NEW ORLEANS -- Imagine the excitement of knowing you had a baby girl on the way and then the heartache when something goes wrong, and the baby is born way too soon, causing a disability.
Now one family is the first to join a new study that can help their daughter, and other children and teens can benefit too.
Sarah Derbes just started preschool this week. Her mother Tammy says she is bright and smart at 5 years old, but she knows it's hard for her to keep up with the other children on the playground.
"We didn't find out she had the seizures or cerebral palsy, we found out she had the cerebral palsy about a year and a half," said Tammy Derbes, Sarah's mother.
After three healthy children, Derbes calls Sarah her 40th birthday surprise. She had problems with her placenta, and Sarah was born 10 weeks early at only 2 pounds, three ounces. Her lungs were not fully developed.
She spent the first four months of her life at Lakeside and then at Children's Hospitals.
"It was a different way of life, because you have, you know, a child that has a disability, and it's hard because, you know, we have her sister that comes over with her little friends and she wants to run outside and play with them, you know, and go get on the swing," said Derbes.
"Right now, it's because we've gotten so good at saving very tiny babies, so about 50 percent of the children who have cerebral palsy were born probably 2 pounds or less, or born before maybe 27 weeks, 30 weeks (into the pregnancy,)" said Dr. Ann Tilton, an LSUHSC professor of neurology and pediatrics and section head of child neurology who practices at Children's Hospital.
Dr. Tilton has been with Sarah at Children's Hospital since the beginning. She says cerebral palsy usually happens within the first two years of life, when some sort of deficit or injury happens to the brain or central nervous system.
The children mostly have a problem with motor skills. It's called spastic cerebral palsy. There are no jerky spasms though. Spastic means that the muscles are locked or tight and resist against you when you try to move them. That is why Sarah has braces, because her calves are tight and don't move normally.
For many years, thousands of children around the world as well as Dr. Tilton's patients have had the tight muscles injected with a drug that successfully relaxes them.
Now Sarah is the first child to sign up for a study to get FDA approval for that drug to be used on legs.
You've probably heard of the drug Dysport before, but in a totally different context. It is related to Xeomin and Botox, a drug that adults usually use to temporarily get rid of wrinkles up in the forehead and around the crows feet near the eyes.
But this study is going to use it in a completely different way.
"It's FDA-approved for crossed eyes, eyelid spasms. People know it because of adults with neck spasms, and a lot of other reasons, even cosmetic preparation, but in Europe it's approved in multiple countries for spasticity for children," said Dr. Tilton, who said it is also used to reduce sweat under the arms.
Dr. Tilton is looking for children and teens with this condition who have problems walking. They will get Dysport injections and physical therapy free.
"So if you have a limitation of moving your arm because it's tight or walking because your muscles are tight, you walk up on your toes. You can't extend your leg fully. Then the hope is that you can use one of the medicines and make them move more freely, learn that motor pattern, and over time, the balance between those muscles that are relaxed and those muscles that are tight, works out better," she said. "And it shows to in other studies that it actually allows the muscle to lengthen."
Sarah said her favorite part of school is playing with friends. This drug could give her more freedom to do that.
The study is going on in four countries: France, Mexico, Turkey, and the U.S. To qualify, children have to be between two and 17 years old with spastic cerebral palsy. Call 504-894-5377 for more. You will be compensated for gasoline.
The study is also going on at several other sites in the U.S.