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Feature Stories

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.

Michael J. Fox Foundation Funds Research Project that Leverages NCATS Chemical Screening Approach and Resources

image of parkin screen

A key NCATS approach to solving translational problems is to focus on improving scientific methods and tools that can speed the translation of laboratory discoveries into new treatments for patients. With support from the Michael J. Fox Foundation for Parkinson's Research, James Inglese, Ph.D., director of NCATS' Assay Development and Screening Technology Laboratory, and Richard Youle, Ph.D., of 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 Pre-Clinical Innovation

Spotlight on Collaboration: A Journey From Biological Probes to
Potential Therapeutics

High-throughput screening robot.

Researchers studying the biology of diseases often use scientific tools called biological probes, which are small molecules that change biological processes or characteristics of a disease in some way. Since 2004, scientists at the NIH Chemical Genomics Center, which is now part of NCATS, have worked closely with academic, nonprofit and biotech researchers on more than 300 collaborative probe development projects. The efforts of one project team exploring an enzyme family called lipoxygenases culminated in the discovery of three novel small molecule chemical probes with the potential to treat diabetes, stroke and thrombosis.

NCATS Support: NIH Chemical Genomics Center

NCATS Researchers Propose Innovative Approach to Test Drugs in Rare Diseases

Untreated cells from children with the genetic disease progeria (left) compared to similar cells treated with farnesyltransferase inhibitors.

Discovering treatments for rare diseases is challenging because the small numbers of patients with these diseases make it difficult to find enough people who can participate in clinical trials. The small numbers of rare disease patients also make gathering information about the diseases difficult. As a result, scientists often know little about the symptoms and biology of these conditions, which adds to the complexity of designing drug studies. In a paper published in the June 6, 2014, issue of Nature Biotechnology, NCATS scientists 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

Marathon runners in the city

The IRB review process can be especially cumbersome for researchers and IRB staff conducting multisite research (i.e., conducted at several locations). Yet, multisite studies are critical to translational research because they provide researchers with access to larger numbers of participants, which can enhance the validity of results. Multisite studies are designed to foster collaboration among investigators, which can improve problem-solving and idea-generation. Addressing these challenges is a key part of NCATS’ mission to make translational research more efficient. NCATS’ Clinical and Translational Science Awards (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

Petra Kaufmann Joins NCATS as Clinical Innovation Director

Petra Kaufmann, M.D., M.Sc.

On May 4, 2014, Petra Kaufmann, M.D., M.Sc., joins NCATS to direct its Division of Clinical Innovation. In this role, she will oversee the Clinical and Translational Science Awards program with the aim of improving the effectiveness and efficiency of the process of translation from scientific discovery through clinical research to improved health outcomes. Kaufmann brings a broad range of academic experience from laboratory to clinical research.

NCATS Support: Office of the Director

Screening Platform Is a Launch Pad for Novel Treatment Combinations

Screening resultsIn the Jan. 27, 2014, early edition of the journal Proceedings of the National Academy of Sciences, NCATS and National Cancer Institute investigators published the results of 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. Investigators chose a difficult-to-treat variety of diffuse large B-cell lymphoma, a type of blood cancer, as the model for the study.

NCATS Support: Division of Pre-Clinical Innovation

NCATS Welcomes McInnes and Zuk to Leadership Team

Pamela McInnesand Dorit Zuk

In January 2014, NCATS Director Christopher P. Austin, M.D., 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 already is benefitting from the enormous expertise and experience they bring to the Center.

NCATS Support: Office of the Director

Tissue Chip Projects Highlighted in Major Journal

embryonic smooth muscle cellOn 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. Stem Cells on Bioengineered Microphysiological Platforms for Disease Modeling and Drug Testing features reviews of the progress NCATS grantees have made to date on innovative cell resources and model systems. Following a brief introduction authored in part by NCATS’ Danilo Tagle, Ph.D., who oversees the program, 18 projects are highlighted.

NCATS Support: Tissue Chip for Drug Screening

Rare Diseases Research Advocate Retires

Stephen Groft

Long-time rare diseases research champion 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. He is recognized by many as a luminary and true champion for rare disease patients and families.

NCATS Support: Office of Rare Diseases Research

Understanding the Brain’s Response to Sugar Could Help Treat Obesity

MRI images of brain response to sugar

Finding new biomarkers to better prevent, diagnose and treat diseases is one of many translational science challenges that NCATS is working to overcome. Yale researchers, supported in part by a Clinical and Translational Sciences Award (CTSA) from 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 and could lead to the development of new biomarkers for the condition.

NCATS Support: Clinical and Translational Science Awards

Five CTSAs Enable NIH-Funded Research on Innovative Allergy Therapy

Peanuts are one of the most common and deadly food allergies, causing more than half of all deaths from food-related allergic reactions. Finding a new way to treat and possibly prevent the severest of these reactions — and those related to a number of other common foods — 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 the challenge of finding a viable treatment approach.

NCATS Support: Clinical and Translational Science Awards

NCATS Spotlight: Toxicology in the 21st Century (Tox21) Program

graphic of DNA and associated codeWe are exposed to thousands of different chemicals during our lifetimes, from consumer products to food additives to pharmaceutical drugs. Yet scientists lack the data to predict how these chemicals affect our bodies and their potential for harm. New system-wide methods for assessing chemical toxicity can improve the way scientists evaluate environmental chemicals and develop new medicines. The Tox21 program, a collaborative initiative among NIH, the Food and Drug Administration, and the Environmental Protection Agency, 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

Retinal ganglion cells

Understanding of the causes and characteristics of many diseases has improved dramatically over the past several decades, but in most cases, new interventions based on this understanding have not kept pace. NCATS focuses on ways to speed the development of diagnostics and therapeutics to unlock the promise of science for patients. The NCATS approach — which is based on innovation in how science is done as well as what is done — now has led to dramatic success for a project focused 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

blood spot card with baby feetA team of researchers at the University of California, Davis, found that more people have gene changes linked to Fragile X syndrome (FXS) 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. Accurate and timely diagnosis of FXS by such newborn screening may allow early educational and behavioral intervention, which can improve functional outcomes in FXS children.

NCATS Support: Clinical and Translational Science Awards

NCATS Research Team Identifies Possible Treatment for
Niemann-Pick Type C1

An NPC cell

Translational research is a team sport. 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. The research team recently showed that patient skin cells treated with a form of vitamin E, called delta-tocopherol, have lower accumulation of cholesterol. The team’s findings represent another potential ingredient in a treatment cocktail for Niemann-Pick type C1.

NCATS Support: Division of Pre-Clinical Innovation

TRND Research Leads to NIH Trial to Test Drug for
Niemann-Pick Type C1

NPC researchers

Niemann-Pick disease type C1 is a rare, inherited disease characterized by progressive impairment of motor and intellectual functions in early childhood. To date, the disease is incurable, and no drugs approved by the Food and Drug Administration are available to treat it. Now, a promising new treatment is on the horizon. NIH has initiated a Phase I clinical trial to evaluate the safety and effectiveness of cyclodextrin as a potential therapy for Niemann-Pick type C1.

NCATS Support: Therapeutics for Rare and Neglected Diseases

Pitt Researchers Work to Restore Function in Paralysis Patients

Jan Scheuermann and robot arm

A multidisciplinary team of researchers at the University of Pittsburgh (Pitt) 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. The collaboration relied on help from four federal agencies along with support by a private foundation, two academic research centers and a private company.

NCATS Support: Clinical and Translational Science Awards

Patients with Rare Muscle Disorder Benefit from Repurposed Heart Drug

Boy playing baseball

Patients with a rare genetic muscle disorder have new help: a generic drug called mexiletine, which normally is prescribed to treat heart disorders. With assistance from NIH’s Rare Diseases Clinical Research Network and CTSA program, researchers at seven institutions in four countries were able to recruit enough patients with this rare disease for a clinical trial to test the drug.

NCATS Support: Rare Diseases Clinical Research Network and Clinical and Translational Science Awards

CTSA Training Enables Research on the Effects of Antibiotics on Body Fat

Ilseung Cho

A core component of the CTSA program is training, cultivating and sustaining future leaders in the biomedical research workforce. New York University School of Medicine’s Ilseung Cho, M.D., M.S., attests that his school’s CTSA support has made crucial and ongoing contributions to his professional growth and achievements.

NCATS Support: Clinical and Translational Science Awards

A New Method to Help Scientists Better Identify Drug Candidates

Gloved hand holding pipette with orange liquid over assay plate

In the past few years, researchers have discovered that the use of reporter genes, a powerful technique widely used in drug discovery screening, can produce misleading results and lead to wasted effort and inefficiency in the drug discovery process. Now, researchers from NCATS 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 Pre-Clinical Innovation

NCATS Science Showcased at NIH Research Festival

Raisa Jones (right) and attendee

Researchers and staff from the National Center for Advancing Translational Sciences (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, Md.

NCATS Support: Division of Pre-Clinical Innovation

NCATS Collaborative Project Wins Award for Excellence in Technology Transfer

Lili Portilla, Elizabeth Ottinger, Alan Hubbs, Forbes Porter, and Steven Silber

A collaborative research team, including nine experts from NCATS, was honored last month 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 genetic disease of cholesterol storage that eventually leads to neurodegeneration.

NCATS Support: Therapeutics for Rare and Neglected Diseases

Activating Key Cancer Enzyme Blocks Tumor Growth in Mice

PKM2 enzyme

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: NIH Chemical Genomics Center

Collaboration May Help Uncover Treatments for Rare Neurologic Disease

Sung-Wook Jang

A research collaboration including scientists from NCATS and the University of Wisconsin–Madison, helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease (CMT), a genetic neurological disease for which there are no current treatments.

NCATS Support: NIH Chemical Genomics Center

An Epigenetic Strategy to Kill Cancer Tumors

Yanming Wang and Gong Chen

Penn State professors Yanming Wang (left) and Gong Chen (right), supported in part by CTSA 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

New Guide to Help Researchers Develop Therapeutic Screening Tests

Assay Guidance Manual

On May 2, 2012, NCATS and Eli Lilly and Company jointly released an online Assay Guidance Manual designed to provide researchers with step-by-step guidance through the complex process of turning a basic research finding into an assay that will start the process of discovering pharmacological tools and drugs. Assays are laboratory tests that enable researchers to examine thousands of compounds using state-of-the-art high-throughput screening systems critical to drug discovery.

NCATS Support: NIH Chemical Genomics Center, Assay Development and High-Throughput Screening

New Drug for Rare Type of Cystic Fibrosis

Medical professionals look at x-ray film on a lightbox.

Building on decades of NIH support for cystic fibrosis research, 10 CTSA 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

Converting Brain Signals into Action

Brain surgery patient

A multidisciplinary team of researchers at the University of Pittsburgh received critical help from CTSA regulatory experts to develop a micro-electrocorticography grid that may help paralyzed individuals move again.

NCATS Support: Clinical and Translational Science Awards

Lighting a Path for Improved Cancer Treatment

Michelle Bradbury

CTSA 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

Designing Solutions to Improve Health for All

Matt Callahan

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