ALS (amyotrophic lateral sclerosis) is a disease where nerve cells controlling voluntary movement gradually die. About 5,000 people in the US are diagnosed each year. Treatment has historically meant managing symptoms, though two drugs approved in the past decade can slow progression modestly in some people.
What's actually going on in research
Trials are testing drugs that target different aspects of ALS biology — reducing inflammation in the brain and spinal cord, protecting motor neurons from stress, blocking toxic protein buildup, and addressing genetic forms of the disease. Gene therapy approaches for specific mutations and stem cell injections to support dying neurons are both in human testing.
SOD1-targeted therapies
Tofersen, approved by FDA in 2023, targets a specific genetic mutation (SOD1) that causes about 2% of ALS cases. Other gene-silencing drugs for different ALS mutations are now in trials.
Stem cell approaches
Researchers are injecting stem cells into the spinal cord to see if they can protect motor neurons or slow disease progression. Early trials suggest the approach may be safe, and larger studies are testing effectiveness.
Combination strategies
Because ALS involves multiple disease processes, some trials are testing two or three drugs together. The idea is that attacking the disease from several angles might work better than single drugs.
What to know before you search
Eligibility typically depends on how recently you were diagnosed, how fast the disease is progressing, breathing function, and for some trials whether you have a specific genetic mutation.
What types of trials are currently open
- Treatment trials — Testing new drugs, often pills or infusions, to see if they slow disease progression or extend survival compared to placebo or current treatment.
- Gene therapy trials — Testing therapies that target specific genetic mutations responsible for familial ALS, usually delivered as a one-time injection into spinal fluid.
- Stem cell trials — Studies injecting stem cells into the spinal cord or elsewhere to see if they can support or protect dying motor neurons.
- Biomarker studies — Tracking people with ALS to find biological markers that predict disease progression or treatment response, which helps design better trials.
- Symptom management trials — Testing treatments for specific ALS symptoms like muscle cramps, excess saliva, difficulty speaking, or breathing problems.
Recently added ALS trials
Receive blood filtering sessions to reduce harmful proteins in ALS
10-20% of patients with ALS have anti-NRIP autoantibody and the titer of anti-NRIP autoantibody is correlated with motor functional decline and mortality in ALS. The PALADIN2 clinical trial is a single arm study, which intends to enroll 20 ALS patients having anti-NRIP autoantibody in plasma. Patients will receive 3 courses of plasmapheresis per 3 months in order to maintain low concentration of anti-NRIP autoantibody in plasma. The study will follow up these patients for another 6 months after plasmapheresis. This project will potentially confirm the efficacy and safety of plasmapheresis for ALS patients having anti-NRIP autoantibody.
FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS
The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS. TRCN-1023 is an investigational medicine given as a single injection into the fluid surrounding the spine (intrathecal injection). The trial will also assess how the body processes the drug and whether it shows early signs of benefit over 24 weeks.
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