An experimental oral medication, combining an antibiotic with an anti-inflammatory drug, may offer benefits to patients with amyotrophic lateral sclerosis (ALS), according to the results of a clinical trial. ALS is a progressive neurodegenerative disease that destroys motor neurons, leading to gradual loss of movement and eventually paralysis and death.
Motor neuron disease (MND) is a fatal neurodegenerative condition, with ALS being the most common form.
Even though relatively rare, affecting approximately 1.6 people per 100,000 adults, ALS has a significant impact. The disease affects nerve cells (motor neurons) in the brain and spinal cord, causing progressive degeneration. As these connections break down, muscles weaken and become rigid, leading to difficulties with walking, speaking, eating, and breathing.
Currently, there is no cure for ALS, and most individuals diagnosed live between three and five years after receiving a diagnosis. This underscores the urgent need for new treatment options for this devastating condition.
Researchers evaluated an experimental treatment for the disease in a randomized, mid-phase clinical trial.
The treatment was designed to target inflammation in the nervous system, excessive iron accumulation, and disruptions in the activity of microRNAs – molecules that control gene expression and are implicated in ALS.
This stage of the study was designed to assess the safety of the treatment, rather than its effectiveness.
A total of 68 participants were included in the trial, receiving either the experimental drug, PrimeC, or a placebo for six months.
The study continued for an additional 12 months without a control group, with all participants receiving the experimental treatment, a combination of celecoxib and ciprofloxacin.
Although the study wasn’t designed to prove effectiveness, patients treated with the combination showed improved functional outcomes, particularly in their ability to speak and swallow.
Patient status was assessed using the ALS Functional Rating Scale Revised, a tool that measures daily functioning on a scale from 0 to 48 points, with a maximum score indicating normal function.
After six months, patients treated with the experimental medication achieved scores 2.23 points higher than those in the placebo group. After 18 months, the average difference increased to 7.92 points in favor of those receiving the treatment.
Early initiation and long-term continuation of the treatment was also associated with a 64% reduction in the risk of disease-related complications, including hospitalization, respiratory failure, or death.
patients who received the experimental therapy showed lower levels of ferritin, a protein involved in iron storage in the body, as well as reduced levels of microRNAs associated with the disease.
The data support advancing the medication to a Phase 3 trial to evaluate the treatment’s efficacy and safety in a larger population, according to the study authors.
The treatment is being developed by biotechnology company NeuroSense Therapeutics, based in Israel, in collaboration with Recipharm, a pharmaceutical company in Sweden.
The study results were published on Monday, in the journal JAMA Neurology.
