A practical roadmap

Getting into an ALS clinical trial

Being diagnosed is the start. What actually opens trial doors is being plugged into an expert centre, knowing the genetics, and moving before eligibility windows close. Here's the sequence, for both the US and France.

Why speed matters. Many disease-modifying trials favour people earlier in the disease and with preserved breathing and function. The steps below overlap — you don't finish one before starting the next. Start the centre referral and the genetic test on the same week if you can.

  1. Get to an expert ALS centre

    Specialised multidisciplinary centres are where trials actually enrol — and they measurably extend survival, most of all in bulbar-onset disease. Ask the diagnosing neurologist for a referral now.

    France: the FilSLAN national network of reference centres — find one on the map at portail-sla.fr. Care is covered under the ALD (long-term illness) scheme.
    United States: an ALS Association Certified Treatment Center (directory at als.org) and/or a site in the NEALS research consortium, which runs most US trials.

  2. Order genetic testing straight away

    A comprehensive multi-gene panel (C9orf72, SOD1, FUS, TARDBP, and others such as TBK1) is the single highest-leverage test, because it can unlock gene-specific trials — and one gene-targeted therapy, tofersen (Qalsody), is already approved for SOD1-ALS in both the US and EU. About 1 in 10 cases are familial, but the same mutations turn up in people with no family history, so testing is recommended for everyone at diagnosis.

    The centre arranges this. Ask specifically for a full panel, not just one gene.

  3. Start standard therapy — it's not either/or with trials

    Approved medicines can usually run alongside trial participation (a trial may have washout rules — the study team will say). Riluzole is standard everywhere and recommended from diagnosis; in the US, edaravone (Radicava) is also available. Liquid and dissolvable forms exist for swallowing difficulty.

  4. Capture the baseline numbers trials screen on

    Trials commonly gate on time since first symptom, ALSFRS-R score, and breathing capacity (FVC/SVC), plus weight. Get these recorded early and tracked over time — they determine which trials she qualifies for, and the window can close as they change.

  5. Do the voice and swallowing groundwork early

    Voice/message banking while speech is still clear preserves her own voice for later communication tools. Discuss feeding-tube timing before major weight loss rather than in a crisis. These protect quality of life and keep her eligible and strong enough for trials.

  6. Search trials and turn on alerts

    Use the finder to search ClinicalTrials.gov and the EU CTIS together, filtered to disease-modifying and bulbar-relevant studies. Subscribe to email alerts so new matches come to you. Leave the country filter open — with homes in the US and Europe, you want to see everything.

  7. Shortlist, then review eligibility with the centre

    Export your shortlist (CSV) and go through it with the centre team. They can read the fine print of the inclusion/exclusion criteria, judge realistic fit, and often know trials before they're widely listed. Save promising ones to Our plan.

  8. Contact study coordinators and prepare records

    For the top choices, reach out to the listed coordinator. Have ready: the formal diagnosis, genetic panel results, current medications, and the baseline measures above. Being organised speeds screening.

  9. Screening, consent, enrolment

    The study team confirms detailed eligibility, explains risks and commitments (visit frequency, procedures, placebo odds), takes informed consent, does baseline assessments, then enrols. Ask what happens at the end of the trial (open-label extension access).

  10. Sort the logistics — you're flexible on both continents

    Some trials run the same protocol at sites in both the US and Europe, so she may qualify on either side. Being established at a centre in each country (French sécu / a US ALS clinic) is what makes relocating workable. Ask each coordinator about travel reimbursement and which study costs are covered. Because moving takes time, get established somewhere first and be ready to act, rather than moving and then searching.

  11. If no trial fits — ask about early/compassionate access

    When someone isn't eligible for a trial, there are sometimes routes to an investigational drug outside one: Expanded Access in the US, and accès précoce / accès compassionnel in France. The centre can tell you what's realistic for a given drug.

Approved treatments as of 2026 — quick reference

Standard-of-care options, separate from trials. Your centre decides what's appropriate.

DrugWhereWhat it's for
RiluzoleUS + EUStandard disease-modifier for all ALS; from diagnosis. Liquid/film forms for swallowing.
Edaravone (Radicava)US onlySlows functional decline; withdrawn in EU. Benefit shown in bulbar-onset too.
Tofersen (Qalsody)US + EUGene-targeted — only for SOD1-ALS. Why genetic testing matters.
Dextromethorphan/quinidine (Nuedexta)US onlyPseudobulbar affect; also used off-label for bulbar symptoms.
Keep your own copy. Track where you are on these steps, her details, and your trial shortlist in the private Our plan area — visible only to your family. The Library has the full guides on bulbar care, centres, and treatments.
This is general information to help you organise, not medical advice, and it can go out of date. Decisions about treatment, testing, and trial eligibility belong with your mum's ALS care team.