University of North Florida researchers have achieved a significant breakthrough in cancer treatment, developing a targeted compound that causes cancer cells to self-destruct while sparing healthy tissue. The team, led by Dr. Bryan Knuckley, Department of Chemistry and Biochemistry chair and professor, along with Dr. Corey Causey and Dr. Fatima Rehman, has received a U.S. patent for their innovative peptoid compound and is preparing to advance to animal trials this fall.
Revolutionary Mechanism Targets Cancer at the Source
The research focuses on a group of proteins called protein arginine methyltransferases (PRMT), which become overproduced and highly activated in certain cancers. When these proteins bind with other proteins in the body, they create a chemical mark called methylation that causes previously suppressed cancer genes to become active, essentially "turning cancer on."
"We're still early on in the process, but this is an exciting development, and the research shows promising intervention for the treatment of some aggressive cancers," said Knuckley, who began this research shortly after joining UNF's faculty in 2012.
The team's peptoid compound specifically targets these PRMT proteins, disrupting the methylation process that activates cancer genes. This targeted approach represents a fundamental shift from conventional cancer treatments.
Selective Cancer Cell Destruction
The compound's most remarkable feature is its ability to cause specific cancer cells to explode while having minimal to no effect on healthy cells. This selectivity could potentially eliminate the harsh side effects typically associated with chemotherapy and radiation treatments.
Dr. Fatima Rehman played a crucial role in testing the efficacy and specificity of these novel compounds in human cancer and normal cells, assessing their potential as a targeted cancer therapy. The research has shown promise against breast, colon, and lung cancers specifically.
Patent Achievement and Future Development
UNF is among the very first studies to explore the use of peptoids for early cancer diagnosis and potential treatment, and is the only known university conducting this type of study. The team has received their first patent focused on the treatment of cancers using peptoids targeting PRMTs, with a second patent for the specific peptoid compounds awaiting approval within the year.
Knuckley spearheaded the project by identifying and developing new peptoid compounds and conducting biochemical studies to validate their targeting of PRMT proteins. Causey created the essential building blocks needed for the synthesis of these innovative peptoids. Several undergraduate students have also been involved in the research since 2019.
Expanding Therapeutic Potential
Beyond cancer treatment, the researchers note that their compound shows promise for other conditions, including dementia. The team is currently examining the exact mechanism of action through which these peptoids kill cancer cells specifically, while conducting further testing of individual peptoids designed during the study.
"This is a lifetime project to contribute to ending cancer," said Knuckley. "We want to continue our research and testing to better understand how these proteins work and how we can improve upon the inhibitor to develop new therapies or pharmaceuticals."
Scientific Validation and Next Steps
The discovery emerged through an unexpected pathway, as Knuckley noted: "In science, you have these hiccups, these things that come up where it doesn't work, and so you can be disappointed. No different than when we originally found this compound. It was through disappointment that we realized, oh wait -- this is something good."
The team has spent the past year in laboratory testing their revolutionary compound, with results published in the Journal of Biological Chemistry. Animal trials are scheduled to begin this fall, marking the next critical phase in translating this laboratory discovery into potential clinical applications.
The significance of this targeted approach lies in its potential to revolutionize cancer treatment by eliminating the collateral damage to healthy cells that characterizes current therapeutic approaches, potentially offering patients effective treatment without the debilitating side effects of conventional cancer therapies.
