First cancer treatment developed at UofL now entering clinical trials

by magazine staff last modified Sep 19, 2008 07:12 AM

First cancer treatment developed at UofL now entering clinical trials

The compound was developed by UofL scientists John Trent and Paula Bates (left) and Donald Miller (inset), director of the university's James Graham Brown Cancer Center.

A new cancer treatment developed by researchers at the University of Louisville's James Graham Brown Cancer Center was given to a patient at the center for the first time this fall.

The compound, AGRO100, was approved by the Food and Drug Administration for Phase I clinical trials in October.

The trial, scheduled to end in the spring, is the first human testing of new compounds called GROs, or Guanine-Rich Oligonucleotides, discovered by UofL researchers Paula Bates, Ph.D., Donald Miller, M.D., Ph.D., and John Trent, Ph.D.

"One of the reasons that we are so excited about the potential of these GROs is that they work by a new mechanism that is completely different from other agents that are currently used to treat cancer," Bates explained. "These compounds are very effective in blocking the growth of cancer cells but have little effect on the growth of normal cells."

GROs are unusually stable short synthetic pieces of DNA that bind tightly to a specific protein present on the surface of cancer cells, interfering with tumor growth.

"The unique thing about this GRO is that it's cancer selective," Trent said. "It selects cancer cells over normal cells, which should reduce side effects."

"If we can prove that the drug is safe, effectively stunts tumor growth and causes fewer unpleasant side effects for the patient, we will have won a significant battle in the war on cancer," said Damian Laber, M.D., a UofL medical oncologist and principal investigator of the trial.

The trial also marks a significant achievement for the university.

"This trial represents many of the great things happening at the James Graham Brown Cancer Center and UofL," said Miller, the center's director.

"The swift movement of this compound from the research lab to the clinic is exactly the type of translational research outcome for which we strive, and the potential benefit to patients underscores the ultimate goal of everything we do. That we're able to do all of this in Louisville, thereby helping to support the local economy, is an added bonus."

The GRO is one of the technologies that is being developed by Aptamera, a Louisville biotechnology company founded by Bates, Trent and Miller.

The University of Louisville Research Foundation and Miller hold a financial interest in Aptamera. Multiple steps to ensure patient protection and research integrity have been taken consistent with the university's policy for oversight of institutional financial interests in research.

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