Breakthroughs in Children’s Brain Cancer Care

Logan (center), surrounded by representatives from FIU, Nicklaus Children’s, Live Like Bella and elected officials to celebrate the recent research collaboration.

Logan (center), surrounded by representatives from FIU, Nicklaus Children’s, Live Like Bella and elected officials to celebrate the recent research collaboration.

When Logan’s parents learned that his cancer had returned after a brief remission, it was a devastating time for the family. Just two years earlier, during the COVID-19 pandemic, the family celebrated the news that Logan was cancer free. But joy turned to heartache when they learned they would need to battle acute myeloid leukemia a second time.

Fortunately for Logan, now 8, his hematologist/oncologist is Dr. Maggie Fader of Nicklaus Children’s Helen & Jacob Shaham Cancer & Blood Disorders Institute and KIDZ Medical. Dr. Fader was collaborating as the clinical principal investigator with a team of researchers led by Diana Azzam, assistant professor of environmental sciences at the FIU Robert Stempel College of Public Health and Social Work at Florida International University.

The team has developed a revolutionary functional precision medicine approach, published in April in Nature Medicine, that targets cancer by combining genetic testing with a new way to test individual drugs on tumor samples. This approach was used successfully for the first time to guide the treatment of relapsed hard-to-treat pediatric cancer patients, resulting in 83 percent of the participating children – including Logan – showing improvement.

After his second round of treatment and participation in the clinical trial, Logan was finally able to ring the bell in the hospital’s hematology­ oncology unit to celebrate the end of his treatment journey.

“Logan is a courageous boy,” said Dr. Marcos Mestre, chief medical officer at Nicklaus Children’s Hospital. “Thanks to his bravery, his clinical care team at Nicklaus Children’s including his attending physician Dr. Maggie Fader, FIU’s Diana Azzam and their research team, we are advancing pediatric cancer treatment for all kids in Florida and across the region.”

Funding for the research was provided by the Florida Department of Health Live Like Bella Pediatric Cancer Research Initiative. Seed money for the research was provided by STOP! Children’s Cancer of Palm Beach County.

About the Shaham Cancer & Blood Disorders Institute

In April, Nicklaus Children’s announced a $15 million gift from South Florida philanthropists Helen and Jacob Shaham to further elevate world-class pediatric cancer care and research through the hospital’s cancer and blood disorders institute. In recognition of the gift, the institute now proudly bears the name, the Helen & Jacob Shaham Cancer & Blood Disorders Institute.

The gift will elevate pediatric cancer care and research so no child ever has to leave the state for treatment and enhance the hospital’s standing as a leading cancer care provider for children through investments in groundbreaking research focused on new cures, treatments and diagnostic procedures.

New Approach for Devastating Brain Tumors

In other new developments, Nicklaus Children’s Hospital in 2023 became only the third site in North America involved in a Phase I clinical trial studying the role of a new technology called low-frequency focused ultrasound (LoFU) in the care of children with a lethal type of brain tumor that forms within the brainstem.

Diffuse intrinsic pontine glioma (DIPG), also known as diffuse midline glioma (DMG), is an aggressive tumor that occurs within the brainstem and affects approximately 300 children annually, most of them between the ages of 5 and 9. Despite advances in treating other forms of brain tumors in children, DIPGs remain the most lethal brain tumor with a median survival timeframe of less than one year.

“We are constantly seeking new treatment options to advance the care of children with this devastating diagnosis,” said Dr. Toba Niazi, co-medical director of the Nicklaus Children’s Hospital Brain Institute and director of the hospital’s Division of Neurosurgery. “It is our hope that, through this clinical trial, the low-frequency focused ultrasound will temporarily open the blood-brain barrier around the affected area of the brain and allow chemotherapy to gain direct access to the tumor and provide children with a chance for a cure.”

Nicklaus Children’s Hospital, through its Brain Institute and the Shaham Cancer & Blood Disorders Institute is one of only a few centers in the nation with extensive experience in caring for children with complex brain tumors.

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