Nicklaus Children’s Hospital Advances Pediatric Cancer Treatment with Targeted Therapy
Nicklaus Children’s Hospital, in collaboration with Florida International University, is pioneering a targeted therapy approach for pediatric cancer, offering personalized treatment plans based on rapid laboratory testing. This innovative method, which tests over 120 FDA-approved medications on patient samples, aims to improve outcomes by identifying the most effective treatment combinations within 14 days.
As medicine progresses towards personalized care, Nicklaus Children’s Hospital is at the forefront of utilizing targeted therapy to enhance treatment outcomes for pediatric cancer patients. In partnership with Florida International University (FIU), the hospital's Helen & Jacob Shaham Cancer & Blood Disorders Institute is implementing a groundbreaking approach to cancer care.
This innovative treatment strategy begins with the extraction of a cancerous blood or tumor sample from the patient, which is then sent to the FIU laboratory. Here, the sample is placed in an environment conducive to natural cell growth for several days. During this period, the cells are exposed to more than 120 FDA-approved medications, including those not traditionally used for cancer treatment.
Dr. Maggie Fader, a pediatric hematologist/oncologist at Nicklaus, emphasizes the uniqueness of this trial in its ability to rapidly turn around results. "What’s unique about this trial is how rapidly we can turn around results, and use those results to direct our therapy," she stated. Full results are available within 14 days, providing clinicians with a detailed roadmap for treatment planning.
A notable example of this approach's success involves a 3-year-old patient with acute myeloid leukemia. After initial standard treatment led to remission, the cancer returned 14 months later. Instead of proceeding with a standard relapse regimen, Dr. Fader recommended enrolling in the hospital’s ongoing clinical research with FIU. Through this trial, the team identified a combination of medications specifically effective against the child’s tumor, leading to a positive outcome without unnecessary side effects.
This targeted therapy approach represents a significant advancement in pediatric cancer treatment, offering hope for more effective and personalized care. By leveraging rapid laboratory testing and a wide array of FDA-approved medications, Nicklaus Children’s Hospital and FIU are setting a new standard in the fight against childhood cancer.

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Nicklaus hailing targeted therapy for young cancer patients
miamitodaynews.com · Jan 8, 2025
Nicklaus Children’s Hospital, in partnership with FIU, offers a personalized cancer treatment approach. By testing over ...