We Thought It Was Okay

There was a time when testing on children in orphanages was considered acceptable. Not in secret. Not in the shadows. Out in the open, documented, published, justified. At places like Willowbrook Hepatitis Study, children with developmental disabilities were deliberately exposed to disease so researchers could study progression. Elsewhere, in the Tuskegee Syphilis Study, treatment was withheld so the natural course could be observed. Entire groups of people were used to answer scientific questions because they were accessible, because they didn’t have a voice, because someone decided the knowledge mattered more than the individual. And the most unsettling part is this: the people doing it did not think they were doing something wrong. They believed they were advancing science, that the benefits would outweigh the risks, and that it was justified.

History didn’t agree. And guess who stood up against these unethical treatments. The fight for justice was carried by mothers and caregivers who refused to stay silent forcing a system built to hide harm into the light, though for many, it came too late. Sound to familar. We are on repeat asking for the same thing in ultra rare and rare diseases.

Some like to believe we are past that now. Pointing to ethics boards, consent forms, oversight committees. Referencing frameworks like the Belmont Report and the Nuremberg Code as proof that we have evolved. And in many ways, we have. What happened decades ago would not be allowed today in the same form. But evolution in ethics is not a finish line. It is a moving boundary, and every generation sits inside its own version of “this is acceptable.”

I think about that a lot lately. Not in a theoretical way. In a very real, very personal way. My own daughter, along with 13 other children, were placed on placebo in a drug trial. Then I met terminal children placed on placebo as well. And I keep coming back to the same question: who decided this was the best method? Because I have watched what happens when a child with a progressive disease is placed on placebo. I have watched time pass that does not come back. I have watched stability questioned, access delayed, and decisions made far from the people living the outcome. And I keep asking the same question: are we sure this is okay?

This is where things get uncomfortable. Because today’s system doesn’t look like the past. There is no obvious villain. No one is intentionally causing harm. The language is careful. The intentions are stated clearly. The goals are aligned around safety, rigor, and evidence. But harm does not require bad intent. It only requires a system that allows it.

Placebo, in many settings, is scientifically clean. It creates a clear comparison. It satisfies a standard that has been used for decades. It makes the data easier to interpret. But that same logic has been pushed to its edge before, and it is still being pushed now. There have been trials where patients were taken into operating rooms, sedated, cut open, and never given the treatment, sham surgeries designed to preserve the integrity of the data. And today, in trials for Huntington’s disease, including gene therapy programs like AMT-130, patients undergo procedures under anesthesia where the scalp is opened and the skull is drilled, but the therapeutic step is withheld. These are long, invasive procedures, sometimes lasting ten hours or more, carrying real risks infection, bleeding, complications yet the patient may receive no active treatment. The system calls this rigor. The patient experiences it as sacrifice. Patients are told this is the only way toward FDA approval.

But in progressive, irreversible conditions, especially in children, the equation changes. Time is not neutral. Disease progression is not reversible. And “waiting to see” is not without consequence. We know more now than we did twenty years ago. We have biomarkers. We have real-world data. We have AI. We have longitudinal patient tracking. We have the ability to measure change in ways that didn’t exist before. And yet, in some cases, we are still asking the same question in the same way: what happens if we give some children nothing?

History has a pattern. It does not judge people by what they intended. It looks at what was allowed. And it asks, later, with more clarity than we have in the moment: could we have done better?

I believe we are repeating the past. I do believe we are standing in a moment that will be looked back on. A moment where science is moving faster than policy. Where patients are speaking louder than systems are used to hearing. Lived experience is challenging long-held assumptions. And that tension, that discomfort, is not a failure. It is the signal. Every major shift in research ethics has started the same way: someone looked at what was being done and said, this doesn’t feel right not because they had all the answers, but because they understood the stakes.

So maybe the question isn’t whether we are ethical. Maybe the better question is whether we are willing to evolve before history forces us to. Because one day, someone will look back at this moment, and they will decide whether we saw it clearly or whether we accepted it because it was standard.

And I think about that a lot.