Over a dozen Batten disease experts from the United States have penned a letter calling on the BC government to restore funding for Charleigh Pollock's life-changing medication.The nine-year-old girl had public funding for Brineura discontinued by the BC NDP in June, and her family has since had to rely on GoFundMe to pay for her bi-weekly infusions.."We write to you as clinicians and researchers from the US-based Batten Disease Centers of Excellence Program and US Batten Disease Clinical Research Consortium," the thirteen MDs wrote. "Collectively, we have cared for the majority of US patients with CLN2 disease treated with enzyme replacement therapy cerliponase alfa (Brineura)."The doctors explained that, "prior to 2017, the late-infantile form of CLN2 disease was universally fatal, with no disease-modifying treatment available," but that, "ERT has demonstrably prolonged survival, slowed progression, improved seizure control, and enhanced quality of life."."Drawing from our collective and unparalleled expertise in CLN2 disease, we strongly oppose the decision to withdraw funding for life-sustaining ERT from Charleigh Pollock and call for a review of the current discontinuation criteria," they declared. "We are concerned by the clinical and scientific justification employed by the BC Ministry in their decision to terminate treatment coverage."The group went on to provide a detailed medical justification for the aforementioned declaration, breaking down each — in their view — erroneous aspect of the BC NDP's decision..'BEYOND EVIL': BC NDP refuses to reinstate funding for nine-year-old girl's life-changing medication."The question of when to discontinue life-sustaining therapy is a profoundly difficult one," they added. "We urge an evidence-based approach, acknowledging the lack of data on the use of M/L scores to discontinue cerliponase alfa, as well as the existing data on the long-term benefit of cerliponase alfa on seizures, survival, and quality of life. The clinical judgment of the treating physician is essential, as previously voiced by clinical experts and caregivers."Health Minister Josie Osborne is set to comment further on the matter in the coming days.
