Gene Therapy May be Key To Unlock Canavan's Disease
Children With Canavan Usually Doomed To Short Life
POSTED: 3:43 pm EDT April 28,
2003
UPDATED: 6:30 pm EDT April 28,
2003
A medical breakthrough is giving a 2-year-old girl a chance for a healthy, normal life.This is the first time that a child this young has received gene therapy in brain surgery.
When Lana Swancey was just 6 months old, doctors told her mother that there was no hope Lana would ever live a healthy, normal life. Canavan's disease is destroying cells in Lana's brain.But now, doctors at Cooper University Hospital in Camden, N.J., may be able to help stop the debilitating disease with experimental gene transfer."We went to Duke University within a month of her being diagnosed. They told us, 'Enjoy what you have. Don't see moonbeams.' That's what they kept telling us," said Michelle Swancey, Lana's mother.That was about 18 months ago. Now, Lana is just two days shy of her second birthday. However, the destruction caused by Canavan's disease has left her body in a stage of development that is more like that of a three-month-old.
The disease attacks the myelin cells -- the part of the brain that helps to transmit and receive impulses.Lana can't hold up her head, can't walk and can't talk. But her family has hope.Dr. Paula Leone is a neuroscientist at Cooper University Hospital. She has been studying Canavan's disease and working on a treatment for most of the last decade."Tomorrow Lana will have a brain surgery, where we are going to have six openings and we are going to lower catheters of the gene that she's missing-- she will actually have the gene -- enough enzyme for the disease to stop (and) for the brain to function normally," Leone said.Leone said that within three months they should know whether the therapy is a success."What we think is going to happen (is) that we are going to be able to see within the first three months, if the brain has accepted the gene, if it's making the gene and if there's myelin being produced," Leone said.Leone hopes that the undamaged cells will be boosted by the treatment -- allowing Lana to develop normal skills. Leone also hopes that someday Lana may be able to run and play like other children her age. "I have not dreamed about her until lately, and I actually dreamed about a black-headed little girl with a big round face sitting on my lap smiling and laughing, without me having to hold her head," Michelle Swancey said.Doctors hope the gene therapy being used in Layna's surgery Tuesday will eventually be able to be done on even younger children to stop the progression of the disease much sooner.There are six other children in Leone's study that will receive the gene transfer this summer.
When Lana Swancey was just 6 months old, doctors told her mother that there was no hope Lana would ever live a healthy, normal life. Canavan's disease is destroying cells in Lana's brain.But now, doctors at Cooper University Hospital in Camden, N.J., may be able to help stop the debilitating disease with experimental gene transfer."We went to Duke University within a month of her being diagnosed. They told us, 'Enjoy what you have. Don't see moonbeams.' That's what they kept telling us," said Michelle Swancey, Lana's mother.That was about 18 months ago. Now, Lana is just two days shy of her second birthday. However, the destruction caused by Canavan's disease has left her body in a stage of development that is more like that of a three-month-old.The disease attacks the myelin cells -- the part of the brain that helps to transmit and receive impulses.Lana can't hold up her head, can't walk and can't talk. But her family has hope.Dr. Paula Leone is a neuroscientist at Cooper University Hospital. She has been studying Canavan's disease and working on a treatment for most of the last decade."Tomorrow Lana will have a brain surgery, where we are going to have six openings and we are going to lower catheters of the gene that she's missing-- she will actually have the gene -- enough enzyme for the disease to stop (and) for the brain to function normally," Leone said.Leone said that within three months they should know whether the therapy is a success."What we think is going to happen (is) that we are going to be able to see within the first three months, if the brain has accepted the gene, if it's making the gene and if there's myelin being produced," Leone said.Leone hopes that the undamaged cells will be boosted by the treatment -- allowing Lana to develop normal skills. Leone also hopes that someday Lana may be able to run and play like other children her age. "I have not dreamed about her until lately, and I actually dreamed about a black-headed little girl with a big round face sitting on my lap smiling and laughing, without me having to hold her head," Michelle Swancey said.Doctors hope the gene therapy being used in Layna's surgery Tuesday will eventually be able to be done on even younger children to stop the progression of the disease much sooner.There are six other children in Leone's study that will receive the gene transfer this summer. Copyright 2006 by NBC10.com. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.
