Many of you may have followed the story of Charleigh Pollock on social media and in the news. I wanted to take this opportunity to share a snippet of my insight over recent months, as shared recently on Facebook.
It’s been three weeks since the Government of British Columbia, Canada, overturned its decision and reinstated life-sustaining treatment for Charleigh. Honestly, I’m still processing the overwhelming events of the past few weeks and months. I have tried - and failed - several times to put it in words (and those who know me, know that I am rarely one lost for words).
This journey has been one of the most emotionally charged and deeply personal advocacy efforts I’ve ever been part of. When the initial decision to discontinue funding for Charleigh’s enzyme replacement therapy (ERT) was announced by the BC Government in February, it felt impossible to comprehend. Looking at Charleigh, and the life, energy and joy radiating from this kid - there was never a question - Charleigh continues to thrive on this treatment.
Our job was clear – as patient advocates, we needed to reach out to the decision-makers, clarify the facts and undeniable, compelling clinical evidence, and have her treatment funding reinstated without delay. Not just for Charleigh and her family. For every life affected by CLN2 Batten disease, whose future depends on access to this life-changing treatment.
Photo: Jori and Charleigh at a recent Brineura infusion day.
Continues from the newsletter here...
To be clear, this treatment, known as Brineura or Cerliponase alfa, is not a cure. It is, however, the only approved disease-modifying treatment currently available for CLN2 or any form of Batten disease. Our global research community is working tirelessly toward gene-based therapies that aim to halt the disease - and they are making impressive progress. But right now, Brineura enables something precious and priceless: a longer, better quality of life for most, and broader, meaningful benefits that we are learning more about as our clinical experience and empirical, scientific evidence grows.
Behind the scenes, we rallied hard for Charleigh. Charleigh’s parents, Jori and Trevor, showed breathtaking, gutsy courage, perseverance, and strength—the superhuman kind that reaches deep long after the tank hits ‘empty’. As a mother, I was profoundly moved watching Jori pour everything she had into this fight for her daughter—and for all the children who may follow. And not that I ever needed a reminder, but these Batten parents… just. wow. They never stop amazing me. These families inspire and energize me in my professional and personal life every single day.
On July 4, we met with the BC Health Minister in her parliamentary office in Victoria BC, to respectfully and professionally present the facts of the case and, what was eventually to be, the pivotal emerging clinical evidence on the benefits of Brineura. We had denials on every count - in February, June and again following this meeting - but we refused to give up.
And then came the letter.
It’s hard to ignore the collective voice of 15 of the world's leading Batten disease clinicians and researchers, who together have provided treatment to the vast majority of CLN2 patients in the US. Their clear and unified opposition to the B.C. Government’s decision, backed by clinical experience and scientific rationale was finally heard (link to letter below ) - and on July 17, Charleigh’s family received a call from the Minister to say her funding for treatment had been reinstated.
That moment of sheer relief and exuberant joy was shared by many—Charleigh’s parents Jori and Trevor, their partners, Lori Brown (BDSRA Canada), Andrew McFadyen (The Isaac Foundation) and the countless family, friends and supporters who stood together in solidarity. We told Charleigh’s story again and again until it could no longer be ignored.
The reversal didn’t come easily. It happened because people stood up, spoke out, and refused to accept injustice. And it doesn’t end there – there is work to do now to ensure reimbursement criteria are formally updated, and that no family goes through this same excruciating uncertainty again.
It’s a win for Charleigh today—but more than that, the events of these past few months exemplify exactly why patient advocates do the work we do. This journey stands as a powerful testament to the strength of the Batten community, the impact of the patient voice, and what’s possible when advocacy is grounded in collaboration, compassion, and evidence-based truth. Above all, it reflects our unwavering belief that every child is worth fighting for.
To everyone who stood with us—thank you. Your voice mattered. Your support made a difference. Together, we’ve shown the world what can be achieved when we refuse to give up.
And most of all, to sweet Charleigh—thank you for allowing us to walk alongside you on this part of your journey. It has been our greatest honour and privilege.
PDF Letter to BC Minister for Health
Read the full press release here: https://bdsrafoundation.org/batten-disease-organizations.../
Photo: Lori Brown (President Canadan BDSRA), Jori Fales (Charleigh's mum) and Dr. Ineka Whiteman (BDSRA Australia & BDSRA Foundation U.S.)