There is something deeply challenging about waiting.
If you’ve sat in a hospital chair, foot tapping the linoleum floor, or refreshed your inbox, hoping for an answer that never arrives, then you can relate to the experience of waiting. For families living with ALS, waiting becomes its own form of grief. What are we waiting for…a diagnosis? decline? news? Yet in 2025, things are different. The waiting room feels different.
We are experiencing, for the first time, a quiet shift in the world of ALS research, fueled by gene therapy, personalized trials, and experimental therapies that finally feel like they are changing the focus of research from a response to symptoms to targeting the root of the disease.
2025 Brings a Shift in How ALS Is Treated And Understood
For decades, ALS has been addressed like a puzzle without a map. Most medications were designed to slow the progression of ALS, as opposed to stop the disease. However, this is changing with recent advances in gene therapy for ALS.
Researchers have begun to focus on specific gene mutations like SOD1 and UNC13A that cause specific types of ALS. New therapies, like antisense oligonucleotides (ASOs), are being developed to target mutations directly through therapies that aim to disrupt the disease process, and not just alleviate symptoms. Some have even advanced to late stage clinical trials, which gives families something more concrete than hope: data.
You can find more about ASOs and their progress via trusted medical sites such as The ALS Association, and Springer’s review of SOD1 gene targeting.
What this translates to is simple, yet seismic. ALS treatment isn’t solely one-size-fits-all anymore. We are beginning to see an emergence of personal, tailored, and predictive treatments for ALS.
Inside The Science: Gene Therapies, Biomarkers, and What’s Next
Gene therapy isn’t the only wave rolling in.
Researchers are also investigating ways to stop the toxic proteins like TDP-43 (one of the more common pathological hallmarks of ALS) from building up in motor neurons. There are promising treatments called intrabodies that are being developed which follow this principle to eliminate the toxic features and slow progression.
Some scientists are working with something called PIKFYVE inhibition, yes it’s a mouthful, but it’s a target that had strong results in preclinical models. And for the first time, doctors are using biomarkers like neurofilament light chain levels better diagnosis and track ALS progression earlier than ever before. This could be a game changer for families as they begin to make care decisions.
While all of this may sound technical, the implications are very intimate. If you’re a caregiver, it means your loved one could qualify for a trial that fits their body’s unique response to ALS. If you’re living with the disease, it means doctors might soon have better answers to questions like:
How long will I walk?
How long will I talk?
When do I need to prepare?
And maybe, someday: What if I didn’t have to lose those things at all?
For more about the future of diagnostics and predictive models, read this open-access review.
What This Means for Families (And Why Let Hope Grow Is Still Here)
Science does not change everything. Not immediately.
You are still taking care of someone who needs assistance with getting dressed. You are still telling your child why Grandpa can’t speak the same way anymore. You are still figuring out the budget for ramps, wheelchairs, and time off from work. And no trial, nor molecule, nor research paper, will ease that.
But you are not carrying that alone. Let Hope Grow is still here. For support. For help when the bills are piling up. For connection when the silence is too loud.
While researchers are moving forward, we keep holding the line. Until one day, the two will converge…the science that stops ALS, and the community that never gave up waiting for it.
Explore our support programs here. Or, if you feel inspired, donate now to help us continue to be that bridge.
A Gentle Push Toward Tomorrow
We are not there yet, but 2025 has given us hope that we may be getting closer.
With every new trial, every new discovery in gene therapy, every family willing to say yes to experimental treatment, something shifts. And in that space between research and reality, we will continue to plant seeds.
Because healing may start in the lab, but it grows in the lives of people who don’t give up.
Let Hope Grow is more than a name. It’s a promise.
And we intend to keep it.