Charleigh Pollock’s mother says she hopes private funding will get her daughter through the summer months, after the province cut off coverage
A 10-year-old Langford girl with a rare terminal disease is scheduled for a privately funded infusion of a life-prolonging drug at Victoria General Hospital on Thursday, after the province cut off coverage last month.
“We’re very relieved that she’s going to be getting the medication that she needs, and that she’s only missed one dose so far,” Jori Fales, mother of Charleigh Pollock, said Monday.
For six years, Charleigh has taken the drug Brineura, which costs about $800,000 a year, to slow the progression of Batten disease (CLN2), a fatal neurodegenerative disorder. Provincial coverage was discontinued on June 19, and her last bi-weekly infusion was July 3.
Several fundraising drives are underway for Charleigh, includingan online fundraising pagepage that has raised $253,000 of its $500,000 goal, with donations ranging from $5 to $10,000.
Island Health has offered the child’s regular bi-weekly infusion date of July 17 at Victoria General Hospital for the privately funded treatment, which takes several hours.
“There’s a lot of emotions today — foremost is gratitude for the community and for them seeing the value of Charleigh’s life and seeing how much more life she still has to live,” said Fales. “My other emotion is anger towards the B.C. government for making this uninformed, inhumane decision that is directly going to affect my daughter’s life.”
Fales said the family can’t pay privately for infusions in the long term, “so now we just feel like a ticking time bomb.”
Fales said Charleigh’s family and medical team believe the drug is improving the child’s quality of life. They have said in the past they would elect to stop the drug if, as the province’s rare disease program experts have maintained, it would not provide “further benefits” for Charleigh.
Coverage decisions under B.C.’s Expensive Drugs for Rare Disease program are based on the advice of a committee of independent experts using clinical criteria established by Canada’s Drug Agency.
The agency said Charleigh met the discontinuation criteria, but in the same report, it noted “evidence is limited regarding advanced disease and when to stop treatment.”
Given that discontinuation criteria — based on motor and language skills — are evolving and Charleigh is the only child in the province with the disease, Fales said she can’t understand why the province is sticking to its decision to end coverage of the drug.
The lifespan for an untreated child with Batten disease is 10 to 12 years.
“I’m very upset about having less time with her, but I’m more so concerned about what her quality of life is going to look like without the medication that has been keeping her out of pain and seizures,” Fales said.
Private funding will hopefully get Charleigh through the summer months, said Fales, adding she has no idea what to do next.
“We flew in experts from around the world. We’ve shown [the province] new research. They had everything to do the right thing, and they still chose to take away the medication from her.”
On July 4, Charleigh’s parents met with Health Minister Josie Osborne, along with neuroscientist Dr. Ineka Whiteman, Canadian Batten Disease Support and Research Association president Lori Brown and Andrew McFadyen of the Isaac Foundation, who helped the government bring Brineura to B.C.
Dr. Angela Schulz, senior physician at the department of pediatrics and adolescent medicine at the University Medical Centre Hamburg-Eppendorf, had said she was available for the meeting but was not contacted.
Schulz is updating the assessment tool used by Canada’s Drug Agency because she says it is no longer an appropriate way to assess disease progression in the mid-to-later stages of CLN2 Batten disease.
As for the names of experts the province made available for media to contact on the drug-discontinuation decision, Whiteman said her team couldn’t find a single publication they were named on with respect to Batten disease research.
“None of those so-called experts were involved in any of the research, and they’re not known to any of the Batten disease community,” said Whiteman, head researcher for the Batten Disease Support and Research Association in the U.S. and Australia.
“So it’s disturbing, it’s deeply disturbing that the minister has stood her ground and used the CDA review as a scapegoat to not go and consider the world experts who maintain that this is an unethical decision.”
Osborne said while the province’s drug review experts did not speak personally to some of the international Batten disease experts that Whiteman and others have suggested, they did review publications from those experts.
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