ALS (amyotrophic lateral sclerosis) progresses faster than most neurological diseases, but the field has shifted from one or two drugs to a wave of trials testing new approaches. Tofersen, a gene-targeted drug for SOD1 ALS, was the first treatment to lower a key disease marker, and platform trials are testing multiple drugs in parallel.
What's actually going on in research
Trials are testing antisense and gene therapies for SOD1, C9orf72, and FUS forms of ALS, stem cell approaches, drugs targeting inflammation and protein clumping, and the HEALEY platform that tests multiple drugs simultaneously. Researchers are also studying earlier diagnosis through blood neurofilament tests and respiratory and feeding support.
Genetic ALS therapies
Drugs like tofersen for SOD1 ALS lower the toxic protein and slow disease in the right patients. Similar approaches are in trials for C9orf72 and FUS ALS.
Platform trials
The HEALEY ALS Platform Trial tests several promising drugs at once with a shared backbone. This design speeds up answers about what works.
Earlier diagnosis
Blood tests for neurofilament light, a marker of nerve damage, are being used to detect ALS earlier and to monitor treatment response in trials.
What to know before you search
Eligibility often depends on time since diagnosis, current function (ALSFRS-R score), genetic testing for SOD1, C9orf72, or FUS, and respiratory function.
What types of trials are currently open
- Treatment trials — Testing new drugs in people with ALS to slow progression or improve symptoms.
- Gene therapy trials — Testing antisense and gene therapies for specific genetic forms of ALS like SOD1 and C9orf72.
- Stem cell trials — Studies of stem cell approaches aimed at supporting motor neurons.
- Symptom trials — Testing treatments for cramps, spasticity, drooling, and breathing support.
- Observational studies — Following people with ALS to understand progression and identify markers for trials.
Recently added ALS trials
Wideband Tympanometry as a Non-behavioral Test of the Speech-weighted Middle Ear Transfer Function.
There is a need for a non-behavioral clinical test that provides an assessment of how speech is conducted through the middle ear for the optimization of middle ear implants. These implants utilize the functionality of the human middle ear and require adequate speech conduction through the middle ear chain. Consequently, the clinical test of this degree of speech transfer can contribute to the pre-operative indication for middle ear implants. Wideband Tympanometry (WT) is a non-invasive diagnostic tool for the clinical assessment of the condition of the middle ear. Unlike classical tympanometry, WT tests the impedance of the middle ear system as a function of both pressure and frequencies. Consequently, a WT measurement contains many times more information about the functionality of the middle ear than classical tympanometry and is ideally suited as a diagnostic tool for the clinical assessment of speech transfer through the middle ear. The WT measurements will be correlated with the Speech Intelligibility Index (SII) collected in normally hearing individuals and patients with isolated conductive hearing loss. This index is a measure of speech transfer through the middle ear. Consequently, a correlation of the WT measurements with the SII addresses the need for a non-behavioral clinical test of speech transfer through the middle ear. Additionally, WT measurements will be performed longitudinally in cochlear implant candidates, one of the populations that can benefit from the developed clinical test. These longitudinal WT measurements, which will be performed both pre- and post-operatively, allow for the evaluation of the impact of middle ear surgery on the predicted SII.
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility for more details) to control and receive feedback from assistive devices.
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