The Personalized Medicine Initiative at Nicklaus Children’s and Miami Children’s Health System is one of the nation’s first programs focused on precision and personalized medicine to support children’s medical care, health and wellness. At Nicklaus Children’s, we know that children are not just small adults and that every child and family deserves personalized care.
The Personalized Medicine Initiative (PMI) involves a comprehensive approach to tailoring medical management to each specific child’s individual characteristics, needs and preferences at all stages of care, including prevention, diagnosis, treatment and follow up.
What is Personalized Medicine?
The idea of personalized medicine is not new. Clinicians have long recognized that different patients respond differently to medical interventions.
What is new? Transformational developments in science and technology offer promise for developing targeted diagnostics and tools for predicting who will respond to a medical therapy and who will experience side effects.
Illnesses may be rooted not only in a child’s genes, but also within environmental, genetic, social and cultural influences. This combination of factors underscores the need for a comprehensive approach to personalized medicine that includes assessment at the molecular, cellular, tissue and organ, and community levels.
Integrated into Every Service
The Personalized Medicine Initiative is not a separate service of the health system, but is woven into all hospital service lines and outpatient centers to optimize and personalize care.
The PMI is focusing on emerging imaging technologies, with current focuses on 3D printing and surface imaging, to tailor care to specific patient characteristics. 3D models can be used to create personalized medical devices and serve as important tools guiding surgeons in the diagnosis and treatment of congenital heart defects and other medical challenges.
Surface imaging systems provide visual reference to capture anatomically precise images for surgical assessment and intervention. The sequential-3D systems support plastic surgeons in optimizing care practice for patients with craniofacial differences and more. The team is also assessing the potential of virtual reality and augmented reality, along with other emerging technologies.
Biobanks and Tissue Repository
The hospital’s biobank and tissue repository is an essential resource for personalized medicine research efforts, enabling the study of both health and disease over time. The Nicklaus Children’s biobank collects samples and health information from volunteers, regardless of health history. Once a participant becomes part of the Biobank, he or she contributes to ongoing health research.
The biobank partners with national leaders on specimen storage, management and integration with participant health information.
Pediatric Genomic Medicine
The hospital is the beneficiary of a generous contribution from philanthropist Denny Sanford and Sanford Health to launch concerted efforts in pediatric genomic medicine. In addition, Nicklaus Children’s is a member of the Sanford Children’s Genomics Consortium, a group of seven children’s hospitals from around the U.S. committed to assuring that children benefit from the promise of genomics.
The following are projects currently in development:
- Utilization of rapid whole genome sequencing for children receiving care in the hospital’s three intensive care units to support diagnosis and treatment of this critically ill population.
- Development of whole genome sequencing to help patients and families on a “diagnostic odyssey” – a protracted search for a diagnosis of what may be a rare genetic condition – offering answers to help them move forward with medical care.
- Implementing pharmacogenomics or matching a patient’s genetics to support medication selection.
Minority populations, including Hispanics and African Americans, are underrepresented in genomics. An estimated 96 percent of all known genetic information comes from people of Northern European heritage. In a multi-ethnic community like South Florida, this creates challenges in applying genomics to medicine.
Research studies within the PMI will garner information that will help build a database of common genomes from underrepresented minorities to support understanding of the population’s predisposition to certain diseases and responses to medications.
Patients and families do best in a care environment sensitive to cultural needs. Nicklaus Children’s is developing a curriculum for staff and trainees to support cultural competency to enhance support South Florida’s diverse patient population as well as the many international patients who receive care at the hospital.
Community Outreach and Education
This focus is aimed at building health literacy, competency and awareness about personalized medicine in South Florida patient and provider populations.
A model is the hospital’s Victor Center for the Prevention of Jewish Genetic Diseases. This center hosts genetic screening and biobanking recruitment events to support identification of those who may be carriers of Jewish genetic diseases and provide counseling and support.
Miami Children's Health System is a Continuing Medical Education provider accredited with commendation by the Accreditation Council for Continuing Medical Education (ACCME). The health system sponsors several regularly scheduled series and yearly conferences at Nicklaus Children's Hospital and in the community.
Policy and Health Economics
Personalized medicine seeks to reduce the burden of disease by targeting prevention and treatment more effectively. By improving the ability to predict and account for individual differences in disease diagnosis, experience and therapy response, personalized medicine offers hope for reducing the duration and severity of illness and improving success rates. It may also reduce healthcare costs by improving clinician’s ability to quickly and reliably identify effective therapies.