AnnualReport2021

Research Informatics with Fast Health care Interoperability Resources (FHIR), will interface FHIR with our existing data resources to allow integration of multiple data sources. The aim is to accelerate research and create tools for personalized and predictive pediatric medicine. For the 2021 calendar year, the research programs department has awarded 32 basic research projects; 20 clinical research projects, involving 18 participating locations; nine developmental projects, involving nine participating locations; six request for proposal projects, involving seven participating locations; seven seed grants with GIT, involving three participating locations, 11 special shared facilities, 12 fellowships, as well as additional system-wide administrative support. Currently, across the Shriners Hospitals for Children system there are 281 active clinical research studies. Of these, 25 projects are multisite. These clinical research projects are focused around the conditions treated by our health care system, with 179 studies focused in orthopaedics, 48 spine studies, 32 burn- related studies, eight studies related to metabolic conditions, four related to plastic surgery, seven associated with motion analysis, and three focused in other areas. Additional highlights of ongoing, outstanding clinical and bench research successes throughout our Shriners research enterprise are presented on the following pages.

The launch of the Genomics and Precision Medicine project at Shriners Hospitals for Children — Mexico in the spring of 2021 represents the first research project ever conducted at the Mexico City hospital. Starting up the research involved several steps including the recruitment of two CRCs; the submission and approval of the clinical research protocol by the Mexico Ministry of Health; the creation of local ethics committees; and development of an electronic entry platform for capture of clinical diagnostic information via a tablet. With the establishment of this new research department, our Mexico hospital will be an important partner in numerous clinical research studies from across the system. The creation of the Genomics Institute and availability of advanced DNA sequencing to our medical and scientific staff system-wide is also driving new areas of clinical research and inspiring multicenter grant applications. One such project recently funded is entitled, Elucidating the Genetic Architecture of Cerebral Palsy (CP). This initiative involves physician investigators from our Northern California, Chicago, Portland and Shreveport locations in a partnership with the Jackson Laboratory, an independent, nonprofit biomedical research institution, in Farmington, Connecticut. Their research will endeavor to gain a greater understanding of the genetic factors involved in CP through clinical characterization and genome sequencing of 500 children affected by CP, as well as their parents, which represents the largest group of CP patients and their families ever to be investigated in this way. The research programs department is establishing a number of partnerships to build an infrastructure for maximizing collaboration and data sharing. Working with GIT and other partners, our department’s informatics team is building a big data platform in Microsoft Azure cloud platform and launching tools to capture, access, share and analyze clinical research information. This project, Accelerating

EB, a devastating inherited disease of the skin that makes the skin very fragile, resulting in a lifetime of blisters, chronic ulcers and infections. There are currently no adequate treatments other than prevention of trauma and a lifetime of relentless care of wounds and infections. A highly experienced multidisciplinary team in translational regenerative medicine under the auspices of and with support from the Howard Green Center for Children’s Skin Health & Research plans to use gene and stem cell therapy to treat EB. The research requires the implementation of several innovative technologies, including the production of clinical-grade gene transfer vectors, as well as coordination with regulatory bodies, such as the FDA. The team of investigators includes researchers from our Boston hospital, Massachusetts General Hospital and Harvard Medical School. Physicians and scientists from our Galveston and Northern California locations will also participate in this project in an attempt to set the stage for first-in-human clinical validation of Good Manufacturing Practices and FDA compliant processes developed in this project. THE GENOMICS INSTITUTE The Shriners Hospitals for Children Genomics Institute continues its mission to perform DNA sequencing of samples from our patients and their families who voluntarily enroll in the Shriners Hospitals for Children Genomics and Precision Medicine project. For this study, a saliva sample is obtained and shipped to our Genomics Laboratory located within the University of South Florida Research Park in Tampa, Florida. More than 1,000 patient and family samples have been obtained from hospitals and outreach clinics in our health care system, and a major ramp-up effort is occurring. With the Joint Boards’ full and generous support, the laboratory is fully equipped and performs sequencing of a study participant’s entire genome via installation of a top-of-the-line Illumina NovaSeq instrument, combined with a robotics suite to process large sample numbers.

Sincerely,

Marc Lalande, Ph.D. Vice President, Research Programs Shriners Hospitals for Children

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