The Center for Precision Medicine (CPM) at Nicklaus Children's Hospital is proud to one of the nation's first programs to focus on precision and personalized medicine to support children's medical care, health and wellness. It offers the latest precision medical offerings to support the assessment and treatment of children with rare conditions, and is part of Nicklaus Children's commitment to offering the most innovative, personalized care to the children and families we are privileged to serve.
Who We Are
The Center for Precision Medicine is a collaborative multidisciplinary program enhancing translational research, providing access to the best molecular diagnostics, and implementing precision-medicine-based clinical treatment in the fabric of routine care. The center also provides education and advocacy in precision medicine and connects the diverse South Florida community to providers and researchers.
This program brings together clinicians and scientists to perform innovative, clinically relevant research in rare and undiagnosed diseases, pediatric cancers, and pharmacogenetics. This is a continuation of the multi-year Personalized Medicine Initiative at Nicklaus Children's Health System, which started in 2017. As of 2023, CPM includes the Undiagnosed Diseases Clinic, Nicklaus Children's Biobank, Pharmacogenomics Program, and Precision Medicine Analytics.
What is Precision Medicine?
The idea of precision medicine is not new. Clinicians have long recognized that different patients respond differently to medical interventions.
What's 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.
The overarching goal of the center is to promote integrated precision care across the Nicklaus Children's Hospital and use the personalized profile of each child to guide value-based therapeutics management strategies with enhanced efficacy and safety. The integration of precision medicine in patient care includes the following key programmatic elements.
- Bridging the gap by bringing precision medicine to underserved populations to address health disparities in diverse and predominantly Hispanic South Florida populations.
- Building infrastructure to maximize synergies. Implementation of precision medicine requires a transdisciplinary framework that cuts across organizational silos and fosters strong collaborations between clinicians, researchers, information technology, administrators, community organizations, and government agencies.
- Advanced molecular diagnostics as a front door for rare undiagnosed disorders at the Undiagnosed Disease Clinic, led by clinical geneticists. Early diagnoses of monogenic diseases in critically ill infants and children on diagnostic odyssey can help improve care and lessen suffering and mortality.
- Development of pharmacogenomic and other precision medicine tools to identify critical biomarkers for disease progression and drug responses. The Pharmacogenomics Program tailors gene-specific drug treatments to increase efficacy and mitigate the risk of adverse reactions. CPM conducts discovery and translational research to help patients who do not respond well to traditional therapies.
- Outreach and education of healthcare professionals, researchers, patients, and the public on precision medicine, pharmacogenomics, rare diseases, and preconception screening.
- Facilitating research opportunities to access high-quality biospecimens through Nicklaus Children's Biobank and research data through Precision Analytics.
- Promoting research and industry collaborations, access to clinical trials, gene therapy, enzyme replacement, and other new therapy options. This approach is especially valuable to health disparity populations, who may not respond to standard diagnostic or treatment strategies developed from clinical trials lacking representative study participants.
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.
Integrated into every Service Line