As medicine continues to progress toward personalized medicine, innovative targeted therapy methodologies show promise to improve outcomes for children experiencing cancer. Through a partnership with Florida International University (FIU), the Helen & Jacob Shaham Cancer & Blood Disorders Institute at Nicklaus Children's Hospital offers a groundbreaking cancer treatment approach that wields the power to change cancer care forever.

“In treating cancers, some people think that more is better — that it will be more aggressive and more likely to kill the tumors,” says Maggie Fader, MD, pediatric hematologist/oncologist with the Sarcoma & Solid Tumors Program at Nicklaus Children's Hospital. “What we've seen in the lab doesn't prove that.”

Targeting a Second Chance

The study's approach, pioneered by FIU cancer researcher Diana Azzam, provides a clear path toward targeted therapy.

The process to identify appropriate targeted therapy through the trial includes the following:

• A clinician withdraws a cancerous blood or tumor sample on-site and immediately submits the sample to the FIU laboratory.
• FIU researchers place the sample in an environment that promotes natural cell growth, where cells are cultured for a few days.
• While in this environment, the cells are exposed to more than 120 FDA-approved medications, some that typically treat cancer and others that do not. Researchers may test multiple medications simultaneously in order to find the most successful combination of therapies to treat the specific cancer.
• Full results are available within 14 days, providing a roadmap for clinicians to follow in treatment planning.

“What's unique about this trial is how rapidly we can turn around results,” Dr. Fader says, “and use those results to direct our therapy.”

Reduced Risk, Improved Results

Diagnosed at age three with acute myeloid leukemia (AML), Logan initially underwent the standard treatment routine. His disease soon entered remission, only to return 14 months later. Instead of launching into a standard relapse regimen, which can have some variations, Dr. Fader talked with the boy's parents about the possibility of enrolling Logan in the hospital's ongoing clinical research with FIU. Logan's parents were excited about the possibility and agreed to enroll their son.

By enrolling Logan in the clinical trial, Dr. Fader and her team learned what specific medications had the greatest effect on Logan's tumor before beginning therapy. This allowed the team to give individualized therapy that met Logan's very specific needs, without the risk of additional side effects or future complications that may have accompanied additional, unnecessary medications.

Based on laboratory testing, FIU researchers identified a combination of medications best suited to treat the specific cancer affecting Logan. Dr. Fader administered the combination therapy, and Logan achieved a positive outcome. Others have realized similar results, with 83 percent of children enrolled showing improvement, according to study results published in Nature Medicine.

“Thanks to Logan's bravery, his clinical care team at Nicklaus Children's and FIU's research team, we are advancing pediatric cancer treatment for all kids in Florida and across the region,” says Marcos Mestre, Chief Medical Officer at Nicklaus Children's.