Approximately 15,000 children age 19 and younger develop cancer every year. Although the five-year survival rate has increased for those patients in recent decades, the effects of treatment can last a lifetime for some patients.

“The one thing that has created progress not just in cancer survival but in quality of life over time has been a hard push toward including patients in clinical trials as often as possible,” says Maggie Fader, MD, Director of the Sarcoma and Solid Tumor Program at Nicklaus Children's Hospital. “That's why our department is committed to enrolling as many of our patients as possible in clinical trials, even if a child has a good prognosis. It may be that we know they have a 95 percent chance of success, but they have a 40 percent chance of having late effects, and this new trial might taper down those late effects.”

This long-standing commitment to research is why the Nicklaus Children's Cancer and Blood Disorders Institute remains one of the premiere destinations for pediatric cancer treatment in Florida and the U.S. Most importantly, this dedication has led to multiple partnerships that provide access to life-changing clinical trials and advanced treatment protocols.

Children's Oncology Group: A Vital Collaboration

Collaboration is at the core of the program's research, starting with a longstanding membership in the Children's Oncology Group (COG), the largest consortium for pediatric cancer research.

“As a member institution, we try to open the majority of the active protocols at our facility, so our kids have access to every trial,” Dr. Fader says. “Through COG, we can offer clinical trials for common and uncommon pediatric cancers, including treatment for rare sarcomas, neuroblastoma, rhabdomyosarcoma, leukemia, lymphoma and brain tumors.”

Nicklaus Children's is currently participating in approximately three dozen COG clinical trials, but that's only the beginning.

“In addition to participating in clinical trials, our COG affiliation has opened the door to establishing other collaborations with like-minded organizations dedicated to the care of children's cancer,” says Thomas Temple, MD, pediatric orthopedic-oncologic surgeon at Nicklaus Children's and professor of orthopedic surgery and pathology at the University of Miami Miller School of Medicine.

Leading Research Efforts Into Pediatric Brain Tumors

The Cancer and Blood Disorders Institute also partners with the Collaborative Network for Neuro-oncology Clinical Trials (CONNECT), which focuses on high-risk brain tumors, including:

• Atypical teratoid rhabdoid tumors
• Diffuse intrinsic pontine glioma
• High-grade gliomas
• High-risk medulloblastoma

CONNECT, which comprises a global network of pediatric cancer centers, conducts pilot studies to evaluate adding new therapies to established treatment protocols, working in tandem with phase I consortia and large clinical trial groups. This program can help assess the potential uses of novel agents in small groups of patients without the extensive resources needed for phase 2 and 3 trials.

The Cancer and Blood Disorders Institute is also part of the Beat Childhood Cancer Research Consortium, formerly known as the Neuroblastoma Medulloblastoma Translational Research Consortium.

Advances in Personalized Medicine

The laboratory of the Cancer and Blood Disorders Institute includes hundreds of patient cell cultures for screening.

Nicklaus Children's is further delving into personalized medicine through a research affiliation with Florida International University.

“We recently opened a trial for personalized medicine that uses samples of a patient's tumor for molecular profiling to search for mutations or fusions that might contribute to that patient responding better to a targeted therapy,” Dr. Fader says. “The study also includes ex vivo drug sensitivity testing of the tumor to see whether certain agents or combinations of agents exhibit positive activity against the tumor and may have better success than standard treatment protocols.”

Lifesaving Research

Affiliations with COG and other groups help patients today and in the future.

“I had one young patient who had a pathologic fracture from an osteosarcoma, and scans showed pulmonary metastasis and other sites of disease,” Dr. Temple says. “After chemotherapy through a COG protocol, we were able to remove her tumors with novel surgical techniques and expand her lower extremity with a magnetized implant. Now she's in college, doing extremely well and able to walk normally.”

The oncology team has also successfully used new therapies with compassionate-use FDA approval.

“Over the last few years, we've been able to create individualized treatment plans for patients, using drugs that were not available commercially for pediatric patients,” Dr. Fader says. “These patients were not doing well on standard-of-care treatments. These drugs made a huge difference for them, eradicating most, if not all, of their tumors.”

Clinical Trials Are Critical

In addition to offering the potential for improved prognosis in patients with devastating forms of pediatric cancer, clinical trials through COG and other consortia have provided crucial lifelines for South Florida families.

“If patients don't have to leave home to access trials, that improves their quality of life,” Dr. Fader says. “It also allows parents to stay close to their communities and continue to work while providing their kids with the same opportunities available at larger cancer centers.”

“My hope for the future is that people like me are out of business,” Dr. Temple says. “We hope all this research will lead to innovations that allow us to cure cancer without the difficult side effects.”

To learn whether our current clinical trials and other research protocols might benefit your patients, email our physician liaison.