Why choose Cook Children’s Precision Medicine Program

At Cook Children’s Hematology and Oncology Center, precision medicine means caring for each child based on their unique genetic makeup, environment and life story, rather than using a one-size-fits-all approach. By understanding the specifics of each child’s condition, our team can diagnose faster, choose more targeted treatments and reduce long-term side effects whenever possible. 

What you can expect

Precision medicine doesn't replace conventional treatments, it uses advanced genetic testing tools like massively parallel sequencing and cytogenetic technology to guide care. These techniques help identify the exact changes in a tumor or a child’s DNA, so treatments can be selected or adjusted with greater confidence. Every detail we uncover helps us design the most thoughtful approach for your child.

When you trust us with your care, you can expect:

A team dedicated to each child

Behind the scenes, a large multidisciplinary team—including physicians, pharmacists, genetic counselors, nurses, social workers, data and research specialists—meet regularly in a molecular tumor board and precision medicine conference to review each complex case. This collaboration helps ensure that every child benefits from the latest evidence, clinical trials and institutional expertise. This teamwork helps turn complex information into clear and confident treatment decisions for your family. 

The precision medicine program brings together multiple specialties to support children and families across many conditions.​ Your child's care may involve surgery, chemotherapy, radiation therapy or a mix of these treatments which may be given as standard care or through participation in clinical trials. Our goal is finding the best approach for your child. 

  • Oncology and genetic oncology (Genetic Oncology Clinic) clinics working together to understand each child’s cancer at the molecular level.​
  • Pharmacogenomics support analyzes how a child’s genetic makeup affects their ability to process treatments such as chemotherapy or pain medications.
  • Genetics services collaborate with experts worldwide to advance our understanding of rare disease and provide counseling on hereditary forms of cancer.
  • Adolescent and Young Adult (AYA) Program and other specialized services provide comprehensive support tailored to every age and need. 

 

A cancer diagnosis affects the whole family. We offer emotional support, resources and practical assistance to reduce uncertainty, preserve possibilities and help your child maintain their sense of childhood. Explore our Hematology and Oncology resources.

Our experts drive scientific advancement through research, education and collaboration with leading institutions. These efforts continuously improve care for patients here and everywhere.

For children with cancer, precision medicine can help identify targeted therapies and predict who may be at higher risk for side effects. For example, research at Cook Children’s includes a trial using trametinib in Langerhans Cell Histiocytosis (LCH) (study details) and a study screening for genetic susceptibility to chemotherapy‑induced heart and hearing problems.

Education is central to this program, from simple explanations of DNA and genetic variants to podcasts, conferences and a precision medicine certification to spread knowledge. Families are encouraged to ask questions, learn about their child’s specific genetic findings, and partner with the care team in every decision.

 

Understanding tumor genetics 

Cancer often begins when the “instruction book,” called DNA, inside cells develops changes, or variants. These changes can be small typos in a single letter, extra or missing pages, rearranged chapters, or invisible “on/off switches.” Ultimately, these changes affect the way the cells work and can influence how a tumor grows and responds to treatment.

 

Inherited and acquired changes

Some genetic changes are inherited and present in every cell from birth (germline), which can increase the risk for certain cancers. Other changes are acquired only in tumor cells during a child’s life (somatic), often driven by aging, environment, or random events and these are the main drivers of most cancers. 

Our program actively leads and participates in clinical trials and research studies focused on targeted therapy, ctDNA monitoring and reducing toxicities like cardiotoxicity and ototoxicity. Findings from these efforts have led to national and international conference presentations and publications that help shape how children with cancer are treated worldwide.

Your specialized care team

Our team includes specialists, researchers, case workers and more.

  • Andrea Fagerman
  • Diana Carrasco, MD (Clinical Genetics)
  • Danielle Miller, PharmD, BCPPS
  • Claudette Fraire, PhD
  • Megan Gibbs, BSN, RN, CPHON
  • Kelly Vallance, MD (Hematology-Oncology)
  • Amanda Kennedy, LCSW
  • Kaitlin Smith, MSN, RN, CPHON
  • Aly Anthony, MSN, RN, CPN
  • Reena John, MSN, RN
  • Heidi Trinkman, PharmD, BCOP
  • Anish Ray, M.D. (Hematology-Oncology)
  • Maya Derhake, MS, CGC
  • Sunshine Morales
  • Jason Wang, MD (Molecular Pathology)
  • Kim Nguyen, MSIT, MLS (ASCP)CM

Advancing personalized medicine for children

Our program helps guide care for a broad range of pediatric cancers and blood disorders. Our precision medicine team meets weekly to review test results and identify treatment options to update your child's personalized care pathway. This map outlines first-line treatments, targeted therapy options and relevant clinical trials. We ensure you leave your consult with a plan for both immediate and future care.

Precision medicine, or personalized medicine, customizes care by examining your child’s unique genetic and biological makeup. Through advanced tests such as molecular profiling and next-generation sequencing, we reveal details at the cellular level to guide therapy.

These efforts help us:

  • Select cancer treatments based on specific tumor features for optimal success.
  • Match medications to your child’s genetics for safer, more effective drug choices with fewer side effects.
  • Detect early signs of recurrence through sensitive blood tests, sometimes even before imaging can.

We use some of the most sophisticated tools available to analyze tumors and blood cancers for genetic changes. Our methods help us understand cancer's behavior and predict your child's response to treatment. Tailored services include:

  • Next-generation sequencing (NGS): We use NGS to analyze hundreds of genes from one sample, uncovering variations that affect cancer's response to treatment.
  • Pharmacogenomics: This testing evaluates how your child's genetics impact their responses to medications. It enables us to choose the safest and most effective therapies.
  • Genetic Oncology Clinic: Our Genetic Oncology Clinic supports children and families with inherited cancer risks. We provide genetic testing, counseling and ongoing care.
  • Measuring circulating tumor DNA: These sensitive blood tests detect trace cancer DNA, monitor treatment effectiveness and check for recurrence.
  • Molecular profiling: We examine genes and proteins to determine what drives a tumor's growth and inform targeted therapy choices.

Once your child's analysis is complete, our team develops a customized treatment plan that includes recommendations, clinical trial opportunities and supportive services. We share this mapping with your child's entire care team to ensure coordinated and informed care.

  • Efunkoya, T., Trinkman, H., Ray, A., et al. “Assessing the risk: predictive value of asparaginase assay levels for Calaspargase-Induced hypersensitivity in Pediatric patients”. Children’s Oncology Group, September, 2025.
  • Franz, C., Ray, A. et al. "Implementing Improvements in AYA Care for Vulnerable Populations". AYACC 2025 Conference. 
  • Grace, M., Kiley, M., Ray, A. “ Discontinuation of trametinib in LCH patients - a careful process”. Histiocyte Society Conference, September 2025
  • Wojciechowska, N., Ray, A. et al.“Class III MAP2K1 Mutations Do Not Respond to MEK Inhibition; Or Do They?”. Histiocyte Society Conference, September 2025
  • Shibu, C., Ray, A. “Development Of Thyroid Carcinoma As Second Malignancy In Patients With Neuroblastoma”. ASPHO, 2025
  • Kumar, S., Ray, A. et al. “Factors Affecting Radiation Induced Dermatitis In The Pediatric Population”. ASPHO, 2025.
  • Wojciechowska, N., Greer, C.,  Ray, A. et al. "ctDNA Monitoring In Neuroblastoma Highlights Disease Dynamics And Precision Oncology Challenges".  ASPHO, 2025 - oral presentation.
  •   Bolds, A., Collis, S., Ray, A. “Mediastinal Gray Zone Lymphoma Complicated By Nivolumab-Associated Pneumonitis In A Teenager”. ASPHO, 2025.
  •  Richards, L., Ray A., et al. “Treatment For Children With LCH Treated With Trametinib Have Sustained Remission”. ASPHO, 2025.
  • Zumwalt, L., Schluterman, H., Ray, A. “Role Of Medical Students In A Pediatric Oncology Precision Medicine Clinic”. ASPHO, 2025.
  • Richards, L., Ray A., et al. “Brentuximab Vedotin-Associated Encephalopathy: A Case Report”. ASPHO, 2025.
  • Wojciechowska, N., Diaz, M. Ray, A.“Children with Langerhans cell histiocytosis treated with MEK inhibitor, trametinib front-line and at relapse”. Histiocyte Society annual Conference, 2024. 
  • Wojciechowska, N., Ray, A. “Immunotherapy in Pediatrics”. ASPHO, 2024.
  • Wojciechowska, N., Murray, L., Aguilar, R., Ray, A. “Osteoblastoma: a case series”. ASPHO, 2024.
  • Wojciechowska, N., Ray, A. “Congenital multi-system langerhans cell histiocytosis responding to targeted therapy with trametinib”. ASPHO, 2024.
  • Keane, M., Wojciechowska, N., Ray, A.“Role of medical students in a pediatric oncology precision medicine clinic”. ASPHO, 2024.
  • Orr, K., Ray, A. “Creation of a tumor molecular testing registry: an update”. ASPHO, 2024.

Recent publications and presentations include:

  • Wojciechowska, N., Ray, A. et al. “Targeted Therapy in Pediatric Langerhans Cell Histiocytosis: Describing a Novel Strategy to Minimize Long-Term Exposure While Maintaining Efficacy”. Pediatric Blood and Cancer, 2025.
  • Wojciechowska, N., Ray, A. et al. “Class 3 MAPK Mutation in Langerhans cell histiocytosis and Trametinib—Reporting Two Contrarian Responses”. Pediatric Blood and Cancer, 2025.
  • Keane, M., Wojciechowska, N., Ray, A. et al. “Development and implementation of a pediatric oncology precision medicine clinic to enhance personalized approaches for diagnosing and treating pediatric solid tumors”. Oncology Research, 2025.
  • Naymagon, L., Roehrs, P., Ray, A., Kumar, A. et al. “Perspectives on the current diagnostic and treatment paradigms in secondary hemophagocytic lymphohistiocytosis.” Orphanet Journal of Rare Diseases, 2025.
  • Henderson, L., Abraham, R., Ray, A. et al. “Multidisciplinary approach to treating complex immune dysregulation disorders: an adaptive model for institutional implementation”. Frontiers in Immunology, 2025.
  • Kumar, S., Gonzalez, A., Farbo, D., Albritton, K., Ray, A.”The effects of proton and photon radiation therapy on the development of pediatric dermatitis”. Radiation, 2024.
  • Ray, A., Levitt, M., Efunkoya T., Trinkman H. “Precision Medicine in Acute Lymphoblastic leukemia”. Children, 2024. https://doi.org/10.20944/preprints202410.0191.v1
  • Zumwalt, L., Schluterman H., Ray, A. “Role of medical students in a pediatric oncology precision medicine clinic”. BMC Medical Education, 2024.
  • Zumwalt, L., Schluterman H., Ray, A. “Life-threatening lymphatic malformation with somatic activating NRAS mutation successfully treated with trametinib: a case study”. Journal of Pediatric Hematology/Oncology, 2025.
  • Chandrakasan, S., Ray, A. et al. “Emapalumab Treatment in Patients With Rheumatologic Disease-Associated Hemophagocytic Lymphohistiocytosis in the United States: A Retrospective Medical Chart Review Study”. Arthritis and Rheumatology, 2025. 
  • Wojciechowska, N., Burke, S., Ray, A. “MEK Inhibition in the Treatment of Congenital Langerhans Cell Histiocytosis: A Case Report and Review of the Literature”. Journal of Pediatric Hematology/Oncology, 2024.
  • Wojciechowska, N., Orr, K.,  Albritton, K.,  Heym, K., Vallance K., Ray, A. “Immune Checkpoint Inhibition in Pediatric Oncology Patients: A Single-Institution Experience”. Hemato, 2024.
  • Chandrakasan, S., Jordan, M., Ray, A. et al. “Real-world treatment patterns and outcomes in patients with primary hemophagocytic lymphohistiocytosis treated with emapalumab.” Blood Advances. 2024
  • Li D, Wang Q, Bayat A, et al. Spliceosome malfunction causes neurodevelopmental disorders with overlapping features. J Clin Invest. 2024;134(1):e171235. Published 2024 Jan 2. doi:10.1172/JCI171235
  • Layo-Carris DE, Lubin EE, Sangree AK, et al. Expanded phenotypic spectrum of neurodevelopmental and neurodegenerative disorder Bryant-Li-Bhoj syndrome with 38 additional individuals. Eur J Hum Genet. 2024;32(8):928-937. doi:10.1038/s41431-024-01610-1

We've developed a certification program for learners eager to deepen their understanding of precision medicine. This initiative spreads awareness about precision medicine tailored for pediatric care.

 

Access the program