Director's Message
I often talk about how NCATS' mission and programs are different from those of other organizations in the research ecosystem. One of these differences is that NCATS is "disease-agnostic": rather than focusing on a single type of condition or biological system, we look for what is common among diseases and the translational process. This systematic approach is especially important for investigating rare diseases, which number in the thousands; only a few hundred have treatments approved by the Food and Drug Administration. NCATS' Rare Diseases Clinical Research Network aims to address many of the unique challenges in developing rare disease therapies, including difficulties in diagnosis, widely dispersed patients and scientific experts, and a perceived high risk and cost for developing such treatments.
Read more of the latest Director's Message.
Christopher P. Austin, M.D.

What's New at NCATS?
NCATS Selects New Pre-Clinical Drug Development Projects
NIH Announces Grants for Frontotemporal Degeneration Research
NCATS Launches Three New Therapeutic Projects for Rare Blood Disorders and Infectious Diseases
New Rare Diseases Clinical Research Network Awards
NCATS Becomes a Partner in Global Translational Research Collaboration
NIH to Offer Webinars in November for New Grant Applicants
Collaborate with NCATS Scientists


NCATS Selects New Pre-Clinical Drug Development Projects
On Oct. 29, 2014, NCATS announced support for two Bridging Interventional Development Gaps (BrIDGs) pre-clinical drug development projects. The new projects are aimed at finding potential treatments for diabetic keratopathy — a condition that often leads to blindness — and severe heart attacks. Through the BrIDGs program, NCATS provides scientists with access to pre-clinical resources such as toxicology studies. Read the full announcement.


NIH Announces Grants for Frontotemporal Degeneration Research
On Oct. 23, 2014, NIH announced $5.9 million in 2014 funding for three large, five-year projects on frontotemporal degeneration, a form of dementia that affects approximately 50,000 Americans. NCATS is funding one of the projects: the Frontotemporal Lobar Degeneration Clinical Research Consortium, part of the Rare Diseases Clinical Research Network. This project and the other two, which are supported by the National Institute of Neurological Disorders and Stroke and the National Institute on Aging, will help advance research through collaboration and development of novel strategies for prevention, diagnosis and treatment. Read the NIH news release.


NCATS Launches Three New Therapeutic Projects for Rare Blood Disorders and Infectious Diseases
On Oct. 15, 2014, NCATS announced it will begin work on three new pre-clinical drug development projects aimed at finding treatments for malaria, Lassa fever and two blood disorders: beta-thalassemia and sickle cell disease. Supported through the Center's Therapeutics for Rare and Neglected Diseases program, the research also could lead to innovations that broadly improve and accelerate the translational science process. Read the full announcement.


New Rare Diseases Clinical Research Network Awards
On Oct. 8, 2014, NCATS announced awards — totaling about $29 million in fiscal year 2014 NIH funding — to continue and expand the Rare Diseases Clinical Research Network (RDCRN). The RDCRN is led by NCATS and supported by 10 other NIH institutes and offices. Through this support, multidisciplinary physician scientists at 22 consortia, including six new ones, will collaborate with representatives of 98 patient advocacy groups to advance clinical research and investigate new treatments for patients with rare diseases. Read the full news release.

NCATS Becomes a Partner in Global Translational Research Collaboration
NCATS is part of a new collaborative initiative designed to identify and address globally relevant problems in translational research — such as high development failure rates and low reproducibility of data — that thwart worldwide disease preventions, treatments and cures. The agreement is among NCATS, EATRIS ERIC (the European Infrastructure for Translational Medicine, part of the European Research Infrastructure Consortium), Therapeutic Innovation Australia, and the Canadian Centre for Drug Research and Development. Read more about the collaboration.

NIH to Offer Webinars in November for New Grant Applicants
In early November, NIH will host four free webinars for new grant applicants to help demystify the application and peer review processes. Each will focus on a different type of NIH grant. For dates and topics, view NOT-OD-15-002.

Upcoming Events
December
Cures Acceleration Network (CAN) Review Board Set to Meet December 12
On Dec. 12, 2014, the CAN Review Board will convene a virtual meeting by teleconference, in open session at 11:00 a.m. ET. Freda C. Lewis-Hall, M.D., CAN Review Board chair, will lead the meeting. In accordance with Public Law 92-463, the session will be open to the public. For more information, visit the CAN Review Board page of the NCATS website.
January
NCATS Advisory Council/Cures Acceleration Network (CAN) Review Board Set 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 others about the Center's initiatives, policies, programs and future direction. For more information, visit the NCATS Advisory Council page and the CAN Review Board page of the NCATS website.

NCATS in the News
- NCATS Bridges Research Gaps to Develop Treatments for Diabetes-Related Blindness and Severe Heart Attacks • NCATS • Oct. 29, 2014
- Could a Robot Do Your Job? • USA Today • Oct. 28, 2014
- NIH Announces Grants for Frontotemporal Degeneration Research • NINDS, NCATS, NIA • Oct. 23, 2014
- New Diagnostic Test Helps UCLA Doctors Pinpoint Mutations That Cause Rarest Genetic Diseases • University of California, Los Angeles • Oct. 23, 2014
- You Won't Believe What's In These Pills • NIH Director's Blog • Oct. 21, 2014
- UNMC Researcher Awarded $3.3 Million for Study of Rare Childhood Diseases • Omaha World-Herald • Oct. 20, 2014
- NCATS Announces New Research Projects to Improve Treatments for Rare and Neglected Diseases • NCATS • Oct. 15, 2014
- Platelet Transfusions in Newborn Babies • Wauwatosa Now • Oct. 15, 2014
- NIH Offers Grantees Tools to Succeed in Business • BioWorld • Oct. 13, 2014
- NIH Allocates $29M to Study 200 Rare Diseases • Genetic Engineering & Biotechnology News • Oct. 9, 2014
- Kidney Researchers Lead $10 Million Effort to Study Nephrotic Syndrome • Newswise • Oct. 9, 2014
- $6.25M NIH Award to Lead Brittle Bone Disease Clinical Research Consortium • Baylor College of Medicine • Oct. 8, 2014
- Orphan Disease Research from NIH to Help People with the World's Rarest Disorders • Medical Daily • Oct. 8, 2014
- NIH Funds Research Consortia to Study More than 200 Rare Diseases • NCATS • Oct. 8, 2014
- NIH Launches "I-Corps" to Grow Drug, Diagnostic Startups • Genetic Engineering & Biotechnology News • Oct. 7, 2014
- Sabin Announces Completion of Human Hookworm Vaccine Candidate Phase 1 Trials • Outbreak News Today • Oct. 7, 2014
- Scientists Put Living Human Tissue on Silicon Chip • Voice of America • Oct. 2, 2014

Collaborate with NCATS Scientists
NCATS researchers are seeking collaborators in the following areas:
NCATS Chemical Genomics Center (NCGC)
NCGC is one of the centers in the Molecular Libraries Probe Production Centers Network (MLPCN), which is an NIH Common Fund initiative. Through the MLPCN, NCGC offers biomedical researchers access to large-scale screening capacity along with the medicinal chemistry and informatics expertise necessary to identify 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.
