How to read this site

Every value in the tables comes from a public source: a trial registry, a regulator, a published paper, a company announcement or reputable news. Click any cell to see where it came from.

What each column means

Company

The company, university or organisation running the drug's development.

How it works

The idea behind the drug, in plain words. The popup gives the scientific name.

Stage

Where the drug is in testing. Phase 1 checks safety, Phase 2 looks for early signs it works, Phase 3 is the large trial meant to prove it. For drugs a regulator has decided on, it also shows each country's decision. See theglossary.

Evidence

An overall verdict based on all key studies, positive and negative, plus the most favourable result reported. Seehow the verdict works.

Who it's for

Which people with ALS the drug is meant for, such as everyone or only people with a particular gene, and how it is taken.

What's next

The next public milestone, such as trial results or a regulatory decision, and when it is expected.

Can I join?

Whether a trial is recruiting, where, and whether there is an expanded access (compassionate use) programme.

Signal

How often and how reliably the company gives public updates. How it is calculated.

Checked

The date we last confirmed the information for that drug, shown under its name.

How the evidence verdict works

A single good result can make a drug look better than it is. So for every drug we list all the key studies we can find, including failed trials and independent studies, and mark each one by whether it met the main goal it set for itself before it started. The verdict is worked out from that list, the same way for every drug:

• Consistent benefit Two or more controlled trials met their main goal, and none found the opposite.
• One positive trial One controlled trial met its main goal, but no second trial has confirmed it yet.
• Mixed evidence At least one controlled trial was positive, but other trials or large independent studies found no benefit.
• Hints only Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.
• Early signal only Only small, open-label or historical-comparison studies so far. Promising at best; not proof.
• Not shown to work Controlled trials found no benefit.
• Results pending Trials are running, but there are no efficacy results yet.

Placebo-controlled trials count most, because they are the only reliable way to tell whether a drug works. Large independent real-world studies that found no benefit also count against a positive trial. Open-label studies, comparisons with past patients and analyses done after a trial ended can only ever give an "early signal".

We only use green for consistent benefit. Approval by a regulator is not shown as green either: regulators use different standards and sometimes disagree. When they do, the drug is marked Regulators disagree.

Early pipeline drugs

On the Early pipeline page we list drugs in their first trials in people (Phase 1). These trials check whether a drug is safe and how the body handles it, often in a few dozen healthy volunteers. They are not designed to show whether it slows ALS, so these drugs have no evidence verdict.

We list them because news about them travels fast, and it helps to see plainly how early they are. Most drugs that start Phase 1 never reach approval, and many that work well in mice do not work in people. We only include a drug once a registered trial has dosed its first person, and it moves to the main table when a trial in people with ALS starts measuring whether it works.

Reading effect numbers

Many ALS trials measure the ALSFRS-R, a score of everyday abilities. "35% slower decline" means the score dropped about 35% less than in the placebo group over the trial, for example 6.5 points lost instead of 10. It does not mean the disease stopped, or that 35% of people improved.

These numbers cannot be compared across drugs. Each trial enrols different people (early or later disease, fast or slow progression), lasts a different length of time, and measures things differently. A big number from a small, short trial is much less certain than a smaller number from a large one.

Percentages also make modest effects sound large. That's why we give the effect in points too, for example "2.5 points less decline over 6 months on the 48-point scale". For a sense of scale, people with ALS lose on average about one point a month, though this varies a lot from person to person.

How Signal is calculated

Signal answers one question: how often, and how reliably, do we hear from the company about this drug? It is calculated automatically from the drug's timeline, counting only updates the company itself published (press releases and investor updates), and from the milestones the company has publicly promised. It is not a measure of whether the drug works.

Active 70 or more ·Steady 45 or more ·Quiet 20 or more ·Silent below 20

Large companies often say little about a single drug, and academic trials usually update only at major moments, so a "Quiet" signal is common and not necessarily bad news. A drop from Active to Silent, or missed promises, is worth noticing.

How the data is kept up to date

Every week we check each drug's company newsroom, trial registry entries, regulators and ALS news. We add new events to the timelines, update anything that changed with its new sources, and list the changes on the updates page. If you spot something wrong or missing, please tell us.

Limits

This is a summary for non-specialists, not a complete database. We focus on drugs in Phase 2 or later, plus a few notable earlier ones. Company press releases are useful but are written to present results in the best light; we say when a result has not yet been independently published.