A novel treatment for botulism that may tame the toxin with therapeutics that have the potential to reverse the deadly paralysis — all coming out of Boston Children’s Hospital. The research on mice is being called a “botulism breakthrough” by Science Daily, the first to publish the work.
With fewer than 1,000 confirmed cases a year in the United States, botulism is a rare poisoning caused by toxins produced by Clostridium botulinum bacteria. It can be fatal and requires immediate emergency medical care.
Botulism can occur in infants, be spread in food, or infect a wound. Symptoms include difficulty swallowing or speaking, facial weakness, and paralysis. The paralysis usually affects muscles used for breathing, making it necessary for patients to be placed on ventilators.
Toxins produced by Clostridium botulinum can become fatal once they get into the neurons because at that point they’ve not been treatable.
These are the toxins that cause deadly paralysis and are found in contaminated foods from improper home canning or from spores in honey, soil, and dust. Clostridium botulinum spores colonize in the intestines and produce toxins.
Anti-toxins are available but are only effective before the toxins enter the motor neurons. A researcher with the Urology Department at Boston’s Children’s Hospital has reported on the first therapy that effectively eliminates the toxins after they’ve entered the neurons.
Children’s Min Dong, Ph.D., reports that in mice, researchers have produced a novel treatment that neutralized the toxin with a second, modified botulinum toxin that delivered a mini antibody into the cells, reversing the paralysis.
Human tests should show whether the new approach represents a breakthrough in treating botulism. In mice, the treatment got inside the neurons and reversed the muscle paralysis within hours. Mice withstood botulinum toxins that in the past were fatal.
Shin-Ichiro Miyashita, a postdoctoral fellow in Dong’s lab, says two barriers were overcome — getting across the cell membrane and targeting specific cell types. The new approach rests on a new toxin, botulinum neurotoxin X, which was developed in 2017 Unlike previous toxins, botulinum neurotoxin X shows no toxicity and is a safe delivery tool, according to Dong.
The new botulism treatment may also be used for “botox reversal” where botox has caused unwanted muscle paralysis as a side effect, sometimes lasting for months.
Dong says it will potentially be possible to inject the therapeutic protein and replace the botox in a few hours.
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