From the Director | What's New at NCATS? | Research Opportunities Volume 04 • Issue 11 • November 24, 2015

Director's Message

Christopher Austin

At this Thanksgiving time of year, we turn our attention to how very different our family members can be despite sharing the same genes. Diseases turn out to be like families this way — a discovery with profound implications for translational science.

Read more in the latest Director's Message.

Christopher P. Austin, M.D.

What's New at NCATS?

NCATS Seeks Applications to Repurpose Existing Drugs

Rocket Men Design Device to Analyze Breath of Critically Ill Children

NCATS Seeks Input for Strategic Plan

NIH Staff Receive Award for Teaming with Industry to Treat Niemann-Pick Disease Type C

Training Investment Supported by CTSA Program Enables Thirty Million Words® Initiative

NCATS Now Offers Patenting and Licensing Expertise

Groft Honored with RDLA Lifetime Achievement Award

Small Businesses: Apply for NCATS Funding

Meet NCATS Staff

Upcoming Events

NCATS in the News

Collaborate with NCATS Scientists

Female researcher in a laboratory.

NCATS Seeks Applications to Repurpose Existing Drugs

NCATS is seeking applications for rigorous, pre-clinical research projects that are based on repurposing existing drugs or biologics. Through this new funding opportunity, NCATS anticipates committing $4.3 million in fiscal year 2016 to issue 10 to 15 awards in support of studies that establish the rationale for a clinical trial. Read the full announcement.

Mitchell Spearrin, Victor Miller (seated) and Christopher Strand with their device to measure ammonia levels.

Rocket Men Design Device to Analyze Breath of Critically Ill Children

Three Stanford University rocket-combustion experts, known as the "rocket men," have designed and tested a breathalyzer-like device to help detect a rare genetic disorder by measuring toxic ammonia levels in critically ill children. The device, primarily funded by the Stanford Center for Clinical and Translational Research and Education (Spectrum) via NCATS' Clinical and Translational Science Awards (CTSA) Program, may someday quickly and noninvasively detect everything from diabetes to cancers.

The experts chose a difficult molecule to measure (ammonia), a disease caused by a rare genetic defect with little commercial potential (hyperammonemia) and a hard-to-test patient population (infants). In just one year, the team went from a rough idea on paper to a working prototype that was tested with patients.

Now, the team is planning a second, larger trial. Since finishing their first prototype, the team has formed a company, Lumina Labs, funded by a grant from NIH's Small Business Technology Transfer program. The team also is preparing articles for publication describing the underlying spectroscopy, the device and, ultimately, their clinical studies. The Stanford Office of Technology and Licensing has filed a provisional patent. Read more in Stanford Medicine magazine.

Collage of a researcher in a laboratory and a doctor with a patient.

NCATS Seeks Input for Strategic Plan

NCATS currently is seeking input on the scientific and operational opportunities, challenges and research needs in translational science to help set the Center's strategic priorities and inform the development of a five-year strategic plan. Details about how to provide feedback are outlined in NOT-TR-16-002. The deadline for comments is Jan. 8, 2016.

2015 FLC Excellence in ‪Technology Transfer‬‬‬‬‬‬‬‬‬‬‬ Award winners.‬

NIH Staff Receive Award for Teaming with Industry to Treat Niemann-Pick Disease Type C

On Nov. 3, 2015, staff members from NCATS, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Cancer Institute (NCI) and Vtesse, Inc., were honored with an Excellence in ‪Technology Transfer Award from the Federal Laboratory Consortium for Technology Transfer.

The team was honored for its role in successfully transferring technology to Vtesse to further develop treatments for ‪Niemann-Pick disease type C and other ‪lysosomal storage disorders. The awardees are: NCI's Alan Hubbs, Ph.D.; NCATS' Krishna Balakrishnan, Ph.D., Juan Marugan, Ph.D., Charles Niebylski, Ph.D., J.D., Elizabeth Ottinger, Ph.D., Lili Portilla, M.P.A., and Wei Zheng, Ph.D.; NIH Office of Technology Transfer's Richard Rodriguez, M.B.A., Fatima Sayyid, M.H.P.M., and Suryanarayana Vepa, J.D., Ph.D.; NICHD's Forbes Porter, M.D., Ph.D.; and Vtesse's Bernardus Machielse, M.S., and Ravi Venkataramani, Ph.D.

Dana Suskind works with children who have cochlear implants.

Training Investment Supported by CTSA Program Enables Thirty Million Words® Initiative

Preparing researchers to be successful translational scientists is a significant focus of NCATS' Clinical and Translational Science Awards (CTSA) Program. Dana Suskind, M.D., professor of surgery and pediatrics and director of the pediatric cochlear implant program at the University of Chicago Institute for Translational Medicine (ITM), successfully made that transition. Through a CTSA Program-supported ITM KL2 Career Scholar Award, Suskind leveraged her early research on the language skills of children with cochlear implants and later findings on the impact language has on children's brain development to establish the Thirty Million Words® Initiative. Through this effort, she and other researchers are developing and disseminating evidence-based, parent-directed programs to encourage early brain development.

Most recently, Suskind published a book that translates much of what she has learned in her research into information parents can use to boost their children's language and development skills. Thirty Million Words: Building a Child's Brain recently reached number one on the best-seller list for parenting and family reference on Amazon. The KL2 support Suskind received helped her leverage her training and expertise, and her research continues to inform her scientific peers and the broader public, demonstrating the promise of the CTSA Program training investment. Read the full story in ITM News.

A street sign labeled Technology Boulevard and Research Bouelvard.

NCATS Now Offers Patenting and Licensing Expertise

NIH has realigned its technology transfer mission to better serve the pace and progress of science and to deliver benefits to patients faster. As of Oct. 1, 2015, NIH granted authority and responsibility for patenting and licensing activities to the specific NIH Institutes and Centers (ICs) that fund and direct the science behind these discoveries. As a result, NCATS will oversee its patenting and licensing activities directly rather than through the NIH Office of Technology Transfer.

"These organizational improvements will enable NCATS to better align its technology transfer expertise with its mission and programs, to the ultimate benefit of patients," said Lili Portilla, M.P.A., director of strategic alliances at NCATS.

The technology transfer reorganization also will:

To learn more about strategic alliances and technology transfer at NCATS, contact NCATSPartnerships@mail.nih.gov.

Stephen Groft receives the RDLA Lifetime Achievement Award.

Groft Honored with RDLA Lifetime Achievement Award

On Nov. 4, 2015, Stephen Groft, Pharm.D., former director of NCATS' Office of Rare Diseases Research, received  a Lifetime Achievement Award at the Rare Disease Legislative Advocates (RDLA) RareVoice Awards gala. He was honored for his dedication to stimulating rare diseases research and advancing development of therapies during his tenure at the Food and Drug Administration and NIH. Read the RDLA news release.

Small Businesses: Apply for NCATS Funding

NCATS has multiple funding opportunities available through the Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs, which aim to help entrepreneurs speed the translation of innovative technologies from the lab to the clinic.

In addition to the opportunities linked below, current NCATS SBIR/STTR Phase 1 grantees can apply for the I-Corps™ at NIH Program and the Niche Assessment Program. Current NCATS SBIR/STTR Phase 2 grantees are eligible to participate in the newly launched Commercialization Readiness Pilot Program.

2015 SBIR/STTR Omnibus Solicitation

SBIR Direct to Phase II

Platform Delivery Technologies for Nucleic Acid Therapeutics

Development of Appropriate Pediatric Formulations and Drug Delivery Systems

Visit the Open Small Business Opportunities for more information.

Henrietta Hyatt-Knorr and Joan Davis Nagel.

Meet More of NCATS' Key Staff

Henrietta Hyatt-Knorr, M.A., is a senior program and policy analyst in the Office of Rare Diseases Research; Joan Davis Nagel, M.D., M.P.H., is a medical officer in the Division of Clinical Innovation. Learn more about Hyatt-Knorr and Nagel — and many other NCATS staff members — via NCATS' Staff Profiles. Check back often to meet more of our team!

Upcoming Events

Cures Acceleration Network (CAN) Review Board Meeting

On Dec. 11, 2015, the CAN Review Board will meet by teleconference from 11:00 a.m. to 1:30 p.m. ET. Freda C. Lewis-Hall, M.D., CAN Review Board chair, will lead the meeting. For more information, visit the CAN Review Board page.

NCATS Advisory Council/CAN Review Board Meeting

On Jan. 14, 2016, NCATS will hold a joint meeting of the NCATS Advisory Council and the CAN Review Board from 8:30 a.m. to 5:00 p.m. on the NIH campus in Bethesda, Maryland. The meeting will feature reports from NCATS Director Christopher P. Austin, M.D., and others about the Center's initiatives, policies, programs and future direction. For more information, visit the NCATS Advisory Council and CAN Review Board pages.

Rare Disease Day at NIH

Rare Disease Day at NIH will take place on Feb. 29, 2016. The event aims to raise awareness about rare diseases, the patients they affect and the research collaborations that are addressing rare disease challenges. Sponsored by NCATS and the NIH Clinical Center, the annual event will take place from 8:30 a.m. to 3:30 p.m. in Masur Auditorium in Building 10 on the NIH campus in Bethesda, Maryland. The day will feature tours, posters and exhibits, and presentations. More information, including registration details, will be available soon.

Visit the NCATS Events page for more information about all Center-related events and activities.

NCATS in the News

Collaborate with NCATS Scientists

NCATS researchers are seeking collaborators in the following areas:

Bridging Interventional Development Gaps (BrIDGs)

Through the BrIDGs program, NCATS assists researchers in advancing promising therapeutic agents through late-stage pre-clinical development toward an Investigational New Drug application and clinical testing. NCATS now is accepting proposals on an ongoing basis to collaborate with BrIDGs scientists. For more information, contact BrIDGs@mail.nih.gov.

NCATS Chemical Genomics Center (NCGC)

NCATS' NCGC offers biomedical researchers access to large-scale screening capacity and medicinal chemistry and informatics expertise to develop chemical probe molecules. These resources can help scientists study the functions of genes, cells and biochemical pathways. NCGC also features assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav.

NIH RNA Interference (RNAi) Initiative

Through the NIH RNAi initiative, NCATS provides state-of-the-art, high-throughput RNAi genome-wide screens for humans and mice to NIH intramural researchers. For more information, contact Madhu Lal-Nag, Ph.D.

Pfizer's Centers for Therapeutic Innovation (CTI) for NIH Researchers

NCATS is facilitating Pfizer's CTI program at NIH, which pairs NIH intramural researchers and clinicians with Pfizer resources to pursue scientific and medical advances through joint therapeutic development of biologic compounds. To apply, submit a completed pre-proposal brief to your NIH Institute or Center's technology transfer office by February 2016. For more information, contact NIH-PfizerCTI@mail.nih.gov.

Therapeutics for Rare and Neglected Diseases (TRND)

The TRND program provides collaborators with access to significant in-kind resources and expertise to develop new therapeutics for rare and neglected diseases. NCATS now is accepting proposals on an ongoing basis through the TRND program for collaborative projects that focus on pre-clinical and early clinical development of new drugs for rare and neglected tropical diseases. For more information, contact TRND@mail.nih.gov.

Toxicology in the 21st Century (Tox21)

The goal of the Tox21 program is 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. Proposed assays must be compatible with the high-throughput screening guidelines described in the assay guidance criteria. To suggest an assay, submit a nomination form (PDF - 44KB) to Menghang Xia, Ph.D.

Research Opportunities and Announcement Highlights

New since last month in reverse chronological order

NIH Precision Medicine Initiative Cohort Program Funding Opportunities • NIH • Nov. 17, 2015

Pre-application: Stimulating Peripheral Activity to Relieve Conditions (SPARC): Comprehensive Functional Mapping of Neuroanatomy and Neurobiology of Organs (OT1) • RFA-RM-15-003 • Nov. 16, 2015

Limited Competition - Stimulating Peripheral Activity to Relieve Conditions (SPARC): Comprehensive Functional Mapping of Neuroanatomy and Neurobiology of Organs (OT2) • RFA-RM-15-018 • Nov. 16, 2015

Pre-application: Stimulating Peripheral Activity to Relieve Conditions (SPARC): Foundational Functional Mapping of Neuroanatomy and Neurobiology of Organs (OT1) • RFA-RM-15-019 • Nov. 16, 2015

Limited Competition - Stimulating Peripheral Activity to Relieve Conditions (SPARC): Foundational Functional Mapping of Neuroanatomy and Neurobiology of Organs (OT2) • RFA-RM-15-020 • Nov. 16, 2015

Advance Notice: NIH Plans to Implement the Federal Awardee Performance and Integrity Information System (FAPIIS) Requirements Beginning January 1, 2016 • NOT-OD-16-019 • Nov. 4, 2015

Request for Information (RFI): Inviting Comments and Suggestions on the Priorities Specified for the NIH-wide Rehabilitation Research Plan • NOT-HD-15-032 • Nov. 3, 2015

SBIR/STTR Commercialization Readiness Pilot (CRP) Program: Technical Assistance (SB1) • PAR-16-026 • Nov. 2, 2015

Innovation Corps (I-Corps) at NIH Program for NIH and CDC Phase I Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) Grantees (Admin Supp) • PA-16-019 • Oct. 29, 2015

NCATS Is Accepting Proposals for the Bridging Interventional Development Gaps Program • Oct. 22, 2015

NCATS Is Accepting Proposals for the Therapeutics for Rare and Neglected Diseases Program • Oct. 22, 2015

Pre-clinical Research Based on Existing Repurposing Tools (R21) • RFA-TR-16-001 • Oct. 21, 2015

Request for Information (RFI): Undiagnosed Diseases Research • NOT-RM-16-001 • Oct. 9, 2015

Request for Information (RFI): Soliciting Input for the National Center for Advancing Translational Sciences (NCATS) Strategic Planning Process • NOT-TR-16-002 • Oct. 8, 2015

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

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.