The Latest in Pediatric Cancer Clinical Trials

Chair, Department of Pediatric Oncology
Tuesday, October 17, 2017 - 12:48pm

Clinical trials are a key reason why childhood cancer treatments and survival rates have improved significantly in recent years. Today we cure more than 75 percent of children with cancer – a rate much higher than for adult cancers. However, to make major advances, we need patients to participate in these trials. While pediatric cancer clinical trials have a 60-70 percent participation rate compared with only 5-10 percent for adults with cancer, we strive to continue growing that number. Parents should recognize that most clinical trials in pediatric cancer are about fine-tuning care. We do not test new therapies in children unless there is a proven track record for those therapies in adults. The purpose of pediatric clinical trials is to make treatments more precise and less toxic.

Oral Chemotherapy Treatments

We’re thrilled to offer a few exciting new studies here at Roswell Park. One, which is unique to Roswell Park, will look at adherence to oral chemotherapy for children with Acute Lymphoblastic Leukemia (ALL). We know from previous research that missing chemotherapy doses more than 10 percent of the time is a leading risk factor for relapse. Many ALL patients are on oral chemotherapy for more than two years, so it’s understandable that they may miss doses — but we want to reduce the number.

Thanks to generous donations, Elizabeth Gage-Bouchard, PhD, and I were awarded a grant from the Roswell Park Alliance Foundation to study parent and caregiver compliance for oral chemo treatments. We purchased special medicine bottles with caps that track dosages. We’re also diving into health literacy and social connectedness. We want to see what parents and caregivers understand about oral chemotherapy and find out if compliance is stronger when they have a network of support.

Immunotherapy for Hodgkin Lymphoma

We’re also ready to launch a new immunotherapy trial for pediatric and young adult patients with Hodgkin lymphoma. This trial will be the first of its kind in many ways. I collaborated with Children’s Oncology Group & EURONET to launch the study, which will be the first transatlantic immunotherapy trial for pediatric Hodgkin lymphoma. Only a handful of sites will offer this, so I am excited that we will be on the forefront of some exciting research. This is the first pediatric immunotherapy study looking at checkpoint inhibitors.

Reducing Treatment-Related Toxicities for ALL Patients

We’re collaborating with Dana-Farber Cancer Institute on a trial aimed at improving treatment for ALL, the most common type of cancer in children, and we are one of only seven sites in Canada and the United States to offer the study. Historically, we have classified patients into two or three main groups, and treated them accordingly. Now we are trying to categorize patients’ risk of relapse based on biological markers, so we can provide more personalized treatments.

It Takes a Village

Pediatric cancer researchers have led the way in multicenter collaborative clinical trials. Childhood cancer researchers at NCI-designated cancer centers, like Roswell Park, are often held up as the best example of how to best facilitate cancer clinical trials. Because childhood cancer is fairly rare, we have to come together and pool our efforts. Our strong network of cancer centers and academic medical centers that care for children that has made it possible for us to improve prognosis for our youngest patients.