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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.

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 (RDCRN) and Clinical and Translational Science Awards (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 C, 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