Glucocerebrosidase Activators for the Treatment of Gaucher Disease, Parkinson's Disease, and Other Proteinopathies Lead Inventor: Juan Marugan (NCATS) Inventors: Ehud Goldin (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS) Ref. No.: E-144-2012/0 Abstract: Gaucher disease is a rare lysosomal storage disease that is characterized by a loss of function of the glucocerebrosidase (GCase) enzyme, which results in a decreased ability to degrade its lipid substrate, glucocerebroside. The intracellular build up of this lipid causes a broad range of clinical manifestations, ranging from enlarged spleen/liver and anemia to neurodegeneration. In Gaucher disease, the loss of GCase function has been attributed to low levels of the protein in the lysosomal compartment, resulting from improper GCase folding and transport. Also, mutations in the GCase gene have been linked to some forms of Parkinson's disease, and may also be involved in other proteinopathies. Publications: Discovery, Structure-Activity Relationship, and Biological Evaluation of Noninhibitory Small Molecule Chaperones of Glucocerebrosidase • Journal of Medicinal Chemistry • June 28, 2012 • Probe Development Branch, NCATS Chemical Genomics Center High Throughput Screening for Small Molecule Therapy for Gaucher Disease Using Patient Tissue as the Source of Mutant Glucocerebrosidase • PLoS One • Jan. 17, 2012 • Probe Development Branch, NCATS Chemical Genomics Center A High Throughput Glucocerebrosidase Assay Using the Natural Substrate Glucosylceramide • Analytical and Bioanalytical Chemistry • January 2012 • Probe Development Branch, NCATS Chemical Genomics Center Evaluation of Quinazoline Analogues as Glucocerebrosidase Inhibitors with Chaperone Activity • Journal of Medicinal Chemistry • Feb. 24, 2011 • Probe Development Branch, NCATS Chemical Genomics Center |
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