Scientists discover small molecules up Botox efficacy

By Simon Pitman

- Last updated on GMT

Related tags Protein

US researchers say they have discovered several small molecules
that can 'superactivate' botulinum neurotoxin (BoNT), which in turn
could greatly improve its effectiveness as an anti-wrinkle

According to scientific research carried out at the Scripps Research Institute in La Jolla, California, the smaller activating molecules bind to specific sites on the neurotoxin protein, which increases protease activity and enhances the toxin effect.

The scientists say they have proved this by assessing the increase in protease activation. Proteases are enzymes that act as cellular catalysts, breaking up proteins into smaller elements such as amino acids and reducing the amounts of energy needed for activation.

Any increase in protease activation will essentially speed up the efficacy of the BoNT formulation, leading to quicker and more pronounced results.

In some of the studies carried out in the Institute's laboratories, the scientists reported that the efficacy of the molecules increased protease activation by as much as fourteen-fold. The researchers say that prior to these results the greatest ever increase in protease activation was only two-fold.

"Since the botulinum neurotoxin is the most poisonous toxin known, finding a compound to activate it might seem somewhat counterproductive,"​ said Jin Janda, head of the lab study.

"But the range of clinical uses for the toxin have increased well beyond its cosmetic use - multiple sclerosis, stroke, cerebral palsy, migraine, and backache are just a few of the conditions for which BoNT has proven surprisingly effective,"​ he added.

"The discovery of small molecule activators may ultimately provide a valuable method for minimizing dosage, reducing resistance, and increasing its clinical efficacy."

As well as being a highly effective treatment, the fact that botulinum neutrotoxins are one of the most lethal poisons known, with side effects including the freezing of muscles, paralysis and even eventual death, great caution has to be shown during its administration.

Medical experts also say that one of the side effects of repeated low-dose therapies is that it could eventually lead to the development of a significant immune response.

Bearing this in mind, the Scripps researchers say that as well as improving efficacy, they believe that there findings could also reduce risk for the treatment.

"We hypothesized that the use of BoNT in combination with a small molecule that could superactivate the action of the toxin would allow for lower doses, and reduce the patient's immune response,"​ Janda said.

"As the importance of BoNT in medicine continues to expand, we need to find some way to counter these unintended immune responses. Compounds like the ones we discovered, which produced the greatest protease activation ever recorded, may point the way to a potential solution."

Doctors first began to use Botox in the 1950s to treat facial twitches. The injections comprise tiny amounts of bovine-sourced poisons that were used to freeze the facial muscles and in turn prevent spasms. However, in administering the treatment it was also discovered that the treatment helped to minimize wrinkling.

Botox started to be used as an anti-wrinkle treatment in the US some ten years ago, since then it has evolved in to a multi-million dollar business. It has proved to be particularly popular for women in the 40 to 60 age group, however, as the treatment has become more and more popular, so too has the diversification of people taking up on the treatment, with younger women as well as men now opting for the treatment.

Costing around $400 a session for a treatment that lasts up to 9 months, it is currently estimated that the number of treatments being carried out in the US every year is now will over 3 million.

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