Live

Watch CBSN Live

Gene Therapy As A Treatment For Patients Who Suffer From Fanconi Anemia

Gene therapy is a relatively new medical field, one that some people believe has the potential to ultimately control or even cure a variety of diseases. Perhaps that’s why, despite the many safety issues that have been raised, a lot of hopeful people continue to volunteer themselves as human guinea pigs for scientific research.


Eleven year old Angelica has Fanconi anemia, a fatal genetic disorder whose victims seldom survive beyond their 20s. Their bodies simply loose the ability to produce life-sustaining blood cells. She and her family have come from Italy to North Carolina, in hope that Dr. Chris Walsh of the University of North Carolina has a cure for her disease.


Dr. Walsh has been testing a possible solution, using a virus as a delivery van to insert healthy genes into defective blood-forming cells he removed from Angelica.


If it works, the good genes will find Angelica’s bone marrow and start to produce an abundance of healthy cells. But she’s not the first to try. The first was Lynn Mendenhall, who had the distinction of being the oldest known Faconi survivor.


Last Valentine’s Day, Lynn Mendenhall lived her dream, pioneering gene therapy for Fanconi anemia.


"The fact that this worked in the mouse, and I’m convinced that we did everything the right way, tells me that this will work," said Dr. Walsh at the time. "The number of the stem cells that we recovered for her were very low. We still had enough to transfer the gene and we have results that show that the transfer did work," But the healthy genes had no affect on Lynn. Five months after receiving them, Lynn Mendenhall died of a consequence of Fanconi anemia, cancer. Still, she’d proven the process was safe.


In a decade of trying, researchers hadn’t been able to make it work in humans and the challenge became even tougher in the death of the 18 year old Jessie Gelsinger. In a gene therapy experiment at the University of Pennsylvania, Jesse became the first death ever of a volunteer of this brave New World research.


His death led to Washington hearings on how closely gene therapy experiments are being monitored. The bad news seemed to deflate ten years of hype about this potential cure. But then, good news started to trickle in from French researchers treating children with immune deficiencies to American scientists growing blood vessels in diseased hearts.


Dr. W. French Anderson of the University of Southern California speaks on gene therapy: " It’s not living up to all the hype yet, but now we can see that we’re turning the corner, we’re starting to see success."


"She wanted to be as helpful as she possibly could for other patients," says Dr. Walsh of Lynn Mendenhall. Patients like Angelica Synder and her parents. Dr. Walsh is analyzing Angelica’s bone marrow now, to see if healthy new blood cells have begun to appear. All of Angelica’s mother’s dreams and hopes are in gene thrapy. But Angelica’s father realizes that they are participating in a scientific trial and it may not be enough.


It would be a medical dream come true and a new life for Angelica.

©MMII CBS Worldwide Inc. All Rights Reserved. This material may not be published, broadcast, rewritten, or redistributed

View CBS News In
CBS News App Open
Chrome Safari Continue