New hope arises for children battling neuroblastoma as a clinical trial is about to sweep the nation. The C17 Council and Helen DeVos Children’s Hospital consortium, known as Beat Childhood Cancer (BCC), partner to bring an innovative clinical trial to Canada that will investigate whether a repurposed drug could prevent relapse in patients diagnosed with high-risk neuroblastoma who are currently in remission. The first clinical site will be at the Centre Hospitalier Universitaire de Sainte-Justine, Montreal, Quebec, and has been approved to commence recruiting patients.
This repurposed drug, 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 that projected positive results and led to a subsequent trial that further demonstrated possible patient benefit. From what history has shown, about 40 per cent of patients with high-risk neuroblastoma relapse within four years; and with less than 15 percent of children receiving DFMO relapse during the initial study, this sounds like a promising procedure.
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,” says 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,” says 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.”
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.
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, shars 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.”
Through the support of patient advocates like Andrew Cuttle, funding through Beat NB Foundation and partnerships with Helen DeVos Children’s Hospital’s consortium, there is renewed hope for Canadian patients and their families as pediatric doctors and oncology researchers strive for the ultimate goal: improving survival and quality of life for children with childhood cancers.