Drug trial for rare childhood cancer lands in Montreal
Ste-Justine’s first site in Canada for promising experimental therapy
An Ontario family whose son has a rare childhood cancer will be travelling to Montreal, joining a handful of Canadian patients who are eligible for a promising but experimental therapy that until now was only available in the United States.
For Leslie and Ludger Hacault, whose son Marcus, 6, has been battling neuroblastoma for more than two years, the Montreal site is a blessing following two harrowing years in which their family life was turned upside down.
“We are so grateful,” Leslie Hacault told the Montreal Gazette. Marcus is the eighth of 10 children, and he needed specialty care, first in Toronto and then in the U.S. “It was hard to leave them,” she said of her other children.
“It means quite a lot to know that there are families that won’t have to go through what we did.”
Their ordeal began when Marcus complained of stomach pain. A scan in the emergency room of a Kingston hospital in January 2016 showed he had an abdominal tumour, and the cancer had already spread to his bones. He was diagnosed with a high-risk neuroblastoma, a cancer that has a statistical, five-year survival rate of about 40 per cent.
Marcus needed surgery first, then high-dose radiation and chemotherapy, followed by immunotherapy.
The family was divided for the better part of two years. While Leslie Hacault’s parents and her sister-in-law moved to Kingston to look after the children, Marcus started treatment in Toronto, accompanied by his parents.
“It was quite extensive,” Hacault recalled. “He had two bone marrow transplants. Not one, but two, to eradicate the disease.”
In March, the family got good news. A scan showed the disease was in remission. “It’s a big deal,” she said. “Some children don’t make it to remission.”
By then, Hacault, who was desperately searching the internet, had found a small Facebook group of parents. The high-risk cancer affects about 40 Canadians each year, usually children under age six. The social media group had found a clinical trial of an experimental drug known by its initials, DFMO.
DFMO is a repurposed medication that for decades has been used to treat African sleeping sickness. The trial is being conducted at a pediatric hospital in Grand Rapids, Mich.
The parents discussing the latest treatments available for neuroblastoma included Andrew Cutter of Toronto, whose son was among the initial handful of children worldwide — and the first Canadian — to complete a twoyear DFMO treatment course in Michigan.
“He’s doing fantastic now,” Cutter said of his son. “You wouldn’t know to look at him that he had front-line chemo and radiation therapy.”
Cutter was instrumental, along with other Canadian parents and a small group of dedicated pediatric oncologists, in bringing DFMO to Canada.
It gives parents a lot of hope, especially those who can’t afford to travel, Cutter said.
However, physicians caution that the drug is used as maintenance treatment. Only children whose cancer is in remission brought about by the initial frontline treatments are considered eligible for the DFMO trial. Patients take the medication twice a day for two years.
When physicians declared Marcus’s cancer in remission, DFMO was not yet available in Canada. Patients are supposed to start the treatment within one month of being declared in remission, Hacault said. “We couldn’t wait for it to come to Canada.”
So six months ago, the Hacault family began making the trek to Michigan. They were under the care of Dr. Giselle Sholler, pediatric oncologist at Helen DeVos Children’s Hospital, who pioneered the treatment. Sholler found that about 40 per cent of patients with high-risk neuroblastoma relapse within four years. In the initial study, less than 15 per cent of children receiving DFMO relapsed.
“We are thrilled to be able to offer our study to children across Canada and work with their clinical teams,” said Sholler, who was born and raised in Pointe-Claire.
In September, Marcus will transfer to the care of pediatric hematologist-oncologist Pierre Teira at Ste-Justine Hospital, the first Canadian clinical trial site approved to recruit young patients for the DFMO trial. It’s also available at the Montreal Children’s Hospital. The study sites are expected to expand to Laval and Toronto.
“What’s particular about this treatment is that the parents really pushed to start this in Canada,” said Teira, also assistant professor of pediatrics at the Université de Montréal. “The new treatment seems effective and is well tolerated. There’s very little toxicity. It’s much less toxic than the treatments they were getting before, and it’s targeted therapy. It blocks the way the cells produce energy to support their cancerous evolution.”
While the initial data seems promising, there weren’t enough patients to approve the drug outside of a clinical trial and so the U.S. Food and Drug Administration asked Dr. Sholler to expand the investigation into a bigger study and multiple centres, Teira explained. Hacault said she and her family are thrilled to be able to access the care in Montreal.