Professor's research, a lifesaving option for pediatric cancer patients

A little girl has late-stage Rhabdomyosarcoma, an aggressive cancer that forms in the muscles. A board of specialists in emergency medicine, radiology, oncology, surgery, pathology, infectious diseases and pharmacy unite to understand the treatment options and decide on a plan. This is standard course of action when a child is diagnosed with cancer— an all-hands-on-deck approach to provide the best opportunities for the health and survival of a child diagnosed with cancer. 

“The specialists come together to present the best treatment. But, unfortunately, there is not a lot of pediatric cancer research being conducted, which means there are little-to-no options currently available when the standard course of treatment doesn’t work. That typically leaves the families and physicians without additional options,” said Diana Azzam, research assistant professor at the Robert Stempel College of Public Health & Social Work and recipient of a Live Like Bella Initiative research grant.

To better understand the process a child and family undergo after a cancer diagnosis, Stempel College — in partnership with the Live Like Bella Childhood Cancer Foundation, which was funded by the Florida legislature —hosted its first-ever "Mock Tumor Board" to place elected officials in the shoes of decision-makers at the hospital when it comes to treating a child with cancer. The exercise also showcased Azzam's research, still in clinical trials.

The board was guided in their understanding of the case and decision making by Raymond Rodriguez-Torres, Bella’s father and chairman of the Live Like Bella Childhood Cancer Foundation. The “doctors” on the board included Rep. Mario Diaz-Balart, Sen. Anitere Flores, Rep. Cindy Polo and their staff members, along with representatives from the offices of Rep. Debbie Mucarsel-Powell, Rep. Donna Shalala, Sen. Marco Rubio and Sen. Rick Scott. 

Rodriguez-Torres presented the case, following much of the same process his daughter Bella underwent when she was diagnosed with cancer at the age of four. On the screens, he presented some of Bella’s radiology scans as part of the historic exercise. The elected officials and staff members, read assigned roles and made treatment decisions and assigned a specific medical specialty. The emotions in the room were palpable, all-knowing the end of the case: Bella ultimately passed away in 2013 at the age of 10. 

Then, Rodriguez-Torres presented what he called, “a new hope,” another option when children and their families have run out of options. 

Led by Azzam, in collaboration with Daria Salyakina, Dr. Ziad Khatib, Dr. Maggie Fader, and other doctors from Nicklaus Children’s Hospital, the clinical trial supported by the Live Like Bella Foundation is showing promising results. Able to test a tumor against hundreds of medications, Azzam and her team are finding interventions that are helping physicians treat cancer patients and leading to remission.  

“We are working to give families more options, options that make it possible for pediatric cancer patients to survive cancer with the best-individualized treatment that can get to the core of the tumor,” Azzam said.

“Now, we need the support of our officials to ensure that this trial can continue so that we can see these children thrive. The results are fast, in only five days, we can understand the best course of action,” Azzam added.

The goal is that one day, these results will be available during real tumor boards— and physicians can be even better equipped to make the best decisions for their patients from the start. 

While it is too late for Bella, her legacy lives on through the foundation – working to find lifesaving options for the more than 15,000 children who are diagnosed with cancer each year in the United States and, eventually, help children diagnosed around the world.

“The worst nightmare of a parent is hearing that they have run out of treatment options and Dr. Azzam’s work is finding those additional options and giving hope to families. Thank you to all who took part in such an important exercise and informative session. Together we will make childhood cancer a treatable and curable disease,” Rodriguez-Torres said.