8083 |
Funding & Notices |
NCATS Funding & Collaboration Opportunities
NCATS provides support for research projects, core facilities, scientific resources and tools, scientific conferences, and collaboration opportunities for small businesses and other partners as well as grants programs and in-kind services.
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Funding & Notices |
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8082 |
News & Media |
NCATS has a wide variety of resources for the news media and general public. Follow the links below to learn about the latest news, research and media coverage involving NCATS and its supported investigators.
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NCATS has a wide variety of resources for the news media and general public. Follow the links below to learn about the latest news, research and media coverage involving NCATS and its supported invest |
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News & Media |
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8080 |
NCATS Hosts Early Career NIH Scientists for Translational Science Training Program |
The NIH Translational Science Training Program (TSTP) provides NIH graduate students and postdoctoral scholars with opportunities to learn about the scientific and operational principles of the full spectrum of translation. On March 31 and April 1, 2016, trainees participated in a “boot camp” on the NIH campus in Bethesda, Maryland, that focused on topics from early stage drug discovery to marketing of an approved drug. NCATS speakers included Christopher P. Austin, M.D., director; Sitta Sittampalam, Ph.D., senior advisor; Madhu Lal-Nag, Ph.D., research scientist; and Todd Wilson, D.O., medical officer.
In addition, on April 27, 2016, NCATS hosted a TSTP Preclinical Development Workshop at its intramural laboratories for participants representing 11 NIH Institutes and Centers. NCATS staff led the workshop, with a focus on the early development stages of translation, and challenged participants to create a plan to translate their own basic research. Topics included assay design, medicinal chemistry and the experiments required to launch a human clinical trial. Participants also toured the NCATS high-throughput drug screening facility and received constructive feedback on their translational research plans in small group discussions with NCATS drug development scientists.
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8078 |
FDA-NIH BEST Biomarker Glossary Now Available |
Precise, clear communication across all scientific research disciplines is crucial for more efficient translational of laboratory discoveries into new treatments for patients. To this end, NIH and the Food and Drug Administration (FDA) recently published an open access textbook: the Biomarkers, EndpointS and other Tools (BEST) Resource. NCATS’ Pamela McInnes, D.D.S., M.Sc. (Dent.), Holli Hamilton, M.D., and Monica Shah, M.D., M.H.S., M.S.J., as well as the National Cancer Institute’s Lisa McShane, Ph.D., represented NIH on the FDA-NIH Joint Leadership Council whose members collectively identified the need for and then developed this new resource.
The first phase of BEST comprises a glossary that clarifies important definitions, captures the distinction between biomarkers and clinical assessments, and describes some of the hierarchical relationships, connections, and dependencies among the terms. NIH and FDA plan to use the BEST Resource glossary when communicating about biomarkers to help ensure a consistent use of terms and a common understanding of issues.
For more information, see NIH’s Under the Poliscope, JAMA and FDA Voice.
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FDA-NIH BEST Biomarker Glossary Now Available |
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8077 |
NCATS Seeks New Drug Development Partners and Pharmaceutical Assets |
NCATS is seeking new drug development partners and pharmaceutical assets to expand its Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. Through this program, NCATS matches researchers with a selection of pharmaceutical industry assets to test ideas for therapeutic uses.
NCATS coordinates the solicitation of ideas from the academic community, conducts NIH peer review of the ideas and funds the Phase II clinical trial of meritorious projects. NCATS’ drug development partners provide their drug or biologic to funded investigators who will explore the compounds to test for potential new uses. The partners also provide documentation so that funded investigators can file or cross-reference an Investigational New Drug application with the Food and Drug Administration.
Potential partners, including companies with at least three assets available that have been in Phase 1 clinical trials, should email NCATS at newtherapeuticuses@mail.nih.gov by June 9, 2016.
Note that while NCATS is not soliciting New Therapeutic Uses applications at this time, more information will be provided late this year.
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NCATS Seeks New Drug Development Partners and Pharmaceutical Assets |
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8076 |
Translational Science Representatives Meet to Advance Field |
NCATS Director Christopher Austin, M.D., and Taylor Gilliland, Ph.D., an NCATS health scientist, recently joined other translational science experts in Vancouver, Canada, to discuss global efforts in education and training, reproducibility and attrition, cross-sector collaboration, and data sharing. Other organizations represented included the Canadian Centre for Drug Research and Development, European Infrastructure for Translational Medicine, the Czech Institute of Molecular and Translational Medicine, and Therapeutic Innovation Australia.
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Translational Science Representatives Meet to Advance Field |
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8072 |
New Chapters, Updates to the Assay Guidance Manual |
In July 2016, authors of the Assay Guidance Manual (AGM) ― a free, best-practices online resource devoted to the successful development of robust, early-stage drug discovery assays ― added two new chapters and revised two others. Updated quarterly, the AGM provides step-by-step guidance for high-throughput screening, lead optimization and early phases of regulated drug development projects.
The new chapters are:
Cellular Thermal Shift Assay (CETSA), in the In Vitro Cell-Based Assays section. The CETSA methodology provides a powerful and unprecedented approach to validate target engagement within the context of a living cell. This information provides guidance for adapting CETSA to a microplate format to support high-throughput screening and structure-activity relationship studies.
Interferences with Luciferase Reporter Enzymes, in the Assay Artifacts and Interferences section, focuses on artifacts associated with luciferase reporters, which are heavily used in a variety of primary and counter assays for drug discovery. The goal is to provide researchers with help developing and implementing the best possible primary and counter assays for high-throughput screens.
In addition, two chapters have been revised: Early Drug Discovery and Development Guidelines: For Academic Researchers, Collaborators, and Start-up Companies and Cell Viability Assays. The next update is scheduled for October 2016.
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The Assay Guidance Manual is a free, best-practices online resource devoted to the successful development of robust, early-stage drug discovery assays. |
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New Chapters, Updates to the Assay Guidance Manual |
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8071 |
Pfizer’s Centers for Therapeutic Innovation Proposal Portal Opens September 6 |
On Sept. 6, 2016, Pfizer’s Centers for Therapeutic Innovation (CTI) staff will begin accepting research proposals from NIH intramural scientists. Led by NCATS, the Pfizer CTI program at NIH is designed to help bridge the gap between early scientific discovery and its translation into new medicines through public-private resource sharing. The program pairs leading NIH intramural researchers with Pfizer resources to pursue scientific and medical advances through joint therapeutic development. Goals include identifying biologic compounds with activity in a pathway or target of interest to both NIH and Pfizer and then together moving the compounds into the clinic to test them.
The deadline for submitting applications to the relevant NIH Institute or Center technology transfer offices is Oct. 7, 2016. Visit the CTI Call for Proposals page for details about how to apply, key deadlines, therapeutic areas of interest and criteria for success. To set a meeting that can include non-confidential discussion about pre-proposal ideas, contact Pfizer CTI Representative Nader Halim, Ph.D., at nader.halim@pfizer.com, and copy NIH-PfizerCTI@mail.nih.gov.
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On Sept. 6, 2016, Pfizer’s Centers for Therapeutic Innovation (CTI) staff will begin accepting research proposals from NIH intramural scientists |
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Pfizer’s CTI Proposal Portal Opens September 6 |
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8070 |
NCATS Funds Feasibility Assessment to Develop Biomedical Data Translator |
Scientific experts from 11 institutions are collaborating to assess the feasibility of developing NCATS’ Biomedical Data Translator (Translator). The goal is to identify and design innovative tools to integrate and leverage the vast amounts of medical research data currently available — all of which could bring about rapid, high-impact change that could revolutionize translational research and the translational science process.
NCATS is funding Translator through its Cures Acceleration Network (CAN), which includes Other Transaction (OT) authority. With CAN, NCATS has more funding flexibility, especially using OT authority, when grants, contracts and cooperative agreements may not adequately meet scientific need. NCATS staff will work closely with the newly awarded experts to expand and modify — and if needed, discontinue — activities based on program needs.
During the feasibility assessment, the research team will evaluate the translational gap between the scientific molecular and cellular biology and the clinical signs and symptoms produced in diseases. More specifically, they will identify data integration and inclusion barriers and develop and test a plan for data quality control and updates. They also will explore the development of models used in artificial intelligence research to gain new insights into biology, health and disease.
To pressure test various aspects of feasibility, cross-cutting research questions that have been difficult to address by other means will be addressed in demonstration projects to evaluate the functionality of the newly integrated data. The type of functionality explored includes understanding which drugs have the potential to interact in unexpected ways; examining the impact of environmental exposures on the onset or worsening of disease; evaluating the ability to help patients for whom existing approaches have failed to identify the origin of their symptoms; and better understanding relationships between rare and common diseases.
The longer-term goal is to create a ground-breaking, publicly available resource that brings together all biomedical and health data types using an informatics platform approach. Ultimately, researchers will have access to a powerful system that enables discovery of complex relationships among data, which will help scientists better understand disease and develop new treatments for patients.
Learn more about Translator or contact the Translator team.
Posted October 2016
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Scientific experts from 11 institutions are collaborating to assess the feasibility of developing NCATS’ Biomedical Data Translator. |
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NCATS Funds Feasibility Assessment for Biomedical Data Translator |
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8069 |
Rare Disease Patient Profiles |
The video clips below feature individuals who participated in the 2016 Rare Disease Day at NIH event and shared their stories of living with a rare disease. Hear rare disease patients and caregivers talk about the challenges they face and how NIH’s collaborative research efforts offer hope.
Description
Preview
Jillian Clegg shares the story of her two sons, who have MED23 gene defect
Jillian Clegg has two sons with MED23 gene defect, a condition that causes intellectual disability and affects the neuromotor system.
Runtime: 3:31
Date: February 2016
Right-click to download this video (MP4 - 223MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/nfpRgIdSlDA">https://www…;
Rachel Daniels describes what life is like for her daughter with congenital generalized lipodystrophy
Rachel Daniels’ daughter Alani has congenital generalized lipodystrophy. Alani’s body is unable to store fat under her skin, and any fat she does take in can connect to organs such as the heart and liver, causing health complications.
Runtime: 3:29
Date: February 2016
Right-click to download this video (MP4 - 220MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/dPb-6csezzE">https://www…;
Marcela Ferrada shares her story of living with relapsing polychondritis
Marcela Ferrada was diagnosed with an autoimmune disease called relapsing polychondritis, a rare degenerative disease characterized by recurrent inflammation of the cartilage in the body. Deterioration of the cartilage may affect any part of the body where cartilage is present. The ears, larynx and trachea may become “floppy,” and the bridge of the nose can collapse into a "saddlenose" shape. The aortic heart valve may also be affected.
Runtime: 2:35
Date: February 2016
Right-click to download this video (MP4 - 164MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/YMReF4QxpOc">https://www…;
Michael and Rachel Harris discuss their son’s life with Vici syndrome
Michael and Rachel Harris’s son has Vici syndrome, a multisystem congenital disorder characterized by agenesis (failure to develop) of the part of the brain called the corpus callosum. Symptoms can include cataracts, hypopigmentation of the eyes and hair, cardiomyopathy and combined immunodeficiency.
Runtime: 2:10
Date: February 2016
Right-click to download this video (MP4 - 137MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/PkNq5ay7YBk">https://www…;
Jessica McQuillen discusses her life with hereditary hemorrhagic telangiectasia (HHT)
Jessica McQuillen has a condition called HHT that causes massive nosebleeds and can cause bleeding elsewhere in the body.
Runtime: 3:07
Date: February 2016
Right-click to download this video (MP4 - 198MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/fhQ6uB4NHao">https://www…;
Joe Murray shares his daughter’s experience with epidermolysis bullosa (EB)
Joe Murray’s daughter is affected by a connective tissue disorder called EB that causes the skin to be very fragile. The slightest friction can cause open blisters, wounds and chronic pain.
Runtime: 3:43
Date: February 2016
Right-click to download this video (MP4 - 236MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/sd-dksx5fgY">https://www…;
Megan O’Boyle tells the story of her daughter, who has Phelan-McDermid syndrome
Megan O’Boyle’s daughter Shannon O’Boyle suffers from Phelan-McDermid syndrome, a rare condition that can cause autism, epilepsy and intellectual disabilities.
Runtime: 2:45
Date: February 2016
Right-click to download this video (MP4 - 174MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/VOMSq1D24Fc">https://www…;
Jennifer Payne discusses living with phenylketonuria (PKU)
Jennifer Payne discusses living with PKU, a liver enzyme deficiency caused by the inability to metabolize an essential amino acid called phenylalanine. Left untreated, PKU results in psychological disorders, neurological deterioration, mental illness and brain damage.
Runtime: 2:48
Date: February 2016
Right-click to download this video (MP4 - 177MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/btSQQYcxnjk">https://www…;
Stephanie Perron shares the story of her son’s life with a mitochondrial disease
Stephanie Perron’s son, Teddy, has been diagnosed with MELAS (mitochondrial disease complex 3 with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes) syndrome that affects many of the body’s systems, particularly the nervous system and muscles.
Runtime: 1:45
Date: February 2016
Right-click to download this video (MP4 - 110MB)
Video courtesy of FasterCures.
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/C7CM7PuDd4s">https://www…;
Suzanne Ripley discusses what life is like for her two sons, who have glutathione synthetase disorder
Suzanne Ripley has two sons who suffer from an ultra-rare condition, glutathione synthetase disorder, which is a deficiency of the enzyme glutathione synthetase. The severe form of the disease can affect infants, causing progressive neurological symptoms, such as impairment in the acquisition of skills requiring the coordination of muscular and mental activities (psychomotor retardation), varying degrees of intellectual disability, seizures and diminished coordination of voluntary movements.
Runtime: 3:45
Date: February 2016
Right-click to download this video (MP4 - 239MB)
YouTube embed video: <a href="https://www.youtube-nocookie.com/embed/e6wqR-NJWmE">https://www…;
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Rare Disease Patient Profiles |
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