By Daniel Casciato

Pediatric oncology is undergoing a rapid transformation, driven by precision medicine, advanced technologies, and collaborative research. At the forefront of this evolution is the Nicklaus Children’s Helen & Jacob Shaham Cancer & Blood Disorders Institute, where innovation is reshaping how childhood cancers are diagnosed, treated, and managed long term.

“We are proud at Nicklaus Children’s Helen & Jacob Shaham Cancer & Blood Disorders Institute to be a leader in pediatric cancer care,” says Matthew A. Love, president and CEO of Nicklaus Children’s Health System. “Our specialists provide state-of-the-art medical and surgical care for more pediatric cancer patients than any other facility in South Florida, leading the way through investments in groundbreaking research focused on new cures, treatments and diagnostic procedures.”

One of the most impactful advances currently improving outcomes is the Ex Vivo Drug Sensitivity Test, a breakthrough research tool that evaluates how a patient’s tumor cells respond to a wide range of FDA-approved therapies. This approach allows oncologists to select treatments based on real-time tumor response rather than trial and error. Alongside genetic therapy, immunotherapy, molecular tumor analysis, and targeted treatments, this innovation enables truly individualized care plans.

Precision medicine plays a central role in the Institute’s oncology program. “At Nicklaus Children’s, our medical experts use a holistic and personalized approach to treat each patient’s diagnosis,” says Dr. Ziad A Khatib, medical director of the Hematology/Oncology program at Nicklaus Children’s.“This includes each one of our patients undergoing genetic testing of their cancer, which helps identify inherited gene mutations that could increase the risk of developing certain diseases.”

In more complex or resistant cases, Ex Vivo Drug Sensitivity Testing evaluates tumor cells against more than 120 FDA-approved drugs. “Results are sent back to our oncologists with a rank of the effectiveness of each drug against the cancer,” Dr. Khatib says. “The goal is to aid our doctors in finding the best available treatment quickly before having to run down a list of drugs that might not work as effectively.”

This approach is backed by compelling clinical evidence. “The study’s results, published April 11, 2024, in the peer-reviewed medical journal Nature Medicine, found that 83% of patients who received guided treatments showed improvement,” Dr. Khatib notes.

Reducing the physical burden of treatment is particularly critical for pediatric patients. To that end, the Institute has adopted therapies designed to minimize long-term side effects while maintaining effectiveness. “We offer targeted therapy and immunotherapy that’s designed to attack cancer cells more selectively than conventional chemotherapy,” Dr. Khatib says. “This often reduces damage to healthy tissues and leads to fewer, more tolerable side effects.”

Additional protective measures include cardioprotection strategies for children receiving anthracyclines, helping shield the heart from chemotherapy-related damage. For patients undergoing bone marrow transplants, photobiomodulation therapy is used to lessen the severity of mucositis, a painful and common side effect. “As a way to lessen the severity of this particular side-effect, Photobiomodulation is used with bone marrow transplant patients which makes the therapy process more tolerable and less painful,” Dr. Khatib explains.

Technology also continues to elevate diagnostic and surgical precision. In 2015, Nicklaus Children’s Hospital became the first pediatric hospital in Florida to acquire the da Vinci® Xi™ Surgical System. Housed in the Kenneth C. Griffin Surgical Tower, the robotic platform allows surgeons to perform minimally invasive procedures with enhanced control and accuracy.

“With Intraoperative MRIs, our surgeons are able to see in real-time, high-resolution imaging that allows them to more accurately identify tumor boundaries and adjust their approach for safer, more complete removal,” Dr. Khatib says. The Institute also employs magnetic resonance–guided focused ultrasound therapy, offering select patients a noninvasive option to treat brain tumors with pinpoint accuracy.

Research and collaboration remain essential to advancing pediatric oncology. “Many of our oncologists are also actively researching new and emerging treatments for different types of tumors, the underlying biology of these cancers, along with supportive care and survivorship best practices,” Dr. Khatib says. Through participation in national and global research networks such as the Collaborative Network for Neuro-oncology Clinical Trials (CONNECT) and the National Experimental Therapeutics Consortium (NEXT) Consortium, physicians collaborate across institutions to accelerate discovery and share promising therapies.

As survival rates improve, long-term care has become a growing priority. The Institute’s Survive & Thrive survivorship program provides comprehensive follow-up for childhood cancer survivors. “Within the program, survivors meet with dedicated specialty physicians, nurse coordinators, psychologists, school coordinators, social workers and registered dietitians to ensure these children have the best quality of life as they grow into adulthood,” Dr. Khatib says.

Through innovation, research, and compassionate care, Nicklaus Children’s continues to redefine what is possible in pediatric cancer treatment—ensuring that children and families have access to world-class care without ever leaving South Florida.

For more information, visit nicklaushealth.org.