Despite breakthroughs, gene therapy still a long way from mainstream

University of Pennsylvania researchers published preliminary evidence on a  breakthrough in gene therapy this week. By engineering blood cells to destroy cancer cells, they say two leukemia patients are cancer-free after a year.

However, despite recent breakthroughs, the field of gene therapy is still in its experimental stage. The first approved human gene therapy experiment was conducted in 1990 on a child with a rare immune disease. White blood cells were removed from her body and injected with a missing gene, then put back into her body. Nine years later, the field was shaken when a teenager enrolled in a Penn gene therapy trial died.

“The focus of the last ten years has really been to establish safety in these treatments,” said Dr. Barry Byrne, head of the Powell Gene Therapy Center gene therapy center at the University of Florida.

He said now, the field is moving beyond proving safety, and advances have been made, significantly in treating blindness and inherited immune disorders but also in cancer and HIV.

“It’s important to add all those together and see that as a field we see the development of effective treatments,” Byrne said.

Dr. Louis Weiner is head of the Lombardi Comprehensive Cancer Center at Georgetown University and a gene therapy researcher.  He said larger clinical trials are needed before gene therapy treatments like the one out of Penn will win approval for widespread use.

“We now know what can happen when things go right, we now have to determine how frequently things go right,” Weiner said.

After that, complicated and expensive techniques need to be scaled up and researchers would need to win approval and pharmaceutical company support for new treatments. Weiner says the hurdles are significant, but if the Penn case is indeed an example and not an exception, he sees them easily cleared.