Sylvester Comprehensive Cancer Center Pairs Scientific and Clinical Excellence to Improve the Lives of Pediatric Cancer Patients
Summary
- Scientists across Sylvester Comprehensive Cancer Center continue to lead the way toward an even brighter future for children with cancer.
- Dr. Julio Barredo noted the expansion of the pediatric bone marrow transplant program, the introduction of cellular therapies and the implementation of the region’s only phase 1 clinical trials program as developments that improved pediatric patient outcomes.
- Dr. David Lombard found that SIRT5 plays a role in Ewing sarcoma, which mostly affects children and young adults, and is now working to develop small-molecule inhibitors of SIRT5.
When Daniel Armstrong, Ph.D., professor of pediatrics and director of the Mailman Center for Child Development at the University of Miami Miller School of Medicine, arrived in Miami in 1985, the survival rate for children with cancer was about 50% in the first year after diagnosis.
Dr. Armstrong got to work collaborating with other researchers and clinicians at Sylvester Comprehensive Cancer Center, part of the Miller School. He studied how cancer treatments affected patients’ developing brains, and he witnessed the growth of clinical trials to advance treatments.
Soon, he noticed his hospital rounds were taking longer.
“We were still seeing new patients, but my caseload was climbing exponentially because children were surviving,” Dr. Armstrong said.
Today, the survival rate is about 85% for all pediatric cancers, and many types are more than 90%, said Dr. Armstrong, now chair of the Sylvester Children’s Cancer Initiative, which is working to build on the cancer center’s strengths.
Faculty across Sylvester continue to lead the way toward an even brighter future for children with cancer.
“As South Florida’s only National Cancer Institute-designated cancer center, Sylvester is committed to weaving together paradigm-shifting research and patient-focused care that together improve outcomes for children in our state and beyond,” said Sylvester Director Stephen D. Nimer, M.D., professor of medicine, biochemistry and molecular biology, executive dean for research at the Miller School and the Oscar de La Renta Endowed Chair in Cancer Research.
The Value of Collaboration
Julio Barredo, M.D., credited with bringing alex’s place to life, has also seen incredible advances for patients in his 20 years at the cancer center. Dr. Barredo is the director of Sylvester’s Pediatric Oncology Program, chief of the Miller School’s Division of Pediatric Hematology-Oncology and Toppel Family Endowed Chair in Pediatric Hematology-Oncology. He noted the expansion of the pediatric bone marrow transplant program, the introduction of cellular therapies and the implementation of the region’s only phase 1 clinical trials program, which focuses on early-stage treatments.
Dr. Barredo experienced a shift in how clinicians specialize and collaborate. Everybody used to be a general oncologist, he said, but Sylvester is now home to an array of specialists.
These specialists don’t work alone. Sylvester partners with Holtz Children’s Hospital, where pediatric oncologists work with surgeons, radiation oncologists, radiologists and more. For retinoblastoma, one of Sylvester’s specialties, oncologists collaborate with ophthalmologists at Bascom Palmer Eye Institute.
“We changed the model to provide the best and most advanced care possible in a multidisciplinary way,” Dr. Barredo said. “Without a doubt, we provide the best pediatric cancer care in the region.”
Sylvester’s emphasis on collaboration extends beyond Miami.
A recent example of statewide partnership is the Sunshine Genetics Act Pilot Program, which unites researchers at Sylvester and the Miller School with experts across Florida to expand newborn testing for rare diseases, improve early intervention and build a national model for genomic medicine.
On a global scale, Sylvester is part of the Children’s Oncology Group, the world’s largest pediatric cancer research organization. Sylvester researchers not only learn from clinical trials around the nation, but also contribute their own advances, Dr. Barredo said.
Innovation Begins in the Lab
Sylvester’s excellence in patient care is intertwined with the science that enhances understanding of cancer and drives more effective, less toxic treatments.
One of Sylvester Children’s Cancer Initiative’s working groups focuses on opportunities in basic and translational sciences, which are essential for developing new, precision-medicine approaches, Dr. Armstrong said.
David Lombard, M.D., co-leader of Sylvester’s Cancer Epigenetics Program and professor and vice chair of clinical and translational research in the Miller School’s Department of Pathology and Laboratory Medicine, is investigating one new discovery.
Dr. Lombard historically studied aging and cancer, focusing on a protein called SIRT5. His team realized that SIRT5 also plays a role in Ewing sarcoma, which mostly affects children and young adults. They’re now working to develop small-molecule inhibitors of SIRT5.
While the research is still in its early stages, Dr. Lombard said his team has an eye on the future.
“I think one of the real strengths of Sylvester is the way clinicians and basic researchers can work hand in glove to identify new treatments and deploy them to the clinic,” he said. “Certainly, that’s our goal, too.”
Antonio Iavarone, M.D., deputy director of Sylvester, director of the Sylvester Brain Tumor Institute and professor of neurological surgery, biochemistry and molecular biology at the Miller School, is in the process of translating basic science into clinical treatments for pediatric brain tumors.
Brain tumors that fall under a single diagnosis may be different from a molecular standpoint, Dr. Iavarone said. Advanced technologies allow scientists to understand why a particular tumor developed and what genetic alterations are allowing it to grow.
Dr. Iavarone’s lab is using these tools to prospectively provide real-time therapeutic opportunities for patients. His team is collaborating with Nicklaus Children’s Hospital in Miami to receive pediatric brain tumor samples, profile them and match the tumor with the therapies that are most likely to be effective.
“What we’re trying to do right now is make sure that we can provide these opportunities, possibly to the entire community of pediatric cancer patients in Florida,” Dr. Iavarone said.
Beyond Established Therapies
Sylvester researchers also take a science-driven approach to improving therapies that are already in regular use.
For example, Glenn Flores, M.D., FAAP, the Miller School’s chair of pediatrics and senior associate dean of child health, highlighted that Sylvester and the Department of Pediatrics have the longest continuously accredited pediatric stem cell transplant program in the region that offers cellular and gene therapy. He said the newest opportunities involve modifying a patient’s own stem cells.
“The applications of cellular therapy are so important for children because they provide effective and curative options for cancer, chronic hematological disorders, immune deficiencies, inherited disorders and viral infections,” said Dr. Flores, also a professor of pediatrics and public health sciences and the George E. Batchelor Endowed Chair in Child Health at the Miller School. “Newer approaches are more targeted and have fewer complications than traditional stem-cell transplant techniques, and the demand and applications for such cellular therapies continue to rapidly expand.”
Now that more and more children are surviving cancer, Sylvester also has a growing survivorship program to help patients throughout their lives.
Influence for Patients Everywhere
These projects and more are building momentum at Sylvester. Dr. Iavarone, who came to Sylvester two years ago, said he was drawn by the exceptional opportunities.
“The best physicians and scientists want to go where there are also other scientists,” he said. “So we have started to create a very successful community.”
A key investment in the community is the Kenneth C. Griffin Cancer Research Building.
Momentum at Sylvester has ripple effects for pediatric cancer patients everywhere. Sylvester helps train physicians from around the world, and advances in care that work in Miami can work around the globe, Dr. Armstrong said.
“The reason for doing anything that we do is to see the children and families who seek care at our institution—or in institutions that are influenced by what we do—have better outcomes, have lives that are fulfilling, have as few limitations as they possibly can,” he said.
Tags: alex's place, Batchelor Children's Research Institute, brain tumors, clinical trials, Department of Pediatrics, Division of Pediatric Hematology-Oncology, Dr. Antonio Iavarone, Dr. Daniel Armstrong, Dr. Glenn Flores, Dr. Julio C. Barredo, gene therapy, Mailman Center for Child Development, Newsroom, pediatric cancer, pediatric stem cell transplantation, pediatrics, Sylvester Brain Tumor Institute