Research – Botulism breakthrough? Taming botulinum toxin to deliver therapeutics

Boston Childrens Hospital

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While rare, botulism can cause paralysis and is potentially fatal. It is caused by nerve-damaging toxins produced by Clostridium botulinum — the most potent toxins known. These toxins often lurk in contaminated food (home canning being a major culprit). Infants can also develop botulism from ingesting C. botulinum spores in honey, soil, or dust; the bacterium then colonizes their intestines and produces the toxin.

Once paralysis develops, there is no way to reverse it, other than waiting for the toxins to wear off. People with serious cases may need to go on ventilators for weeks or months. But a new botulism treatment and delivery vehicle, described today in Science Translational Medicine, could change that.

“Currently, there are anti-toxins, but these only work before the toxins enter the motor neurons,” says Min Dong, PhD, a researcher in the Boston Children’s Hospital Department of Urology and corresponding author on the paper. “What we have developed is the first therapy that can eliminate toxins after they get inside neurons.”

If proven in humans, the approach would represent a breakthrough in treating botulism. In mice, the treatment successfully got inside neurons and reversed muscle paralysis within hours. It also enabled mice to withstand doses of botulinum toxin that would otherwise be lethal.

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