According to the American Childhood Cancer Organization, almost 13,000 children under the age of 21 are diagnosed with cancer annually in the United States. In recognition of September’s Childhood Cancer Awareness Month, SMA recently spoke with Joseph Pressey, MD, Associate Professor of Pediatrics at the University of Alabama at Birmingham, (UAB) and Director of UAB’s Developmental Therapeutics Program at Children’s of Alabama, about pediatric cancer and the state-of-the-art therapies that are resulting in groundbreaking treatment advancements.
About the Developmental Therapeutics Program
Established in 2006, the Developmental Therapeutics Program, a part of the Alabama Center for Childhood Cancer and Blood Disorders, is a partnership between the UAB Division of Pediatric Hematology/Oncology and Children's of Alabama. By partnering with patients and their families, the program researches the latest medical innovations and therapies related to pediatric cancers through the involvement of leading physicians, scientists and researchers.
Through its inclusion in the Children’s Oncology Group (COG) Phase 1 & Pilot Consortium, a leading group of 21 pediatric oncology programs in North America, the Developmental Therapeutics Program is able to provide patients with unique treatment opportunities not widely available. The primary mission of the Consortium is to conduct Phase 1 trials involving new therapeutic agents that have never been studied in children. “These agents have typically been found to be promising in adult cancers and often in preclinical work in pediatric cancers; however, these often represent the first trials in children,” Dr. Pressey explained. “We are offering them to children whose cancers are recurrent or refractory and have no known curative options. This is very select group of patients who have exhausted all proven therapies.” The primary goal of these trials is to determine how tolerable the agents are and to find the appropriate dosing in children. A secondary objective is to determine how well the agents work in pediatric cancers. “Admittedly, only a minority of patients have a documented response to Phase I therapies,” Dr. Pressey acknowledged, “But it’s exceptionally rewarding and exciting when a child who has no known curative options is exposed to a new agent and responds well to it.”
UAB/Children’s is involved in most of the COG’s nearly 100 active clinical trials, focusing primarily on Phase 2 and Phase 3 trials. “The Phase 1 program is [somewhat] of a separate entity because it is so specialized,” Dr. Pressey said. Currently, there are dozens of Phase 2 and Phase 3 trials open and UAB/Children’s, with at least 12 Phase 1 trials also taking place at this time.
In addition to its involvement with the COG Phase 1 & Pilot Consortium, UAB is a member of the Sarcoma Alliance for Research Through Collaboration (SARC) –a consortium focusing on connective tissue cancers. While SARC involves both adult and pediatric patients, Dr. Pressey noted that UAB/Children’s is one of the few institutions that participates as a pediatrics-only program. “We are able to offer a few studies specifically for sarcomas through that consortium,” he said, “And it is another opportunity to offer cutting-edge therapies to our patients from here in Alabama and from surrounding states.”
Innovative treatments are not only being offered in Birmingham, they are also being developed at UAB, with several new initiatives taking place at the university. Currently, there are projects underway that focus on rhabdomyosarcoma, osteosarcoma, and immunotherapy. “In all situations, we are taking findings in the lab and doing ‘bench-to-bedside’ type trials where we are initiating pilot studies to investigate findings that have been made here at UAB,” Dr. Pressey said. “We are developing some of our own ideas and discoveries that we hope to take to the larger pediatric oncology community once these are better characterized and proven to be worthy of further study.”
State-of-the-Art Treatment, In State
While Birmingham is often recognized as either the “Steel City” or the “Magic City”, many people are unaware that it is home to world-renowned oncology research and treatment. Sometimes people come in and say, “We have a diagnosis of cancer, so where do you need to send us?,” Dr. Pressey noted. “Of course, we want to match patients with the best therapy available wherever that might be; yet typically we find that with the programs such as Developmental Therapeutics offered at UAB/Children’s of Alabama that it is quite rare that a child needs to go elsewhere for therapy.”
Why Pediatric Oncology?
With myriad aspects of medicine and health care to consider, what drew Dr. Pressey to pediatric oncology? To him, the answer is simple. “It was an opportunity, as I saw it, to do something really meaningful for a population of patients who still desperately needed new therapies.” He further explained that while approximately 80% of children with cancer are ultimately cured, not only is there a substantial subset of patients who cannot be cured with existing therapies there is still much work to be done as it relates to life after treatment. “There is a significant proportion of patients who have long-term adverse effects from their therapy, despite having survived their cancer,” Dr. Pressey noted. “[With] these late effects from chemotherapy, radiation, or surgery, there is a huge frontier to identify less toxic therapies.”
The prospect of translating bench research findings to clinical studies also led Dr. Pressey to his chosen field. “The opportunity to translate scientific findings into cutting-edge clinical initiatives in pediatric oncology holds a lot of the appeal for me,” he said.
As a physician who spends his days actively researching and developing new treatments to fight cancer, Dr. Pressey stresses that the well-being of the patients always come first. “Being mindful of the journey our patients are going through is imperative,” he said. “While our goal is always to cure patients, we also recognize that it not always possible. When that is the case, we want to offer hope through experimental therapies but also be especially mindful of the well being and goals of the patients and their families.”
Other articles in the Focus on Childhood Cancer Series:
In recognition of Childhood Cancer Awareness Month