Kitamaat toddler's life saved thanks to clinical trials at B.C. Children's Hospital
Emmett Doolan was born with a rare brain tumour. She received treatment support through clinical trials held at B.C. Children's Hospital. Photo by B.C. Children's Hospital Foundation
Source: Vancouver Sun
Written By: Denise Ryan
Emmett Doolan, a 2½-year-old of Haisla and Nisga’a heritage from Kitamaat village, was diagnosed before birth with hydrocephalus, a buildup of fluid in the brain. After travelling to Vancouver for further tests, a scan showed a black mass sitting on her brain.
She wasn’t expected to live.
It was devastating news for her moms, Lena and Lana Doolan.
“At two days old, she had her first brain surgery,” said Lena Doolan. “Seven days later, she was diagnosed with diffused midline glioma, stage 4.”
A biopsy showed a unique genetic alteration in her brain tumour. She was one of just three children to have been diagnosed with that cancer.
At two months old, Emmett was approved for a clinical trial of a form of chemotherapy called Larotrectinib.
Thanks to that clinical trial, and the clinical trials super hub, a new program at B.C. Children’s Hospital that supports access to clinical trials for children when there is no standard treatment available — and facilitates delivery of those treatments across B.C. and the Yukon — Emmett is back home in Kitamaat with her family.
Lana and Lena Doolan credit clinical trials held at B.C. Children’s Hospital with helping their daughter Emmett. Photo by B.C. Children's Hospital Foundation.
Emmett is able to remain on the clinical trial as the medication is taken orally, and be home with her family.
While the treatment is not a cure, Emmett is hitting all her milestones and confidently chattering in Nisga’a, Haisla and English.
“She’s thriving,” said Doolan.
The hospital clinical trials super hub came about, in part, due to the resolve of Dr. Quynh Doan, a pediatric emergency physician and senior executive director of B.C. Children’s Hospital Research Institute.
“Clinical trials save lives,” said Doan.
They also require infrastructure, safeguards, expertise, and adherence to stringent Health Canada regulations.
“That makes it very difficult for investigators to embark on a clinical trial of their own. We have to have resources and infrastructure to get them done efficiently,” said Doan. “If we are not equipped, they go elsewhere to conduct their clinical trials, and our kids lose out.”
Typically, teams are assembled for different trials, “starting from scratch each time,” said Doan. Once the clinical trial is over, those experts move on to other jobs.
Her dream was to create a permanent super hub for clinical trials at the hospital, “so no child will have to travel out of province to access lifesaving treatment, and no child will die waiting for one.”
Dr. Quynh Doan at B.C. Children’s Hospital. Photo by Jason Payne /PNG
Doan said it became clear if they wanted more access to clinical trials for desperately sick children, they would have to create something new, and fund it themselves.
“The government did not prioritize this, but as a community we came together” said Doan.
In 2023 and 2024 the Crystal Ball, the annual B.C. Children’s Hospital Foundation fundraiser for research and care, raised over $8 million for the super hub, while other community efforts, including private donations and $2 million raised at the foundation’s For Children We Care Gala, have put the project within striking distance of its $12 million goal.
The super hub will house trials, and eventually expand, so those same clinical trials can be delivered to children throughout the province and the Yukon. While many children with leukemia and cancer already receive treatment through clinical trials, the goal is to expand access to clinical trials for other rare pediatric diseases and conditions, said Doan.
Most clinical research focuses on adults, leaving children as therapeutic orphans, in part due to issues of ethics and consent, but also because the market for pediatric drugs is not attractive or lucrative enough for some pharmaceutical companies to invest in trials.
Doan knows too well the pain parents feel when their children need treatment. Her own son, now an adult, had a serious health challenge as a child. There was no pediatric treatment, and no clinical trials were available to him.
After he turned 18, he was able to access the treatment he needed, said Doan.
Lana and Lena Doolan, with daughter Emmett, credit B.C. Children’s Hospital for clinical trial. Photo by B.C. Children's Hospital Foundation.
Some kids are not so lucky.
“From the moment your child gets sick, as a parent you feel so much guilt and anxiety. You are constantly wondering, how do I fix my child? When you are told there is nothing that can be done, and you have to watch it progress is a feeling of failure. It’s redeeming as a parent, and as a scientist to be able to facilitate something like this.”
Doan said that although the super hub was her dream, it was by no means a solo project.
“This is coming together because the community has responded so positively: my teammates at B.C. Children’s Hospital Foundation, my colleagues, the kids and families we work with. This is a shared accomplishment.”