Features and Briefs

NCATS programs and initiatives are enabling scientists to transform the way research discoveries are turned into new tools, methods and treatments. A collection of these stories and other items of interest are featured below.

View NCATS features by year:

2020

Tissue Chip Trio to Orbit Earth for Answers to Osteoarthritis and Muscle Diseases
Three tissue chip experiments exploring osteoarthritis, muscle wasting and heart tissue damage will launch into space for the Tissue Chips in Space initiative.
NCATS Support: Tissue Chips in Space

NCATS Patent Book Showcases Opportunities for Collaborations and Licensing
NCATS’ online book, Innovation at NCATS, describes 22 patents issued to NCATS and its collaborators between October 2018 and July 2020. NCATS seeks commercial partners interested in licensing and using the patents to accelerate biomedical research.
NCATS Support: Office of Strategic Alliances, Division of Preclinical Innovation

Target Watch Sheds Light on Overlooked Proteins
The Illuminating the Druggable Genome program, supported by the NIH Common Fund and administered through NCATS and the National Institute of Diabetes and Digestive and Kidney Diseases, has partnered with Nature Reviews Drug Discovery to create Target Watch, which highlights understudied protein targets.
NCATS Support: Illuminating the Druggable Genome

Teaching Children About Translational Science and Clinical Trials
University at Buffalo’s CTSA Program researchers created “Sofia Learns About Research,” a coloring and activity book that introduces children and their guardians to translational science and teaches them about the importance of clinical trials.
NCATS Support: Clinical and Translational Science Awards Program

High-Throughput Screening Helps Advance Melanoma Therapy
Finding the right compounds for new uses in treating a disease or improving a therapeutic approach can be time consuming. To speed up the process, NCATS scientists used high-throughput screening to help advance a therapy for melanoma.
NCATS Support: Early Translation Branch

The Beat Goes On — Even 258 Miles Above Earth
By using tissue chips to model cardiac dysfunction in space, researchers can advance heart disease research on Earth.
NCATS Support: Tissue Chips in Space

Rare Disease Day at NIH Fosters Connections Across the Community
The annual event — held this year on Feb. 28, 2020 — focused on forging connections in the rare diseases community, shortening the journey toward diagnosis and pursuing personalized medicine as a therapeutic approach for treating rare diseases.
NCATS Support: Rare Diseases Research

Easing the Way to Diagnosing Alzheimer’s and Rare Syndromes
Contributions from the NCATS-led Rare Diseases Clinical Research Network helped develop a new blood test that may make it easier to diagnose Alzheimer’s disease and rare neurodegenerative syndromes.
NCATS Support: Rare Diseases Clinical Research Network

A Multipronged Approach Toward Treating DMD
Experts in high-throughput screening and lysosomal biology collaborate on a multipronged approach to lessen the symptoms of Duchenne muscular dystrophy.
NCATS Support: Division of Preclinical Innovation, Early Translation Branch, Rare Diseases Research

Making Daily Pills a Thing of the Past to Improve Medication Adherence
About half of all patients in the developed world don’t take medicines as prescribed. A star-shaped capsule manufactured with support from NCATS’ Small Business Innovation Research program could help keep people from missing a dose.
NCATS Support: Small Business Innovation Research

Grants Help Move Promising Rare Disease Interventions to Clinical Trials
To close the gap between development of candidate interventions for rare diseases and clinical testing, NCATS and NICHD created rare-disease Clinical Trial Readiness grants. Funds support projects that collect data needed to move on to clinical trials.
NCATS Support: Rare Diseases Clinical Research Network

Harnessing the CTSA Program to Advance Telehealth
CTSA Program-supported researchers are harnessing their resources and expertise to evaluate access to high-quality telehealth care for rural and underserved children.
NCATS Support: Clinical and Translational Science Awards Program

2019

View Slideshow of NCATS’ 2019 Successes and Activities
Collaboration is integral to our approach for breaking down barriers in the translational science process, as illustrated in these highlights from 2019.

NCATS-Supported Consortium Plays Pivotal Role in New Porphyria Drug
Years of NIH-supported studies contributed to the U.S. Food and Drug Administration approval of a new drug for acute intermittent porphyria—a rare, debilitating condition. The NCATS-supported Porphyrias Consortium played a pivotal role in its development.
NCATS Support: Rare Diseases Clinical Research Network

Small Business Awards Propel "One-of-a-Kind" Technology
A new tissue chip technology that can predict the effects of drugs on the human nervous system is the latest discovery advanced by a small business innovation research award. Nerve-on-a-Chip® is considered a first-of-its-kind tissue chip technology.
NCATS Support: Small Business Innovation Research, Tissue Chip for Drug Screening

Ketogenic Diet Leads to Cognitive Improvement in Patients with Mild Alzheimer’s Disease
CTSA Program-supported researchers tested the hypothesis that ketones could provide a new source of energy for the brain in people with Alzheimer’s disease (AD). The study found improved brain function in people with mild AD after a three-month ketogenic diet.
NCATS Support: Clinical and Translational Science Awards Program

Following a Translational Science Path
NCATS supports several training and career development programs that not only inspire students to become scientists but also help grow the translational science workforce. Read about one student’s journey to becoming a translational scientist.
NCATS Support: Translational Science Education & Training; Early Translation Branch

NCATS BioPlanet: A Resource for Discovery
Researchers can better study how compounds and drugs affect cells with an NCATS resource that combines all pathways in human cells into one database. BioPlanet lets users easily browse, retrieve and analyze the pathways and explore pathway connections.
NCATS Support: Division of Preclinical Innovation - Early Translation Branch and Toxicology in the 21st Century (Tox21)

Research Speeds Diagnosis of Genetic Diseases in Seriously Ill Children
NCATS-supported researchers have developed an automated approach to diagnosis of genetic diseases in seriously ill children to allow faster diagnosis and initiation of treatment, and, ultimately, better outcomes. The study appeared in the April 24, 2019, issue of Science Translational Medicine.
NCATS Support: Clinical and Translational Science Awards Program

An Itch to Scratch: Scientists Identify a Potential New Approach to a Chronic Problem
People with constantly itchy skin, take heart. NCATS and National Institute of Dental and Craniofacial Research scientists report that blocking a receptor on the surface of spinal cord neurons may help relieve the chronic need to scratch. The study appeared online on July 10 in Science Translational Medicine.
NCATS Support: Assay Development and Screening Technology

CTSA Program Researchers Aim to Improve Health Care from All Sides
A research framework enables the recruitment of hospitalized patients for a wide range of studies to identify optimal care approaches during and after hospitalization. Undergraduate students play an integral role in the research as they recruit patients, conduct follow-up activities, and learn about clinical research.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Innovation Continues Through Latest HHS Ignite Team Selections
Three NCATS teams selected through the latest round of HHS’ Ignite Accelerator IDEA incubator program will begin projects to emphasize happiness as an essential component of health, establish a system to track and coordinate clinical and translational science best practices, and optimize NCATS’ Genetic and Rare Diseases (GARD) Information Center. Teams will receive support and training to develop their innovations.
NCATS Support: Division of Clinical Innovation and Office of Rare Diseases Research

CTSA Program Support Enables Development of Life-saving Blood Loss Monitor
NCATS-supported researchers have developed an innovative device that detects internal bleeding and monitors a patient’s response to blood loss. The monitor guides appropriate treatment before a patient goes into life-threatening shock. The device was approved by the U.S. Food and Drug Administration in 2018 and is in clinical use today.
NCATS Support: Clinical and Translational Science Awards Program

New Method Could Help Find Potential Treatments for Rare Diseases
Scientists in NCATS’ Assay Development and Screening Technology Program are developing new methods for screening small molecules that could help researchers test possible drugs and develop new treatments for many diseases.
NCATS Support: Assay Guidance Manual

Working Together Is the Name of the Game at Rare Disease Day at NIH
Rare Disease Day at NIH, held on Feb. 28 in 2019, exemplifies NCATS’ approach to rare diseases research: involving patients as partners throughout the translational research process to ensure that research and interventions address patients’ needs.
NCATS Support: Rare Diseases Research

NCATS Assay Recognized in Top Ten List of Innovative Scientific Achievements
NCATS developed a screening test to identify molecules that block the process of growing new blood vessels. With the test, NCATS scientists found both known and potential new anticancer drugs. The work earned a spot on the Society for Laboratory Automation and Screening’s top 10 innovative scientific achievements list for 2018.
NCATS Support: Toxicology in the 21st Century (Tox21)

Brigham and Women’s Hospital and NCATS Scientists Dial Up an Approach Against Out-of-Control Inflammation
Scientists at Brigham and Women’s Hospital collaborated with NCATS high-throughput screening experts to investigate a different approach against chronic inflammation. The team identified four compounds, including two novel synthetic molecules. This finding provides a potential opportunity to develop a new kind of drug to reduce inflammation.
NCATS Support: Division of Preclinical Innovation; Early Translation Branch

Opioids Increase the Risk of Pneumonia
NIH-supported research suggests a new danger associated with opioids: They can increase a person’s risk for pneumonia that is severe enough to warrant hospitalization.
NCATS Support: Clinical and Translational Science Awards Program

Researchers Convene to Take Stock and Take Action in the Opioid Crisis
In February, NCATS co-hosted a two-day symposium for pain and addiction researchers as part of The NIH Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM. The event highlighted key challenges and innovative approaches to finding better treatments for addiction and pain.
NCATS Support: NIH HEAL Initiative, Division of Preclinical Innovation, Assay Guidance Manual

NCATS Spearheads a New Resource for Natural Products
Canvass, a screening library for natural products, was created by NCATS in collaboration with academic and industry researchers to uncover the potential medical use of these compounds and enable the creation of new treatments for diseases. The researchers have already found some compounds with unexpected biological activities.
NCATS Support: Division of Preclinical Innovation

CTSA Program Supports Emerging Research on Health Effects of Plastics
An early-career investigator has developed techniques to study how chemicals used in medical devices affect the still-developing hearts of pediatric patients.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Releases Updated Translational Road Maps
An expanded, interactive beta version of the Drug Discovery, Development and Deployment Maps provides a more dynamic map interface for enhanced usability compared with the static version. Users can zoom into and out of areas of the process to view different levels of complexity, find information and best practices, and connect to relevant NCATS programs and resources. NCATS invites the public to explore the map and provide feedback on the current version.
NCATS Support: Office of the Director

NCATS-Supported Researchers Find Cell Source Matters for Tissue Chips
Researchers from the Tissue Chip Testing Center at Texas A&M University have evaluated a kidney-on-a-chip developed by researchers at the University of Washington and Nortis Bio. The kidney chip was able to perform the same functions as real kidneys, helping to validate this innovative research model.
NCATS Support: Tissue Chip for Drug Screening

NCATS-Supported Researchers Find Exercise May Help Protect DNA
Researchers supported by NCATS’ Clinical and Translational Science Awards (CTSA) Program who studied older caregivers found that those who exercised had longer telomeres (the caps that protect the ends of DNA). These findings may lead to better health outcomes for older adults as they age.
NCATS Support: Clinical and Translational Science Awards Program

2018

Charting a New Path: NCATS Internship Helps Promising Student Soar
Through NIH-wide training programs, NCATS scientists mentor students to strengthen their skills across research disciplines and help prepare them for successful scientific careers. These efforts can help increase diversity in the pool of translational scientists, including individuals from underrepresented groups and geographic areas. Learn more about former NCATS intern Bryan Queme.
NCATS Support: Assay Development and Screening Technology

Tissue Chip Researchers Create a Working Model of a Heart Chamber
Researchers supported by NCATS’ Tissue Chip for Drug Screening program have developed a 3-D working model of a human heart chamber.
NCATS Support: Tissue Chip for Drug Screening

A Model for Accelerating Translational Research in Real Time
Investigators at a Clinical and Translational Science Awards (CTSA) Program hub have devised and piloted an innovative approach called Real-Time IRB that reduces the time between study submission to the institutional review board and final approval by 70 percent.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Makes Science Fun for Local Middle Schoolers
As part of the 10th annual Frontiers in Science and Medicine Day on Nov. 9, 2018, nearly 100 middle school students experienced a glimpse of what it is like to be a scientist at NCATS, including hands-on experiments and lab tours.
NCATS Support: Division of Preclinical Innovation

NCATS Collaborations Lead to Potential New Drug Combinations for Childhood Cancers
To help address the challenges in treating children with cancer, a team of researchers in NCATS’ Chemistry Technology and Matrix Screening programs is rethinking treatment approaches for several difficult-to-treat childhood cancers.
NCATS Support: Matrix Combination Screening, Chemistry Technology

NCATS Researchers Develop New Approach to Identify Potential Cancer Drugs
NCATS researchers overcame a translational roadblock by developing a series of assays (tests) to identify compounds that disrupt a driver of several cancers. The work, using NCATS’ high-throughput screening technology to test thousands of compounds at once, provides a template for other scientists to discover new compounds that could be useful as cancer therapies.
NCATS Support: NCATS Chemical Genomics Center

NCATS Day 2018 Participants Share Perspectives to Advance Research Engagement
NCATS Day is a cornerstone of the Center’s efforts to engage and collaborate with a variety of diverse stakeholders, particularly patients and their communities, to advance translational science.
NCATS Support: NCATS Day 2018: Engaging Patients and Communities for Smarter Science

NCATS-Supported Research Shows Potential for Altering Body Fat
Researchers supported in part by the Clinical and Translational Science Awards Program at Columbia University have discovered a new way to take white fat from a mouse, turn it into brown fat and return it to the mouse. These findings could have implications for treating obesity.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Researchers Study How Robots Can Help Chronically Sick Children Attend School
Clinical and Translational Science Awards (CTSA) Program researchers explore how using robots in the classroom to help chronically ill children attend school could improve learning and better connect them socially.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Scientists Prioritize Compounds to Advance Research on Mitochondrial Damage
NCATS scientists collaborated with other federal agencies to develop an approach that enables researchers to prioritize environmental chemicals according to their ability to disrupt mitochondrial activity.
NCATS Support: Toxicology in the 21st Century

CTSA Program Supports Early Development and De-Risking of Innovative Heart Valve Technology
NCATS’ Clinical and Translational Science Awards (CTSA) Program support enabled the early development and “de-risking” of an innovative device that may help patients avoid complications of heart valve replacement.
NCATS Support: Clinical and Translational Science Awards Program

NCATS, FDA Co-Host Workshop on Gene Therapy for Rare Diseases
NCATS and the Food and Drug Administration are collaborating to identify bottlenecks to gene therapy development and share best practices for advancing gene therapies.
NCATS Support: Office of Rare Diseases Research

Templates for Success: Speeding the Formation of Public-Private Partnerships
NCATS is overcoming key operational hurdles in the research process. By using standardized legal agreements, the Center has accelerated private-public research collaborations, enabling smarter, faster science.
NCATS Support: Discovering New Therapeutic Uses for Existing Molecules, Strategic Alliances, Stimulating Peripheral Activity to Relieve Conditions

3-D Tissue Bioprinting: An Emerging Path to Better Drug Development
NCATS scientists are developing 3-D tissue models that more closely mimic the complexity of tissues in the human body in a reproducible, automated and scalable manner using bioprinting techniques. These innovative, human-like tissue models are used for compound testing and could accelerate drug development for treatments of both rare and common diseases.
NCATS Support: 3-D Tissue Bioprinting

CTSA Program Researcher Studies Barriers to HIV Prevention
At Washington University in St. Louis, an NCATS Clinical and Translational Science Awards (CTSA) Program hub, Rupa R. Patel, M.D., M.P.H., is studying the barriers that keep young adults from obtaining an HIV intervention called pre-exposure prophylaxis.
NCATS Support: Clinical and Translational Science Awards Program

NCATS-Supported Research Shows Promise for Stretchable, Wearable Electronics
Researchers at the Altman Clinical and Translational Research Institute, an NCATS Clinical and Translational Science Awards Program hub at the University of California, San Diego, have found a way to make stretchable electronics in 3-D, opening up potential diagnosis and treatment avenues.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Develops New Technique to Look Inside Cells Growing in 3-D
NCATS intramural researchers outline a simple, fast and automated process to make 3-D tissues transparent. The new method, described in the July issue of Scientific Reports, enables many types of observations within 3-D growth environments, such as locating molecules inside a cell or finding damaged DNA, and it can do so quickly in thousands of cells in a tissue-like structure.
NCATS Support: Division of Preclinical Innovation: 3-D Tissue Bioprinting, Automation, and Informatics programs

NCATS Creates Drug Development Data Portal
To help researchers find the information they need more easily, NCATS created an online tool called NCATS Inxight: Drugs. This open-access, easy-to-use and evolving portal aggregates reliable, curated drug development data from multiple existing sources, all in one place.
NCATS Support: Division of Preclinical Innovation

NIH-Supported Researchers Use Robots, Stem Cells to Produce Organ Models
Researchers at the University of Washington, supported by NCATS’ Tissue Chip for Drug Screening program and the National Institute of Diabetes and Digestive and Kidney Diseases, have created an automated procedure for producing kidney organoids on a large scale and in a format that works with current screening tools used in drug discovery.
NCATS Support: Tissue Chip for Drug Screening

New NCATS-Supported Test Could Provide Faster Diagnosis of Deadly Disease
Through Phase 1 and 2 Small Business Innovation Research grants, NCATS helps fund the development and analysis of a simple paper test that can detect seven common causes of diarrhea in about 30 minutes. 
NCATS Support: Small Business Innovation Research

CTSA Program Researchers Partner with Barbershops to Cut High Blood Pressure
Black men are more likely to die from complications of high blood pressure than any other group in the U.S. To tackle this disparity, researchers at Cedars-Sinai Medical Center and the University of California, Los Angeles, part of NCATS’ Clinical and Translational Science Awards (CTSA) Program, are pairing pharmacists with barbershops to help prevent heart attacks and strokes.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Researchers Shed Light on Improved Melanoma Treatment
Researchers at the University of California, Los Angeles, an NCATS Clinical and Translational Science Awards (CTSA) Program hub, discovered four subtypes of melanoma — pointing to new treatment approaches.
NCATS Support: Clinical and Translational Science Awards Program

Finding What’s Common Among Diseases, Seeing the Bigger Picture
To find cures for diseases, scientists traditionally study one disorder at a time. But scientists at NCATS take a different approach. By working to address translational research obstacles through projects across many diseases, NCATS teams aim to learn more about what different diseases have in common. This knowledge of the bigger picture can help scientists advance the overall translational science process.
NCATS Support: Division of Preclinical Innovation

NCATS-Supported Study Highlights Troubling Trend in Opioid Prescribing
Researchers at the University of California, Los Angeles, supported in part by NCATS’ Clinical and Translational Science Awards Program, discovered a troubling opioid prescribing trend. Patients who took a type of anti-anxiety medicine were more than twice as likely as the general public to receive new opioid prescriptions despite a higher overdose risk.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Ignites Translational Science Spark for Young Investigators
Meet three graduate students working collaboratively on research projects with NCATS and university scientists from England and Sweden.
NCATS Support: Division of Preclinical Innovation

CTSA Program Support May Lead to Blood Test for Colon Cancer Screening
University of Alabama at Birmingham researchers, supported in part by NCATS’ Clinical and Translational Science Awards (CTSA) Program, teamed up with scientists from the HudsonAlpha Institute for Biotechnology to identify markers in the blood that could be used to detect precancerous polyps in patients.
NCATS Support: Clinical and Translational Science Awards Program

Repurposed Drug Approach May Thwart Spread of Cancer Cells
NCATS preclinical researchers helped NIH Extracellular RNA Communication program-supported scientists use a new approach to find drugs that may be effective in preventing cancer cell progression. The team found that antibiotics, antifungal medicines and anti-inflammatory agents were effective in preventing advanced prostate tumor cells from releasing exosomes or in blocking exosome production.
NCATS Support: Division of Preclinical Innovation, Extracellular RNA Communication

New Study Shows Medication-Based Treatment After Opioid Overdose Can Save Lives
Researchers from Boston University, an NCATS Clinical and Translational Science Awards Program hub, have found a strong link between the use of medications to treat opioid use disorder after an overdose and a reduced risk of subsequent overdose death.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Researchers Advance Heart Condition Study Through Precision Medicine and Digital Health
Researchers at the Scripps Translational Science Institute in San Diego, part of NCATS’ Clinical and Translational Science Awards (CTSA) Program, are working to address the challenge of diagnosing atrial fibrillation, a common heart condition with complex causes. The team’s efforts can ultimately help advance precision medicine approaches to preventing and diagnosing other types of complex diseases.
NCATS Support: Clinical and Translational Science Awards Program

NIH-Supported Scientists Discover New Personalized Approach to Diagnosing and Treating Rare Food Allergy Disease
A team of scientists from Cincinnati Children’s Hospital Medical Center and the Consortium of Eosinophilic Gastrointestinal Disease Researchers has developed a new personalized approach to diagnosing and treating a rare food allergy disease called eosinophilic esophagitis.
NCATS Support: Rare Diseases Clinical Research Network

NIH-Supported Scientists Uncover Clues to Spinal Cord Development, Neurodegenerative Disease
Researchers at Cedars-Sinai, supported by NCATS and the National Institute of Neurological Disorders and Stroke, used stem cells and 3-D tissue chips to mimic conditions in the early human spinal cord. The results could provide a better understanding of the development of some neurodegenerative diseases.
NCATS Support: Tissue Chip for Drug Screening

NIH-Supported Research Helps Advance a Potential Rare Disease Gene-Editing Treatment into Clinical Trials  
RDCRN-supported research helps chart how several rare, inherited disorders progress in patients over time. This information facilitated the advance of a potential rare disease gene-editing treatment into clinical trials.
NCATS Support: Rare Diseases Clinical Research Network

CTSA Program Hubs Collaborate to Assess Community Engagement
The Clinical and Translational Science Awards (CTSA) Program hub at the University of Rochester has launched a pilot study to develop and test a community engagement assessment tool to help academic health centers evaluate and enhance their work. 
NCATS Support: Clinical and Translational Science Awards Program

Translator: Unique Collaborative Approach to Advancing Biomedical Data Sharing
Through the Biomedical Data Translator program, NCATS is leading a unique collaboration between academic and private-sector partners to create a data-mining computational resource that will use many types of biomedical information to help facilitate translational innovation in disease prevention, diagnosis and treatment.
NCATS Support: Biomedical Data Translator

Closing the Protein Knowledge Gap to Identify New Treatments 
As part of the NIH Common Fund’s Illuminating the Druggable Genome (IDG) program, a team of NCATS and other scientists recently discovered that about one in every three proteins is poorly understood. To speed new research along, NCATS scientists developed Pharos, an online portal that provides access to the protein information collected by IDG investigators.
NCATS Support: Illuminating the Druggable Genome

NCATS Funds Drug Repurposing Projects, Seeks New Industry Partners
NCATS is seeking additional industry partners to participate in future Discovering New Therapeutic Uses for Existing Molecules funding opportunities.
NCATS Support: Discovering New Therapeutic Uses for Existing Molecules

CTSA Program Support Enables Innovative Study of Brain Function
A Clinical and Translational Science Awards (CTSA) Program​-funded researcher tests innovative training to help people with schizophrenia “correct” disordered brain waves. Promising results suggest the training could potentially help improve memory.
NCATS Support: Clinical and Translational Science Awards Program

Patient Perspectives and Hope of Research Defined Annual Rare Disease Day at NIH
On March 1, 2018, patients, scientists, policymakers and others participated in the annual Rare Disease Day at NIH. The event emphasized the importance of involving patients and their perspectives at every stage of the translational science process to help ensure research outcomes that will be most relevant and more readily adopted.
NCATS Support: Rare Diseases Research

NCATS Supports Award-Winning Technology for Drug Development
NCATS supports an innovative platform technology to precisely deliver nutrients and hormones to cells for a preclinical therapeutics testing program. The adaptability of the technology to other translational science applications led to additional funding for its commercialization and supports NCATS’ goal of making drug development more efficient. 
NCATS Support: Small Business Innovation Research, Tissue Chip for Drug Screening

CTSA Program Scholar Learns New Research Approaches Through Externship
Through the NCATS-Eli Lilly externship, Clinical and Translational Science Awards (CTSA) Program​ Scholar Josephine Taverna, M.D., learns new approaches to drive her research forward.
NCATS Support: Clinical and Translational Science Awards Program

New CTSA Program Informatics Center to Create a Nationwide Data Ecosystem
NCATS established the National Center for Data to Health to develop standardized approaches and best practices that address operational and institutional barriers to sharing data.
NCATS Support: Clinical and Translational Science Awards Program

Rare Disease Patients and Families Find Hope in Research
NCATS supports rare disease patients and their communities by providing translational research funding, tools and other resources that help address their unique challenges.
NCATS Support: Rare Diseases Research

NIH Collaboration Leverages CTSA Program for Research Mentoring
NCATS’ Clinical and Translational Science Awards Program is collaborating with the National Institute of Dental and Craniofacial Research to provide oral, dental and craniofacial scientists with translational research training opportunities early in their careers.
NCATS Support: Clinical and Translational Science Awards Program

NCATS’ Preclinical Collaboration Enables Gene Therapy for Rare Muscle Disease to Advance to Clinical Trial 
NCATS Therapeutics for Rare and Neglected Diseases (TRND) researchers and scientists at Duke University’s Clinical and Translational Science Institute (CTSI) have helped advance a gene therapy for Pompe disease into clinical testing for the first time.
NCATS Support: Therapeutics for Rare and Neglected Diseases

NCATS-Supported Consortium Charts New Course for Rare Immune Diseases
Scientists with an NCATS-supported research consortium are pioneering new studies to better understand and develop therapies for rare immune system disorders. The Primary Immune Deficiency Treatment Consortium conducts natural history studies and other research as part of the NCATS Rare Diseases Clinical Research Network.
NCATS Support: Rare Diseases Clinical Research Network

CTSA Program Fosters Next Generation of Translational Scholars
NCATS’ Clinical and Translational Science Awards (CTSA) Program fosters the next generation of translational science scholars by providing trainees with needed skills, knowledge and core competencies.
NCATS Support: Clinical and Translational Science Awards Program

2017

CTSA Program Supports Research on Translating Evidence into Practice
Clinical and Translational Science Awards (CTSA) Program-supported researchers explore the dissemination and clinical implementation of therapies, interventions and policies to ultimately improve human health.
NCATS Support: Clinical and Translational Science Awards Program

NCATS-Supported Researchers Demonstrate Value in Community-Based Research Consultation Service
Clinical and Translational Science Awards (CTSA) Program-supported researchers developed a community-based research evaluation model to determine the effectiveness of consultation services that help translate clinical practice observations into new treatment approaches.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program-Supported Investigators Validate Biomarkers for NPC1 Treatment
With support from the Sharing Partnership for Innovative Research in Translation (SPIRiT) consortium, Daniel Ory, M.D., of Washington University in St. Louis, and Charles Vite, D.V.M., Ph.D., of the University of Pennsylvania, collaborated to validate biomarkers intended to monitor the response to therapy for Niemann-Pick diseases type C1 (NPC1).
NCATS Support: Clinical and Translational Science Awards Program, Therapeutics for Rare and Neglected Diseases

NCATS Explores Science with Middle School Students
NCATS staff engaged nearly 60 local middle school students in science experiments and led tours of the Center’s laboratories during the ninth annual Frontiers in Science and Medicine event on Oct. 27, 2017.
NCATS Support: NIH

Michael G. Kurilla Joins NCATS as Clinical Innovation Director
Michael Gregory Kurilla, M.D., Ph.D., will direct the NCATS Division of Clinical Innovation beginning Dec. 10, 2017. Kurilla’s new role will include leading the Clinical and Translational Science Awards (CTSA) Program.
NCATS Support: Office of the Director, Clinical and Translational Science Awards Program

NCATS Unveils Patient-Focused Therapy Development Toolkit
NCATS showcases web-based platform of patient-focused rare diseases translational science resources, many of which also are applicable to other diseases, at NIH event on Sept. 8, 2017.
NCATS Support: Office of Rare Diseases Research

CTSA Program-Supported Researchers Test Low-Cost Treatment for Deadly Illness
Virginia Commonwealth University researchers have discovered a low-cost and potentially lifesaving treatment for sepsis. Support from the Clinical and Translational Science Awards (CTSA) Program helped streamline translation of this promising therapy to multisite clinical studies.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Collaboration Aims to Improve Brain Cancer Drug Testing
Clinical and Translational Science Awards (CTSA) Program-supported scientists work to develop novel technique to test drugs against the most common form of brain cancer, glioblastoma multiforme.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Scientists Participate in NIH Research Festival
NCATS scientists highlighted their recent work at the NIH Research Festival, held Sept. 13-15, 2017, on the NIH campus in Bethesda, Maryland. 
NCATS Support: NCATS

NCATS Staff to Test Innovative Ideas for HHS
The U.S. Department of Health and Human Services chose two NCATS teams for its Ignite Accelerator program to improve the way their program, office or agency works.
NCATS Support: Office of Rare Diseases Research, Division of Clinical Innovation

NYU CTSA Program Hub Releases Research Study Recruitment E-Book
The New York University Clinical and Translational Science Institute has developed an electronic how-to book aimed at helping researchers recruit and retain participants in research studies.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Furthers Efforts to Create a Data Ecosystem to Explore Disease Connections
Biomedical Data Translator scientists are working to create a computational tool to improve connections among biomedical data types and provide insights into disease biology and treatments. 
NCATS Support: Biomedical Data Translator

NCATS Enables Scientists, Community Clinicians to Collaborate on Health Initiatives
NCATS’ Clinical and Translational Science Awards (CTSA) Program supports improvements in public health by enabling innovative research partnerships between university scientists and community clinicians.
NCATS Support: Clinical and Translational Science Awards Program

NCATS-Supported Study Shows Lipoic Acid May Benefit Multiple Sclerosis Patients
Rebecca Spain, M.D., M.S.P.H., a Clinical and Translational Science Awards (CTSA) Program scholar at Oregon Health & Science University, found evidence suggesting that an over-the-counter antioxidant, lipoic acid, can help multiple sclerosis (MS) patients.
NCATS Support: Clinical and Translational Science Awards Program

NYU CTSA Program Hub Hosts Science Café
The New York University Clinical and Translational Science Institute ― an NCATS Clinical and Translational Science Awards (CTSA) Program hub ― hosted a “science café” featuring informal, open dialogue about research needs and collaboration opportunities.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Hub Expands Successful Radio Show with Podcast
Since 2013, the Clinical and Translational Science Awards (CTSA) Program hub at the Medical College of Wisconsin has aired a monthly radio segment, Discovery Radio. It’s now a successful podcast with a worldwide audience.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Supports Novel Methods to Improve Institutional Review Board Efficiencies
NCATS’ Clinical and Translational Science Awards (CTSA) Program support helped a University of Wisconsin–Madison Ph.D. student discover new ways of identifying factors affecting institutional review board review times.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Mentoring Helps Engineers Develop Innovative Rehabilitation Therapies
NCATS-supported KL2 program enables early-stage investigators to conduct team-based clinical research and helps them jumpstart independent careers in translational research.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Announces Quarterly Update to Its Assay Guidance Manual
In July 2017, authors of NCATS’ Assay Guidance Manual (AGM) added two new chapters and revised two others. Updated quarterly, the AGM provides step-by-step guidance for high-throughput screening, lead optimization and early phases of regulated drug development projects.
NCATS Support: Assay Guidance Manual initiative

First NCATS Day Demonstrates Commitment to Patients Through Smarter Science
On June 30, 2017, NCATS hosted an inaugural event to identify patients’ needs and brainstorm ways to foster engagement at every step of the translational science spectrum.
NCATS Support: NCATS

Small Business Award Spurs Innovation to Improve Data Use
Through its Small Business Innovation Research program, NCATS supported the development of a technology to use electronic health records data to advance value-based and enhanced patient care.
NCATS Support: Small Business Innovation Research program

NCATS and Eli Lilly Collaborate to Bridge Cultural, Research Divide
NCATS and Eli Lilly partnered on the NCATS-Eli Lilly scholars externship program, which pairs scholars, trainees and investigators from NCATS’ Clinical and Translational Science Awards (CTSA) Program with an Eli Lilly project team for up to one year.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Supports Expanded Eye Screening for Underserved Diabetics 
Through a community partnership between the Clinical and Translational Science Awards (CTSA) Program hub at the University of California, Los Angeles, and the Los Angeles County Department of Health Services, researchers discovered that offering diabetic retinopathy screening in primary care settings reduced examination waiting periods and increased the number of patients served. 
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Investigators Collaborate to Enhance Newborn Screening
With support from NCATS’ Clinical and Translational Science Awards (CTSA) Program, the Early Check initiative has been created to screen newborns for fragile X syndrome and other rare genetic diseases. 
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Community Mentorship Helps Advance Multiple Sclerosis Research in Latinos
Through an NCATS-supported community mentorship program, a neurologist strengthens her translational science skills by increasing her engagement with an underserved population.
NCATS Support: Clinical and Translational Science Awards Program

NCATS SMART IRB Team Launches Online Reliance System
The NCATS Streamlined, Multisite, Accelerated Resources for Trials Institutional Review Board (IRB) team launched an Online Reliance System to help streamline the ethics review conducted by IRBs. 
NCATS Support: Clinical and Translational Science Awards Program

NCATS Trial Innovation Network Investigators Tackling Clinical Trial Inefficiencies
Through NCATS’ new Trial Innovation Network, Clinical and Translational Science Awards (CTSA) Program-supported investigators are working collaboratively to tackle inefficiencies in clinical trials.
NCATS Support: Clinical and Translational Science Awards Program, Trial Innovation Network

CTSA Program Mentoring Paves Way for Brain Cancer Therapy
Clinical and Translational Science Awards (CTSA) Program mentoring support sparked an interdisciplinary translational research team that developed a potential therapy for brain cancer. 
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Support Helps Talented Investigators Re-Establish Their Careers
Clinical and Translational Science Awards (CTSA) Program support helped Anandi Krishnan, Ph.D., get back into a research career after taking a leave of absence to care for her child. 
NCATS Support: Clinical and Translational Science Awards Program

From ER Physician to Translational Scientist via CTSA Program Support
Clinical and Translational Science Awards (CTSA) Program hub (funded by NCATS) at the University of Florida helps physician pursue research project in sepsis patients.
NCATS Support: Clinical and Translational Science Awards Program

NIH, Academia and Patient Advocate Collaboration Speeds Niemann-Pick Type C1 Research
Patient-focused translational science collaboration among government and academic scientists, patients, and their advocates advances a potential treatment for the rare disease Niemann-Pick type C1.
NCATS Support: Therapeutics for Rare and Neglected Diseases

CTSA Program Collaboration in Action
Update on three Clinical and Translational Science Awards (CTSA) Program-hub projects from Northwestern University, Ohio State University and Indiana University.
NCATS Support: Clinical and Translational Science Awards Program

Unprecedented Trans-NCATS Collaboration Enables Rapid Advancement of Rare Lung Disease Therapy to Human Trials
aPAP is a rare lung disease with a difficult treatment. With this collaboration, Dr. Trapnell is optimistic that a potentially life-changing treatment is one step closer to benefitting patients.
NCATS Support: Clinical and Translational Science Awards Program, Office of Strategic Alliances, Rare Diseases Clinical Research Network, Therapeutics for Rare and Neglected Diseases

2016

NCATS-Supported Researchers Recruit Citizen Scientists to Help Mine Biomedical Literature
A team of bioinformatics scientists from the Scripps Translational Science Institute (STSI) invented a web-based technology platform to arrange biomedical literature into a format that is easier for computers to organize and analyze.
NCATS Support: Clinical and Translational Science Awards Program

NIH I-Corps Training Helps NCATS-Supported Companies Find Their Way
The NIH I-Corps program is a collaborative effort to support the development and commercialization of biomedical technologies. It was expanded to focus on moving technologies developed through SBIR/STTR funding to the marketplace. NCATS is among 17 participating Institutes and Centers from NIH and the Centers for Disease Control and Prevention.
NCATS Support: Small Business Innovation Research

NCATS Presents Early Plans for Trial Innovation Network, Solicits Project Proposals
Efforts to align and harmonize three Trial Innovation Centers (TICs) and the Recruitment Innovation Center (RIC) are underway within the NCATS Trial Innovation Network, a new collaborative initiative made possible through the Clinical and Translational Science Awards (CTSA) Program. The initial framework was shared with CTSA Program hub, TIC, and RIC representatives at the Trial Innovation Network kickoff meeting held on Oct. 26, 2016, in Chicago.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Supports New Ultrasensitive Diagnostic Test Development
Stanford University researchers have developed a new test for diagnosing diseases, including thyroid cancer, HIV and type 1 diabetes. The method appears to be many times more sensitive than some traditional diagnostic tests, meaning that it potentially can detect illnesses earlier, enabling clinicians to treat patients sooner and possibly slow disease progression.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Small Business Award Seeds App to Improve Medication Adherence
Too many individuals take their medications incorrectly or do not take them at all. A new smartphone application helps patients take the right medication at the right time. The artificial intelligence technology was developed with funding from an NCATS small business award.
NCATS Support: Small Business Innovation Research

Doctoral Students Use NCATS Resources to Investigate Promising Disease Therapies
Since 2012, the NIH Oxford-Cambridge Scholars Program has enabled future scientists to work with NCATS researchers to explore the translation of promising new therapies for cancers and tuberculosis.
NCATS Support: NIH Oxford-Cambridge Scholars Program

CTSA Program-Supported Research Uncovers Genetic Components of Healthy Aging
Older adults often face aging-related ailments that can be costly and shorten lifespans. However, some people live long lives without encountering these common health problems. What sets apart healthy agers from their peers? Researchers at Scripps Translational Science Institute are conducting a study of the “Wellderly” to find out.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Scholar Receives Prestigious NIH Grant
Clinical and Translational Science Awards (CTSA) Program Scholar support enabled Ying Liang, M.D., Ph.D., an assistant professor of toxicology and cancer biology at the University of Kentucky, to obtain her first NIH Research Project Grant (R01). The R01 grant will fund her research on the role of a protein called latexin in preventing cancer therapy-induced stem cell damage.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Resources Help UCLA Scientists Visualize Key Enzyme in Cancer and Aging
Clinical and Translational Science Awards (CTSA) Program resources help UCLA scientists produce clearest-ever image of key enzyme in cancer and aging. This knowledge could inform the development of anti-cancer and anti-aging therapies.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Introduces Plans for New Single IRB Reliance Model
NCATS held a workshop on May 2, 2016, to educate participants about using its new single institutional review board (IRB) reliance model ― the NCATS Streamlined, Multisite, Accelerated Reliance for Trials (SMART) IRB Model ― for multisite clinical studies.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Supports Streamlined Clinical Trial Recruitment
Researchers at the University of New Mexico (UNM) Clinical and Translational Science Center (CTSC) recently established the Participant Recruitment Service (PRS). Supported through NCATS’ Clinical and Translational Science Awards (CTSA) Program, the goal is to streamline and improve recruitment rates for clinical trials while maintaining patient privacy.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program-Supported Researcher Is Translating Laboratory Discoveries into Better Hearing for Cochlear Implant Patients
In 2015, Suhrud Rajguru, Ph.D., presented his work at a Clinical and Translational Science Awards (CTSA) Program-supported translational research poster competition, in which he was among the winners. As part of his award, he participated in a course designed to help scientists move research innovations into commercial space.
NCATS Support: Clinical and Translational Science Awards Program

Breathing Easier: First Treatment for Rare Lung Disease Approved
Research coordinated by The LAM Foundation and carried out with scientific partners in NCATS’ Rare Diseases Clinical Research Network culminated in the first treatment for lymphangioleiomyomatosis (LAM) approved by the Food and Drug Administration.
NCATS Support: Rare Diseases Clinical Research Network

NCATS' Small Business Funding Helps Launch New Platform for Rare Diseases Drug Discovery
A Ph.D. student at the University of Utah was developing an approach to screen existing drugs for their potential to treat a rare disease when he saw a way to expand that method into an enterprise to discover hundreds of treatments for thousands of diseases.
NCATS Support: Small Business Innovation Research and Small Business Technology Transfer

CTSA Program Supports Research Network in Geographically Isolated Region
With support from NCATS’ Clinical and Translational Science Awards (CTSA) Program, the University of New Mexico Clinical and Translational Science Center (UNM CTSC) was a founding partner in the Mountain West Research Consortium (MWRC), currently a network of 11 universities spanning seven states. The MWRC’s goal is to build and enhance the geographically isolated region’s clinical and translational research capacity.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Collaborations Spark Broad Public Engagement in Common Rule NPRM
Clinical and Translational Science Awards (CTSA) Program representatives recently collaborated with patient advocacy groups to host four national meetings promoting public engagement in the Notice of Proposed Rulemaking (NPRM) on the Federal Policy for the Protection of Human Subjects.
NCATS Support: Clinical and Translational Science Awards Program

CTSA Program Collaboration Provides Investigators with Access to High-Throughput Screening and Drug Discovery Expertise
The Drug Rescue, Repurposing and Repositioning Network (DR3N) provides Clinical and Translational Science Awards (CTSA) Program investigators with access to state-of-the-art technology, innovative tools, and guidance in translating pilot projects from preclinical to clinical stages via drug repurposing.
NCATS Support: Clinical and Translational Science Awards Program

Tox21 Researchers Publish Analysis of 10,000 Potentially Toxic Chemicals
In the January 26 issue of Nature Communications, researchers from the Toxicology in the 21st Century (Tox21) program published their first comprehensive analysis from screening a library of more than 10,000 chemicals for toxicity in 30 different human cell–based assays (tests).
NCATS Support: Toxicology in the 21st Century (Tox21)

Former CTSA Program Scholar Develops Interactive End-of-Life Decision Tool
Ronald L. Hickman Jr., Ph.D., R.N., ACNP-BC, FAAN, an associate professor at Case Western Reserve University’s Frances Payne Bolton School of Nursing and an acute care nurse, is applying the knowledge and guidance he gained as a Clinical and Translational Science Awards (CTSA) Program Clinical Research Scholar (KL2) to help develop a new tool called Interactive Virtual Decision Support for End of Life and Palliative Care (INVOLVE).
NCATS Support: Clinical and Translational Science Awards Program

2015

Data Released on Drug Combinations to Treat Malaria
NCATS researchers tested and released data on thousands of combinations of known and newly identified drugs to potentially treat malaria.
NCATS Support: NCATS Chemical Genomics Center, Matrix Combination Screening

Modeling the Female Reproductive Tract in 3-D: The Birth of EVATAR™
Researchers are developing EVATAR™, a miniaturized 3-D representation of the female reproductive tract and liver on a handheld, interconnected platform for use in drug testing and to study the basic biology of female reproduction.
NCATS Support: Tissue Chip for Drug Screening

NCATS-FDA Team Receives HHS Innovation Ventures Support to Crowdsource Information on Treating Tropical Diseases
The NCATS-FDA team recently received support from the HHS Innovation Ventures Fund — an HHS IDEA Lab program — to enhance the Web-based Collaborative Use Repurposing Engine (CURE). The highly competitive awards provide growth-stage funding and 15 months of mentoring as well as tools to help grow and sustain CURE.
NCATS Support: NCATS Chemical Genomics Center

Partnering with Disease Foundations to Speed Drug Discovery
Designing high-throughput screening assays is a science in itself. The team must have in-depth familiarity not only with assay technology but also with the target disease and its unique challenges. To increase the likelihood of success, postdoctoral researchers who are knowledgeable about specific diseases join project teams through fellowships sponsored by patient groups and foundations.
NCATS Support: Assay Development and Screening Technology

Stopping Metastasis in Its Tracks: New 3-D Cell Model Enables Closer Look at Cancer Progression
NCATS and University of Chicago researchers worked together to create, adapt and validate an innovative 3-D ovarian cancer cell model used to identify small molecules that can prevent cancer cells from spreading to new sites in the body. The new model includes connective tissue cells and the extracellular matrix (a collection of supportive molecules outside cells), creating a more lifelike simulation of the human body environment. It represents an exciting advance in research technology.
NCATS Support: NCATS Chemical Genomics Center

Allergy Drug Could Treat Hepatitis C
Recently, NCATS scientists, in collaboration with researchers from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), discovered that an over-the-counter drug used to treat allergy symptoms also limited hepatitis C virus (HCV) infection in human liver cells. The findings suggest that the drug, chlorcyclizine HCl (CCZ), potentially could be used to treat this chronic infection in people.
NCATS Support: Division of Preclinical Innovation

NCATS Plate Washing Initiative Saves Money, Keeps Plastic Out of Landfills
High-throughput robots help researchers run screening experiments quickly and efficiently, but until recently, they produced a significant amount of environmental waste in the form of thousands of single-use plastic plates. With an innovative idea and a commercial dissemination partner, NCATS is changing that story and keeping unnecessary waste out of landfills.
NCATS Support: Division of Preclinical Innovation, Small Business Innovation Research

NCATS Support Leads to Clinical Trial to Test Repurposed Cancer Treatment as Alzheimer’s Therapy
On March 31, 2015, NCATS announced that scientists at Yale University have found that a compound originally developed as a cancer therapy potentially could be used to treat Alzheimer’s disease. Now the team is testing the compound’s effectiveness in humans.
NCATS Support: Discovering New Therapeutic Uses for Existing Molecules

Tox21 Collaboration Generates an Innovative Platform for Testing Individual Differences in Chemical Sensitivity
A research team including NCATS scientists used the Center’s robotic screening capabilities to test the toxicity of 179 chemicals using cells from more than 1,000 people to investigate the range of human sensitivity to environmental chemicals.
NCATS Support: Toxicology in the 21st Century

Study Demonstrates Success of NCATS’ Rare Diseases Therapeutic Development Programs
A study found that two NCATS rare diseases programs have led to the reduced cost of developing new drugs and reduced financial risks.
NCATS Support: Therapeutics for Rare and Neglected Diseases and Bridging Interventional Development Gaps

CTSA Program Consortium Tackling Clinical Trial Recruitment Roadblocks
The Clinical and Translational Science Awards (CTSA) Program Accrual to Clinical Trials initiative was launched to develop a nationwide network of sites that share electronic health record data to further multisite study feasibility and recruitment efforts.
NCATS Support: Clinical and Translational Science Awards Program

Searching Saliva for Signs of Disease
ExRNA researchers at the University of California, Los Angeles have found that saliva offers as many clues to a person’s health as blood. Now, they are using this information to develop a noninvasive diagnostic test for stomach cancer.
NCATS Support: Extracellular RNA Communication

2014

NCATS Repurposing Test Identifies 53 Drugs that May Block Ebola Infection
A team of researchers from NCATS and the Icahn School of Medicine at Mount Sinai worked to approach the Ebola public health problem by testing existing drugs. The results yielded 53 drugs that may block Ebola virus-like particles from entering and infecting cells.
NCATS Support: Division of Preclinical Innovation

NCATS Screening Technologies Enable Identification of Potential Treatment Target for Neurological Disorder
A recent advance made possible through a project collaboration involving NCATS experts, academic researchers, a patient advocacy group and a pharmaceutical company enabled a research team to identify a potential drug target for Charcot-Marie-Tooth disease.
NCATS Support: Division of Preclinical Innovation

NCATS Science Featured at 28th NIH Research Festival
Researchers from NCATS and other NIH Institutes and Centers gathered at the 28th NIH Research Festival Sept. 22 – 24, 2014, to share insights on important scientific advances made by intramural investigators.
NCATS Support: Division of Preclinical Innovation

Improved Disease Model Leads to Potential Therapy for Rare Disorder
A team of researchers from NCATS and the National Human Genome Research Institute made a major advance in the understanding and treatment for Gaucher disease, creating a new model that not only helps identify treatments for patients, but also is useful in studying other diseases.
NCATS Support: NCATS Chemical Genomics Center

Tox21 Researchers Analyze Potential Negative Effects of Thousands of Chemicals on Human Health
Find out how Tox21 researchers have developed faster, cheaper and more effective testing methods to analyze the effects of chemicals on human health.
NCATS Support: Toxicology in the 21st Century

Michael J. Fox Foundation Funds Research Project that Leverages NCATS Chemical Screening Approach and Resources
Researchers from NCATS and the National Institute of Neurological Disorders and Stroke are leading a project that showcases how NCATS’ chemical screening resources can advance development of potential therapeutics for a broad range of diseases.
NCATS Support: Division of Preclinical Innovation

Spotlight on Collaboration: A Journey From Biological Probes to Potential Therapeutics
Scientists at the NCATS Chemical Genomics Center work closely with academic, nonprofit and biotech researchers to pursue collaborative probe development projects, including the discovery of three novel small molecule chemical probes with the potential to treat diabetes, stroke and thrombosis.
NCATS Support: NCATS Chemical Genomics Center

NCATS Researchers Propose Innovative Approach to Test Drugs in Rare Diseases
Scientists often know little about the symptoms and biology of rare conditions, which adds to the complexity of designing drug studies. NCATS scientists have propose a new way to test drugs and develop better treatments for rare disease patients by targeting common molecular mechanisms across multiple diseases.
NCATS Support: Office of Rare Diseases Research

IRB Reliance: A New Model for Accelerating Translational Science
The institutional review board (IRB) review process can be especially cumbersome for researchers and IRB staff conducting multisite research. The CTSA Program is designed to encourage collaboration across institutions and to remove or minimize common translational barriers, such as those involved in IRB oversight.
NCATS Support: Clinical and Translational Science Awards Program

Petra Kaufmann Joins NCATS as Clinical Innovation Director
On May 4, 2014, Petra Kaufmann, M.D., M.Sc., joined NCATS to direct its Division of Clinical Innovation.
NCATS Support: Office of the Director

Screening Platform Is a Launch Pad for Novel Treatment Combinations
NCATS and National Cancer Institute investigators published a collaborative study that demonstrated how a new combination drug screening platform developed at NCATS can quickly narrow down a long list of potential drug combinations to find those with the most potential to help patients.
NCATS Support: Division of Preclinical Innovation

NCATS Welcomes McInnes and Zuk to Leadership Team
In January 2014, NCATS welcomed two new members of the Center’s leadership team. Pamela M. McInnes, D.D.S., M.Sc.(Dent.), is the NCATS deputy director, and Dorit Zuk, Ph.D., is the director of the Office of Policy, Communications and Strategic Alliances.
NCATS Support: Office of the Director

Tissue Chip Projects Highlighted in Major Journal
On Dec. 20, 2013, the journal Stem Cell Research & Therapy published a supplement that provides an overview of projects funded through the Tissue Chip for Drug Screening program.
NCATS Support: Tissue Chip for Drug Screening

Rare Diseases Research Advocate Retires
Stephen C. Groft, Pharm.D., director of the NCATS Office of Rare Diseases Research, announced his retirement effective Feb. 8, 2014. Groft leaves a legacy as a public servant for more than four decades and a tireless advocate for rare diseases research.
NCATS Support: Office of Rare Diseases Research

2013

Understanding the Brain’s Response to Sugar Could Help Treat Obesity
Yale researchers, supported in part by the Clinical and Translational Science Awards (CTSA) Program at NCATS, have used imaging technology to look at how the brain responds to sugar. This approach could improve scientists’ knowledge of the brain’s role in obesity.
NCATS Support: Clinical and Translational Science Awards Program

Five CTSAs Enable NIH-Funded Research on Innovative Allergy Therapy
Finding a new way to treat and possibly prevent the severest food allergy reactions could improve the lives of millions. A consortium of scientists at five institutions that receive NIH funding, including through NCATS’ Clinical and Translational Science Awards (CTSA) Program, took up this challenge.
NCATS Support: Clinical and Translational Science Awards Program

NCATS Spotlight: Toxicology in the 21st Century (Tox21) Program
Scientists lack data to predict how many chemicals affect our bodies and their potential for harm. The collaborative Tox21 program aims to test 10,000 chemicals and evaluate their potential to cause health problems.
NCATS Support: Toxicology in the 21st Century

NCATS and Johns Hopkins Researchers Identify New Therapeutic Strategy for Eye Diseases
The NCATS approach to speeding the development of diagnostics and therapeutics — based on innovation in how science is done as well as what is done — has led to dramatic success for a project on degenerative diseases of the retina.
NCATS Support: Assay Development and High-Throughput Screening, RNA interference

CTSA Resources Support Largest U.S. Newborn Screening Study for Fragile X Mutations
A team of researchers at the University of California, Davis, found that more people have gene changes linked to Fragile X syndrome (FXS) than anticipated. Accurate and timely diagnosis by such newborn screening may enable early intervention.
NCATS Support: Clinical and Translational Science Awards Program

TRND Research Leads to NIH Trial to Test Drug for Niemann-Pick Type C1
Niemann-Pick disease type C1 is a rare, inherited disease characterized by progressive impairment of motor and intellectual functions in early childhood. NIH initiated a Phase I clinical trial to evaluate the safety and effectiveness of cyclodextrin as a potential therapy. (See related story below.)
NCATS Support: Therapeutics for Rare and Neglected Diseases

NCATS Research Team Identifies Possible Treatment for Niemann-Pick Type C1
To help streamline the scientific process, NCATS is collaborating with government agencies, academic scientists, the pharmaceutical industry and patient support groups to find potential treatments for a rare disease called Niemann-Pick disease type C1. (See related story above.)
NCATS Support: Division of Preclinical Innovation

Pitt Researchers Work to Restore Function in Paralysis Patients
A team of researchers at the University of Pittsburgh and its medical center have published breakthrough brain-computer-interface research that provides hope to nearly 6 million paralyzed individuals and another 1.7 million amputees nationwide.
NCATS Support: Clinical and Translational Science Awards Program

2012

Patients with Rare Muscle Disorder Benefit from Repurposed Heart Drug
With RDCRN and Clinical and Translational Science Awards (CTSA) Program support, researchers at seven institutions in four countries were able to recruit enough patients with a rare genetic muscle disorder for a clinical trial to test a potential repurposed drug therapy called mexiletine, normally prescribed for heart disorders.
NCATS Support: Rare Diseases Clinical Research Network and Clinical and Translational Science Awards Program

CTSA Training Enables Research on the Effects of Antibiotics on Body Fat
New York University School of Medicine’s Ilseung Cho, M.D., M.S., attests that his school’s Clinical and Translational Science Awards (CTSA) Program support has made crucial and ongoing contributions to his professional growth and achievements.
NCATS Support: Clinical and Translational Science Awards Program

A New Method to Help Scientists Better Identify Drug Candidates
Reporter genes are a powerful tool widely used in drug discovery screening that can produce misleading results. NCATS researchers have designed a novel method that increases the odds of identifying candidate compounds with true activity against biological or disease targets.
NCATS Support: Division of Preclinical Innovation

NCATS Science Showcased at 26th NIH Research Festival
Researchers and staff from NCATS highlighted some of the Center’s recent science advances and new initiatives at the 26th Annual NIH Research Festival October 9–12 on the NIH campus in Bethesda, Maryland.
NCATS Support: Division of Preclinical Innovation

NCATS Collaborative Project Wins Award for Excellence in Technology Transfer
A collaborative research team, including nine experts from NCATS, was honored by the mid-Atlantic regional Federal Laboratory Consortium for Technology Transfer for work on an investigational treatment for Niemann-Pick disease type C1, a rare neurological disease.
NCATS Support: Therapeutics for Rare and Neglected Diseases

Activating Key Cancer Enzyme Blocks Tumor Growth in Mice
A team that includes nine NCATS researchers has identified compounds that delay tumor formation in mice. The compounds target a specific form of pyruvate kinase, called PKM2, which governs how cancer cells use glucose.
NCATS Support: NCATS Chemical Genomics Center

Collaboration May Help Uncover Treatments for Rare Neurologic Disease
Scientists from NCATS and the University of Wisconsin–Madison have helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease, a neurological disorder.
NCATS Support: NCATS Chemical Genomics Center

An Epigenetic Strategy to Kill Cancer Tumors
Researchers at Penn State University, supported in part by Clinical and Translational Science Awards (CTSA) Program pilot funds, created a promising new strategy for “reactivating” genes that causes cancer tumors to shrink and die.
NCATS Support: Clinical and Translational Science Awards Program

New Guide to Help Researchers Develop Therapeutic Screening Tests
NCATS and Eli Lilly have jointly released an Assay Guidance Manual designed to provide step-by-step guidance through the complex process of turning a basic research finding into an assay (test) that will start the process of discovering pharmacological tools and drugs.
NCATS Support: NCATS Chemical Genomics Center, Assay Development and Screening Technology

New Drug for Rare Type of Cystic Fibrosis
Building on decades of NIH support for cystic fibrosis research, 10 Clinical and Translational Science Awards (CTSA) Program institutions provided resources and partnered with the Cystic Fibrosis Foundation and Vertex Pharmaceuticals to develop the first targeted therapy for a rare type of this deadly disease.
NCATS Support: Clinical and Translational Science Awards Program

Converting Brain Signals into Action
A multidisciplinary team of researchers at the University of Pittsburgh received critical help from Clinical and Translational Science Awards (CTSA) Program regulatory experts to develop a micro-electrocorticography grid that may help paralyzed individuals move again.
NCATS Support: Clinical and Translational Science Awards Program

Lighting a Path for Improved Cancer Treatment
Clinical and Translational Science Awards (CTSA) Program pilot funds often help jump-start innovative ideas — Michelle Bradbury, clinician-scientist at the Memorial Sloan-Kettering Cancer Center’s Nanotechnology Center, is taking a new nanoparticle into the clinic.
NCATS Support: Clinical and Translational Science Awards Program

Designing Solutions to Improve Health for All
The OneBreath ventilator was designed by a team in Stanford University’s Biodesign program for use in emergency pandemic situations and for patient care in resource-poor countries.
NCATS Support: Clinical and Translational Science Awards Program