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Gene Therapy: Science Fiction or Science Fact?

Children's Health
Author name: Dr. Britt Stroud


Gene therapy sounds like science fiction: Introducing healthy genes into a person who lacks them to treat disease. Then again, video chatting, bionic limbs, and space travel were "far-out" sci-fi imaginings that have become as common as the house fly.

So how does gene therapy work?

If a patient has a bad version of a gene, gene replacement therapy can deliver a good version of that gene into the patient's cells without surgery or drugs, explains Dr. Britt Stroud, a pediatric neurologist with Lee Health.

"Gene replacement offers the potential to treat a variety of rare and common diseases such as ALS, Parkinson's disease, retinal disease, hemophilias, and cancers," Dr. Stroud says.

The Food and Drug Administration (FDA) approved the first gene replacement therapy to treat children less than 2 years old with spinal muscular atrophy (SMA).

The rarest of children's diseases -- it occurs about one in 10,000 kids worldwide – SMA (type 1) is the deadliest. SMA affects the motor nerve cells in the nervous system—making children unable to walk, eat, or breath. Studies report that only 8 percent of children diagnosed with SMA live to 20 months old.

The FDA-approved gene replacement therapy, called Zolgensma, is keeping children with SMA and their hopes alive—and Golisano Children's Hospital of Southwest Florida has it. In fact, the children's hospital is only one of five pediatric centers in Florida and the only one between Tampa and Miami to offer the ground-breaking gene therapy treatment.

"We're working constantly to bring world-class care to this region," says Armando Llechu, chief administrative officer of Golisano Children's Hospital. "That we offer gene therapy is another example of our effort to ensure that the children of Southwest Florida have access to the best medical care possible."

How does Zolgensma work?

  • Zolgensma is available for children with SMA who are less than 2 years of age.
  • Zolgensma replaces the nonworking or missing gene that causes SMA.
  • It transfers into the cells a new, fully functional gene that instructs the cells to make the survival motor neuron (SMN) protein.
  • Patients only require one dose of Zolgensma gene therapy, which is given through an IV in a 60-minute treatment.

Is this treatment right for your child?

Dr. Stroud stresses that, as with any medical decision, the right choice is the one a family makes with a doctor they trust after fully understanding its potential benefits and risks of treatment.

"To get the best outcome, it's critical that treatment start before symptoms occur and before irreversible motor neuron loss happens," he cautions. "This is especially true for kids who have SMA type 1. Without the treatments available today, these kids eventually would have died prematurely or needed permanent ventilation."

"We're proud to bring innovative treatments for SMA to patients of the Golisano Pediatric Neurology program," Dr. Stroud says. "Golisano Children's Hospital remains dedicated to bringing progressive, evidence-based care to the children of Southwest Florida."

Talk with your pediatric neurologist to learn more about this therapy.

If you would to contribute to Healthy News, please contact us at [email protected]

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