| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
|---|---|---|---|---|---|---|---|---|---|---|
| 7111 | Addressing Health Disparities | The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care. Read the latest in CTSA Program news about underserved populations in translational research across the lifespan. January 2020 November 2019 January 2019 June 2018 June 2017 January 2017 August 2016 March 2016 October 2015 July 2013 December 2012 March 2012 January 2020 Harnessing the CTSA Program to Advance Telehealth CTSA Program-supported researchers are harnessing their resources and expertise to evaluate access to high-quality telehealth care for rural and underserved children. November 2019 The Road to Improving Rural Health Outcomes One out of every five Americans lives in a rural area, and many have difficulties accessing health care. To improve rural health outcomes, our CTSA Program is working to address health disparities unique to these areas and promote health equity. January 2019 NCATS-Supported Researchers Find Exercise May Help Protect DNA CTSA Program-supported researchers who studied older caregivers found that those who exercised had longer telomeres (the caps that protect the ends of DNA). These findings may lead to better health outcomes for older adults as they age. June 2018 CTSA Program Researchers Advance Heart Condition Study Through Precision Medicine and Digital Health Researchers at the Scripps Translational Science Institute in San Diego, a CTSA Program hub, are working to address the challenge of diagnosing atrial fibrillation, a common heart condition with complex causes. The team’s efforts can ultimately help advance precision medicine approaches to preventing and diagnosing other types of complex diseases. June 2017 CTSA Program Investigators Collaborate to Enhance Newborn Screening Efforts are now underway to add rare diseases to newborn screenings through Early Check, a research program in which voluntary screening for fragile X syndrome and other genetic conditions yet to be determined will be offered to up to 120,000 families each year in North Carolina. NCATS is supporting Early Check through a CTSA Program Collaborative Innovation Award (CCIA). January 2017 Unprecedented Trans-NCATS Collaboration Enables Rapid Advancement of Rare Lung Disease Therapy to Human Trials NCATS helped shepherd a potential new drug for a deadly lung disease, called autoimmune pulmonary alveolar proteinosis, through many steps of the translational science process. Center support provided a bridge in moving the promising therapeutic from preclinical to clinical research. August 2016 CTSA Program-Supported Research Uncovers Genetic Components of Healthy Aging Older adults often face aging-related ailments that can be costly and shorten lifespans. However, some people live long lives without encountering these common health problems. What sets apart healthy agers from their peers? Researchers at Scripps Translational Science Institute are conducting a study of the “Wellderly” to find out. March 2016 CTSA Program Supports Research Network in Geographically Isolated Region With support from NCATS’ CTSA Program, the University of New Mexico Clinical and Translational Science Center was a founding partner in the Mountain West Research Consortium (MWRC), currently a network of 11 universities spanning seven states. The MWRC’s goal is to build and enhance the geographically isolated region’s clinical and translational research capacity. October 2015 CTSA Program Domain Task Forces: Spotlight on Integration Across the Lifespan The CTSA Program Integration Across the Lifespan Domain Task Force goals are to integrate translational science across the entire lifespan to achieve health improvements, launch efforts to study special population differences in the progress and treatment of disease processes, and develop a seamless, integrated approach to translational science across all phases of research. July 2013 CTSA Program Resources Support Largest U.S. Newborn Screening Study for Fragile X Mutations A team of researchers at the University of California, Davis, found that more people have gene changes linked to Fragile X syndrome than anticipated. Accurate and timely diagnosis by such newborn screening may enable early intervention. December 2012 Patients with Rare Muscle Disorder Benefit from Repurposed Heart Drug Patients with nondystrophic myotonia, a rare genetic muscle disorder, often have problems with debilitating muscle stiffness, fatigue, and sometimes even episodes of paralysis. Now, these patients have new help: a drug called mexiletine, normally used to treat heart disorders. With assistance from NIH’s Rare Diseases Clinical Research Network and the CTSA Program, researchers at seven institutions in four countries collaborated to demonstrate in a clinical trial that the heart drug is effective in treating patients with this rare disease. March 2012 New Drug for Rare Type of Cystic Fibrosis Patients with a rare type of the deadly lung disorder cystic fibrosis may be able to breathe easier thanks to a new drug that targets the defective protein causing the disease. Researchers from 13 universities and hospitals, including 10 CTSA Program institutions, partnered with the Cystic Fibrosis Foundation and the drug manufacturer Vertex Pharmaceuticals to conduct clinical trials and obtain Food and Drug Administration approval for the drug Kalydeco as a new treatment. | Promote the Integration of Special and Underserved Populations in Translational Research Across the Human Lifespan | /sites/default/files/ctsa-goal3.jpg | CTSA Program Goal 3 | Promote the Integration of Special and Underserved Populations in Translational Research Across the Human Lifespan | /sites/default/files/ctsa-goal3.jpg | CTSA Program Goal 3 | ||
| 7109 | Promoting Impactful Partnerships and Collaborations | The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care. Read the latest in CTSA Program community engagement news. | Engage Patients and Communities in Every Phase of the Translational Process | /sites/default/files/ctsa-goal2.jpg | CTSA Program Goal 2 | Engage Patients and Communities in Every Phase of the Translational Process | /sites/default/files/ctsa-goal2.jpg | CTSA Program Goal 2 | ||
| 7108 | Promoting Training and Career Support | The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care. Read the latest in CTSA Program training news. June 2020 June 2020 Teaching Children About Translational Science and Clinical Trials CTSA Program researchers from the University at Buffalo created “Sofia Learns About Research,” a coloring and activity book that introduces children and their guardians to translational science and teaches them about the importance of clinical trials. | Train and Cultivate the Translational Science Workforce | /sites/default/files/ctsa-goal1.jpg | CTSA Program Goal 1 | Train and Cultivate the Translational Science Workforce | /sites/default/files/ctsa-goal1.jpg | CTSA Program Goal 1 | ||
| 7093 | Frequently Asked Questions About RFA-TR-17-002 and RFA-TR-17-003 | Application Information Should there be an introduction page with a response to the written feedback from the X02? Having a small amount of the asset would enable investigators to quickly generate preliminary data for a UG3/UH3 application prior to the receipt date. Can investigators execute an agreement now to facilitate transfer of materials for preliminary data generation? Should applicants include a support letter from the pharmaceutical partner? The time frame for grants may not be optimal for certain disease conditions that are age-dependent and chronically progressive (such as neurodegenerative disorders), in which case both the preclinical and clinical studies (e.g., Phase I clinical trial) could take longer. What flexibility do applicants have to propose more time for the application? Should steering committee members be named in the application? Can the formulation of an asset be changed? Pediatric Indications What types of studies are acceptable for pediatric indications? Are there any limitations on the use of assets in pediatric populations? Confidential Disclosure and Collaborative Research Agreements Will NIH accept a UG3/UH3 application if a confidential disclosure agreement (CDA) and collaborative research agreement (CRA) have not been executed? NCATS indicates that the collaborative research agreement (CRA) must be in place with the pharmaceutical partner for the full UG3/UH3 application to be accepted. If more than one institution is involved, do all institutions need to have a CRA in place with the pharmaceutical partner? Would a CRA with one institution and/or lead investigator be permitted if there are multiple principal investigators or co-investigators from different institutions? Application Review What are the submission requirements for UG3/UH3 application acceptance? Budget/Funding Will the asset be provided by the pharmaceutical partner free of charge, or does this need to be negotiated and accounted for during budgeting? How will the UG3/UH3 grant applications be selected for funding? Application Information Should there be an introduction page with a response to the written feedback from the X02? No. The full application is not considered a resubmission; therefore, an introduction should not be included. Having a small amount of the asset would enable investigators to quickly generate preliminary data for a UG3/UH3 application prior to the receipt date. Can investigators execute an agreement now to facilitate transfer of materials for preliminary data generation? No. Pharmaceutical partners should not be asked to provide an asset before the application receipt date. To be fair to all applicants, no application is expected to contain preliminary data on the asset with the proposed new use. Should applicants include a support letter from the pharmaceutical partner? Yes. A letter indicating that applicants will have access to the asset (if the application is funded) is required. Applications that do not include such a letter will not be accepted for review. The time frame for grants may not be optimal for certain disease conditions that are age-dependent and chronically progressive (such as neurodegenerative disorders), in which case both the preclinical and clinical studies (e.g., Phase I clinical trial) could take longer. What flexibility do applicants have to propose more time for the application? As with any funding opportunity, the solicitation will not be optimal for all projects. For adult indications, the UG3 must be completed within 12 months, and the UH3 must be completed within 36 months. For pediatric indications, the UG3 must be completed within 24 months, and the UH3 must be completed within 36 months. The clinical outcomes measured in the application must conform to these timelines. Should steering committee members be named in the application? No. The committee will not be identified until after an award is made. Applicants should NOT name or contact potential steering committee members; only describe their role, experience and function. Can the formulation of an asset be changed? No. Only the formulations listed for specific assets provided by the pharmaceutical companies are available. The only exception is for pediatric indications, which may need to be formulated as a solution/suspension for oral administration (ages 6 to 11) or a small tablet/capsule (ages 12 to 18). Palatability issues also may have to be addressed for pediatric administration. Pediatric Indications What types of studies are acceptable for pediatric indications? Applicants should refer to the Table of Assets for Pediatric Indications to determine if the asset will be considered by the pharmaceutical company for pediatric use. After clicking on the asset of choice in the first column of the table, refer to the "Additional Characteristics" row of the more detailed asset information chart. This row provides information on the types of pediatric indications that the pharmaceutical company will consider. Applicants must provide NIH with documentation of access to the asset and associated data needed for conducting the proposed preclinical studies and pediatric clinical trials and for filing an investigator-sponsored Investigational New Drug application to conduct the proposed clinical trials in a UG3/UH3 application (e.g., letter from the pharmaceutical company providing access to the asset for the indicated use). Are there any limitations on the use of assets in pediatric populations? Yes. In general, pediatric populations to be considered for this funding opportunity announcement (FOA) refer to disease populations for which there is no adult equivalent and thus no adult population in which the drug could be tested prior to testing it in children. However, trials in pediatric or juvenile populations for indications that also have an adult population (e.g., type 2 diabetes, autism, osteoarthritis) may be considered if there is a strong scientific rationale that justifies why Phase II trials in the pediatric population are required even though an adult patient population exists (e.g., the target in the pediatric population may differ from that in the adult, or treatment of children may reduce progression or severity of the disease). The assets that pharmaceutical companies will consider for use in pediatric populations are listed in the Table of Assets for Pediatric Indications. Applicants must click on the asset code number in the first column of the table to obtain more detailed asset information. To determine the type(s) of pediatric diseases the pharmaceutical company will consider (e.g., only trials in pediatric populations for which there is no adult population; trials for diseases/conditions that have a pediatric and adult population, if the trials in a pediatric population are scientifically justified), open the asset of interest and refer to the “Additional Characteristics” row. Applicants exploring therapies for diseases that occur in children and adults should consider applying in response to one of the companion UG3/UH3 FOA focusing on adult populations. Confidential Disclosure and Collaborative Research Agreements Will NIH accept a UG3/UH3 application if a confidential disclosure agreement (CDA) and collaborative research agreement (CRA) have not been executed? No. UG3/UH3 applications submitted without evidence of access to and the ability to work with the assets, such as evidence that a CRA or equivalent document has been executed, will be deemed incomplete and returned to the applicant without review. NCATS indicates that the collaborative research agreement (CRA) must be in place with the pharmaceutical partner for the full UG3/UH3 application to be accepted. If more than one institution is involved, do all institutions need to have a CRA in place with the pharmaceutical partner? The pharmaceutical partners’ requirements vary in terms of institutions with which they will execute a CRA when multiple institutions are involved. Would a CRA with one institution and/or lead investigator be permitted if there are multiple principal investigators or co-investigators from different institutions? Ultimately, the agreements have to provide the investigative team with access to the asset and associated data. Documentation of that access must be provided as part of the UG3/UH3 application. Application Review What are the submission requirements for UG3/UH3 application acceptance? An application must meet the following criteria to be accepted for review: Use only one asset. Use the same asset or mechanism of action as described in the X02 pre-application. Be the same therapeutic use as proposed in the X02 pre-application. Provide a letter of support from the pharmaceutical company partner documenting access to the asset and associated data needed for conducting the proposed preclinical studies and for filing an Investigational New Drug application for conducting the proposed clinical trials. Budget/Funding Will the asset be provided by the pharmaceutical partner free of charge, or does this need to be negotiated and accounted for during budgeting? The asset and placebo will be provided by the company for the UG3 and UH3 studies at no cost to NIH or the applicant. How will the UG3/UH3 grant applications be selected for funding? The following will be considered in making funding decisions: Scientific and technical merit of the proposed project as determined by scientific peer review Availability of funds Relevance of the proposed project to program priorities | Frequently Asked Questions About RFA-TR-17-002 and RFA-TR-17-003 | Frequently Asked Questions About RFA-TR-17-002 and RFA-TR-17-003 | ||||||
| 7079 | CTSA Program Community Mentorship Helps Advance Multiple Sclerosis Research in Latinos | Translational Science HighlightThrough an NCATS-supported community mentorship program, a neurologist strengthens her translational science skills by increasing her engagement with an underserved population.Lilyana Amezcua, M.D., a University of Southern California (USC) assistant professor and clinical neurologist, investigates the complex genetics of multiple sclerosis (MS) among Latinos in greater Los Angeles. Through the NCATS’ Clinical and Translational Science Awards (CTSA) Program-supported Community Mentorship Program at the Southern California Clinical and Translational Science Institute (SC CTSI), she developed new relationships with Latino patients and communities. This work strengthened her translational science skills and gave her a new understanding of her patients’ experiences, perceptions and struggles with MS.Community engagement is a crucial part of the translational science process, said Cecilia Patino-Sutton, M.D., Ph.D., M.Ed., associate professor of clinical preventive medicine, co-director of the KL2 Program and director of the research career development program at the USC Keck School of Medicine. Under her direction, the medical school has created a program to connect early stage clinical and translational scientists, like Amezcua, with mentors from community-based organizations in Los Angeles who understand the investigators areas of research. USC participants were asked to work with their mentors to identify opportunities for the community to provide feedback to some aspect of the investigators’ research and then present the work to community audiences with expertise in the same topic.“We want to align clinicians and scientists with community members to help [the investigators] understand and incorporate that community perspective in their work,” Patino-Sutton said.New Understanding of Community Mentorship Role in ResearchAt first, Amezcua wondered what she would gain from participating in the mentorship program.“When I was asked to participate in the SC CTSI community mentorship program, I had this notion I already was engaged,” she said. “I had been a fellow with the National MS Society and had participated in educating the community, though not the Latino MS community.”Lilyana Amezcua, center, is presented with the Health Professionals Volunteer Hall of Fame award from the National MS Society’s National Board Chair Eli Rubenstein and President and CEO Cynthia Zagieboylo.Amezcua’s community mentor, Mercy Willard, M.P.H., the director of community engagement and program development at the National MS Society chapter in Los Angeles, helped connect her more closely with the local Latino MS community. Together, they developed and hosted several workshops locally and later on a national level. In addition, they conducted a series of focus groups.“Input from the focus groups made me realize how little I actually knew about how MS-related disabilities affected this population,” Amezcua said. “I learned I had to better understand the community.”Amezcua found that because of a lack of research studies about MS in the Latino population, little is known about how social and cultural factors affect the disease’s severity and progression.“I discovered there are misperceptions about what MS is, including its causes and treatments,” she said. “There also are challenging barriers to care and education.”The experience helped her grow as a translational scientist.“While I had been focused on better understanding the genetic influences of MS for many years, prior to the SC CSTI community mentorship program, I had not realized I needed to spend more time with families living with the disease,” Amezcua said, adding, “My research now includes a focus on the perception and behaviors of the Latino community with MS to better understand how they live with disabilities that come with the disease.”The local workshops helped raise interest at the national level to educate social workers and engage the Latino population.“So what began as a local community mentorship and engagement program became a national outreach effort,” she said.Amezcua has since been invited to be part of a national task force to develop access-to-care principles for the entire MS population, with a particular emphasis on diversity and minorities. In addition, she and her colleagues produced an award-winning short film about the Latino experience with MS.“The film will be used as a rapid intervention tool to better understand perceptions of living with the disease,” she said. “Perhaps it will eventually be used in MS centers across the country.”Amezcua continues to focus on collecting genetic data from the Latino MS community. She is also working closely with colleagues at the University of Miami, and she recently received grants from the National MS Society and the California Community Foundation to use the film as a tool to understand patient and community perceptions of the disease. The National MS Society grant also supports a multisite study to examine genetic, environmental, social and cultural factors and disease severity.The SC CSTI program reach has expanded as well: More than a dozen other NCATS-supported CTSA Program hubs in California, Oregon and other states now collaborate to develop initiatives that connect early stage research scholars with community engagement opportunities that help shape the investigators’ research through interactions with community mentors.Posted May 2017 | Through an NCATS-supported community mentorship program, a neurologist strengthens her translational science skills by increasing her engagement with an underserved population. | /sites/default/files/lilyana-amezcua.jpg | Community Mentorship Helps Advance MS Research in Latinos | Through an NCATS-supported community mentorship program, a neurologist strengthens her translational science skills by increasing her engagement with an underserved population. | /sites/default/files/lilyana-amezcua.jpg | Community Mentorship Helps Advance MS Research in Latinos | ||
| 7066 | News Brief: NCATS SMART IRB Team Launches Online Reliance System | The NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) Institutional Review Board (IRB) team launched an Online Reliance System to help streamline the ethics review conducted by IRBs. Investigators at participating institutions can use the system to request, track and document reliance arrangements among the reviewing IRB and relying institutions for multisite studies. More than 200 institutions are participating in the NCATS SMART IRB.Supported through NCATS’ Clinical and Translational Science Awards (CTSA) Program, the Online Reliance System enables greater IRB efficiencies that will help accelerate the launch of clinical trials nationwide. View the SMART IRB website to learn more. Posted May 2017 | The NCATS SMART IRB team launched an Online Reliance System to help streamline the ethics review conducted by IRBs. | /sites/default/files/smart-irb-news.jpg | NCATS SMART IRB Team Launches Online Reliance System | The NCATS SMART IRB team launched an Online Reliance System to help streamline the ethics review conducted by IRBs. | /sites/default/files/smart-irb-news.jpg | NCATS SMART IRB Team Launches Online Reliance System | ||
| 6949 | May 2017 Council/CAN Review Board Meeting | The May 4, 2017, joint meeting of the NCATS Advisory Council and the Cures Acceleration Network (CAN) Review Board featured the following meeting materials. Agenda (PDF - 109KB) Videocast | May 2017 Council/CAN Review Board Meeting | May 2017 Council/CAN Review Board Meeting | ||||||
| 6874 | NCATS Trial Innovation Network Tackling Clinical Trial Inefficiencies | John N. Clore, professor of medicine and associate vice president for clinical research, speaks with patient Colleen A. Thoma, as she undergoes a test to monitor brain wave activity. (Virginia Commonwealth University Photo/Tom Kojscich) Through NCATS’ new Trial Innovation Network, Clinical and Translational Science Awards (CTSA) Program-supported investigators are working collaboratively — including with the CTSA Program Trial Innovation Centers (TICs) and Recruitment Innovation Center (RIC), as well as with other NIH Institutes and Centers — to tackle inefficiencies in clinical trials. The Trial Innovation Network also is designed to be a national laboratory to study, understand and innovate the processes for conducting multisite studies so that more treatments can reach more patients more quickly. For example, network investigators are establishing a harmonized, central institutional review board (IRB) system among the IRBs at Vanderbilt University Medical Center, the University of Utah, and Johns Hopkins University. Efforts include developing workflows, processes and resources to harmonize network IRB reviews and to pilot the NIH single IRB policy. And, in partnership with the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) IRB team, they will develop metrics to assess the time, costs and resources data required to operationalize an IRB review that uses the SMART IRB Authorization Agreement (PDF - 449KB). Network investigators are also developing a Standard Agreement System for contracting for network trials and studies. Their work includes guidance for using the Federal Demonstration Partnership Clinical Trials Subaward Agreement (FDP-CTSA) and metrics to capture data on the impact of this harmonized template for contracts. In addition, the Trial Innovation Network investigators are implementing a strategic, multidisciplinary, evidence-based approach to optimize participant engagement, recruitment and retention and will develop metrics, milestones and processes. TIC grantees will help ensure protocols address scientifically relevant questions by streamlining study designs, developing realistic eligibility criteria based on target populations and optimizing data collection. The RIC grantees will conduct evidence-based feasibility analyses and support data-driven cohort discovery. RIC grantees are also providing expertise in engaging participants, developing tailored recruitment plans and materials, and using creative approaches to target potential participants. All researchers can access network services and consultations by submitting proposals through the Trial Innovation Network website, which features resources, instructions and materials for the submission process. The website includes information on operationalizing standard agreements and a central IRB, as well as feasibility assessments, recruitment materials and Community Engagement Studios. Trial Innovation Network initial consultations include suggestions on study design, budgets, projected timelines, recruitment and study feasibility. Based on the outcome of an initial consultation, a proposal may be eligible for a comprehensive consultation and development into a protocol that would be implemented in the network. Posted April 2017 | NCATS Trial Innovation Network Tackling Clinical Trial Inefficiencies | NCATS Trial Innovation Network Tackling Clinical Trial Inefficiencies | ||||||
| 6873 | CTSA Program Mentoring Paves Way for Brain Cancer Therapy | Translational Science Highlight CTSA Program mentoring support sparked an interdisciplinary translational research team that developed a potential therapy for brain cancer. Cell biologist Shawn Hingtgen, Ph.D., is convinced that stem cells can effectively treat brain cancer. Using the support he received through the mentored career development program offered by NCATS’ Clinical and Translational Science Awards (CTSA) Program, he has collaborated with neurosurgeons, oncologists, stem cell experts, drug development specialists and others to create a potential therapy for glioblastoma multiforme, a deadly type of brain cancer. Hingtgen, an assistant professor in the Eshelman School of Pharmacy at the University of North Carolina (UNC) at Chapel Hill, previously was an NCATS KL2 Mentored Clinical Research Scholar at the North Carolina Translational and Clinical Sciences (NC TraCS) Institute. Among other things, this award supports mentored career development for investigators who have recently completed professional training and are early in their research careers. “The multidisciplinary nature of [the] NC TraCS KL2 program enables junior faculty to work with experts in many different fields to solve problems and provide guidance in the process of translating basic science knowledge to clinical medicine,” said Kim Boggess, M.D., UNC professor of obstetrics and gynecology and co-director of the NC TraCS KL2 program. Shawn Hingtgen, Ph.D., sits in front of a fluorescent microscope that is used to examine stem cells in his laboratory. Hingtgen, a former CTSA Program KL2 scholar at the University of North Carolina at Chapel Hill, is developing personalized tumor-homing stem cell therapies for cancer. Glioblastoma is an insidious disease that infiltrates the brain, killing approximately 14,000 people annually in the United States. The cancer sends out tendrils into brain tissue that make it nearly impossible to completely eradicate with drugs or surgery. The current therapy for glioblastoma is surgery, radiation and chemotherapy, with most patients only surviving one to two years after diagnosis. Stem cells are a promising new approach to deal with shortcomings of current therapies. Hingtgen and his team are taking advantage of a technique known as “direct reprogramming.” During this process, fibroblasts, skin cells responsible for making collagen and connective tissue, are converted into induced neural stem cells, which have an innate ability to target cancer cells in the brain. “When neural stem cells are put into the brain, they track down the cancer tentacles and roots that spread through the brain,” Hingtgen said. But that’s not enough to kill the cancer cells, so Hingtgen’s group has engineered stem cells to carry and deliver drugs to the cancer cells. The researchers are studying two approaches. In one approach, stem cells are modified to carry an enzyme trigger that can activate a drug inside the cells, killing nearby tumor cells. In the second approach, neural stem cells are engineered to carry and continuously pump out tumor-killing proteins that can bind to tumor cell surfaces, killing those cells. “We’re developing a personalized tumor-homing drug carrier with anti-cancer agents that will seek out residual cancer that chemotherapy and surgery miss,” he said. Hingtgen collaborated with a surgeon and an oncologist to create a way to deliver the cells to a tumor site. Instead of being injected, the stem cells are placed on a customized, implantable patch or bandage, which is placed at the surgical site. The stem cells then migrate to the cancer. While much of the research to date has been done with mouse cells, more recent research has focused on using human skin cells. Mentoring Improves Research Translation Hingtgen began his stem cell therapy research as a postdoctoral fellow at Massachusetts General Hospital in Boston. Shortly after arriving at UNC-Chapel Hill in 2012, he was accepted into the KL2 scholar program. As a KL2 scholar, Hingtgen attended seminars on a range of topics, from how to manage a lab and write grants to understanding the drug development process and therapeutic product development. A mentoring team, including Matthew Ewend, M.D., chair of neurosurgery at UNC Hospitals, helped with all aspects of translation and the product development process for Hingtgen’s work. The group frequently met to discuss how to translate the stem cell technology into something useful for patients. “NC TraCS advisors connected me with surgeons, engineers and others to discuss what a clinical product should look like. We discussed how to personalize the therapy and found a way to get the stem cells into the brain cavity. After several months, we arrived at the first design of our clinical product,” Hingtgen said. Scientists determined the anti-tumor effects of neural stem cells in 3D culture models. Stem cell aggregates, or spheroids (blue), were placed next to brain cancer cell spheroids. Fluorescent images captured over seven days showed that the stem cell therapy decreased the volume of cancer spheroids. Researchers are working to advance the therapy towards first-in-human clinical trials. In addition to classroom work, Hingtgen’s training and mentoring included work with the NC TraCS 4D (Drugs, Devices and Diagnostic Development) program, which enabled him to consult with experts in pharmaceutical product development to explore commercial opportunities and industry partnerships. At the same time, the joint NC TraCS-Research Triangle International Regulatory Service advised on preclinical regulatory requirements for the product. With NC TraCS’ guidance, Hingtgen wrote a research protocol and received UNC institutional review board approval to collect human cancer tissue for testing with the experimental stem cell therapy. Hingtgen received a $50,000 TraCS pilot grant to develop a stem cell bandage, enabling him to partner with research leaders and generate preliminary data that led to a 2016 grant from the National Institute of Neurological Disorders and Stroke to further develop the technology. Perhaps just as important, the KL2 program specialists helped Hingtgen hone his presentation skills. “Through the KL2 program, I also met with communications experts who helped me develop a five-minute sales pitch — sometimes called an ‘elevator speech’ — which helped me land a $750,000 award from the UNC Eshelman Innovation Fund and a $300,000 grant from the state of North Carolina to further my team’s research,” he said. The 4D program experts also helped him work with UNC’s intellectual property office to file two patents for stem cell work and, subsequently, start a company, Falcon Therapeutics, based on the stem cell technology. The company recently licensed the technology from the university. Hingtgen is continuing to gather data and refine the stem cell technology, and he is using his elevator speech as a springboard for discussions with the U.S. Food and Drug Administration. He credits the KL2 experience with teaching him the value of collaboration, team building and communication, all invaluable skills critical to his growth as a translational scientist. “I have always wanted to create solutions in the lab to help improve people’s lives,” he said. “We’re making a therapy that has the potential to help thousands of cancer patients, and our goal is to get it to patients as fast as we can.” Posted April 2017 | CTSA Program mentoring support sparked an interdisciplinary translational research team that developed a potential therapy for brain cancer. | /sites/default/files/hingtgen_shawn%28900px%29.jpg | CTSA Program Mentoring Paves Way for Brain Cancer Therapy | CTSA Program mentoring support sparked an interdisciplinary translational research team that developed a potential therapy for brain cancer. | /sites/default/files/hingtgen_shawn%28900px%29.jpg | CTSA Program Mentoring Paves Way for Brain Cancer Therapy | ||
| 6872 | CTSA Program Support Helps Talented Investigators Re-Establish Their Careers | Anandi Krishnan, Ph.D., is a blood/thrombosis researcher returning to science following an NCATS (2016-18) re-entry award. Krishnan’s current work is in identifying platelet transcriptomic signatures in myeloproliferative neoplasms. Investigators who take a break from research for personal reasons — such as to care for a family member — can have a difficult time returning to academia. To address this challenge and cultivate the best minds to tackle problems in translational science, NCATS funds career re-entry supplements through its Clinical and Translational Science Awards (CTSA) Program. One recipient is Anandi Krishnan, Ph.D., who was completing a successful postdoctoral fellowship before needing time off to care for her newborn child in 2011. One year later, Krishnan was ready to return to work, but despite having a stellar publication record, she was unable to obtain a faculty researcher position. Instead, she accepted an administrative position within Spectrum, the Stanford Center for Clinical and Translational Research, a CTSA Program hub. Still, she missed being a hands-on researcher. When Krishnan learned of NCATS’ career re-entry awards, she collaborated with faculty at Spectrum to successfully apply for a supplement to study myeloproliferative neoplasms (MPNs), a group of rare blood cancers. These diseases carry changes in blood cells’ genetic code that may contribute to complications such as blood clotting. Thanks to the funding, Krishnan is now examining platelets of MPN patients to identify genetic signatures, and she is compiling data to provide a better understanding of the diseases. Her work could ultimately help clinicians detect these diseases earlier and tailor treatments. And how does it feel to be actively engaged in research again? “Spectacular!” Krishnan said. “I am thankful for the support provided through the CTSA Program, and for my outstanding mentors, our dedicated research support staff, collaborators and, above all, patients who offer their samples. It really does take a village to do research.” Posted April 2017 | CTSA Program support helped Anandi Krishnan, Ph.D., get back into a research career after taking a leave of absence to care for her child. | CTSA Program Support Helps Investigators Re-Establish Careers | CTSA Program support helped Anandi Krishnan, Ph.D., get back into a research career after taking a leave of absence to care for her child. | CTSA Program Support Helps Investigators Re-Establish Careers |
Last updated on