Research Is Yielding A Reason To Hope Cystic Fibrosis Could Be The First Disease Conquered By Gene Therapy. If That Happens, Managing Other Diseases In That Way Could Follow. Scientists, Including Some At Penn, Are Moving Closer To That Goal.

July 23, 1995|By Donald C. Drake and Jim Detjen, INQUIRER STAFF WRITERS

Six scientists sat patiently as Yiping Yang, a young researcher at the University of Pennsylvania Medical Center, prepared to show color slides of mouse lung and liver tissue.

The slides involved a complicated study of a new type of cystic fibrosis

drug, but no scientific training was needed to interpret them:

Pink was bad; blue was good.

Pink meant that the scientists had no idea why the drug stopped working after a few weeks. Blue meant they had just found out why. This would be a breakthrough in their quest to wipe out disease with a new type of treatment called gene therapy.

Story continues below.

Yang turned on the slide projector and a huge picture of mouse tissue, stained with dye, appeared on the screen at the end of the conference table. It was fuzzy. Even before Yang focused the projector, it was obviously pink. He showed another slide, again pink.

James M. Wilson, his boss, looked at the slides without comment. He knew it was unlikely they would easily solve a problem fundamental to the entire field of gene therapy.

Yang explained that the pink slides were of tissue taken from normal laboratory mice. Now he wanted to show slides from mice that were identical, with one exception. They had no immune systems.

Yang flashed the first one onto the screen. It was blue. So was the next. And the next. They were all blue.

Yang snapped off the machine and sat down. His colleagues at the Institute for Human Gene Therapy went on with the meeting, taking turns to report on their own research. But Wilson was no longer listening.

He was stunned. His mind was filled with the implications of the blue slides. Yang's finding would alter the course of his laboratory's research. Wilson was now convinced that he knew how to make gene therapy work.

As Wilson left the meeting that day last December in elation, his mind kept coming back to one word:

Cure.

*

The age of gene therapy has dawned.

More than 80 trials involving almost 600 patients have started or will soon begin in the United States amid predictions that wondrous things are about to happen.

"Gene therapy will create a revolution in the practice of medicine and substantially alter the pharmaceutical industry," said Kenneth W. Culver, a gene therapy pioneer and executive director of the Human Gene Therapy Research Institute in Des Moines, Iowa.

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