Sylvie Brandicourt knew the shadow of illness loomed large. Her father succumbed to Charcot’s disease, and several family members share a rare, inherited form of this devastating neurodegenerative illness. So, when diagnosed in 2019, she wasn’t entirely surprised. She’d already adapted her home in Boën-sur-Lignon accordingly. The recent decision by France’s High Authority for Health (HAS), however, sent shockwaves through her: the green light for a treatment she’d been receiving for two years was denied. This therapy, administered monthly via spinal injections at the CHU of Saint-Étienne, was developed by an American laboratory and targets a protein responsible for neuronal degeneration.
Sylvie is a living testament to its success. “There’s no question about it, the disease has stabilized for the past two years. I continue to do the same things I always have,” she shares, a vibrant 63-year-old who still drives and enjoys riding her electric tricycle for miles. While she acknowledges the need for careful exertion, relying on her wheelchair or walking aids when necessary, she knows the threat of amyotrophic lateral sclerosis (ALS), the medical term for Charcot’s, remains ever present. But compared to the painful decline she witnessed in her father, diagnosed in the 1980s, her progress is remarkable. “He was completely bedridden within five years,” she remembers, offering a stark contrast to her own experience.
“After five years, I’m still active. Morally, knowing that we gain precious years of life and quality of life makes a world of difference,” she says with resolute hope. So when the HAS decision arrived, “the world crumbled,” Sylvie confesses, her thoughts turning to her two children and grandchildren. Authorities cite insufficient proof from studies demonstrating the drug’s effectiveness, developed by Biogen, which argue it doesn’t address a significant unmet medical need.
But Sylvie and the ARSLA, an organization dedicated to ALS research, are fighting back. ARSLA boasts over fifty individuals benefiting from this treatment, including Sylvie, who became a beacon of hope for many. Over 46,000 signatures have poured into a petition addressed to President Emmanuel Macron, a desperate plea urging him to prioritize the needs of those suffering from this debilitating disease.
“The hope is within reach, within grasp, and France, formerly a leader in rare diseases, is turning away,” the ARSLA petition proclaims. Sylvie, unwavering in her determination, reached out to every parliamentarian in the Loire region. While some responded swiftly, others remained silent. Yet, Senator Jean-Claude Tissot has pledged to raise the issue during a question-answer session with the government.
Interview: The Editor of Time.news vs. Sylvie Brandicourt on the Impact of ALS Treatment Denial
Editor: Welcome, Sylvie! Thank you for joining us today and sharing your story with us. Your experience with the denial of your treatment must be incredibly challenging. Could you begin by telling us a bit about your journey with ALS and what the past few years have been like for you?
Sylvie: Thank you for having me. My journey began in 2019 when I was diagnosed with ALS, which is also known as Charcot’s disease. It runs in my family, so I had a feeling it might be a possibility, but the diagnosis still hit hard. In the last few years, I made adjustments to my lifestyle and my home in Boën-sur-Lignon to accommodate the illness. The treatment I had been receiving via spinal injections had made a significant difference in my day-to-day life—I was able to maintain my independence and continue doing things I love, like driving and riding my electric tricycle.
Editor: It sounds like you’ve shown incredible resilience in adapting to your condition. Can you explain the impact of the treatment that was denied by France’s High Authority for Health?
Sylvie: Absolutely. The treatment I was on targets a specific protein responsible for neuronal degeneration, which is crucial for individuals with ALS. For me, it stabilized the disease, and I felt that I could manage my symptoms well. The denial of this treatment felt like a cruel twist of fate after two years of stability and progress. It’s difficult to come to terms with the fact that something that has worked so effectively for me is no longer available, especially considering the implications it has for my quality of life.
Editor: That must be incredibly frustrating. What has the response been from the medical community and your support network regarding this decision?
Sylvie: The response has been mixed. My doctors at CHU of Saint-Étienne were just as shocked and disappointed as I was—many were advocating for the continued use of the treatment. On a community level, my support network has been wonderful. We are all trying to raise awareness about the challenges facing ALS patients when it comes to treatment access. Many people don’t realize how critical these decisions are, and we’re hoping to garner support from the public and health authorities to revisit this decision.
Editor: It’s so important that patients’ voices are heard in these discussions. Given your experience, what message do you hope to convey to others who may be facing similar challenges with ALS or other rare diseases?
Sylvie: I hope to convey the importance of resilience and advocacy. Patients must advocate for their own health and treatment options. It’s crucial to stay informed and connected with your healthcare team and support network. I also want to emphasize that communication and understanding from health authorities are essential. We are not just statistics; we are individuals with dreams, lives, and loved ones. Every decision impacts our ability to live our lives fully.
Editor: That’s a powerful message, Sylvie. As a final thought, what do you think the future holds for ALS research and treatment, especially in light of your experience?
Sylvie: I remain cautiously optimistic. There are ongoing research efforts around the world, and new treatments are being developed. However, I believe it’s vital for authorities to recognize the lived experiences of patients when making decisions. We need more collaboration between researchers, medical professionals, and patients to find effective treatments and ensure they are accessible. My hope is that through sharing my story, it sparks dialogue and brings about change.
Editor: Thank you so much for your time today, Sylvie. Your insights are invaluable, and your advocacy is inspiring. We hope to see positive developments in ALS treatments moving forward.
Sylvie: Thank you—it’s been a pleasure. I appreciate the opportunity to share my story and raise awareness about ALS.