• Discovery’s Edge: Plugging into drug discovery

The research laboratory of Mayo Clinic pediatric oncologist Richard J. Bram, M.D., Ph.D., was enjoying the kind of success many researchers hope for. His team in Rochester, Minnesota, had long been interested in studying brain tumors, particularly those in children.

“They’re the second most common malignancy in children, but the development of cures has lagged,” Dr. Bram says.

Dr. Bram’s research team made a big discovery: They found that a particular protein called cyclophilin B turned up in abundance in glioblastomas and medulloblastomas, two deadly nervous system tumors.

The protein seemed to be critical to these two cancers. When researchers blocked the protein in cell samples, the tumors died. Even more impressively, when they genetically eliminated the protein in mice, the mice continued to behave normally.

“The findings suggested the protein could be a good target for a drug, selectively killing tumor cells and having a minimal effect on normal ones,” Dr. Bram explains.

But elation was quickly tempered by practicality. Researchers at Mayo Clinic had long taken note of the formidable gulf that often exists between discoveries in the lab and the development of new drugs. Read the rest of the article.
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