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Photos from the National Center for Advancing Translational Sciences (NCATS)
2013
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Drug Combinations
This grid shows the results of an assay that tested 10 concentrations of ibrutinib in combination with 10 concentrations of another drug called BKM-120. The bright red areas indicate drug combinations that killed all or most of the cancer cells, whereas the dark areas indicate combinations that did not affect survival of the cancer cells.
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Identifying New Therapies for Patients
Diagram of identifying new therapies for patients through (1) pharma, which create drugs, provide agents, sign agreements with researchers and form alliances with NIH/NCATS; (2) researchers, who provide new therapeutic use ideas, access patient populations, conduct clinical trials, sign agreements with pharma and receive funding from NIH/NCATS; and (3) NIH/NCATS, which posts agent information, develops agreement templates, crowdsources ideas, provides funding to researchers and forms alliances with pharma.
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Sickle cell disease (SCD)
Sickle cell disease (SCD) is a genetic blood disorder that alters red blood cells. The disease affects millions worldwide and about 80,000 patients in the United States, in particular, one in every 500 African American births. A defect in hemoglobin (a protein that helps the cells carry oxygen through the body) causes red blood cells to become rigid and take on a crescent (sickle) shape, blocking small blood vessels and causing decreased blood flow, inflammation, pain and strokes in children. To date, the only drug approved by the Food and Drug Administration (FDA) to treat SCD is hydroxyurea, an anticancer drug that is indicated for use only in adults. Hydroxyurea is only moderately effective and has undesirable side effects that limit its use.
2012
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Cell-to-cell Communication
The National Institutes of Health awarded $17 million in 2013 for 24 research projects designed to improve scientists’ understanding of a newly discovered type of cell-to-cell communication based on extracellular (outside the cell) RNA, also called exRNA. Through these awards, scientists are exploring basic exRNA biology and developing tools and technologies that apply new knowledge about exRNA to the research, diagnosis and treatment of diseases. ExRNAs move in body fluids including fluid surrounding the brain and spinal cord, urine, and blood.
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Niemann-Pick Disease
NIH scientists began a clinical trial in January 2013, to test whether the drug cyclodextrin could be used as a possible treatment for a rare and fatal genetic disease, called Niemann-Pick disease type C1 (NPC). Scientists from the NIH’s National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) are conducting the clinical trial at the NIH Clinical Center. Reaching this stage required collaboration among government, industry, patient advocacy groups and academic researchers. In the image on the left, fibroblasts homozygous for mutations in NPC1 demonstrate an increased accumulation of red Lysotracker staining indicative of the storage disease. On the right, addition of cyclodextran rescues this lysosomal storage defect.
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RNA Interference (RNAi) Technology
Scientists at the National Institutes of Health have used RNA interference (RNAi) technology to identify dozens of genes which may represent new therapeutic targets for treating Parkinson’s disease. The findings also may be relevant to several diseases caused by damage to mitochondria, the biological power plants found in cells throughout the body. NIH scientists used RNAi to find genes that interact with parkin (green), a protein that tags damaged mitochondria (red). Mutations in parkin are linked to Parkinson’s disease and other mitochondrial disorders.
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RNA Molecules
Scientists have harnessed the power of RNA interference to study the function of many individual genes by reducing their activity levels, or silencing them. This process enables researchers to identify genes and molecules that are linked to particular diseases. To do this, researchers use small interfering RNAs, which are RNA molecules that have a complementary chemical makeup, or sequence, to that of a targeted gene. While the gene is silenced, researchers look for changes in cell function to learn about what it normally does. By silencing genes in the cell one at a time, scientists can explore and understand their complex relationship to other genes.
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Christopher P. Austin, M.D.
On September 14, 2012, NIH Director Francis S. Collins announced the appointment of Christopher P. Austin, M.D., as director of the National Center for Advancing Translational Sciences. Austin succeeded former acting director of NCATS and former director of the National Institute of Mental Health Thomas R. Insel, M.D., on September 23, 2012.
2011
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2012 mid-Atlantic regional Federal Laboratory Consortium for Technology Transfer Award
Several recipients of the 2012 mid-Atlantic regional Federal Laboratory Consortium for Technology Transfer Award (left to right): Lili Portilla, M.P.A., acting director of the NCATS Office of Policy, Communications and Strategic Alliances; Elizabeth Ottinger, Ph.D., TRND project manager at NCATS; Alan Hubbs, Ph.D., technology transfer specialist at the National Cancer Institute; Forbes Porter, M.D., Ph.D., clinical director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development and team lead for the project; and Steven Silber, M.D., vice president of established products, and Mark Kao, Ph.D., team lead in preclinical development drug safety sciences at J&J Pharmaceutical Research & Development, LLC.
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Bringing a New Drug to Market
The process of testing and approving drugs is broken into four phases: drug discovery, preclinical testing, clinical trials and FDA review. Due to high attrition rates, mainly during preclinical translation, bringing one new drug to market comes at a high cost in terms of time and resources, and in the human cost to patients and their families. One drug typically involves the investigation of up to 10,000 compounds and takes about 14 years to be approved. NCATS aims to eliminate the bottlenecks in the translation process.
