From the Director | What's New at NCATS? | Research Opportunities Volume 03 • Issue 08 • November 25, 2014

Director's Message

Christopher Austin

Developing interventions for better human health differs from developing other consumer goods in a fundamental way: Interventions to improve health generally are developed without direct input from the people they are meant to benefit. I believe this odd fact of history is responsible for much of the inefficiency and ineffectiveness of translational science. Imagine for a moment that developers of a new snack food locked themselves away in a lab where the criteria for success were novelty and appeal to fellow developers, but they did not consult any consumers. The result could be an innovative snack food that would delight the developers but taste terrible; no one would buy it. This kind of scenario happens often in intervention development.

Read more of the latest Director's Message.

Christopher P. Austin, M.D.

What's New at NCATS?

NCATS Screening Technologies Help Identify Potential Treatment Target for Neurological Disorder

CTSA-Supported Network Aided Boston Marathon Victim Otologic Study

Portilla Receives NIH Office of the Director Honor Award for Tech Transfer Efforts

NCATS Collaborates with Foundation on Patient Registry Software

NCATS Researchers Participate in China's First Systems Toxicology Workshop

NCATS Engages Students at Frontiers in Science and Medicine Event

Proposed Regulation and Policy Changes to Improve Clinical Trials

Upcoming Events

NCATS in the News

Collaborate with NCATS Scientists

Hands of a CMT patient

NCATS Screening Technologies Help Identify Potential Treatment Target for Neurological Disorder

Improving scientific methods and tools is a key part of the NCATS approach to solving translational problems. A recent advance in this area — made possible through a collaboration involving NCATS experts, academic researchers, a patient advocacy group and a pharmaceutical company — already has enabled researchers to identify a potential new drug target for Charcot-Marie-Tooth disease, an inherited neurological disorder. Read the full feature.

Marathon runners in the city

CTSA-Supported Network Aided Boston Marathon Victim Otologic Study

In the days following the Boston Marathon bombing in April 2013, area clinicians treated large numbers of patients with blast traumas to the ear. Researchers at Massachusetts Eye and Ear Infirmary, affiliated with Harvard Medical School, immediately set out to study the extent of these injuries. Fortunately, with support from NCATS' Clinical and Translational Science Awards (CTSA) program, Harvard already had an institutional review board (IRB) reliance network in place that enabled the study to proceed efficiently.

Researchers enrolled nearly 100 of the injured individuals in an eight-institution study. Participants completed an otologic/noise exposure history, a current symptom assessment and quality-of-life questionnaires at initial visits and six months later. The findings were published online in November 2014.

Nearly all of the study participants reported good hearing quality before the explosions but suffered long-term ear and hearing problems after. Forty-eight patients in the study had ruptured eardrums, and damage to the eardrum healed on its own in more than one-third of the cases. Some victims underwent surgery or other therapies to treat the damage, and others suffered permanent damage.

The researchers concluded that blast-related ear injuries constitute a major source of ongoing morbidity (affecting quality of life) following the bombings. In future studies, the researchers plan to look more closely at the range of hearing damage patients experienced, particularly those who may be suffering from "hidden hearing loss"Ě that cannot be identified by standard testing.

This study likely would not have happened without the Harvard IRB reliance framework that enabled researchers to avoid a great deal of the redundancy and overlap that occurs during the traditional IRB review process used to initiate clinical studies and trials. To learn more about the Boston Marathon bombing study, the related IRB process, and CTSA efforts underway to streamline the implementation of clinical studies and trials, read the May 2014 NCATS feature story.

Portrait of Lili Portilla, NCATS' director of strategic alliances

Portilla Receives NIH Office of the Director Honor Award for Tech Transfer Efforts

Congratulations to Lili Portilla, M.P.A., NCATS director of strategic alliances, who received an Honor Award from the NIH Office of the Director on Nov. 19, 2014. Portilla received the award for her "exceptional creativity and tenacity in developing plans to reorganize the NIH technology transfer enterprise for the 21st Century."

NCATS Collaborates with Foundation on Patient Registry Software

The Marshfield Clinic Research Foundation has developed a computer software program that soon will support the NIH/NCATS Global Rare Diseases Patient Registry Data Repository (GRDRSM) program. The software is designed to help advance rare diseases research. Read the Marshfield news release.

NCATS staff Dan Tagle, Ph.D., holds a tissue chip

NCATS Researchers Participate in China's First Systems Toxicology Workshop

NCATS researchers and grantees collaborate to develop better methods for predicting which drugs and chemicals may be harmful to people. Center representatives recently traveled to Beijing for China's first systems toxicology workshop, which NCATS helped organize. The focus was on exchanging knowledge and lessons learned with international colleagues. Danilo Tagle, Ph.D., NCATS associate director for special initiatives, presented a session titled "3-D Cell Culture Models" that included discussion about progress in the NCATS-led Tissue Chip for Drug Screening program. In addition, Anton Simeonov, Ph.D., acting NCATS deputy scientific director, led a presentation on "Overview of Structure, Processes and Research Model for the Tox21 Collaborative Program." Review the workshop guide.

NCATS researcher shows children science experiment

NCATS Engages Students at Frontiers in Science and Medicine Event

On Oct. 24, 2014, NCATS staff joined other Washington, D.C.-area companies to participate in the sixth annual Frontiers in Science and Medicine Day at the Shady Grove Life Sciences Center, Rockville, Maryland. Designed to encourage children to pursue careers in science and medicine, the event featured more than 600 local seventh-graders. In this photo, NCATS medicinal chemist Paresma "Pinky" Patel, Ph.D., demonstrates how polymers (large, strong molecules composed of many repeated parts) are formed by pulling long strings of nylon from the interface of two liquid chemicals. Read more about the event.

Proposed Regulation and Policy Changes to Improve Clinical Trials

Medical advances would not be possible without participants in clinical trials, and transparency about trials in process and subsequent results ensure potential participants can make informed decisions about enrollment. Federal efforts now are underway to improve information access. Read the NIH news release.

Upcoming Events

December

Cures Acceleration Network (CAN) Review Board to Meet December 12

On Dec. 12, 2014, the CAN Review Board will meet by teleconference and in open session at 11:00 a.m. ET. Freda C. Lewis-Hall, M.D., CAN Review Board chair, will lead the meeting, which will feature presentations by board members and NCATS leadership, including updates on phase II of the Tissue Chip for Drug Screening program and general discussion. For more information, visit the CAN Review Board page of the NCATS website.

January

NCATS Advisory Council/CAN Review Board to Meet January 15

On Jan. 15, 2015, NCATS will hold a joint meeting of the NCATS Advisory Council and the CAN Review Board on the NIH campus in Bethesda, Maryland. The meeting will feature reports from NCATS Director Christopher P. Austin, M.D., and other presenters about the Center's initiatives, policies, programs and future direction. For more information, visit the NCATS Advisory Council and CAN Review Board pages of the NCATS website.

NCATS in the News

Collaborate with NCATS Scientists

NCATS researchers are seeking collaborators in the following areas:

NCATS Chemical Genomics Center (NCGC)

NCGC offers biomedical researchers access to large-scale screening capacity along with the medicinal chemistry and informatics expertise necessary to develop chemical probe molecules and to study the functions of genes, cells and biochemical pathways. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav.

NCGC researchers also seek collaborators for assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. Learn more.

NIH RNA Interference (RNAi) Initiative

The NIH RNAi initiative, administered by NCATS, provides state-of-the-art, high-throughput RNAi genome-wide screens for humans and mice. This resource is available only to NIH researchers. Scientists interested in performing high-throughput RNAi screens can contact Scott Martin, Ph.D., for more information.

Toxicology in the 21st Century (Tox21) Program

The Tox21 program aims to test 10,000 chemicals and evaluate their potential to cause health problems. Any investigator may propose the development of biological assays for high-throughput screening.

To suggest an assay, submit an assay nomination form to Menghang Xia, Ph.D. Proposed assays must be compatible with the high-throughput screening guidelines as described in the assay guidance criteria.

Research Opportunities and Announcements

Visit the NCATS Open Opportunities page for a complete list of funding and program announcements.

NIH Request for Public Comments on the Draft NIH Policy on Dissemination of NIH-Funded Clinical Trial Information • NOT-OD-15-019

Publication of Notice of Proposed Rulemaking for Clinical Trials Registration and Results Submission under FDAAA • NOT-OD-15-018

Request for Information (RFI): Input on Direction of the Second Phase of the "Illuminating the Druggable Genome" Program • NOT-RM-14-018

Notice of Corrections to RFA-TR-14-009 "Clinical and Translational Science Award (U54)" • NOT-TR-14-013

Notice of Revised NIH Definition of "Clinical Trial"Ě • NOT-OD-15-015

Reminder: NIH Requires the Research Performance Progress Report (RPPR) for All Type 5 Progress Reports • NOT-OD-15-014

NIH Offers Niche Assessment Program to SBIR and STTR Phase I Awardees • NOT-OD-15-003

Webinars on What You Need to Know About NIH Application Submission and Review • NOT-OD-15-002

Reminder Notice Regarding Requirements of the Bayh-Dole Act and the NIH's Implementing Regulations • NOT-OD-15-004

Use of New Inclusion Management System Required as of October 17, 2014 • NOT-OD-15-005

NIH Operates Under a Continuing Resolution FY2015 • NOT-OD-15-001

Notice of Change in the Application Due Date in RFA-TR-14-009 "Clinical and Translational Science Award (U54)" • NOT-TR-14-012

Clinical and Translational Science Award (U54) • RFA-TR-14-009

Onsite Tools and Technologies for Heart, Lung, and Blood Clinical Research Point-of-Care STTR (R41/R42) • RFA-HL-14-017

Onsite Tools and Technologies for Heart, Lung, and Blood Clinical Research Point-of-Care SBIR (R43/R44) • RFA-HL-14-011

Platform Delivery Technologies for Nucleic Acid Therapeutics (R41/R42) • PA-14-308

Platform Delivery Technologies for Nucleic Acid Therapeutics (R43/R44) • PA-14-307

Bioreactors for Reparative Medicine (R41/R42) • RFA-HL-15-004

Bioreactors for Reparative Medicine (R43/R44) • RFA-HL-15-008

We Want to Hear from You

We welcome your feedback to ensure that we are meeting the needs of all of our stakeholders. Please e-mail us directly at info@ncats.nih.gov, follow us on Facebook and Twitter, view our YouTube channel, and join the NCATS e-mail list for other Center announcements.

For language access assistance, contact the NCATS Public Information Officer.