Dear President Donald Trump,
My name is Kelly Stacy. I am a mother of four sons, two of whom, Elijah and Kai, are still living with Duchenne muscular dystrophy, a rare and fatal disease that steals their ability to walk at the earliest of ages, their independence by their teenage years, and their life by their 20s.
Three of my four sons inherited Duchenne, and now I face Stage IV pancreatic cancer. It is a cruel lottery no one talks about, but it is all too real for families such as mine.
I am not writing to ask for anything for myself. I am writing because my time is short, and I need to know I did everything in my power to protect my children while there may still be a chance to change their course.
We lost our third son, Max, too soon. Elijah, my second son, is now entering cardiac decline — his most recent cardiology appointment showed measurable worsening of his heart function, a grim confirmation of where the disease is heading. This is the stage of Duchenne that almost always determines survival. Kai, our youngest, is already in a wheelchair. Both face the same cruel trajectory.
There is a possible treatment — an investigational therapy called deramiocel, developed by Capricor. Unlike existing medications that only slow decline, deramiocel has shown in multiple years of follow-up that it can stabilize the heart, preserving left ventricular function, the single most important predictor of survival in Duchenne.
Kai participated in the HOPE-3 trial. I saw changes: more energy, more strength, and the ability to get in and out of bed faster. In Duchenne, even small gains are life-changing.
Yet this summer, the Food and Drug Administration rejected Capricor’s application. In its complete response letter, the FDA admitted that deramiocel helps the heart but dismissed the data as “exploratory” because they weren’t prespecified — in plain English, officials acknowledged the benefit, then rejected it on a paperwork technicality.
Mr. President, this makes no sense.
In the spirit of your right-to-try law, which you championed to ensure that terminally ill patients are not denied access to promising treatments, this situation demands the same courage. While this is not technically a right-to-try application, the principle is identical: When lives hang in the balance, bureaucracy cannot be allowed to block hope. Families such as mine cannot wait years for perfection in trial design when the evidence already shows hearts being preserved.
The FDA has already shown flexibility. Sarepta’s Duchenne drugs were approved despite missed primary endpoints, based on secondary data and biomarkers. And in oncology, trials have shifted endpoints so that overall survival, the outcome that truly matters, could be prioritized. The principle is simple: when a disease is lethal and time is short, the trial must measure what truly determines life and death. In Duchenne, that measure is cardiac function.
If flexibility could be shown in cancer, and if flexibility could be shown in Duchenne before, why not now? The precedent is already set: The FDA can and has approved therapies when the data were imperfect but the biological plausibility and patient impact were clear.
Meanwhile, Elijah is a tireless patient advocate — as a consultant to Capricor, a book author, and a nonprofit founder. My youngest son has already shown benefit from this drug. And yet, because of bureaucracy, they may still be denied access.
Families such as mine know the truth: no ACE inhibitor, beta blocker, or other standard medication has ever stopped Duchenne hearts from declining. Deramiocel is the first therapy that truly appears to help.
Mr. President, I am asking you to cut through this red tape. You have done it before. You know that when bureaucrats say, “Wait,” what they really mean is, “Accept more loss.”
All I want is a clear message: that the FDA will judge this therapy with the same compassion and consistency it has shown before — and give children like mine the chance to live longer, fuller lives.
Since my diagnosis, I have already spent 25 of the past 62 days in the hospital — nearly half of my time away from my children and family. This is how serious, how urgent, and how hard this already is. My husband will soon face life without me. Please don’t make him and our children face it without hope.
Sincerely,
Kelly Stacy
Mother to Wil, Elijah, Max (2004–2019), and Kai
Wife, advocate, and caregiver