Beat Childhood Cancer consortium's clinical trial offers new hope to children in Canada

Aug 8 2018

Children in Canada who are battling neuroblastoma, a form of cancer, have been granted new hope toward a better prognosis and long-term survival. Helen DeVos Children's Hospital's consortium, known as Beat Childhood Cancer (BCC), announced today that through a partnership with The C17 Council, the organization's first Canadian clinical trial site, Centre Hospitalier Universitaire de Sainte-Justine, Montreal, Quebec, Canada, has been approved to begin recruiting patients for a pivotal clinical trial investigating the potential of a repurposed drug to prevent relapse in patients diagnosed with high-risk neuroblastoma who are currently in remission.

DFMO (eflornithine HCl) has been commissioned by the World Health Organization for decades in the treatment of patients with African sleeping sickness and has a robust safety profile for use in children. Through translational research, it was discovered that a common pathway in neuroblastoma tumors may be inhibited by DFMO. This discovery led to a Phase I clinical trial in a handful of patients. The results were promising, and led to a subsequent trial that further demonstrated possible patient benefit. Historically, about 40 percent of patients with high-risk neuroblastoma relapse within four years; on the initial study less than 15 percent of children receiving DFMO relapsed.

The current trial has enrolled 132 patients from around the world at centers in the United States. Now children in Canada will have easier access to this potentially lifesaving drug, making it more readily available to patients who do not have the means to travel to the United States for treatment.

"We are thrilled to be able to offer our study to children across Canada and work with their clinical teams," said Giselle Sholler, MD, chair of Beat Childhood Cancer and Haworth Director of Innovative Therapeutics at Helen DeVos Children's Hospital. "Collaboration and partnerships are essential in pediatric cancer to make a difference for our children."

"Parents and doctors in Canada are very excited to announce that the DFMO trial developed by the Beat Childhood Cancer group is now available in Canada for all children suffering from high risk neuroblastoma," said physician investigator for the study Pierra Eteira, MD, MSc, assistant professor of pediatrics at the University of Montreal. "This new treatment appears very promising and very well tolerated. As a pediatric oncologist, I'm convinced that this new collaboration with Dr. Giselle Sholler and her team at Helen DeVos Children's Hospital will help to cure children with neuroblastoma in Canada."

Andrew Cuttle, a father of a child who battled neuroblastoma who has recently completed this study traveling from Canada to Michigan for the past two years, and a key parent advocate who helped bring DFMO to Canada, shared the following: "What this means to families is monumental. As this clinical trial rolls out to more hospitals across Canada, families can make the choice to enroll without the barrier of financial burden, travel and time away from their families. It gives parents a voice, a choice and to most, a glass full of hope. We need to break down the borders when it comes to children and their rights to access some of the most promising treatments towards life threatening cancers."

The availability of DFMO through this study in Canada is already expanding, with five additional hospitals currently in the process of seeking approval to recruit patients as well. The data collected from patients enrolled in this trial will be presented to the U.S. Food and Drug Administration to create a compelling argument to make DFMO available to patients without the need to enroll in a clinical trial. Through the support of patient advocates like Andrew Cuttle, funding through Beat NB Foundation and partnerships with organizations like The C17 Council, hope for these patients and their families is reaching further every day.

BCC is a growing consortium of more than 40 research centers and children's hospitals headquartered at Helen DeVos Children's Hospital in Grand Rapids, MI that offers an international network of childhood cancer clinical trials. The mission is to act as a national and international collaborative effort of researchers, oncologists and family advocates to bring forward new therapies and technologies with the goal of improving the survival and quality of life of children with childhood cancers.