Until recently, ALS (amyotrophic lateral sclerosis) was treated with just a handful of medications that provided modest benefit. Families often heard the phrase “no disease-modifying therapies” far too often.
That is beginning to change.
In 2025, ALS care is entering a new era with:
- gene-targeted therapies such as tofersen for SOD1-ALS,
- expanded interest in combination and precision approaches,
- and a renewed focus on symptom-supportive medication that patients can actually take safely (especially as swallowing becomes more difficult).
This article explains what tofersen is, who it’s for, what it does and what it does not do plus where personalized medication and compounding can support ALS care.
This is educational information, not a treatment recommendation. All decisions about ALS therapy should be made with a neuromuscular specialist.
The Starting Point: What Are the “Traditional” ALS Drugs?
For many years, ALS treatment meant:
- Riluzole: an oral medication that modestly slows disease progression.
- Edaravone: originally IV, now also available as an oral formulation, designed to reduce oxidative stress.
- Sodium phenylbutyrate/taurursodiol (AMX0035): aimed at protecting motor neurons (availability and regulatory status have evolved by region).
These therapies can help some patients, but they are not cures. That’s why the approval of tofersen drew so much attention.
What Is Tofersen and Who Is It For?
Tofersen (Qalsody™) is an antisense oligonucleotide (ASO): a synthetic strand of nucleic acid that binds to messenger RNA and reduces production of a target protein. In this case, the target is the mutant SOD1 protein in people with SOD1-ALS, a genetic form of ALS caused by mutations in the SOD1 gene.
Key points:
- Tofersen is given intrathecally (into the cerebrospinal fluid via lumbar puncture).
- It was granted accelerated FDA approval in April 2023 based on evidence that it reduces neurofilament light chain, a biomarker of neuronal injury, in people with SOD1-ALS.
- Ongoing analyses in 2024 – 2025 have shown encouraging trends in slowing functional decline when treatment is started early in the disease course, although results vary.
This therapy is only for patients with confirmed SOD1 mutations, which represent a small fraction of all ALS cases.
What Tofersen Means, and Doesn’t Mean for ALS Patients
Tofersen has been described as a “proof of concept”:
- It demonstrates that gene-targeted therapies can affect ALS biology, at least for some genetic subtypes.
- It opens the door to additional antisense and gene-modifying therapies aimed at other genetic forms (e.g., C9orf72) under active research.
However, important limitations remain:
- It is not appropriate for people without SOD1 mutations.
- It requires repeated lumbar punctures and specialized centers.
- It is not a cure; it may slow progression, particularly when started early, but does not stop the disease entirely.
That means symptom management and supportive medication remain crucial for all people living with ALS, whether they qualify for gene-targeted treatment or not.
Symptom Management and Medication Challenges in ALS
Many ALS symptoms are addressed with medications originally designed for other conditions:
- Muscle cramps and spasticity: often treated with agents like baclofen or tizanidine.
- Excess saliva or drooling: may require anticholinergic medications.
- Pain, anxiety, sleep disturbances: often require tailored regimens.
- GERD and swallowing issues: frequently need careful acid-suppression strategies and reflux management.
As ALS progresses:
- swallowing becomes more difficult,
- aspiration risk increases,
- and tablets/capsules may be hard or unsafe to take.
That’s where personalized formulations can be helpful.
Where Compounding Can Help Support ALS Care
A 503A compounding pharmacy like AllMedRx cannot provide tofersen (that’s a proprietary, highly specialized biologic). But compounding can support ALS care in other ways:
1. Alternate dosage forms for symptom medications
For example:
- converting a necessary medication from tablet to liquid or other suitable form,
- simplifying administration for patients with feeding tubes,
- reducing choking risk when swallowing has become compromised.
2. Tailored strengths
In patients who are particularly sensitive to medication side effects, compounding can:
- create intermediate doses not commercially available,
- allow slower titration to find the balance between symptom relief and tolerability.
3. Excipient considerations
For some people, dyes, lactose, or other excipients can worsen GI symptoms or cause intolerances. Personalized formulations can:
- remove certain dyes,
- reduce or alter excipients when clinically appropriate.
Any such changes must be guided by the prescriber and handled by a licensed, quality-focused pharmacy. For help evaluating pharmacies:
How to Evaluate a Compounding Pharmacy in 2025
The Emotional Side – Supporting Mental Health in ALS
Understanding new therapies also has an emotional impact:
- Hope can coexist with grief, uncertainty, and fear.
- Not every patient meets eligibility criteria for gene-targeted treatments.
- Some families feel left behind when a new therapy is not applicable.
Depression and anxiety are common and deserve direct attention and treatment, just as motor symptoms do.
See: Depression condition page
Final Thoughts – A New Era, but Not the End of the Story
ALS care in 2025 is more complex, and more hopeful than it was a decade ago:
- We now have a gene-targeted therapy (tofersen) for SOD1-ALS.
- Additional precision treatments are under development.
- Symptom-supportive medications remain central to quality of life.
In this environment, medication safety, personalization, and clear communication are crucial. At AllMedRx, our role is to work with neurologists and ALS teams to:
- provide personalized formulations when needed,
- reduce barriers to taking essential medications,
- and stay aligned with the latest standards and safety expectations.
For questions about personalized medication support in ALS, clinicians and caregivers can contact: