When I was a little girl, my father was my hero. I remember jumping into his arms when he came home from work, him tickling me as I burst into laughter, or curling up next to him on the couch. I loved him so much, and I knew he loved me.
I don’t remember the exact moment I realized I was losing the father I knew and loved. Over time, he changed, slowly, because before his earthly life was over, he was taken from me by a terrible disease that is now part of my future as well.
Father’s Day is approaching. Like many daughters, I’ve been looking through old family photos. In every picture, my father is smiling back at me. I also see two dads: the one I knew when I was young, and the person Huntington’s disease destroyed.
There are about 30 million American families facing Huntington’s and other rare diseases, like Duchenne muscular dystrophy or Sanfilippo or Hunter syndrome. Although each family’s details are unique, we are united in pain — and in concern that the Food and Drug Administration ignored that pain when it rejected emerging treatments that brought hope for rare disease patients.
There was encouraging news this week when the FDA reversed course and indicated it may accept an accelerated approval filing for Huntington’s gene therapy AMT-130 without requiring the kind of extreme placebo-controlled brain surgery trial it had previously seemed to demand. That is a step in the right direction. But for rare disease families, one positive decision is not enough. We need new FDA leadership to consistently embrace scientific innovation and move with the urgency our diseases demand.
Every extra month matters because I have seen what Huntington’s disease does when time runs out.
Grief usually begins when someone dies. It started much earlier for me. In the last year of my father’s life especially, I watched, powerless, as the brilliant man who could fix anything, who loved his family deeply … broke. An inherited neurological disorder that robs people of speech, memory, mobility, and eventually life, Huntington’s disease is heartless, stealing the very essence of a person.
It’s a grief few people talk about. How do you mourn someone who is still alive?
Dad was carrying so much more than we imagined, and could not face the horror of slowly losing his independence to an incurable disease that had already taken the life of my grandmother, his mother.
Knowing what Huntington’s would continue to do to his body and mind, Dad took his own life. His suicide was devastating, and the ultimate indication that he knew what was coming. He knew things would only get worse.
The finality of Dad’s death was painful, but not the end of my grief story — just the end of a chapter.
Just months after my father’s death came the news I had always wondered would be my story too. I tested positive for the Huntington’s gene.
Testing positive for Huntington’s changed how I see my father and our shared story. I no longer mourn only what I lost. I understand what he was losing, too. That is why I fight — for my future and his legacy.
My advocacy work has shown that Huntington’s families are not alone. I have met patients and families fighting for therapies that could alter the course of devastating illnesses, only to face regulatory delays while disease progression continues. Time at the FDA is just another day. Time for rare disease families is the enemy.
As FDA leadership is in a time of change, I cling to hope. I hope that we receive more good news like we did this week — that the agency lets rare disease families decide what treatment is best for them.
Patients wonder how many Father’s Days are in their futures, and so do children and spouses of patients. That is what I think about this Father’s Day — not only what Huntington’s disease took from my father, but what future treatments might give to others.
If Dad were still here, I think he would say the best Father’s Day gift would be that regulatory approvals start embracing emerging science that can improve or extend life.
MISSIONARY PETER STAFFORD HOME IN US AFTER EBOLA BATTLE IN CONGO
This Father’s Day, I choose to remember Dad before the disease. Huntington’s took him, and I don’t want it to take me. Like millions grieving after a rare disease loss, I know it can take our loved ones, but never our precious memories.
But there’s no reason why we shouldn’t get to keep both. That’s why we fight.
Rachel Reising is a Huntington’s disease advocate and nutritional therapy student who has received an HD gene positive diagnosis.