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There could be new hope for ALS management as a promising drug enters phase 3 clinical trials.
ALS is a progressive disease in which the brain loses connection with the muscles, The ALS Association states. This slowly strips away a person’s ability to walk, talk, eat, dress, write, speak, swallow and, eventually, breathe.
The “PREVAiLS” study, which will review the investigational drug pridopidine as a potential ALS treatment, has already enrolled its first participant, according to a press release from Prilenia Therapeutics and Ferrer, developers of the drug.
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The first participant has been enrolled at Mass General Brigham under the supervision of Sabrina Paganoni, MD, PhD, co-director of the Mass General Hospital Neurological Clinical Research Institute and lead researcher in the PREVAiLS trial.
“Pridopidine is a sigma-1 receptor (S1R) agonist,” Paganoni shared in a statement. “The S1R has been shown to play a role in stimulating multiple neuroprotective pathways impaired in neurodegenerative diseases, such as ALS and Huntington’s disease.”
The global clinical trial, which will include 500 participants, aims to evaluate the safety and effectiveness of pridopidine in slowing the progression of ALS in early, rapidly progressive patients. The trial is expected to take place in up to 60 ALS treatment centers across 13 countries.
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This is a follow-up to the phase 2 HEALEY ALS Platform Trial results in 2023, which did not reach its main goal of slowing ALS function over the 24-week study period. That research, however, did see positive results in a subgroup of patients who were early in disease and declining rapidly.
In the HEALEY trial, the drug was generally well-tolerated, with a safety profile similar to placebo. The most common adverse events were falls and muscle weakness, which overlap with ALS symptoms.
PREVAiLS is believed to be the only currently recruiting phase 3 ALS study, according to the release.
Phase 3 incorporates “key learnings” from phase 2, Paganoni shared with Fox News Digital, and is expected to determine whether pridopidine is effective as a potential treatment for the disease.
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“Enrolling the first participant in this confirmatory study is a milestone in our search for potential new therapeutic options that may help to slow disease progression, preserve function, maintain speech and prolong survival – key aims of early ALS therapy,” she said.
“As with all clinical research, definitive conclusions won’t be available until the phase 3 trial is completed and fully analyzed.”
“The earlier we can diagnose and treat ALS, the greater the potential to preserve function and maintain quality of life for longer.”
Kuldip Dave, PhD, senior vice president of research for The ALS Association in New York City, also commented on the “urgent need” for new ALS treatment options.
“The earlier we can diagnose and treat ALS, the greater the potential to preserve function and maintain quality of life for longer, which is key to making ALS livable until we can cure it,” he said in the release.
“It was discouraging to see a lack of overall effect in the phase 2 study population,” he said. “However, we were encouraged to see positive signals emerge from various subgroups, including potential impacts on speech and respiratory function.”
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“Because respiratory decline is a leading cause of ALS morbidity and mortality, even modest preservation of breathing capacity can have a meaningful impact on both quality of life and overall outcomes.”
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The insights gained from the phase 3 trial will be critical in determining whether these early signals translate into “meaningful, consistent benefits for people with early, rapidly progressing ALS,” Dave added.
Dave praised ALS patients for their participation in these studies, along with family members and caregivers for their support. ”Participating in ALS research is one of the most powerful ways individuals can contribute to accelerating scientific discovery and making ALS livable until we cure it.”
What to know about ALS
Early signs of the disease include muscle weakness, stiffness and cramping. Symptom progression and severity are different for each case, and the association notes that there is “no single timeline for ALS.”
The disease only affects motor neurons controlling voluntary movement, so the five senses — sight, touch, hearing, taste and smell — are not affected, nor are the eye muscles or bladder control.
Diagnosis typically occurs between the ages of 40 and 70. Many ALS patients remain “mentally alert and aware” throughout the disease, The ALS Association reported.
ALS is “always fatal,” Dave told Fox News Digital, as most people live only three to five years after diagnosis. About 20% of patients live five years or longer, and only about 5% live longer than 20 years.
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There is currently no cure or treatment to stop disease progression.
Prilenia, a Netherlands-based biotech company, shared with Fox News Digital that as neurodegenerative diseases progress, the damage is “irreversible,” making them difficult to treat.
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“This is why we are focusing on an early, rapidly progressive patient population rather than a broader range of patients, as this provides the best chance of evaluating drug effect within the confines of a time-limited clinical trial,” the company stated.
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