State funding propels local pediatric cancer research

St. Petersburg is home to a comprehensive, innovative pediatric cancer research facility that received a $7.5 million renewable state grant to bolster cutting edge care. 

The funding, announced Monday, will support Johns Hopkins All Children’s Cancer & Blood Disorders Institute. Its director, Dr. Cassandra Josephson, subsequently outlined the hospital’s ambitious plans for the much-needed money. 

She expects the institute to eventually receive $37.5 million from the five-year Florida Cancer Connect Collaborative Research Incubator Grant program. The funding will support All Children’s proposal, dubbed “Florida’s Catalyst for Childhood Cancer Cures.” 

“There have been a lot of patients locally that have to leave because we just don’t have a study open here, or we don’t have the types of studies they need,” Josephson told the Catalyst. “We’re going to have more clinical research coordinators and clinical research nurses to run more studies – that is right here in our backyard. 

“If your place is not offering clinical research studies, then you’re really not on the cutting-edge of what you can offer your patients.” 

Dr. Cassandra Josephson, director of the Johns Hopkins All Children’s Cancer & Blood Disorders Institute, and an oncology professor at the Johns Hopkins University School of Medicine.

That innovative work is already occurring at All Children’s. The hospital is Florida’s only Pediatric Early Phase Clinical Trials Network site, as designated by the global Children’s Oncology Group. 

All Children’s is also the state’s top-ranked pediatric hospital for the third consecutive year and No. 37 for child cancer nationally, according to U.S. News & World Report. Josephson said a key focus of the grant program is ensuring “no patient has to leave the State of Florida to get care.” 

Florida’s pediatric population ranks third nationally, underscoring the need for cutting edge childhood cancer care. Josephson credited the governor, his wife and state lawmakers for championing the incubator initiative, designed to accelerate those efforts at All Children’s, Nicklaus Children’s Hospital in Miami, Nemours Children’s Hospital in Orlando and Wolfson Children’s Hospital in Jacksonville. 

Josephson stressed that clinical research improves patient outcomes, which is why some cancer survival rates surpass 90%. However, studies typically require several years of specialized treatment and monitoring, and funding remains an issue. 

While cancer is the leading cause of death by disease among children after infancy, only about 4% of National Cancer Institute Funding supports pediatric research. 

“The return on investment for the private sector is just not there,” Josephson said. “Pharmaceutical companies do not have a drive to go and develop things that are going to help a very small population of people – even though that small population of people may be their child.” 

All Children’s will use the funding to:

• Expand access to and participation in innovative clinical trials;
• Develop a statewide interactive database portal to match patients with appropriate trials;
• Create a statewide network to move research discoveries into the clinical trial phase more rapidly;
• Study how to enhance the immune system to fight cancer;
• Generate, test and deploy next-generation gene and cellular therapies to combat pediatric cancer. 

“The private sector is not going to do this for you,” Josephson said. “If we don’t step in, who’s going to make sure we find all these cures for cancers?”

All Children’s has invested millions of dollars in collecting and analyzing anonymous patient data. The hospital operates the state’s only pediatric biorepository, which stores biological specimens for research. 

The Cancer & Blood Disorder Institute offers leading therapies for sickle cell disease and hemophilia. Bone marrow transplant techniques improve outcomes for children with leukemia and lymphoma. 

All Children’s was also the first pediatric hospital in Florida certified to provide CAR T-cell therapy, a life-saving treatment for children with acute lymphoblastic leukemia or lymphoma. Josephson said the hospital will use the funding to manipulate patient cells, “infuse it back into their body so it can wake up their immune system and kill the cancer. And they won’t reject it.”

The grant will also support the development of innovative delivery systems, including nanoparticles, that carry gene-regulating RNA from the bloodstream to the brain. If successful, All Children’s could “turn on and off some of the brain tumors,” Josephson said. 

She noted that All Children’s offers studies “right when they walk in the door to learn more about the biology of the cancer.” Some include experimental drugs rather than traditional chemotherapy. 

“If we can toggle the immune system, we can treat things in a much more humane way than even giving chemotherapy,” Josephson added. “If you can wake a person’s own immune system up to help fight the cancer, that’s a game-changer, as far as side-effect profiles and second cancers. 

“Survival is really important – not just to survive the cancer, but to survive the treatment and what happens next in your life. If we make those discoveries here, it can help everybody in the whole world.” 

The hospital is also creating the Florida Collaborative Childhood Cancer Predisposition Program: Protecting Children and Families Through Early Identification and Prevention. A user-friendly, state-of-the-art registry will aid those efforts.