An experimental oral medication combining an antibiotic with an anti-inflammatory drug may be useful for patients with amyotrophic lateral sclerosis, a mid-stage trial suggests.
The randomized trial, designed to prove the combination pill's safety but not confirm its efficacy, involved 68 participants who received PrimeC being developed by NeuroSense Therapeutics and Recipharm, or placebo for six months, followed by a 12-month open-label extension in which all received PrimeC.
PrimeC, a combination of celecoxib and ciprofloxacin, is designed to target the neuroinflammation, excess iron accumulation, and abnormal microRNA gene-regulating activity that occurs in ALS, also known as Lou Gehrig's disease.
Celecoxib is sold under the brand name Celebrex by Viatris.
Although the study was not designed to assess efficacy, participants in the PrimeC arm had better functional outcomes, especially for speech and swallowing, as measured by an ALS Functional Rating Scale Revised score, researchers reported in JAMA Neurology.
On a scale ranging from 0 to 48, with 48 indicating normal functional ability, at 6 months the participants taking PrimeC scored 2.23 points higher than those taking placebo. By 18 months, participants originally assigned to PrimeC scored 7.92 points higher, on average, than their counterparts.
Early, continuous treatment was also associated with a 64% reduced risk of ALS-related complications, including hospitalization, respiratory failure or death.
In addition, those initially assigned to PrimeC had lower levels of ferritin, a key protein involved in storing iron in the body, and lower levels of microRNA molecules that have been linked to ALS, a neurodegenerative disease that causes progressive muscle weakness, paralysis, respiratory failure and eventually death.
"The improved functional and biomarker signals we observed support a Phase 3 study to evaluate PrimeC's effectiveness and safety in a larger population," study leader Dr. Merit Cudkowicz of the Mass General Brigham Neuroscience Institute in Boston said in a statement.
"We are determined to accelerate the development of therapies for people living with ALS," Cudkowicz added.
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