She may be in a wheelchair, but 14-year-old Lanie Hannah doesn’t let that stop her from having fun. She has SMA. It attacks the body’s motor neurons and causes paralysis.
“It affects my daily life, like not being able to do certain things,” she said.
“Put her in bed, take her out of bed, dressing, I mean it affects everything that she does,” Lana Hannah, Lanie’s mom said.
There is no cure for SMA but doctors are studying an experimental therapy that targets mutated SMN genes, responsible for SMA.
“With this treatment, we are targeting the disease,” said Susan T. Iannaccone, MD, Pediatric Neurologist, Children’s Medical Center in Dallas and UT Southwestern Medical Center.
The gene therapy is injected into the spinal fluid. The idea is the drug will enter into the nerve cells and change the protein production so that the normal SMN gene is created. It could mean fewer symptoms and more strength for patients.
“Our hope, absolutely, is that someday these children will not experience the symptoms,” Dr. Iannaccone said.
Lanie was in the phase I trial. She hopes this therapy will be the answer she’s been waiting for, because she has big plans for the future.
“I want to go to college. I want to be a teacher, and I want to teach English,” Lanie said.
“Everything that she does amazes me,” Lana Hannah said.
Lanie has a milder form of the disease, but she has never been able to walk. Researchers say the next step is to study more patients in a phase II trial to see if the gene therapy does what they hope it will.