| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
|---|---|---|---|---|---|---|---|---|---|---|
| 3344 | ETB Scientific Capabilities | The ETB operates in small, focused teams composed of biologists, chemists and informatics scientists, each with specialized areas of expertise and access to shared scientific resources. For more than 10 years, the ETB’s drug discovery teams have offered collaborators unique access to the facilities and expertise needed to uncover new small molecule therapeutics and advance translational medicine. These capabilities include: Assay design and optimization/miniaturization. Dose-response–based high-throughput screening and support. Advanced biochemical, cellular and biophysical assay validation. Medicinal chemistry to support large-scale hit optimization driven by structure-activity relationships. Absorption, distribution, metabolism and excretion (ADME) and pharmacokinetics/pharmacodynamics testing to support preclinical studies. Cheminformatics and computational chemistry. Bioinformatics. Molecular modeling and structure-guided docking. | ||||||||
| 3337 | ETB in Action | ETB staff collaborate with more than 200 investigators at NIH and in the academic, biopharmaceutical and nonprofit sectors to generate probes for studying a diverse cross-section of human biology, focusing specifically on new targets and untreatable diseases. Read the latest news about these collaborations below. May 2020 High-Throughput Screening Helps Advance Melanoma Therapy Finding the right compounds for new uses in treating a disease or improving a therapeutic approach can be time consuming. To speed up the process, NCATS scientists used high-throughput screening to help advance a therapy for melanoma. February 2020 Reprogramming Immune Cells to Fight Tumors Tumors can co-opt the body’s immune defenses to escape detection and boost cancer growth. To overcome this, researchers found a way to reprogram immune cells to stop tumor growth and kill cancer cells. A Multipronged Approach Toward Treating DMD Experts in high-throughput screening and lysosomal biology collaborate on a multipronged approach to lessen the symptoms of Duchenne muscular dystrophy. September 2019 Following a Translational Science Path NCATS supports several training and career development programs, including a postbaccalaureate program that not only inspires students to become scientists but also helps grow the translational science workforce. Read how one student’s summer internship and fellowship experience at NCATS turned her onto a career path in translational science. March 2019 Brigham and Women’s Hospital and NCATS Scientists Dial Up an Approach Against Out-of-Control Inflammation Scientists at Brigham and Women’s Hospital collaborated with NCATS high-throughput screening experts to investigate a different approach against chronic inflammation. The team identified four compounds, including two novel synthetic molecules. This finding provides a potential opportunity to develop a new kind of drug to reduce inflammation. NCATS Spearheads a New Resource for Natural Products Canvass, a screening library for natural products, was created by NCATS in collaboration with academic and industry researchers to uncover the potential medical use of these compounds and enable the creation of new treatments for diseases. The researchers have already found some compounds with unexpected biological activities. December 2018 NCATS Researchers Develop New Approach to Identify Potential Cancer Drugs NCATS researchers overcame a translational roadblock by developing a series of assays (tests) to identify compounds that disrupt a driver of several cancers. The work, using NCATS’ high-throughput screening technology to test thousands of compounds at once, provides a template for other scientists to discover new compounds that could be useful as cancer therapies. July 2018 NCATS Ignites Translational Science Spark for Young Investigators Through NIH’s Graduate Partnerships Program, translational research scholar Dorian Cheff collaborates with scientists at ETB and the Karolinska Institutet in Sweden on an understudied family of enzymes. May 2018 NIH, Northwestern Scientists Develop Potential New Approach to Stop Cancer Metastasis NCATS scientists from ETB and the Bridging Interventional Development Gaps program collaborated with National Cancer Institute and Northwestern University researchers to identify a new compound that blocks the spread of several types of cancer in animal models. The new compound, called metarrestin, potentially could be effective as a therapy after cancer surgery. February 2018 NCATS, Karolinska Institutet Scientists Attack Cancer’s Defenses Description: Scientists from NCATS and Sweden’s Karolinska Institutet have developed a potential new approach to fighting cancer by breaking down a defense system used by cancer cells. January 2018 NCATS-Led Team Identifies Potential Strategy to Fight Huntington’s Disease A team of NCATS scientists and collaborators have uncovered a new potential strategy against Huntington’s disease, an inherited, fatal neurodegenerative disorder that has no cure. These findings may also be relevant to a broader range of neurodegenerative disorders. November 2017 Hibernating Ground Squirrels Provide Clues to New Stroke Treatments NCATS and NINDS scientists collaborate to find a molecule that may protect brain cells during a stroke. July 2017 NIH Scientists Find Rare Disease Clues in Cell's Recycling System NCATS scientists and their colleagues have demonstrated how an investigational drug works against a rare, fatal genetic disease, Niemann-Pick type C1 (NPC1). They found that a closely related compound will activate an enzyme, AMPK, triggering a cellular “housekeeping” system that helps reduce elevated cholesterol and other accumulated fats that in the brains and livers of NPC1 patients, which are hallmarks associated with severe neurological problems. June 2017 Researchers Aim to Repurpose Former Experimental Cancer Therapy to Treat Muscular Dystrophy Researchers at NCATS and the University of Nevada, Reno School of Medicine have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD). The candidate drug, SU9516, represents a different kind of approach for treating DMD, a degenerative muscle disease that usually begins in childhood and has no known cure. It is caused by a faulty gene that leads to progressive muscle weakness, with death often occurring around age 25. April 2017 International Scientific Teams Find Potential Approach Against Parasites International scientific teams find potential approach against parasites. Led by NCATS and University of Tokyo scientists, the teams sorted through more than 1 trillion small protein fragments to uncover two that could shut down the enzyme. The finding, reported April 3, 2017, in Nature Communications, could set the stage for the potential development of new types of antimicrobial drugs. May 2016 Ketamine Lifts Depression Via a Byproduct of its Metabolism NIH scientists and grantees have discovered that ketamine lifts depression via a byproduct of its metabolism in mouse study. September 2015 Data Released on Drug Combinations to Treat Malaria NCATS researchers and collaborators from the NIH’s National Institute of Allergy and Infectious Diseases, Georgetown University, and the University of California, San Francisco, have released a large dataset of potential drug combinations for malaria. Using NCATS’ state-of-the-art high-throughput combination drug-screening platform, the Center’s researchers tested 13,910 combinations of known and newly identified antimalarial drugs in three malaria parasite lines. June 2015 NCATS-FDA Team Receives HHS Innovation Ventures Support to Crowdsource Information on Treating Tropical Diseases NCATS-FDA team receives support from the HHS Innovation Ventures Fund — an HHS IDEA Lab program — to enhance the Web-based Collaborative Use Repurposing Engine (CURE). The highly competitive awards provide growth-stage funding and 15 months of mentoring as well as tools to help grow and sustain CURE, which supports crowdsourcing information on treating tropical diseases. May 2015 Stopping Metastasis in Its Tracks: New 3-D Cell Model Enables Closer Look at Cancer Progression ETB experts and University of Chicago researchers work together to create, adapt and validate an innovative 3-D ovarian cancer cell model used to identify small molecules that can prevent cancer cells from spreading to new sites in the body. The new model includes connective tissue cells and the extracellular matrix (a collection of supportive molecules outside cells), creating a more lifelike simulation of the human body environment. August 2014 Improved Disease Model Leads to Potential Therapy for Rare Disorder A team of researchers from ETB and the National Human Genome Research Institute make a major advance in the understanding and treatment of Gaucher disease, creating a new model that not only helps identify treatments for patients with this rare disorder, but also is useful in studying other diseases. June 2014 Spotlight on Collaboration: A Journey From Biological Probes to Potential Therapeutics Scientists in ETB work closely with academic, nonprofit and biotechnology researchers to pursue collaborative probe development projects, including the discovery of three novel small molecule chemical probes with the potential to treat diabetes, stroke and thrombosis. September 2012 Activating Key Cancer Enzyme Blocks Tumor Growth in Mice A team that includes nine ETB researchers identifies compounds that delay tumor formation in mice. The compounds target a specific form of pyruvate kinase, called PKM2, which governs how cancer cells use glucose. July 2012 Collaboration May Help Uncover Treatments for Rare Neurologic Disease Scientists from ETB and the University of Wisconsin–Madison help identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease, a rare neurological disorder. May 2012 NIH and Eli Lilly Publish Guide to Help Researchers Develop Therapeutic Screening Tests NCATS and Eli Lilly and Company jointly release an Assay Guidance Manual designed to provide step-by-step guidance through the complex process of turning a basic research finding into an assay (test) that will start the process of discovering pharmacological tools and drugs. | ||||||||
| 3338 | ETB Operational Model | The majority of ETB work involves collaborations with disease-focused laboratories across the globe. Through an active outreach program, the ETB is continually building new research partnerships. Collaborators work with ETB scientists to develop screens against promising drug targets and to refine these results into small molecule probes and potential therapies. Close communication between external investigators and ETB staff enables the terms of these strong collaborations to remain flexible and specific to each project. These partnerships provide unique resources to both parties and help disseminate high-quality small molecules and cutting-edge knowledge to the broader research community. Learn more about the ETB in action. | ||||||||
| 3339 | ETB Program Goals | The ETB’s goal is to uncover new small molecule therapeutics and advance the process of therapeutic development through a model of collaborative research. For each collaboration, external disease experts team with the ETB’s drug discovery teams, who provide access to small molecule screening, medicinal chemistry and informatics expertise. The Branch is proactive in publishing the results of all its drug discovery campaigns and disseminating small molecule leads to all members of the global health community, who can further explore their potential for development into small molecule therapies. The objectives of the ETB are: Collaborate with both NIH and extramural investigators to perform small molecule screening projects for disease targets (assay development, screening and validation) and help characterize the underlying biology. Pursue new technologies for target-based and phenotypic screening as well as for downstream validation. Provide characterized small molecule probes against identified targets to act as tools for future research and as starting points for drug development campaigns. Leverage ETB expertise in medicinal chemistry and informatics to guide hit-to-lead optimization and other synthetic chemistry efforts. | ||||||||
| 3345 | About ETB | Small molecule chemical compounds, which can be used to test or “probe” the effects of increasing or decreasing the activity of a biological target in cells or animals, are some of the most powerful tools for target validation, which is the process of demonstrating that engaging a target provides meaningful therapeutic benefit. Probes enable researchers to investigate protein and cell functions and biological processes. If appropriate, probes can be optimized to become potential drug candidates. Generating these chemical probes requires specialized expertise and facilities, and NCATS has built world-leading collaborative services to meet these needs. The Early Translation Branch (ETB), formerly known as the NCATS Chemical Genomics Center (NCGC), and originally the NIH Chemical Genomics Center, was created in 2008 as a comprehensive screening center in the Molecular Libraries Probe Production Centers Network, part of the NIH Common Fund’s Molecular Libraries Program. The original goals of the ETB were to translate the discoveries of the Human Genome Project into biology and disease insights and ultimately new therapeutics through small molecule assay development, high-throughput screening, informatics and chemistry. Learn more about the goals of the ETB. ETB staff collaborate with more than 200 investigators at NIH and in the academic, biopharmaceutical and nonprofit sectors to generate probes for studying a diverse cross-section of human biology, focusing specifically on new targets and untreatable diseases. NCATS’ probe development activities also include finding more efficient ways to make probes, using probes to understand diseases and validating targets to treat diseases. Learn more about the ETB operational model. | ||||||||
| 3328 | June 2015 Council/CAN Review Board Meeting | The June 18, 2015, joint meeting of the NCATS Advisory Council and the Cures Acceleration Network Review Board featured the following meeting materials. Presentations NCATS Director’s Report (PDF - 4MB) – Christopher P. Austin, M.D., director, NCATS Discovering New Therapeutic Uses for Existing Molecules Program (PDF - 240KB) – Christine M. Colvis, Ph.D., director, drug development partnership programs, NCATS Inhibition of Fyn Kinase for Disease-Modifying Therapy of Alzheimer’s Disease (PDF - 4MB) – Stephen M. Strittmatter, M.D., Ph.D., professor of neurology and neurobiology; director, cellular neuroscience, Yale University Subcommittee Report on Pharma/BioTech/Venture (PBV) Groups (PDF - 77KB) – Ankit A. Mahadevia, M.D., M.B.A., venture partner, Atlas Venture, and Freda C. Lewis-Hall, M.D., chief medical officer, Pfizer Concept Clearance: NCATS Exploratory Clinical Trials for Small Business (PDF - 111KB) – Lili M. Portilla, M.P.A., director, strategic alliances, NCATS Concept Clearance: Small Business Translational Science Innovation Award Program (PDF - 117KB) – Philip J. Brooks, Ph.D., program director, Division of Clinical Innovation, NCATS, and Lili M. Portilla, M.P.A., director, strategic alliances, NCATS Concept Clearance: Development of Stem Cell- or iPS Cell-Based Assays for Compound Toxicity Evaluation (PDF - 134KB) – Menghang Xia, Ph.D., leader, systems toxicology, Toxicology in the 21st Century, Division of Preclinical Innovation, NCATS; Lili M. Portilla, M.P.A., director, strategic alliances, NCATS; and Anna Rossoshek, M.S., M.B.A., branch and program manager, Chemical Genomics Branch, NCATS Chemical Genomics Center, Division of Preclinical Innovation, NCATS Concept Clearance: Development of Smart Plate Technology (PDF - 118KB) – Sam Michael, Ph.D., acting chief information officer and director, automation and compound management, NCATS; Lili M. Portilla, M.P.A., director, strategic alliances, NCATS; and Anna Rossoshek, M.S., M.B.A., branch and program manager, Chemical Genomics Branch, NCATS Chemical Genomics Center, Division of Preclinical Innovation, NCATS Concept Clearance: R&D Contract Support for NCATS Translational Sciences (PDF - 116KB) – Anton M. Simeonov, Ph.D., acting scientific director, NCATS Concept Clearance: Tissues-on-Chips, Part II (PDF - 222KB) – Danilo A. Tagle, Ph.D., M.S., associate director for special initiatives, NCATS Concept Clearance: Administrative Supplements for Collaborative Innovation in the CTSA Program (PDF - 125KB) – Philip J. Brooks, Ph.D., program director, Division of Clinical Innovation, NCATS The CTSA Program: Collaborative Innovation for Translational Research (PDF - 862KB) – Petra Kaufmann, M.D., M.Sc., director, Division of Clinical Innovation, NCATS Additional Meeting Information Agenda (PDF - 70KB) Videocast Minutes (PDF - 253KB) Federal Register Notice Concept Clearances | ||||||||
| 3309 | Lamirande to Lead NCATS Executive Office | NCATS Director Christopher P. Austin, M.D., has announced Keith R. Lamirande, M.B.A., as the Center’s new associate director for administration and executive officer. Lamirande will join NCATS on Aug. 9, 2015. “Translation is a multifaceted problem that requires team-based solutions at every level, necessitating innovation in both science and administration and seamless coordination between them,” Austin said. “Keith’s training as a scientist, together with his deep and broad accomplishments in administrative areas from budget and human resources to strategic planning and policy, make him a perfect fit for NCATS.” Lamirande’s career at NIH spans more than 25 years, starting in the lab and progressing through a number of administrative disciplines. He currently serves as the executive officer and deputy ethics counselor at NIH’s National Institute on Alcohol Abuse and Alcoholism (NIAAA), where he provides leadership and direction for a wide array of the Institute’s administrative management activities, including financial management, management analysis, general administration and information technology. Lamirande’s other roles during his 14-year tenure at NIAAA included serving as the deputy executive officer, budget officer, deputy budget officer and budget analyst. “I am incredibly grateful to have had the opportunity to serve at NIH for so many years, and I am looking forward to joining the NCATS leadership team,” Lamirande said. “The transformative mission of NCATS to accelerate the delivery of new treatments and cures for disease to patients is exciting and energizing, and I am thrilled to soon be a part of this exceptional and intrepid mission.” Lamirande began his NIH career in 1990 as a biologist with the Veterinary Resources Program of NIH’s former National Center for Research Resources, working in veterinary pathology. A 1993 graduate of the NIH Management Intern program, Lamirande has served on a number of high-profile NIH committees. He currently serves as the chair of the NIH Administrative Training Committee, with oversight of the Management Intern and Presidential Management Fellows programs. Lamirande has been recognized for his work with two NIH Director’s Awards, an NIH Office of the Director Honor Award and the NIAAA Martin K. Trusty Excellence in Management Award. Lamirande earned his master of science degree in business administration from Loyola College in Baltimore and his bachelor of science degree in biology from Bates College in Lewiston, Maine. Posted July 2015 | ||||||||
| 3290 | TIC Frequently Asked Questions (RFA-TR-15-002) | What is the purpose of the CTSA Network Trial Innovation Centers (RICs) funding opportunity announcement (FOA)? Could NCATS please confirm that the Award Budget is to be no greater than $2.4 million (direct costs) annually? The National Institute on Aging (NIA) is a Participating Organization on RFA-TR-15-002. What is the nature of their participation? May a TIC application propose to work with only a subset of CTSA Program hubs? May the TICs work with sites other than CTSA Program hubs? May the TICs provide additional support to CTSA Program hubs for local activities, for example, via subcontracts for participating as sites in CTSA Program data discovery? May several investigators or institutions collaborate to develop and implement central TIC functions? NEW: If we propose more than one Program Director/Principal Investigator (PD/PI), may the commitment of the additional PD(s)/PI(s) be less than three months? NEW: Does an applicant need to be associated with a CTSA Program hub to submit an application? NEW: Are subcontractor facilities and administrative costs excluded from the direct cost limit of $2.4 million per year? NEW: Does the Specific Aims section count toward the 30-page limit for the Research Plan? What is the purpose of the CTSA Network Trial Innovation Centers (TICs) funding opportunity announcement (FOA)? The TICs FOA is one of a suite of recent CTSA FOAs intended to build CTSA Program network capacity. Working in concert with each other, the Recruitment Innovation Centers and network liaisons at the CTSA Program hubs, the TICs will be lead centers of excellence in clinical trials and will facilitate the implementation of multi-site clinical studies by the CTSA Program network. Could NCATS please confirm that the Award Budget is to be no greater than $2.4 million (direct costs) annually? The award budget is no greater than $2.4 million in direct costs annually. The National Institute on Aging (NIA) is a Participating Organization on RFA-TR-15-002. What is the nature of their participation? NIA is co-funding the establishment of a TIC and intends to use this clinical research infrastructure for studies on diseases of aging, in particular Alzheimer’s disease and other aging-related dementias. See NOT-TR-15-016. May a TIC application propose to work with only a subset of CTSA Program hubs? No. The TICs will be expected to work with all CTSA Program hubs. TIC applications that propose to limit themselves to a subset of CTSA Program hubs will not be considered responsive to this FOA. May the TICs work with sites other than CTSA Program hubs? Yes. The TICs may also work with other sites, for example, when studies in rare diseases need additional sites or when additional sites are preferred to ensure adequate inclusion of under-represented populations. TICs may also interface with sites involved in other networks, such as PCORnet. May the TICs provide additional support to CTSA Program hubs for local activities, for example, via subcontracts for participating as sites in CTSA Program data discovery? No. May several investigators or institutions collaborate to develop and implement central TIC functions? Yes. For institutions and organizations proposing multiple program directors or principal investigators, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF424 (R&R) Application Guide (PDF - 3MB). Applicants considering this option are strongly advised to discuss their plan with the NCATS scientific/program contact prior to submitting their application. If we propose more than one Program Director/Principal Investigator (PD/PI), may the commitment of the additional PD(s)/PI(s) be less than three months? Multi-PD/PI applications are accepted. Each PD/PI is expected to commit at least three months’ effort to the grant regardless of whether salary is requested. Does an applicant need to be associated with a CTSA Program hub to submit an application? Eligibility is broadly inclusive (e.g., higher education, nonprofits, for-profits, government agencies, etc.). Association with a CTSA Program hub is not a requirement. However, familiarity with the CTSA Program and/or partnership with a CTSA Program hub would be advantageous. Are subcontractor facilities and administrative costs excluded from the direct cost limit of $2.4 million per year? Yes. Please refer to NOT-OD-05-004. Does the Specific Aims section count toward the 30-page limit for the Research Plan? No, according to PHS SF424 instructions (PDF – 3MB), the Specific Aims section is a separate, one-page document not included with the Research Plan. | ||||||||
| 3272 | News Brief: NCATS-FDA Team Receives HHS Innovation Ventures Support to Crowdsource Information on Treating Tropical Diseases | .caption-left-lg { /* width: 65%; */ width: 40%; /* width: 25%; */ margin: 2% 3% 2% 0; border: 1px solid #ddd; } Each year, 1 billion of the poorest people worldwide suffer from infectious tropical diseases, such as dengue fever, malaria and schistosomiasis. More than 500,000 of those people die. Clinicians often repurpose drugs developed for other uses to treat these diseases; however, clinical observations in the field often are not reported. This means the broader scientific and medical communities cannot use the information to treat patients on a wider scale or to develop better therapies.NCATS researchers and their partners at the Food and Drug Administration (FDA) recognized that the lack of a repository to store this knowledge — a place that could be easily accessed by clinicians around the world — was a critical gap in addressing unmet medical needs. The team collaborated with several public health and research organizations to develop the Web-based Collaborative Use Repurposing Engine (CURE). The platform enables the crowdsourcing of medical information from health care providers to guide potentially life-saving interventions and may help advance the development of new drugs for neglected tropical diseases.Parvesh Paul, M.D., and Arnav Bhagwati, a patient at Jibhi Clinic of Lady Willingdon Hospital, Manali, Himachal Pradesh, India. (FDA Photo/Heather Stone)The NCATS-FDA team recently received support from the U.S. Department of Health and Human Services (HHS) Innovation Ventures Fund — an HHS IDEA Lab program — to enhance CURE. The highly competitive awards provide growth-stage funding and 15 months of mentoring, as well as tools to help grow and sustain CURE.CURE will enable clinicians around the world to share their experiences using existing medical products in new ways. Doctors can use a simple case report form to contribute their knowledge of challenging cases and access a network of experts who can provide guidance about potentially life-saving therapies. Using these data, researchers aim to identify promising products that could be developed formally to treat new diseases. CURE also could enable the rapid identification of available treatments and communication of health outcomes during disease outbreaks.CURE collaborators include the FDA, World Health Organization, Drugs for Neglected Diseases Initiative and Johns Hopkins Center for Clinical Global Health Education. In addition to the HHS Ventures Fund, the FDA’s Medical Countermeasures Initiative and its Center for Drug Evaluation and Research provide grant funding for CURE. Posted June 2015 | ||||||||
| 3252 | Partnering with Disease Foundations to Speed Drug Discovery | When scientists who specialize in drug development have a promising idea for a new disease treatment, they often start by designing biological tests called assays. By using high-throughput (robotically assisted) screening, researchers use the assays to evaluate hundreds of thousands of compounds with the potential to become new treatments. This complex process requires teamwork to involve the right types of expertise and perspectives in the research project team. Designing high-throughput screening assays is a science in itself. The team must have in-depth familiarity not only with assay technology but also with the target disease and its unique challenges. When the disease is rare, limited information can present additional challenges. As director of NCATS' Assay Development and Screening Technology Laboratory, Jim Inglese, Ph.D., leads a team of experts who take on these challenges every day. To increase the likelihood of success, Inglese encourages postdoctoral researchers who are knowledgeable about specific diseases to join project teams through fellowships sponsored by patient groups and foundations. These fellows bring strong disease expertise to NCATS, where Inglese mentors them in broad translational capabilities including assay development and early drug discovery. The overall goal is to develop new technologies and methods to build better disease models that can help advance the search for potential treatments. Several of NCATS’ Assay Development and Screening Technology Laboratory postdoctoral fellows (from left to right): Brittany Wright, Adam Fogel, Melissa Mendez and Michael Iannotti. "The fellows gain experience analyzing large-scale data sets obtained with their assays," Inglese explained. "As biologists, they learn to identify meaningful pharmacological relationships between chemical structures of the compound libraries and the concentration-dependent activity displayed by these substances. Usually, you can only get this kind of training at a pharmaceutical company." The fellowships exemplify NCATS' mission to improve methods and tools to speed the translation of laboratory discoveries into new treatments for patients. They also enable Center researchers to work directly with patient and disease groups as equal partners, each side bringing crucial skills and expertise to the collaboration. Not Your Average Fellowship Inglese has mentored five fellows: Adam Fogel, Ph.D., Michael J. Fox Foundation for Parkinson's Research Michael Iannotti, Ph.D., Alpha-1 Foundation Sung-Wook Jang, Ph.D., Charcot-Marie-Tooth (CMT) Association Melissa Mendez, Ph.D., Hannah's Hope Fund Brittany Wright, Ph.D., CMT Association Training under Inglese prepares the fellows to pursue careers in academia, biotechnology and pharmaceuticals, or the nonprofit sector, thus disseminating the expertise that they gain while at NCATS far beyond the specific research projects they conducted. Fellows have contributed to a number of important research findings as well. For example, Jang was part of a team that identified a potential drug target for a subtype of CMT, an inherited neurological disorder, in 2012. After completing his fellowship, Jang became director of the early discovery initiative at the Cure Huntington’s Disease Initiative. For another project, Inglese, Fogel and colleagues employed a technique developed in Inglese's lab called bioluminescent reporter coincidence detection to develop an assay that can sense subtle increases in the expression of a gene called PARK2. This assay is especially relevant for Parkinson’s disease drug discovery because compounds that change PARK2 expression may help slow or stabilize the disease. "Being able to fund a full-time postdoctoral fellow at NCATS working specifically on our disease is amazing," said Lori Sames, founder of Hannah's Hope Fund, a nonprofit dedicated to research on treatments for giant axonal neuropathy (GAN), a progressive neurological condition. Sames' daughter has GAN, and the organization began supporting a fellowship for Mendez to research potential new treatments at NCATS in 2014. "The expertise, bioinformatics and robotics at NCATS are unparalleled," she added. "To have Dr. Inglese's oversight, with his wealth of experience, is very valuable." Mendez has expertise in neurofilaments and their abnormal buildup in GAN. Neurofilaments, which reside in neurons and run the length of axons, help brain cells talk to each other. Her experience made her a good fit for GAN assay development; working with Sames and meeting GAN patients and families is "a big motivator" for her. "Everything at NCATS has been so collaborative," she said. Looking Beyond the Target Although the assays developed through these fellowships target specific diseases, each assay is based on broadly applicable technology and ultimately may be used to search for potential treatments for other conditions. "There is a drive here, not simply to answer the immediate question, but to recognize that the proximal question is just a piece in a very big puzzle," Mendez explained. In most laboratories, Inglese noted, "You can either focus on the technology or focus on the disease. But at NCATS, we do both. There's a strong emphasis on developing technologies and methodologies that are broad enough to cross diseases and reach more patients." These collaborations provide unique opportunities for the disease foundations, fellows and Inglese himself. "It's gratifying when patients come to the lab and see us working on projects that can benefit them," Inglese explained. "That interaction is something I never expected out of a career in science." Posted June 2015 |
Last updated on