Last Thursday, the British Columbia Ministry of Health announced that public funding for Brineura has been restored for 10-year-old Charleigh Pollock, who takes the medication to treat CLN2 Batten disease.
Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a group of hereditary neurodegenerative diseases that primarily affect children and young adults. Symptoms can include retinopathy, leading to blindness, ataxia and gait problems, drug-resistant epilepsy, cognitive decline, and early death.
In the United States, Brineura is the first FDA (Food and Drug Administration)-approved treatment for CLN2, a form of Batten disease caused by a deficiency of the TPP1 enzyme. Brineura (cerliponase alfa) works by replacing the missing enzyme to help slow the progression of the disease, rather than simply treating its symptoms. It is approved for use in children over three years old who show signs of the condition, according to the New England Journal of Medicine.
Earlier this year, after months of advocacy, public outcry, and the submission of new expert evidence from Charleigh’s family, the British Columbia Ministry of Health had initially decided to discontinue public funding for Brineura. This decision was made despite Charleigh’s family providing unpublished findings and expert opinions suggesting that Brineura was still effectively helping with her seizure management and quality of life.
Health Minister Josie Osborne had previously stated that the decision would stand, based on clinical criteria used by both provincial and national review panels, which determined that the drug was no longer effective at Charleigh’s current stage of the disease. Charleigh’s family, devastated by the initial ruling, had continued to advocate for their daughter’s treatment, arguing that the decision effectively took away her only lifeline.
After further review and continued pressure, the Ministry announced that funding for Brineura would be restored. The decision came after new expert evidence was considered, along with the impact that the medication has had on Charleigh’s seizure management and her overall quality of life.
The family, who lives in Langford, expressed relief and gratitude at the announcement, but they also pointed out that their ongoing fight is not just for Charleigh. They hope this victory will spark change for other families facing similar battles, and that the Canadian healthcare system will become more flexible in evaluating the real-world impacts of treatments for rare and progressive diseases.
While Charleigh’s immediate future looks brighter, her family remains committed to advocating for a more compassionate and nuanced approach to healthcare, one that values the opinions of families and medical professionals rather than relying solely on strict clinical criteria. They also hope this experience will inspire more conversations about better supporting rare disease patients, especially when the stakes are so high.
Sources:
Azpiri, Jon, and Courtney Dickinson. “Mother of Girl with Rare Condition ‘Very Grateful’ After B.C. Reinstates Coverage of $1M Drug.” CBC News, Canadian Broadcasting Corporation, 17 July 2025, http://www.cbc.ca/news/canada/british-columbia/ln2-b-c-funding-brineura-reinstated-1.7588180.
Gammaldi, Nicola, et al. ‘Integrative Human and Murine Multi-Omics: Highlighting Shared Biomarkers in the Neuronal Ceroid Lipofuscinoses’. Neurobiology of Disease, vol. 189, Dec. 2023, p. 106349, https://doi.org/10.1016/j.nbd.2023.106349.
Judd, Amy. “Drug-Funding Decision for B.C. Girl with Rare Disease Will Not Be Reversed.” Global News, Corus Entertainment, 11 July 2025, globalnews.ca/news/11285272/drug-funding-decision-bc-girl-rare-disease-not-reversed/.
Schulz Angela, et al. ‘Study of Intraventricular Cerliponase Alfa for CLN2 Disease’. New England Journal of Medicine, vol. 378, no. 20, Massachusetts Medical Society, May 2018, pp. 1898–1907, https://doi.org/10.1056/NEJMoa1712649.