Ensysce Biosciences says early in vivo experiments using single-walled carbon nanotubes to deliver siRNA drugs to cancer tumors have been so promising, the company will proceed to human clinical trials within the next year or so.
The Houston startup is touting data published recently in the open-access online journal Materials, showing the single-walled, fullerene carbon nanotube tech protects the siRNA drug while carrying it through the blood. Once the treatment reached the tumor target, the company claims that it successfully penetrated the tumor and slammed into the target protein, releasing a drug payload that properly displayed anti-tumor activity. What's more, the delivery system also appeared to generate very little toxicity to other cells.
Based on that data, Ensysce says it will launch human trials within 12 to 18 months, tweaking its drug delivery formulation in the interim, company CEO Lynn Kirkpatrick said in a statement. To get there, Ensysce will be armed, in part, with up to $1.5 million in funding from Texas' Emerging Technology Fund and ongoing collaborations with researchers at M.D. Anderson Cancer Center and Rice University.
We'll see. RNAi drugs hold enormous promise in their potential to turn off bad genes that cause disease, but scientists have struggled to find a competent way to deliver the drugs so they reach their target and do their job. Human trials will be interesting to follow because many of the delivery advances so far have taken place in the preclinical stage. One exception: Alnylam ($ALNY) and Tekmira ($TKMR), which have jointly pursued lipid nanoparticles as a tool to deliver RNAi drugs and produced some promising results in early human trials. The two have also fought bitterly over patent and license issues, highlighting what's at stake with finding a successful RNAi delivery mechanism.
Medtronic ($MDT) also has made some progress, working again with Alnylam. But rather than use nanotech, they've generated encouraging results in preclinical testing using a small implantable infusion system and convection-enhanced delivery to break the blood-brain barrier, and deliver an RNAi-based Huntington's disease treatment to the brain.
- here's the release
- access the published study