Anticipation of a possible new clinical trial has us holding on to hope
Sarepta Therapeutics hopes to 'knock down the protein that causes Huntington’s'
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Hope recently arrived at our house disguised as a press release.
Years ago, my wife, Jill, who is gene-positive with Huntington’s disease (HD), tried to explain what it feels like to live with the emotional ups and downs of a rare disease. She said there are days when news about clinical trials or potential treatments sends her excitement soaring, and she’s filled with hope. Then there are days when disappointment feels like a body blow after a clinical trial has hit a dead end.
As someone who loves roller coasters, she described it this way: “It’s like being on a slow, wooden roller coaster. You climb and climb, and the anticipation reaches a fever pitch, but when you finally get to the top, you get stuck and fall off the track sideways.”
Even though we don’t love the emotional whiplash that comes with each new announcement, Jill and I decided we still needed to get on the roller coaster. She says that living with a rare disease — one that also afflicts our beloved daughter, Alexus — means you don’t always get to choose the ride, but you do get to decide whether to stay buckled in together or watch from the sidelines, eating stale funnel cake.
Building the roller coaster
When we heard that Sarepta Therapeutics, a global biotechnology company headquartered in Cambridge, Massachusetts, had submitted a clinical trial application to Medsafe, the medical regulatory body run by New Zealand’s Ministry of Health, for an investigational HD treatment called SRP‑1005, we decided to climb back into our rickety little car and let it click up the track again.
“Pending approval, Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 [called INSIGHTT] in the second quarter of 2026,” the company recently announced.
SRP-1005 is what scientists call a “small interfering RNA therapy.” It is delivered subcutaneously and designed to reach the deep brain and “knock down the protein that causes Huntington’s disease.” Phase 1 of the trial would test safety and dosing in about 24 participants at multiple centers. In a world where there are still no approved treatments that slow or halt HD, even a small, early‑phase trial feels like watching the roller coaster being built.
The press release came with all the usual cautionary language about risks, uncertainties, and not getting ahead of ourselves. It explained that success in pre-clinical and early clinical studies does not guarantee later success, and that any number of scientific, regulatory, or manufacturing issues could derail the program.
As a longtime journalist, I understand the need for that careful wording, but as a husband and father, my heart skipped straight to the words “novel and potentially paradigm‑changing approach to the treatment of this devastating disease.”
As our hopes lifted, Jill and I talked about what this might mean, not just for us, but for the roughly 40,000 people in the United States living with symptomatic HD, and the more than 200,000 who carry the mutation and are at risk of developing it. Those numbers can feel overwhelming until you remember that behind each one is a family like ours, trying to manage pills, appointments, paperwork, and the effects of a neurodegenerative disease that slowly erodes a person’s mind and body.
The INSIGHTT trial won’t change all of that overnight, but the idea that an injection under the skin might someday help protect the brain feels like something straight out of science fiction.
For years, the HD community has been asking policymakers, companies, and donors to invest more in treatments that could modify the disease, not just manage its symptoms. Seeing another company step into the HD arena with a sophisticated way to deliver medicine across the blood‑brain barrier feels like an answer to those pleas.
Before we ended the conversation, I couldn’t resist a pun: “Looks like scientists are finally hunting down the huntingtin protein,” I said.
Rapid, involuntary eye movement is one of HD’s symptoms, which means Jill often rolls her eyes as a result of being gene-positive. But she’s also been known to simply roll her eyes at one of my (frequent) puns.
After Jill was done groaning and rolling her eyes, I told her that I dream of a day when I can be entirely sure her epic eye rolls are aimed squarely at my terrible, but extremely punny puns and not influenced by her symptoms.
Note: Huntington’s Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Huntington’s Disease News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Huntington’s disease.
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