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| 551 | Matrix Screening Method | NCATS experts implemented and improved the matrix combination screening method to help researchers more quickly identify the best drug combinations. The method includes three major steps described below. Step 1: Generate single agent results: Step 2: Generate 6×6 matrix data to uncover potential synergies among agents: Step 3: Expand good combinations to 10×10 blocks to confirm synergistic combinations and perform self-cross analyses to provide context for activities: | ||||||||
| 550 | video Example | stuff hiiii // | ||||||||
| 548 | CTSA Resources Support Largest U.S. Newborn Screening Study for Fragile X Mutations | Thanks to dramatic advances in genetics and DNA sequencing technologies, today’s clinicians have increased capabilities to more accurately diagnose inherited diseases. They also now know that disease-causing gene changes, or mutations, often are more common than previously thought. A team of researchers at the University of California, Davis (UC Davis) studying the rare disease Fragile X syndrome (FXS) found that, indeed, more people have gene changes linked to this disease than anticipated. FXS is an inherited condition caused by changes in a gene on the X chromosome that lead to intellectual disability, behavioral and learning challenges, and various physical characteristics. Previous estimates suggested that FXS affects one in every 2,500 to 8,000 individuals, but it is not known how many people in the general population have the associated genetic changes. The UC Davis team set out to find a way to determine the prevalence of FXS mutations in the general population before symptoms appear by aiming to show that large-scale newborn screening for FXS mutations was technically and logistically possible and could fill in crucial knowledge gaps. After five years of intensive work and with support from the university’s Clinical and Translational Science Center (CTSC), these researchers made their goal a reality and published findings in the Dec. 21, 2012, issue of Genome Medicine. The National Institutes of Health (NIH) provides funding to the UC Davis CTSC through its Clinical and Translational Science Awards (CTSA) program, which is administered by the National Center for Advancing Translational Sciences (NCATS). NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development funded the project with additional support from the Centers for Disease Control and Prevention and the Association for Prevention Teaching and Research. Using newborn screening to diagnose FXS might enable children with the condition to start treatments earlier, which can improve their quality of life as they get older. Newborn screening is an unbiased way to assess general prevalence of mutations that cause FXS and many other inherited diseases. Identifying mutations at birth can prompt earlier diagnosis and treatment, enable testing for family members who might be affected, and provide those who have mutations but no symptoms with genetic counseling about the risk of passing on the mutation to their children. Having a more accurate prevalence estimate also would help public health officials set aside adequate resources for treatment, patient education and genetic counseling. “This was the very first study in the United States looking at FXS in the general population of newborns,” said Flora Tassone, Ph.D., a professor at the UC Davis Medical Investigation of Neurodevelopmental Disorders (MIND) Institute and lead researcher on the FXS study. “We needed expertise for designing the study and for collecting and analyzing the data on the overall prevalence of FXS mutations.” To meet that need, Tassone’s team turned to Danh Nguyen, Ph.D., who was then the associate director of the Biostatistics, Epidemiology and Research Design (BERD) unit at UC Davis’ CTSC and current director of the BERD unit at UC Irvine. Among many critical resources, CTSA institutions offer expertise in study design and data collection and analysis that can help speed research progress. “Nguyen and the CTSA-supported BERD unit were key players in designing the study,” Tassone said. “Without them, I don’t think we could have done this research.” Understanding a Rare Disease NCATS supports research aimed at improving knowledge of and finding treatments for rare diseases, including FXS. In most FXS patients, a specific DNA segment is expanded within the Fragile X Mental Retardation 1 (FMR1) gene on the X chromosome. Normally, this DNA segment repeats from 5 to about 44 times. In people with FXS, however, the segment repeats more than 200 times and is called a full FMR1 mutation. The abnormal segment turns off the FMR1 gene, and as a result, cells do not produce enough of a protein necessary for proper nervous system function, leading to FXS symptoms. Some people who do not have the full mutation can still have an abnormal number of repeats, which can produce a variety of symptoms. People with 55 to 200 repeats are said to have the FMR1 gene premutation, and people with 45 to 54 repeats have gray zone, or intermediate, expansions. Individuals with a premutation have normal functioning, but sometimes they have autism spectrum disorders, attention deficit-hyperactivity disorder, depression, anxiety, a form of premature menopause and a neurological condition resembling Parkinson’s disease. People who fall within any of these categories — full mutation, premutation or gray zone expansion — can pass on these genetic changes to their children. A Team-Based Approach to the Problem of Prevalence Team-based models like the one at the UC Davis CTSC ensure research projects benefit from knowledge and expertise not otherwise available to a single researcher. For this study, the researchers wanted to carry out a large-scale newborn screening pilot study, but they were unsure of how to design the study and analyze the data. Biostatisticians in the CTSC’s BERD unit provided the statistical expertise necessary for planning and analyzing data from population studies of this size and scope. An NICHD grant enabled the researchers to establish screening sites in three locations: University of North Carolina Hospital in Chapel Hill, led by Don Bailey, Ph.D., of RTI International; Rush University Medical Center in Chicago, led by Elizabeth Berry-Kravis, M.D., Ph.D.; and UC Davis Medical Center. For each newborn in the study, the team collected a few drops of blood, which were stored on filter paper. In many states, infants’ blood spots are routinely screened for developmental disorders and other conditions. Across the three sites, Tassone and her team ultimately collected 14,207 male and female newborn blood spot samples. Coordinating study personnel and data collection across three sites in opposite corners of the country required superb attention to detail. With their epidemiologic and biostatistical expertise, staff from the BERD unit provided much-needed support to the clinical team for “monitoring data collection and making sure that the data were of the quality needed for the analysis,” Nguyen explained. With the data collection and blood analysis complete, Nguyen set about analyzing the data. The results revealed that 1 in 66 females and 1 in 112 males carried the gray zone expansion. The premutation occurred in 1 in 209 females and 1 in 430 males. The data set was too small to detect the prevalence of the full mutation in the general population, but the study’s findings on the premutation and gray zone carriers add important new information to the FXS knowledge base. “BERD’s role in this study was integral to this research project,” said Tiina Urv Ph.D., health scientist administrator at NICHD. “The newborn screening pilot program was a great example of research collaboration. Every person on the research team focused on what they did best. By doing this and by using multiple available resources the scientific process was able to move forward efficiently and effectively. ” Implications for Family and Public Health Identifying FXS premutation carriers has implications beyond just their own clinical futures. “When you identify a baby with the premutation, you identify a whole family that may have a different kind of involvement,” said Randi Hagerman, M.D., UC Davis’ MIND Institute Director and last author on the study. For this reason, MIND Institute researchers have begun recruiting affected family members of screened newborns to participate in ongoing clinical trials. The clinical resource component of the UC Davis CTSC has a hand in this stage of the effort as well. “The CTSC is critically important for the follow-up work we do with the family members, helping us with all of our treatment trials,” Hagerman said. “This work is opening up whole new areas of clinical involvement and allowing us to evaluate premutation carriers in much greater detail.” In addition to the family member follow-up, the multidisciplinary collaborations that enabled the screening study have led to other ongoing research. One such effort, which involves Tassone and other screening study authors, involves checking up on the screened infants when they are 18–24 months old. “In light of this pilot study, it is important to confirm and demonstrate that we see symptoms early in life in the premutation carriers,” Tassone said. “We are seeing different developmental characteristics between carriers and non-carriers.” This pilot study, which began by collecting prevalence data, now has set the stage for a more comprehensive examination of the clinical consequences of FXS-related gene changes. Tassone and Hagerman hope to conduct a larger screening study soon that will confirm the findings of the initial study, follow the identified premutation carriers over a longer period of time and determine the prevalence of the full mutation in the general population. Posted July 2013 | ||||||||
| 547 | About Matrix Combination Screening | The current standard of care for many aggressive diseases, such as cancer and HIV, depends on drug combinations. For example, certain chemotherapy regimens used for the treatment of blood cancers can include up to five or more drugs. Historically, these regimens were established through a lengthy, trial-and-error clinical process. With an increasing number of approved and investigational drugs, the discovery of novel drug combinations through clinical trial-and-error is not feasible. To address this inefficiency, NCATS’ Chemistry Technology team uses matrix combination screening. This platform enables NCATS scientists to quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients. The NCATS’ matrix combination screening platform can rapidly test the effect of different drug combinations on cellular, molecular or biochemical processes that are relevant to a disease of interest. The Chemistry Technology team screens the best pairs to find the optimal concentration of each drug and learn more about their effect on cells, such as whether that combination is toxic. Scientists can use the results of these screens to pursue testing of promising drug combinations in animals and, ultimately, humans. The Matrix Combination Screening Platform The state-of-the-art, qualitative, high-throughput matrix combination screening platform relies on a highly integrated robotics system, custom data analysis software and a unique Web-interface that provides straightforward and intuitive data browsing. Thousands of combination-pairs can be analyzed with this platform in a robust, cost and time-effective way, enabling NCATS scientists to quickly narrow down a long list of drug-pairs to identify the most-effective drug combinations for follow-up and clinical studies. The unbiased discovery of drug combinations through the matrix platform may yield treatments with increased effectiveness; delayed onset of resistance and deliverable at lower, less toxic doses. As of December 2017, NCATS scientists have initiated two clinical trials and several preclinical studies based on the findings from this platform. Top: Heat map representing activities of different drug combinations from a compound library activity derived from an assay determined through quantitative high-throughput matrix screening. The colors indicate different responses (i.e., inhibitory or activation response), and color intensity represents potency. Bottom left: Examples of two compounds by name and chemical structure from the NCATS compound collection. Bottom right: An example of a 10×10 dose-response matrix block experiment.) Matrix Screening Projects NCATS scientists work on a variety of matrix combination screening projects. Learn more about NCATS’ active projects. | NCATS experts use a technology called matrix combination screening to quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients. | /sites/default/files/tox21-chem-tox-robot-carousel.jpg | About Matrix Combination Screening | NCATS experts use a technology called matrix combination screening to quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients. | /sites/default/files/tox21-chem-tox-robot-carousel.jpg | About Matrix Combination Screening | ||
| 530 | New Therapeutic Uses Projects | New Therapeutic Uses for Experimental Assets NCATS matches academic research groups with assets provided by pharmaceutical partners, as part of the New Therapeutic Uses program’s NIH-Industry Partnerships initiative. The goal is to use molecules that have already undergone significant research and development by pharmaceutical companies to more quickly advance new treatments for patients. Awarded projects support preclinical validation studies, clinical feasibility studies or proof-of-concept clinical trials to test whether the selected assets may be effective against a previously unexplored disease. View the 2020 project. View the 2018 projects. Therapeutic/Indication Pairing Strategies NCATS has released a set of funding opportunities to explore a potential new use of an existing investigational therapy, Food and Drug Administration-approved drug, or licensed biologic. Through the Bench-to-Clinic Repurposing initiative, NCATS will support preclinical studies, clinical feasibility studies or proof-of-concept clinical trials to test the utility of an independent crowdsourcing effort or computational algorithm to predict new uses of a drug or biologic. View the 2020 project. View the 2019 projects. View the 2018 projects. View the 2017 projects. View the 2016 projects. Repurposing Off-Patent Drugs Workshop On Dec. 5, 2019, NCATS hosted Repurposing Off-Patent Drugs: Research & Regulatory Challenges to discuss challenges around finding new uses for drugs that are already on the market but lack commercial and regulatory incentives for research and development. | This page features links to projects for the Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. | /sites/default/files/socialcard.jpg | New Therapeutic Uses Projects | This page features links to projects for the Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. | /sites/default/files/socialcard.jpg | New Therapeutic Uses Projects | ||
| 528 | Template Agreements | The template agreements for the Discovering New Therapeutic Uses for Existing Molecules program are available here. They are designed to streamline the legal and administrative process for partnering across multiple organizations. The agreements save time and effort, and they provide a roadmap for handling intellectual property used in or developed through the program. During the pilot program, the templates helped shorten the time required to establish public-private collaborations to about three months instead of the more typical nine months to one year. Memorandum of Understanding Template MOU (PDF - 86KB) Confidential Disclosure Agreements AstraZeneca (PDF - 73KB) Janssen Research & Development, LLC (PDF - 130KB) Pfizer Inc (PDF - 42KB) Collaborative Research Agreements AstraZeneca (PDF - 232KB) Janssen Research & Development, LLC (PDF - 403KB) Pfizer Inc (PDF - 127KB) | ||||||||
| 527 | 2012 Industry-Provided Assets | The funding opportunity related to these assets has expired. View the 2014 industry-provided assets. Assets in this table are alphabetized by mechanism of action. These compounds and biologics have undergone significant preclinical and safety testing in humans. Table of Compounds and Biologics Code Number & Link to More Information Mechanism of Action Original Development Indication(s) Route of Administration Formulation Available (CNS Penetrant+) AVE5530 (PDF - 128KB) canosimibe Acyl-coenzyme A:cholesterol O-acyltransferase (ACAT) inhibitor Cholesterol absorption inhibitor Hypercholesterolemia Oral SSR149744C (PDF - 145KB) celivarone Anti-arrhythmic, Vaughan Williams Class I to IV Maintenance of sinus rhythm in atrial fibrillation patients Prevention of shocks and major clinical outcomes in patients with implanted cardiac defibrillator Oral PF-05416266 (PDF - 92KB) senicapoc (ICA-17043) Calcium-activated potassium channel blocker (KCa3.1), intermediate-conductance Sickle cell disease Asthma Oral ABT-639 (PDF - 87KB) Calcium channel, voltage-gated (Cav3.2, T-type) blocker Pain Oral (Yes) CP-945598 (PDF - 126KB) otenabant Cannabinoid receptor 1 (CB1) antagonist Obesity Oral (Yes) LY2828360 (PDF - 92KB) Cannabinoid receptor 2 (CB2) agonist Osteoarthritis pain Oral (Yes) AZD1981 (PDF - 115KB) Chemoattractant receptor-homologous molecule expressed on Th2 cells (CRTh2)/prostaglandin D2 (DP2) receptor antagonist Asthma Chronic obstructive pulmonary disease Oral SSR150106 (PDF - 151KB) Chemokine receptor antagonist (TNFα release) Rheumatoid arthritis pain Oral AZD2423 (PDF - 96KB) Chemokine (C-C motif) receptor 2 (CCR2) antagonist Chronic obstructive pulmonary disease Pain Oral PF-04136309 (PDF - 169KB) Chemokine (C-C motif) receptor 2 (CCR2) antagonist Chronic osteoarthritis pain Oral (No) CE-326597 (PDF - 152KB) Cholecystokinin 1 receptor (CCK-1R) (or CCKA receptor) agonist Obesity in type II diabetes Oral BMS-562086 (PDF - 89KB) pexacerfont Corticotropin-releasing factor 1 (CRF1) receptor antagonist Major depressive disorder Generalized anxiety disorder Irritable bowel syndrome Oral (Yes) JNJ-39269646 (PDF - 94KB) Fast dissociating D2/D3/5-HT6 antagonist Schizophrenia Oral (Yes) ZD4054 (PDF - 92KB) zibotentan Endothelin receptor A (ETA) antagonist Oncology Pulmonary artery hypertension Oral PF-00913086 (PDF - 200KB) prinaberel (ERB-041) Estrogen receptor β (ERβ, ESR2) agonist/nuclear receptor 3A2 (NR3A2) agonist Endometriosis Oral LY500307 (PDF - 101KB) Estrogen receptor β (ERβ, ESR2) agonist/nuclear receptor 3A2 (NR3A2) agonist Benign prostatic hyperplasia Lower urinary tract symptoms Oral XRP0038 (PDF - 200KB) Riferminogene pecaplasmid, NV1FGF, temusi Non-viral fibroblast growth factor 1 (NV1FGF) Critical limb ischemia Intramuscular (No) AZD7325 (PDF - 153KB) γ-Aminobutyric acid (GABA) A receptor, alpha 2 (GABAA α2) agonist Generalized anxiety disorder Oral (Yes) AZD3355 (PDF - 167KB) lesogaberan γ-Aminobutyric acid (GABA) B receptor, 1 (GABAB1) agonist Gastroesophageal reflux disease Oral PF-05190457 (PDF - 77KB) Ghrelin receptor (growth hormone secretagogue 1a receptor, GHS-1aR) inverse agonist, competitive antagonist Type II diabetes Oral (Yes) AZD1656 (PDF - 1656KB) Glucokinase (GK) activator Diabetes Oral BMS-820132 (PDF - 82KB) Glucokinase (GK) activator Type II diabetes Oral PF-03463275 (PDF - 138KB) Glycine transporter 1 (GlyT1) inhibitor Solute carrier family 6 (neurotransmitter transporter, glycine), member 9 (SLC6A9) inhibitor Schizophrenia Oral (Yes) HMR1766 (PDF - 122KB) ataciguat Soluble guanylate cyclase (sGC) activator Angina pectoris Peripheral artery disease Neuropathic pain Oral GSK1004723 (PDF - 116KB) Histamine H1/H3 receptor antagonist Allergic rhinitis Topical (No) GSK835726 (PDF - 109KB) Histamine H1/H3 receptor antagonist Allergic rhinitis Oral (No) ABT-288 (PDF - 78KB) Histamine H3 receptor antagonist Cognition (cognitive deficits of schizophrenia) Oral (Yes) PF-03654746 (PDF - 192KB) Histamine H3 receptor antagonist Narcolepsy Cognition Attention deficit hyperactivity disorder Allergic rhinitis Oral (Yes) CE-210666 (PDF - 113KB) 5-Hydroxytryptamine 1B receptor (5-HT1B) antagonist Depression Oral (Yes) CP-448187 (PDF - 142KB) elzasonan 5-Hydroxytryptamine 1B receptor (5-HT1B) antagonist Depression Oral (Yes) PH-670187 (PDF - 123KB) dermaciclane, EGIS-3886 5-Hydroxytryptamine 2A/2C receptor (5-HT2A/2C) antagonist Generalized anxiety disorder Oral (Yes) PF-04995274 (PDF - 138KB) 5-Hydroxytryptamine 4 receptor (5-HT4) partial agonist Gastroesophageal reflux disease Alzheimer's disease Oral (Yes) JNJ-18038683 (PDF - 67KB) 5-Hydroxytryptamine 7 receptor (5-HT7) antagonist Major depressive disorder Oral (Yes) LY2590443 (PDF - 70KB) 5-Hydroxytryptamine 7A receptor (5-HT7A) antagonist Migraine Oral MEDI2338 (PDF - 105KB) Interleukin-18 (IL-18) inhibitor Chronic obstructive pulmonary disease Coronary heart disease Intravenous, subcutaneous PF-04191834 (PDF - 130KB) 5-Lipoxygenase (5-LO) inhibitor Asthma Chronic osteoarthritis pain Oral (Yes) SD-7300 (PDF - 137KB) (SC-81490) Matrix metalloproteinase 2, 9, and 13 (MMP-2, -9, -13) inhibitor Post-myocardial infarction cardiac remodeling Disease-modifying osteoarthritis drug Oral AZD1236 (PDF - 78KB) Matrix metalloproteinase 9|12 (MMP9|MMP12) inhibitor Chronic obstructive pulmonary disease Oral BMS-830216 (PDF - 79KB) Melanin-concentrating hormone 1 (MCH1) receptor antagonist Obesity Oral (Yes) AZD5904 (PDF - 93KB) Myeloperoxidase (MPO) inhibitor Chronic obstructive pulmonary disease Multiple sclerosis Oral SAR103168 (PDF - 145KB) Multi-kinase inhibitor Liquid tumors (acute myeloid leukemia) Intravenous ABT-089 (PDF - 107KB) Nicotinic acetylcholine receptor (nAChR) partial agonist (α4β2* subtypes) Alzheimer's disease Attention deficit hyperactivity disorder Oral (Yes) CP-601927 (PDF - 182KB) Nicotinic acetylcholine receptor α4β2 (α4β2 nAChR) partial agonist Multiple CNS Oral (Yes) AZD0328 (PDF - 94KB) Nicotinic acetylcholine receptor alpha 7 (α7 nAChR) agonist Cognitive impairment Oral (Yes) JNJ-39393406 (PDF - 118KB) Nicotinic acetylcholine receptor, α7 (α7nAChR) positive allosteric modulator Cognitive impairment in schizophrenia Oral (Yes) GW274150 (PDF - 110KB) Inducible nitric oxide synthase (iNOS) inhibitor Migraine (acute and prophylaxis) Asthma Rheumatoid arthritis Sepsis Oral (Poor) SD-6010 (PDF - 83KB) (SC-84250) Inducible nitric oxide synthase (iNOS) inhibitor Asthma Osteoarthritis pain Disease-modifying osteoarthritis drug Oral AZD7268 (PDF - 193KB) δ Opioid receptor agonist Anxiety Depression Oral (Yes) AVE8134 (PDF - 117KB) Peroxisome proliferator-activated receptor α (PPARα)/nuclear receptor 1C1 (NR1C1) agonist Type II diabetes Oral AVE0847 (PDF - 189KB) Peroxisome proliferator-activated receptor α/γ (PPARα/γ)/nuclear receptor 1C1/1C3 (NR1C1/1C3) agonist Type II diabetes Oral PF-05019702 (PDF - 139KB) (PRA-27) Progesterone receptor (PR) antagonist/nuclear receptor 3C3C (NRC3C) antagonist Endometriosis Oral AZD9056 (PDF - 115KB) Purinergic receptor 2X, ligand-gated ion channel, 7 (P2X7) antagonist Chronic obstructive pulmonary disease Crohn's disease Osteoarthritis Rheumatoid arthritis Oral LY2245461 (PDF - 71KB) Selective estrogen receptor modulator (SERM) Hot flashes in postmenopausal women Oral AZD0530 (PDF - 65KB) saracatinib Src tyrosine kinase inhibitor Oncology Oral SB223412 (PDF - 122KB) talnetant Tachykinin receptor/neurokinin 3 (NK3) receptor antagonist Cough Chronic obstructive pulmonary disease Schizophrenia Irritable bowel syndrome Overactive bladder Oral (Yes) SAR115740 (PDF - 146KB) Transient receptor potential cation channel vanilloid 1 (TRPV1) antagonist Acute and chronic pain Oral SSR97225 (PDF - 150KB) β-Tubulin-binding agent with dual mechanism Solid tumor Intravenous, injectable suspension AZD2171 (PDF - 123KB) cediranib Vascular endothelial growth factor receptor (VEGFR) 1, 2, and 3 tyrosine kinase inhibitor Oncology Oral +CNS penetrant: Yes, no or unknown. | ||||||||
| 525 | Industry-Provided Assets | table td{padding:5px;} Assets in this table are alphabetized by mechanism of action. These compounds and biologics have undergone significant preclinical and safety testing in humans. 2020 Table of Assets Code Number & Link to More Information Mechanism of Action Original Development Indication(s) Route of Administration Formulation Available (CNS Penetrant+) AZD1656 (PDF - 136KB) Glucokinase (GK) activator Type 2 Diabetes Oral (No/Low CNS) AZD5213 (PDF - 136KB) Histamine receptor 3 (H3) antagonist (inverse agonist) Neuro Oral (CNS) AZD5904 (PDF - 128KB) Myeloperoxidase (MPO) inhibitor Multiple sclerosis (MS), and Chronic obstructive pulmonary disease (COPD) Oral (No/Low CNS) ZD4054 (PDF - 89KB) zibotentan Endothelin type A (ETA) antagonist Oncology Oral (Low CNS) PF-04995274 (PDF - 138KB) 5-Hydroxytryptamine 4 receptor (5-HT4) partial agonist Gastroesophageal reflux disease Alzheimer's disease Oral (Yes) CE-224535 (PDF - 203KB) Purinergic receptor 2 antagonist Rheumatoid arthritis Osteoarthritis Oral JNJ-18038683 (PDF - 67KB) 5-Hydroxytryptamine 7 receptor (5-HT7) antagonist Major depressive disorder Oral (Yes) JNJ-39269646 (PDF - 34KB) Fast dissociating D2/D3/5-HT6 antagonist Schizophrenia Bipolar depression Oral (Yes) | ||||||||
| 523 | Assets & Agreements for NIH-Industry Partnerships | Through the New Therapeutic Uses NIH-Industry Partnerships initiative, NCATS has collaborated with several pharmaceutical industry partners to make a number of partially developed assets available to academic researchers to crowdsource ideas for new uses. Projects using these assets are designed to go directly into Phase II clinical trials, studies that provide data on the preliminary effectiveness for the intended use. Some projects may need a Phase I clinical trial within the target populations to determine dosing, safety and tolerability. Learn more about these industry-provided assets. The private sector holds many of the assets and data needed for efficient drug repurposing. However, because innovative ideas for new uses of these resources can come from a variety of organizations, including NIH and the broader biomedical research community, public-private partnerships and collaborations are critical to this research. Through the New Therapeutic Uses program’s NIH–Industry Partnerships initiative, pharmaceutical companies provide researchers with access to the partially developed therapeutic candidates (referred to as assets) and related data. These assets have undergone significant preclinical and safety testing in humans and are ready for additional testing in patient populations. Making information about these promising assets available to the research community gives scientists an opportunity to explore potential new therapeutic uses in previously unexplored disease areas. NCATS invites collaborating pharmaceutical companies to identify assets suitable for adult indications as well as for pediatric indications. Participating companies provide clinical supplies of drugs and matched placebos to funded investigators at no charge. These companies also provide documentation so that funded investigators can file an Investigational New Drug application with the Food and Drug Administration. To streamline the legal and administrative process for partnering across multiple organizations, NIH created template agreements. These agreements help facilitate complex negotiations among all parties involved in the program, enabling the research to begin faster. Learn more about the template agreements. Current Table of Assets Learn more about becoming a pharmaceutical company partner for the New Therapeutic Uses program. | ||||||||
| 522 | New Therapeutic Uses Contacts | New Therapeutic Uses Program Contacts Christine Colvis, Ph.D. 301-451-3903 Bobbie Ann Mount, Ph.D. 301-435-0824 Strategic Alliances and Licensing Contact Lili Portilla, M.P.A. 301-217-4679 | New Therapeutic Uses Contacts | New Therapeutic Uses Contacts |
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