Goal #4: Improve clinical research

The SC CTSI Regulatory Knowledge and Support (RKS) team in collaboration with the USC + CHLA Alpha Clinic has launched a new self-paced training module, “Cell and Gene Therapy Trials.” This course focuses on implementing quality practices for clinical trials involving these types of regenerative medicine therapies and serves as the inaugural offering in the series, “Special Considerations for Emerging Technologies.”This project was led by RKS Director, Chair and Associate Professor, Eunjoo Pacifici, Pharm.D., Ph.D., and RKS Associate Director Nancy Pire-Smerkanich, D.R.Sc., M.S.. Additional contributors include RKS Associate Director Tony Succar, Ph.D., M.Sc.Med.(OphthSc), along with administrators Annie Ly, M.S., Karen Manrique, M.S., who oversaw development, and Gordon Wimpress, B.Sc., B.A., who oversaw production. The content draws from various sources, such as the FDA, the National Institutes of Health, clinical research organizations, and the expertise and experiences of key stakeholders within the USC + CHLA Alpha Clinic.The module is freely available, and open to the entire clinical research workforce, as well as anyone interested in learning more about ensuring quality in cell and gene therapy trials. It includes four chapters that introduce fundamental concepts in CGTs, examine opportunities and challenges inherent in CGT clinical trials, and provide practical case studies along with actionable resources such as templates and checklists. Upon completion of the module, users receive a digital badge and a certificate of completion, which can be added to resumes, CVs, and LinkedIn profiles.To access this new module, please visit the <a href="https://courses.sc-ctsi.org/product?catalog=RKS-CGT" target="_blank" rel="noopener noreferrer">Cell and Gene Therapy Trials course catalog listing</a>, click the Enroll button, and Login or Create an&nbsp;Account.

Southern California Clinical and Translational Science Institute

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University of Southern California

The SC CTSI Regulatory Knowledge and Support Team and the USC + CHLA Alpha Clinic Launches New Module on Cell and Gene Therapy Trials

New Self-Paced Online Module on Cell and Gene Therapy Trials Now Available!

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Goal #2: Promote partnerships and collaborations

Members from the <a href="https://www.cctst.org/" target="_blank" rel="noopener noreferrer">Center for Clinical and Translational Science and Training (CCTST)</a> recently attended and participated in the collaborative workshop on real-world data (RWD) and real-world evidence (RWE) in Washington, DC. Hosted by the Stanford School of Medicine, NCATS and CCOS, the event convened researchers, patients, industry experts, and government leaders to advance the future of RWD/RWE research.&nbsp;Among the CCTST attendees was Monica Banks, who serves on one of the <a href="https://www.cctst.org/rpag" target="_blank" rel="noopener noreferrer">CCTST’s Research Participant Advisory Groups (RPAGs)</a>. The RPAG includes the <a href="https://www.cctst.org/uc-rpac" target="_blank" rel="noopener noreferrer">University of Cincinnati Research Participant Advisory Council (UC RPAC)</a>, the <a href="https://www.cctst.org/cchmc-rpac" target="_blank" rel="noopener noreferrer">Cincinnati Children's Research Participant Advisory Council (CCHMC RPAC)</a>, and the <a href="https://www.cctst.org/west-end-c-rab" target="_blank" rel="noopener noreferrer">West End Community Research Advisory Board</a>. These groups serve as valuable resources for investigators and research teams at Cincinnati Children's and the University of Cincinnati who are seeking participant and community perspectives to enhance the design, implementation, and impact of research conducted both within healthcare settings and throughout the community.&nbsp;Monica participated in the panel discussion, “Governance Models for Sustainable and Trustworthy RWD,” where she shared her perspective as a parent advocate. Her advocacy journey began when her daughter was diagnosed with lupus at age nine. During her daughter’s treatment, she was supported by a dedicated team of physicians who were deeply involved in research. Throughout the process, she accepted her diagnosis and decided that she wanted to be part of a long-lasting solution, which included research. Over the years, she has participated in numerous research studies, including clinical drug trials. As a pediatric parent, Monica was along for the journey. Today, Monica’s daughter is entering her sophomore year at the University of Cincinnati and is pursuing a path toward becoming a pediatric rheumatologist. She remains involved in research and is currently participating in the Cincinnati Children's Summer Undergraduate Research Fellowship program.&nbsp;Monica emphasized the importance of incorporating the patient perspective throughout the entire research process - from study conception and design to communicating findings back to the patients who contributed their data. Some of the key points she made:<ul><li>Families are often the continuity layer in a fragmented healthcare system. We carry timelines, symptoms, treatment responses, records, and outcomes across institutions and over years.</li><li>Families are willing to share incredibly personal data when they believe the system is stewarding it responsibly, transparently, and ethically.</li><li>Good governance is not just about protecting data. It’s about honoring the people behind it.</li><li>In pediatrics, governance is uniquely complex because children eventually become adults. We need systems that respect both parental stewardship and the future autonomy of the patient.</li><li>Families stay engaged when they feel respected, informed, included, and connected to outcomes over time.</li><li>Reproducibility improves when participation burden is reduced. Otherwise, research will continue to overrepresent families with the time, resources, and stability to participate.</li><li>The future of trustworthy real-world evidence depends not only on better technology, but on stronger partnerships with the families living these experiences every day.</li></ul>&nbsp;Monica’s experience as a parent advocate has brought valuable insight and perspective to the Cincinnati Children’s RPAC. In a recent spotlight feature, Monica shared that serving on the CCHMC RPAC has been one of the most meaningful aspects of her advocacy journey, providing an opportunity to bring the patient and family perspective directly into research discussions. “The best research happens when researchers and families work as partners,” said Monica. “RPAC helps make that partnership possible. Families may not have the scientific expertise of researchers, but they are experts in living the experience. When those perspectives come together, research becomes stronger, more meaningful, and more impactful.”

Cincinnati Center for Clinical and Translational Science and Training

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Cincinnati Children's Hospital Medical Center

CCTST Research Participant Advisory Group (RPAG) member, Monica Banks, participated in a panel discussion at the collaborative workshop on real-world data (RWD) and real-world evidence (RWE), sharing her perspective as a parent advocate.

CCTST RPAG Member Shares Parent Advocate Perspective at National RWD/RWE Workshop

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Goal #3: Address health needs of populations

The fair, which aimed to increase awareness of vital services available to Schuylkill County residents who are retired or entering retirement, did not happen by chance. It was the direct outcome of a dedicated community-academic partnership sparked by a recent Community-Driven Research Day.<h2>The Catalyst: Community-Driven Research Day</h2>Community-Driven Research Days bring together community members, local organizations, and academic researchers to openly discuss pressing local health needs and brainstorm collaborative solutions. Rather than isolated engagements, CDRDs foster sustained collaboration, creating pathways for pilot funding, grant development, and long-term partnerships.During the Schuylkill County event, a clear priority emerged: the need to better support the region's aging population as they navigate the transition into retirement. Recognizing this critical gap, a group of passionate community stakeholders and researchers formed the Schuylkill Healthy Aging Task Force.<h2>Turning Ideas into Reality with CTSI Pilot Funding</h2>Identifying a need is only the first step; taking action requires resources and support. To ensure the momentum from the Community-Driven Research Day translated into tangible community benefits, Penn State CTSI made pilot funds available to support community-led programs designed by the newly established task forces.The Schuylkill Healthy Aging Task Force submitted a compelling proposal titled&nbsp;"Cheers to the Golden Years 2026."&nbsp;Recognizing the potential impact of their initiative, the CTSI leadership team proudly awarded the task force a $2,500 pilot grant to bring their vision to life.<h2>"Cheers to the Golden Years": Connecting Residents with Resources</h2>Fueled by the CTSI grant, the task force successfully organized and hosted the Healthy Aging Fair. The event served as a vibrant hub of information, connecting older adults and soon-to-be retirees with a wide array of local services tailored to their unique needs.Navigating the resources available for healthcare, social engagement, and daily living can be overwhelming for those entering their golden years. The fair broke down these barriers, providing a centralized, accessible, and welcoming environment for residents to learn, ask questions, and build a support network.Jess Petrie, CTSI Education Specialist, and&nbsp;<a href="https://ctsi.psu.edu/ctsi-highlight-healthy-aging-fair-partner-jennifer" target="_blank" rel="noopener noreferrer">Jennifer Youndt</a>, a Food, Families, and Communities Educator from Penn State Extension, co-led the event's planning and execution. Reflecting on the significance of the initiative, Petrie shared:<blockquote>"Being part of the planning process for 'Cheers to the Golden Years' was incredibly rewarding. It is one thing to discuss community needs during a research day, but to see those conversations evolve into a tangible event that directly connects our older residents with vital resources is truly the heart of what we do at CTSI. This task force took a shared vision and turned it into a meaningful support system for Schuylkill County."</blockquote>The success of the "Cheers to the Golden Years" fair represents the core mission of Penn State CTSI: translating scientific discovery and academic resources into real-world health improvements. By providing the platform for dialogue (Community-Driven Research Day) and the resources for action (pilot funding), CTSI empowers communities to lead the charge in improving their own health outcomes.We congratulate the entire Schuylkill Healthy Aging Task Force on a remarkable event. Their work stands as a shining example of how collaborative, community-driven initiatives can make a profound difference in the lives of Pennsylvanians.

Penn State Clinical and Translational Science Institute

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Pennsylvania State Hershey Medical Center

At the Penn State CTSI, we believe that the most effective health interventions begin by listening to the communities we serve. Recently, this philosophy was brought to life in Schuylkill County through a highly successful Healthy Aging Fair, an event that perfectly illustrates the power of community-engaged research and collaboration.

From Community Dialogue to Action: "Cheers to the Golden Years" Fair Supports Healthy Aging in Schuylkill County

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New Self-Paced Online Module on Cell and Gene Therapy Trials Now Available!

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