The Cure Was Built for Children. I Am an Adult.

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I am an adult undergoing gene therapy for sickle cell disease, and I almost did not get the chance. Not because the science was not ready. Because the system was never built for me.

Sickle cell is treated as a childhood disease. That is not a figure of speech. The funding lives in pediatrics. The specialists train in pediatrics. The research centers, the comprehensive programs, the doctors who actually understand this disease — most of them treat children. It makes a kind of sense on paper. Sickle cell is diagnosed at birth. The hardest years for many families are the early ones. So the money and the expertise gathered there and stayed there.

Then we grow up.

Aging out is not a metaphor

Somewhere between eighteen and twenty-six, most of us age out. We age out of the pediatric hematologist who knew our history by heart. We age out of our parents’ insurance. We age out of the children’s hospital that had a sickle cell team, and we land in an adult system that, in most of the country, has almost no dedicated sickle cell infrastructure at all.

The numbers are not gentle about this. When the transition from pediatric to adult care goes badly — and it usually does — hospitalizations roughly double. There is no comprehensive national network of adult sickle cell providers. Surveys have found that only a small share of physicians under fifty feel comfortable treating this disease, and a striking number of adults with sickle cell get their care from neither a primary doctor nor a hematologist. We have, in the plainest terms, no medical home.

I have lived inside that gap. I know what it is to walk into an emergency room in a crisis that would put most people on the floor and be looked at like a problem to be managed rather than a patient to be treated. I know what it is to be quietly labeled a drug-seeker while I was actually undermedicating myself, because the opioids they offer do not really touch this pain and bring a whole set of harms nobody wants to manage afterward. I know what it is to have my care handed to residents who were taught this disease wrong, or taught to distrust the people who have it. I know what it is to be threatened, in the same visit where I came in for help, with the loss of my pain medication and the removal of the people advocating for me.

That is not a bad day. That is the baseline. And it changes how you behave. You stop going. You wait longer than you should. You manage at home in ways no one should have to. And here is the part that matters for what came next: that avoidance, which is a completely rational response to being treated that way, becomes a line on a form that shuts you out of the very research meant to save you.

How the cure left adults behind

The trials that produced these gene therapies did extraordinary things. I do not want to take one ounce away from that. But look at who they were built to enroll. The pivotal studies capped their ages. They required a documented history of severe crises over a set number of years — a criterion that quietly assumes you felt safe enough to go to the hospital every time, so there would be a record. They required organ function healthy enough to survive the chemotherapy conditioning, which is easier to have when you are young and the disease has had fewer years to do its damage.

Put those together and you have a set of doors that many adults simply cannot walk through. Not because we are not sick enough. Because we are too sick, too scarred, too burned by the system to have the clean paper trail the studies wanted. The people with the most accumulated damage — the adults — were the least likely to qualify. So we were underrepresented in the trials, and now we are last in line for the treatments those trials produced.

I spent years trying. The trials that led to approval are no longer enrolling. I reached my treatment on the commercial side, as an adult, in a country with almost no adult sickle cell infrastructure to reach it through. I am one of the very few adults to do that so far. The first commercial patients to make the news were a twelve-year-old and a seventeen-year-old, treated at children’s hospitals. That is not a coincidence. That is the shape of the system showing through.

Why I am documenting all of it

I am writing this down as I go — the science, the fear, the small wins, the parts that still hurt — for a specific reason. Patients are almost never in the room when our cures are designed. We are not there when the trial criteria get written. We are not there when a therapy comes to market and someone decides, on our behalf, whether it can actually be reached. The result is a set of treatments that are real and remarkable and, for a huge share of adults, functionally out of reach.

I can help change that, and not only as a patient. I have spent twenty-five years as an executive working on access and equity inside large institutions. I know how these decisions get made, and I know how to sit at the table where they are made. So I am doing two things at once. I am living this, and I am documenting it, so the next adult does not have to wonder whether the door exists. It does. It is just hidden, and it is guarded, and it should not be.

There is one more thing I want to say plainly, because it is mine to say. The way we define a “cure” for this disease often rests on a single number in the blood. I understand why. But I am a person who meets some of those thresholds and still has crises, still has daily pain, still ends up in the hospital. My own doctors cannot fully explain it. I am not making a claim about anyone’s therapy. I am raising the question the science raises for someone like me: what does a threshold mean when a patient reaches it and still suffers. Patients can help answer that question. We should be asked.

If you build in this world

If you work in this field — a company bringing one of these therapies to patients, an advocacy organization, a hospital finally building adult services — I want to talk. Not to complain. To help. The most useful thing you can do with a story like mine is put it to work: on an advisory panel, in a program design, in the room where the next set of criteria gets written.

Adults with sickle cell have been an afterthought in the research, in the treatment, and in the design of the cures meant to save us. That can change. I am living proof the door exists. Let us make it wider.

— Dr. Dédé Tetsubayashi

If this resonated, subscribe. I will keep writing this as it unfolds. And if you are an adult with sickle cell trying to find your own way to care, you are not imagining how hard it is — and you are not alone in it.