By Brady Huggett

Athersys Inc. is collaborating with the Cleveland Clinic Foundation to discover and functionally validate cancer and inflammation drug targets.

The companies will work to identify novel drug targets that regulate certain cell-signaling and disease pathways, with Athersys having an exclusive option to commercialize any novel therapeutics that result.

The researchers at the Cleveland Clinic Foundation will work under George Stark, chairman of the Lerner Research Institute at CCF, and one of the main reasons Athersys entered the collaboration.

"We were attracted to George Stark's expertise in signal transduction," said Gil Van Bokkelen, president, CEO and chairman at Athersys. "The Cleveland Clinic is right up the street and George and I have passed each other a few times. We knew he was a leader in his field."

Athersys, of Cleveland, will supply its Random Activation of Gene Expression (RAGE) technology to the Cleveland Clinic Foundation researchers. The companies will work to identify regulators of pathways in cells for nuclear factor-kappaB and p53. Nuclear factor-kappaB is a transcription factor involved in inflammation and cancer, among other things, and p53 is a tumor suppressor protein believed to be mutated or inactivated in more than half of all human tumors.

"We will basically do what we are best at," Van Bokkelen said. "We'll turn [leads] over to George and his research team to do the screening. Then we will get together to validate the targets." He added that most of the lead work should be done in the next 12 months.

The RAGE technology is a novel gene expression system that can produce protein from virtually every gene in the human genome without cloning individual genes or using cDNA libraries. Van Bokkelen said RAGE can enable the correlation of a disease process or characteristic with expression of a specific protein. Athersys applies its RAGE technology in four areas: functional genomics, generation of cell lines expressing validated drug targets, production of known therapeutic proteins and gene discovery.

The collaboration has designed research endpoints, Van Bokkelen said, but no money will change hands.

"We are carrying our own costs," Van Bokkelen said. "We construct the libraries, and George's team is responsible for the things on the other end." He added that if Athersys is not interested in resulting therapeutics, then the CCF could develop them, but Athersys would retain an owner's stake. He also said the deal might be expanded.

"There is a good chance of that in the future," he said. "We have mapped out areas that we might be able to expand into, but right now we are taking it one step at a time." n