Botox-encoded mRNA therapy that makes tumors kill themselves

“Our idea was to deliver safe mRNA molecules encoded for a bacterial toxin directly to the cancer cells – inducing these cells to actually produce the toxic protein that would later kill them. It’s like placing a Trojan horse inside the cancer cell,” Dan Peer, one of the study authors and Vice President of TAU’s research and development division, told World Israel News.

How does the toxin mRNA treatment work?

Previous studies suggest that Botox or botulin toxin can make cancer patients more responsive to certain cancer therapies, including chemo. It’s generally safe for human use and can potentially reduce the growth and multiplication of cancer tumors. 

Skin cancer experts report that when scientists used Botox with chemo together, it improved the survival rate of test subjects by 35 percent. This anti-cancer potential of the toxin inspired the TAU team to perform an exciting experiment. 

They extracted Botox from pseudomonas bacteria and encoded it into mRNA molecules. Next, they packaged the molecules inside lipid nanoparticles and injected them directly into tumor cells inside animal models, a feat that has never been achieved. 

The test subjects had melanoma, a type of skin cancer. The mRNA reprogrammed the cancer tumors, producing toxin proteins that eventually killed them. Interestingly, a similar approach was also used to develop mRNA COVID-19 vaccines.

“With a simple injection to the tumor bed, we can cause cancer cells to ‘commit suicide’ without damaging healthy cells,” said Peer. 

This treatment worked so well that it wiped out 40 to 60 percent of cancer cells in the animal models. According to the researchers, the more exciting thing is that many other bacterial toxins could be used to prevent cancer. 

So, for example, scientists can employ another natural toxin if cancer tumors somehow resist Botox. However, no such solution exists for a patient who develops chemotherapy resistance.

“This platform may represent a new class of treatment that can address an unmet clinical need for solid tumors therapy. We hope to further investigate into this aspect and demonstrate improved therapeutic effect in more tumor models in the future.” the researchers note.

The study is published in the journal Theranostics.