A Mother's Race Against Time

My name is Katie Jackson, and my journey with HD is both professional and deeply personal. My husband was diagnosed with HD at just 26. For 14 grueling years, I was his caregiver as he bravely battled this relentless disease, until I lost him at the age of 41. Now, six years later, I am once again staring down the barrel of this nightmare. My children are reaching the age where people are often considering testing, and after 20 years of fighting for them—20 years with no therapy—the fear is overwhelming and urgent. Every day that passes without progress is another day stolen from my family.

If one of my children's diagnoses is positive, it will feel like an even more desperate race against time. I am haunted by the thought of losing my daughter or son to the same fate as their father. For my family, and for countless others in the HD community, time is both precious and a constant source of fear.

Living with HD means living in a constant state of fear and grief. Every new symptom brings fresh waves of anxiety, and the sense of helplessness is crushing. The devastation doesn’t stop with the person diagnosed; depression, isolation, and generational trauma shatter caregivers and families alike. Young people, at risk themselves, often become full-time caregivers for a parent—sometimes developing their own symptoms before their loved one’s battle ends. For many, a lengthy clinical trial isn’t just an inconvenience—it could mean never gaining access to potentially life-saving therapies in time.

Our nonprofit, Help 4 HD, recently asked the community, “What is the number one thing you would like to see in drug development?” The answer was resounding: hope for something—anything—that can slow the progression of HD within their lifetime. To us, every moment matters. Even a slight slowing of the disease means more time to work, to drive, to celebrate holidays, and to simply be with loved ones before HD takes over.

HD is a fatal, irreversible disease with no therapies currently available to modify its course. That’s why it has been so hard lately for the community to understand why the FDA made the decisions it made a couple of months ago. The data from the Phase I/II study, supported by one of the most robust natural history databases in rare disease, should be enough to move us forward.

We have already demonstrated extraordinary commitment as a community. We have contributed to establishing strong databases and have participated in studies that should eliminate the need for further randomized, long, double-blinded trials. We are not asking for certainty, only for the chance to hope.

But this post isn’t about AMT-130; it is about all future trials.

There is new hope that lies in the new FDA leadership, and our deepest hope is that we see change soon.

Requiring only lengthy, double-blinded randomized clinical trials as the sole path to approval delays access to life-changing treatments for patients who need them most. We are not advocating for therapies without evidence, but a rigid insistence on protracted studies risks costing precious time—and lives. It is time to find a middle ground that balances scientific rigor with urgent patient need, so transformative therapies can reach those who need them as quickly and responsibly as possible. That is our hope: that it will come from the ones we trust to make decisions for us on safety and efficacy, our FDA.

To my community as a mother in what feels like a race against time. Time can feel frightening when facing a terminal disease, as each moment is a reminder of its limits and uncertainty. Yet within that same passage of time, there are also beautiful moments—huge joys, meaningful connections, and cherished memories—that remind us of the value and richness of every day we get to spend with our loved ones. I refuse to let HD rob me of the memory that time is precious, and that the majority of the moments I got to spend with my husband were beautiful and meaningful. I will continue to fight, but also remember to cherish every day I get with my beautiful, kind, creative, smart, incredible children.

By Katie Jackson, President of Help 4 HD International