By Katie Jackson
As someone who has dedicated almost two decades now to Huntington's Disease (HD) advocacy, I’ve seen the HD landscape grow from discussions about supplements like fish oil and creatine to a space now filled with promising gene therapies and disease-modifying treatments. The recent wave of clinical trial results has brought both hope and caution because of past trials to our community, and I had the privilege of sitting down with Dr. Sung to help us make sense of it all.
Breaking Down the Latest Developments: The WAVE Trial
Dr. Sung started our conversation with a breakdown of the WAVE trial results. To put it simply, WAVE’s therapy has shown it can selectively reduce the mutant Huntington protein—like a quarterback targeting the right players in a football game, as Dr. Sung metaphorically explained. This specific targeting is promising because it leaves the normal Huntington protein untouched, which is crucial for overall brain function.
However, this breakthrough comes with a caveat: a significant increase in neurofilament light chain (NFL) levels. Now, we’re not talking about football here—NFL, in this context, refers to a marker indicating potential brain cell damage. The rise in NFL levels is concerning because, as Dr. Sung pointed out, it’s like throwing interceptions in a football game: you might be scoring touchdowns by reducing the mutant protein, but those interceptions could still cost you the game.
I believe after talking to many researchers in the HD field there is still a lot of unknowns about NFL and what it means to our community. Research and the brave clinical trial participants that give their bodies and time to clinical trials will help unlock so many questions about NFL pertaining to HD.
One of the most striking aspects of our conversation was the realization that no single therapy will be the panacea for all HD patients. This is why I advocate for a "cocktail" approach to treatment, where different therapies can address various aspects of the disease, from brain degeneration to the broader systemic effects HD has on the body.
The Road Ahead: FDA, EMA, and Clinical Trial Accessibility
The conversation also delved into the differences between regulatory bodies like the FDA in the U.S. and the EMA in Europe. Dr. Sung explained how these organizations have varying thresholds for what they consider ethical in clinical trials, such as the controversial use of sham surgeries. This has significant implications for how and where trials are conducted, and ultimately, how quickly new therapies can reach patients.
For those of you eager to get involved in these trials, Dr. Sung highlighted that Roche's Generation HD2 trial is currently enrolling participants. This trial is focused on earlier-onset patients and involves quarterly spinal injections. If you’re considering participation, now is the time to get informed and see if you qualify.
Final Thoughts
I’ve seen firsthand the power of the HD community's voice. Whether it’s through participation in clinical trials, filling out surveys, or even participating in studies like Enroll-HD, or submitting comments to the FDA, every action we take brings us closer to effective treatments and, eventually, a cure.
To those who’ve participated in clinical trials, I want to extend my deepest gratitude. It’s not easy, and the burden it places on families is significant, but your efforts are the reason we’re seeing progress. For those who haven’t been able to participate, know that your voice still matters. Whether it’s through open dockets, PFDD meetings, or community advocacy, surveys, talking to congress and local government, educating the general public, actually anytime you are talking about HD and your lived experience you are helping shape the future of HD treatment and the HD narrative.
Keep up the hope, and let’s continue to push forward together, side by side.
Stay strong, HD community. We’re in this fight together.
Katie Jackson
You can view the full conversation with Dr. Sung [here](https://www.youtube.com/watch?v=jHk9wZOR3VA).
Comments