Why I’m looking forward with hope for new therapies or even a cure
My strategy is to plan for the future while honoring today
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Living with paroxysmal nocturnal hemoglobinuria (PNH) has a way of teaching you to hold two very different emotions at the same time: gratitude for how far medicine has come, and hope for how much further it still needs to go.
Since my diagnosis in 2017, I’ve learned that PNH is not just a rare blood disorder — it’s a daily negotiation with fatigue, uncertainty, and resilience. It’s waking up some days feeling strong and capable, and other days like your body has quietly rewritten the rules without telling you.
When people hear “bone marrow transplant,” their reactions are usually filled with fear and awe. And honestly, I understand why. A transplant is often described as the closest thing to a “cure” that currently exists for PNH. But it’s not a simple decision or a guaranteed solution. It’s a major, life-altering procedure that comes with serious risks: graft-versus-host disease, infections, long recovery times, and the emotional toll of putting your entire life on pause.
For some patients, a transplant is absolutely the right and necessary choice. For others, especially those who are stable on current treatments, it’s a door one observes carefully, thoughtfully, and sometimes from a distance.
For me, the idea of transplant represents both hope and gravity: hope, because it offers the possibility of a life not shaped around infusions, lab results, and energy calculations; gravity, because it asks you to weigh current quality of life against the unknowns of a very intense medical journey. It’s not a decision made lightly, nor should it be.
The beautiful part of this story, though, is that PNH treatment has already changed so much in the last decade. Targeted therapies have transformed PNH from a life-threatening diagnosis into something many of us can actually live with, manage, and plan around. I’m here, working, building, dreaming, and advocating because of that progress.
So, transplant notwithstanding, will there ever be a cure? I truly believe so. Science is moving fast, with potential gene therapies, complement inhibitors, and a deeper understanding of bone marrow failure syndromes all pointing toward a future where a “cure” might not require such a heavy price. Maybe it will involve correcting the gene mutation that causes PNH at its source. Maybe it will look like a treatment that fully resets the system without replacing it. We don’t know yet, but I think we’re closer than we’ve ever been before.
PNH has taught me to live in hope without denying reality, and to plan for the future while honoring today. Whether the answer is transplant, a breakthrough therapy, or something we haven’t even imagined yet, I believe this: Our stories, voices, and survival are part of what pushes medicine forward. And that, in itself, is powerful.
Note: PNH News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of PNH News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to paroxysmal nocturnal hemoglobinuria.
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