New research shows drug used to treat neuromuscular weakness could counter botulism (Firdapse)

Medical Xpress / Atrium Health Wake Forest Baptist / Molecular Medicine ^ | July 25, 2022 | James B. Machamer et al

[ConservativeMind]

Researchers are investigating a drug used to treat neuromuscular weakness as a potential treatment for botulism.

Botulinum neurotoxins (BoNTs) are a family of bacterial poisons—the most poisonous substances known—responsible for the clinical disease known as botulism. These neurotoxins act within nerve terminals to destroy proteins necessary for muscle contraction, causing paralysis that develops into respiratory arrest and can lead to death.

Currently, the only specific treatment for botulism is early administration with antitoxin. However, antitoxin is only effective if administered before prior botulism symptoms are evident. Once symptoms emerge, three out of four patients require long-term artificial ventilation for survival.

Despite decades of effort, there are no antidotes for the life-threatening consequences of botulism.

The researchers build on their previous work to show that administration of the FDA-approved drug 3,4-diaminopyridine (3,4-DAP) reverses botulism symptoms in a pre-clinical model. The drug is an approved treatment for Lambert Eaton Myasthenic Syndrome, an autoimmune disease caused by reduced acetylcholine release and neuromuscular weakness. Botulism paralysis is caused by reduction of acetylcholine release from motor nerve terminals to subthreshold levels required for muscle contraction.

Acetylcholine is the chief chemical messenger of the parasympathetic nervous system, the part of the autonomic nervous system that contracts smooth muscles, dilates blood vessels, and slows heart rate.

For this study, researchers developed a continuous 3,4-DAP infusion model and measured dose-dependent effects on toxic signs and survival after a lethal dose of botulinum neurotoxin. They found that continuous infusion with the drug produces rapid and sustained therapeutic benefits while survival requires continuous infusion for longer than four days.

"This is the first small-molecule therapy to directly reverse toxic signs and promote survival when administered post-symptomatically after a lethal dose of botulism," said McNutt. "Our data supports the immediate clinical use of DAP in botulism patients."

[ConservativeMind]

The drug goes by the name “Firdapse” in the US and is currently available.

It’s nice to see an effective treatment for such a serious malady.

[ConservativeMind]

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[who_would_fardels_bear]

We need someone to travel around the country hocking these magic pills that have multiple uses...

[allendale]

Have to wonder about long term yet unkown sise effects in people who have themselves injected repeatdly with botulism toxins for cosmetic purposes.

[Gen.Blather]

I wonder if it might work for ALS.