Clinical Trials: Translating Research to Benefit Patients

Pediatric Cancer Clinical Trials

The Pediatric Cancer Clinical Trials Office (PCCTO) was created in 2009 through a five-year, $5 million infrastructure investment from the MACC (Midwest Athletes Against Childhood Cancer) Fund, which supported staff, a translational component (the tissue bank at Children's Hospital of Wisconsin) and the development of a database. The PCCTO now comprises 14 individuals with the expertise needed to run all phases of clinical trials.

Michael Kelly, MD, PhD"We took advantage of this tremendous opportunity afforded by the MACC Fund to expand clinical research, as we had excellent clinical care providers and wonderful patients – but no infrastructure," says Michael Kelly, MD, PhD, (pictured right) associate professor of pediatrics (hematology/oncology) at MCW, director of the PCCTO and director of the Cancer Program at Children's Hospital of Wisconsin. The PCCTO is administered through the hospital and staffed with pediatric cancer specialists from the MCW Cancer Center.

The PCCTO undertakes three different types of clinical trials: treatment trials (exploring whether a treatment or device is safe and effective for humans), registry trials (using observational methods to collect uniform data on specified outcomes in a population defined by a particular disease) and supportive care trials (looking at ways to improve quality of life for cancer patients and survivors).

Dr. Kelly shares that there are more than 150 different types of pediatric cancers, and while most are more aggressive than adult cancers, the incidence is lower. Thus, there is a need to cooperate with other cancer centers around the country in order to build evidence by studying a large enough subset of patients to determine the best treatments. The main source of this collaboration is the Children's Oncology Group (COG), an NCI-supported clinical trials group of more than 250 institutions that comprise the world's largest organization devoted exclusively to pediatric cancer research.

MCW's PCCTO draws patients from southeast Wisconsin, northern Illinois and the Upper Peninsula of Michigan. More than 90 percent of patients are on one clinical trial, and greater than 70 percent are on multiple clinical trials (compared to about 10 percent of adults on clinical trials). During the past five years, the PCCTO has ranked consistently among the top 15 institutions in the COG by total enrollment of pediatric patients in clinical trials.

"We've been able to develop new therapies over the years by recruiting faculty with clinical trial expertise," Dr. Kelly explains. For example, MACC Fund Professor Jeffrey Medin, PhD, is focused on immunotherapies (prevention or treatment of diseases with substances that stimulate the immune response) for pediatric cancers. Michael Burke, MD, is the principal investigator (PI) on two clinical trials for relapsed leukemia. And Julie-An Talano, MD, and Monika Thakar, MD, each are PIs on clinical trials centered on post-hematopoietic stem cell transplantation. "This clearly is a differentiator for MCW. Only a handful of institutions in the COG possess teams that work on discovery through implementation and clinical trials around certain diseases – as we do," Dr. Kelly adds.

Joel Chmielewski and mother Kay
Three-year-old Joel Chmielewski (pictured here with his mother, Kay) has been participating in a clinical trial that adds two additional medications to his conventional leukemia therapy. Diagnosed at 16 months, Joel is in remission and completing treatment to prevent the cancer's recurrence.

"We have two examples of groups focused on discovery-to-clinical implementation for pediatric cancers: the high-risk hematologic malignancy group and the neuro-oncology group. These groups comprise clinical and lab scientists who work together to write grants, publish and recruit top talent," Dr. Kelly explains. "In addition, we are working toward a discovery-to-implementation model across our campus, focused on adolescents and young adults with cancer. Our pediatric oncologists collaborate with medical oncologists and clinical and bench researchers. This cross-campus approach is made possible by the geography of the Milwaukee Regional Medical Center, which is a tremendous benefit to our community and our patients."

– Maureen Remmel • Sara L. Wilkins

MCW Magazine: Clinical story MCW Magazine: Discovery story

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