In recent years, basic scientists have made breathtaking advances in our understanding of the human body's biology and chemistry. The human genome has been sequenced, stem cells understood and RNA interference discovered. All of these advances have been celebrated for holding enormous promise for improving human health. But the road from promise to achievement of health impact — what is called "translation" in medical research terms — is long, complex and full of obstacles. For example, it can take 14 years or more before a basic discovery leads to a new treatment. Another decade can pass before that intervention is available to all patients who need it.
Speeding up this process is a top priority for NCATS, and the Clinical and Translational Science Awards (CTSA) program is key to our efforts. The CTSA program supports a national consortium of medical research institutions that work together to improve the way clinical and translational research is conducted nationwide. CTSA institutions develop new tools, methods, resources and services that catalyze research progress. Because translation can't happen without cooperation and collaboration across disciplines, CTSA institutions also lead training and career development for a new breed of team-oriented scientists and clinicians focused on translation. Earlier this week, NIH announced more than $79 million in fiscal year 2013 funding to support 15 CTSAs.
Other exciting milestones in translation include the participation of NCATS researchers as part of a team that developed a potential vaccine for malaria, a deadly infectious disease affecting an estimated 219 million people worldwide each year. The vaccine already is showing promise in an early-stage clinical trial by NIH's National Institute of Allergy and Infectious Diseases and collaborators at the Walter Reed Army Institute of Research and the Naval Medical Research Center.
And in September, NCATS held its very first Research & Development Day designed to link potential investors with pre-clinical drug development projects. Already, we are seeing benefits from the connections made at this event in Cambridge, Massachusetts. Read more about all of these recent accomplishments in the e-news articles below.
Christopher P. Austin, M.D.
National Center for Advancing Translational Sciences
NCATS Announces 15 Clinical and Translational Science Awards; Expands Consortium's Outreach to New Hampshire
Translating basic discoveries into new treatments that tangibly improve human health requires innovative collaborations and resources, as well as a diverse, highly trained workforce. To help meet these needs, in September, NCATS provided more than $79 million in fiscal year 2013 funding to support 15 Institutional Clinical and Translational Science Awards (CTSAs).
"Science and technology are progressing at an unprecedented pace, and the CTSA program — which represents NIH's largest single investment in clinical research — is helping researchers harness these innovations and deliver improved diagnostics, treatments and cures for disease," said NIH Director Francis S. Collins, M.D., Ph.D.
These latest awards demonstrate NIH's commitment to the CTSA program, which supports a consortium of more than 60 research institutions across the country aimed at strengthening translational research. Under NCATS' leadership, the program is focused on solving roadblocks common to clinical and translational efforts for all diseases.
"The CTSA Consortium is leading national efforts to enhance the efficiency, quality and safety of translational research, no matter the disease or condition" said NCATS Director Christopher P. Austin, M.D. "This aligns with the NCATS mission to create new technologies and methods that can be applied widely to streamline development and implementation of interventions that improve human health."
The 2013 awards expand consortium representation to New Hampshire with an award to Dartmouth College, extending the network to 31 states and the District of Columbia. Read the news release.
Last month, NCATS announced four new pre-clinical drug development projects designed to target a type of hereditary blindness and two diseases characterized by heart problems. The Center selected the projects for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers.
Supported by the Center's Therapeutics for Rare and Neglected Diseases (TRND) program, these projects mark the program's first use of stem cells as well as its first collaboration with a large pharmaceutical company — Eli Lilly — to develop a treatment for a rare disease.
"TRND is grounded in partnerships with academic, government, pharmaceutical and patient advocacy groups. Working in collaboration, scientists conduct pre-clinical development of new drugs and then advance them to first-in-human clinical trials," said NCATS Director Christopher P. Austin, M.D. "Like all NCATS programs, TRND seeks to develop new technologies and more efficient paradigms for translation, in the context of important unmet medical needs."
Two projects seek a treatment for retinitis pigmentosa, a severe form of hereditary blindness. A third project focuses on a potential treatment for hypoparathyroidism, a hormone-deficiency syndrome that can lead to heart problems and convulsions. The remaining project aims to develop a possible therapeutic that targets a heart disorder associated with LEOPARD syndrome, an extremely rare genetic disease that affects many areas of the body.
Read the full news release.
NCATS researchers Jim Inglese, Ph.D., and Patricia Dranchak, Ph.D., are part of a scientific team that helped the biotech company Sanaria, Inc., develop a potential malaria vaccine. Malaria is a deadly infectious disease that affects an estimated 219 million people worldwide each year. The vaccine has shown promise in an early-stage clinical trial conducted by researchers at NIH's National Institute of Allergy and Infectious Diseases (NIAID) and collaborators at the Walter Reed Army Institute of Research and the Naval Medical Research Center.
Inglese leads the Assay Development Screening Technology Group, which includes Dranchak, in NCATS' Division of Pre-Clinical Innovation (DPI). Researchers from Sanaria, which has offices in the same building as DPI in Rockville, Maryland, worked with and relied on the expertise of Inglese and Dranchak to develop a diagnostic assay, or test, for the vaccine. The assay was the key to demonstrating that the malaria vaccine had a positive effect in patient samples.
In August, NCATS Director Christopher P. Austin, M.D., kicked off a two-day conference designed to explore how community engaged research fits within the translational science research process. "Science of Community Engaged Research: Future Directions," which took place August 22–23 in Bethesda, Maryland, emphasized the importance of engaging the community — patients, family members, health care providers, clinical researchers and other stakeholders — in decision-making and participation in all phases of translational research. During his presentation, Dr. Austin outlined the many ways NCATS programs, including the Clinical and Translational Science Awards (CTSA) program, encourage patients and their advocates to participate as research partners.
"Dr. Austin's presence and comments made clear that community engagement is an integral part of translational science," said Syed Ahmed, M.D., M.P.H., Dr.P.H., conference planning committee member and senior associate dean for community engagement at the Medical College of Wisconsin. "It is essential to advancing research across all phases of the translational spectrum."
Keynote speaker Steven H. Woolf, M.D., M.P.H., who is the director of community engagement for the Virginia Commonwealth University (VCU) CTSA, cited a renewed interest among the research community in connecting primary care to public health. Dr. Woolf highlighted the VCU CTSA's "Engaging Richmond" project, in which local community members partner with VCU researchers to provide input on priorities and approaches for health outcomes and disparities research.
Other presentations at the conference — supported in part by NCATS and facilitated by the Duke University Translational Medicine Institute — focused on specific approaches to addressing community engaged research at every stage, including:
- Coordinating data collection and sharing with community partners.
- Partnering with communities to identify research priorities.
- Working with communities to translate data and research into practice.
- Partnering with communities to evaluate and sustain effective action.
- Disseminating and implementing effective practices.
"More than ever before, we are seeing that members of the community are eager to participate in research," said conference facilitator Lloyd Michener, M.D., chair of the Department of Community and Family Medicine at the Duke University School of Medicine. "We now have the opportunity to make a fundamental shift in how we do clinical research in the United States, ensuring that people, patients and communities are involved from start to finish."
NCATS held its inaugural "Research & Development Day" on September 12 in Cambridge, Massachusetts. Hosted by Novartis Institutes for BioMedical Research, the event provided a setting for NCATS researchers to showcase their therapeutic development projects to potential investment partners. Some of the therapeutic candidates included those for sickle cell disease, hereditary inclusion body myopathy, Duchenne muscular dystrophy and cryptococcal meningitis. As a result of this initial event, many connections were made and several are being actively pursued.
More than 100 participants from biopharmaceutical firms, venture capitalism, nonprofits and other organizations learned about collaboration opportunities for pre-clinical drug development projects supported by NCATS' Therapeutics for Rare and Neglected Diseases and Bridging Interventional Development Gaps programs. The goal of both programs is to advance and de-risk pre-clinical projects to the stage where they can attract third-party investors.
A key aspect of the NCATS mission is to play an adaptor role, bridging the translational gap between basic research discoveries and development of interventions that tangibly improve human health. To facilitate this process, NCATS relies on strategic partners to complete the final phases of clinical development by providing financial and technical support to bring potential novel therapeutics to patients.
Stephen Groft, Pharm.D., director of the Office of Rare Diseases Research at NCATS, was honored with a lifetime achievement award at the Global Genes Project's second annual RARE Tribute to Champions of Hope benefit on Sept. 21, 2013, in Newport Beach, California.
Groft received the Henri Termeer Lifetime Achievement Award for his 30-year career, which has been dedicated to advancing research and treatments for those with rare and genetic diseases. Among his many accomplishments, Groft helped establish the Office of Orphan Products Development at the Food and Drug Administration in 1982, and during 1987–1989, he served on the National Commission on Orphan Diseases, which published a report that led to the establishment of the Office of Rare Diseases Research at NIH — an office he still directs today at NCATS. Groft was instrumental in the creation of NIH's Genetic and Rare Diseases Information Center with the National Human Genome Research Institute (NHGRI) in 2002, and the Undiagnosed Diseases Program in 2008, a partnership with the NIH Clinical Center, NHGRI, and other NIH institutes and centers.
"Steve Groft has been a tireless advocate for the patients and families affected by rare diseases for more than three decades," said NCATS Director Christopher P. Austin, M.D. "His efforts have brought about a paradigm shift in both the science and treatment of these diseases and the kind of collaborative approaches that produce the most rapid progress."
Three other individuals received champion awards during the event:
- RARE Champion Award — Patient Driven Science: Honoring Josh Sommer, executive director of the Chordoma Foundation, which Sommer co-founded to speed the pace of research after he was diagnosed with skull-based chordoma in 2006. Chordoma is a rare type of cancerous tumor that can occur anywhere along the spine, from the base of the skull to the tailbone.
- RARE Champion Award — Advocacy: Honoring Rick Guidotti, founder of Positive Exposure, a company that uses photography and video to transform public perceptions of people living with genetic, physical and behavioral differences.
- RARE Champion Award — Advocacy: Honoring Dylan Siegel, a 6-year-old boy who wrote a book called Chocolate Bar to raise money for a cure for his best friend's rare liver condition, glycogen storage disease 1b.
The RARE Tribute to Champions of Hope benefit, which brings together stakeholders in the rare disease community, honors pioneers who advocate for the millions of patients and families affected by rare and genetic diseases.
Global Genes | RARE Project is a leading nonprofit organization advocating for the roughly 30 million Americans and approximately 300 million people worldwide who are affected by rare and genetic diseases.
Peanuts are one of the most common and deadly food allergies, affecting about three in every 500 people across the nation and 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. Scientists have discovered a new way of treating food allergy by placing a small amount of the allergen in liquid form under the tongue. Already widely used and approved in Europe to treat environmental allergies, this method, called sublingual immunotherapy, shows real potential for therapeutic development in the United States. But much work remains before that goal can be achieved, including testing the therapy in people.
A record of successful clinical trials makes innovative treatment methods like this one more attractive to pharmaceutical companies, which can develop the therapy into a product for delivery to patients. By sharing part of the risk involved in developing and testing new interventions, especially those to treat life-threatening conditions, NCATS aims to "de-risk" innovative approaches and methods, enabling more efficient translation of basic scientific knowledge into useful treatments.
In the case of peanut allergy, 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. Led by Hugh A. Sampson, M.D., director of the Institutes for Translational Sciences at Mount Sinai Hospital's Icahn School of Medicine in New York City, the consortium is working with CTSA program experts in multisite clinical trials to build evidence for sublingual immunotherapy as an effective and much needed treatment.
Read the full feature.
In the multidisciplinary research world, innovative scientific advances require effective collaborations. These are especially important when it comes to developing treatments for rare and neglected diseases. Private companies may not pursue new therapies for these diseases because of the low anticipated return on investment. One way to lessen the scientific and financial risk — to "de-risk" a project — is to share the work with partners.
NCATS' Therapeutics for Rare and Neglected Diseases (TRND) program is designed to do just that. TRND enables research partnerships between NCATS scientists and academic investigators, nonprofit organizations, and pharmaceutical and biotechnology companies. These therapeutic development partnerships reduce the risks, time delays and costs of advancing treatments for rare diseases into the first stages of clinical testing. Once projects are "de-risked" to this stage, companies are more willing to adopt them and invest the still-considerable resources needed to complete development and achieve regulatory approval.
TRND can help move rare and neglected disease therapeutics from the idea stage to the proof-of-principle stage. Visit the TRND webpage to learn more about the program or to collaborate with its scientists.
On September 16, NCATS Director Christopher P. Austin, M.D., led a joint meeting of the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board on the NIH campus in Bethesda, Maryland. NCATS Council and CAN Review Board members provide Austin with guidance and direction on the Center's initiatives, programs and policies.
In his director's report, Austin presented updates on the NCATS budget as well as Center leadership hiring statuses. He also reported on the progress of key NCATS programs and related work, including two NIH Common Fund initiatives. One is the Extracellular RNA (exRNA) Communication program, for which NCATS is administering 18 projects that will develop biomarkers from exRNA found in body fluids and explore new ways to use exRNA in treatments for a variety of diseases. "This program is ideal for NCATS because exRNA research could revolutionize our thinking in a number of translational areas, including diagnosis, biomarkers, therapeutics and understanding of disease," Austin said. NCATS also is part of the Common Fund's trans-NIH working group for the Illuminating the Druggable Genome program.
Read the full feature.
Cures Acceleration Network (CAN) Review Board Set to Meet December 12
On Dec. 12, 2013, from 11 a.m. to 2 p.m., the CAN Review Board will meet virtually to discuss how NCATS might exercise its flexible research authority in an optimal manner by using transactions other than grants, cooperative agreements and contracts. Information on how to participate in this Web meeting can be found on the Events page of the NCATS website. For more information about the advisory group, visit the CAN Review Board page.
NCATS researchers are seeking collaborators in the following areas:
Bridging Interventional Development Gaps (BrIDGs) Program
The BrIDGs program makes available, on a competitive basis, certain critical resources needed for the development of new therapeutic agents. The next opportunity to apply to the BrIDGs program tentatively is scheduled for January/February 2014. Visit the BrIDGs webpage for more information.
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 a high-throughput RNAi screen 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.
- Scripps Gets a Big Grant to Study DNA, Digital Devices and Healthcare, KPBS, October 22
- Duke, UNC Receive Hefty NIH Awards, The Herald-Sun, October 22
- University of Utah Awarded $20M for Research on Medical Breakthroughs, Associated Press via The Republic, October 11
- NIH Grant Gives UTHSC the Lead in Regional Health Research, Texas Public Radio, October 10
- 'Researcher Speed Dating' at Tufts Helps Lab Scientists, Doctors, Academics Forge Partnerships, Boston.com, October 10
- Personalized Cancer Vaccine Enters Human Trials, NIH Director's Blog, September 17
- Covance to Collaborate with Indiana University on Trials, PMLiVE, September 16
- Lilly Partners with NIH for Rare-Disease Research, Medical Marketing & Media, September 16
- Looking for Lessons in Cancer's "Miracle" Responders, Reuters, September 15
- NIH Scientists Pursue New Therapies to Improve Rare Disease Drug Development, NCATS, September 12
- NIH Partners with Eli Lilly, Others on Rare Diseases, FierceBiotechResearch, September 12
- Why Redheads Are More Susceptible to Melanoma, NIH Director's Blog, September 4
- NIH Effort Gambles on Mysterious Extracellular RNAs, Science Magazine, August 30