Jefferson Researchers Use Gene Therapy To Treat Rare, Inherited Brain Disease

s. "Within a few years, they cannot feed themselves, walk, or see well." Dr. Freese says that the disease is similar to Tay-Sachs disease in that both are metabolic disorders causing the buildup of toxic compounds that affect normal brain development.

According to Dr. Freese, three years ago, his colleagues at Yale were contacted by Canavan families for their expertise in gene therapy to see about the possibility of developing a therapy for the disease. Drs. During, Leone and Freese developed a gene delivery system based on liposomes and polymers, rather than viruses, to deliver the genetic information for the enzyme into the brain.

They have shown that the system works in the lab and in animals. They then showed in preliminary studies in New Zealand that the Canavan gene could effectively be delivered into the brains of two children with Canavan disease, based on evidence of nerve myelination and on behavioral studies. They have gained federal Food and Drug Administration approval, Dr. Freese notes, to expand the work into a larger Phase 1 clinical trial.

To date, the Jefferson team plans to treat four Canavan disease children.

The therapy will consist of a ventricular catheter implanted under the scalp, allowing access to the brain's ventricles and cerebrospinal fluid, and to allow possible additional treatment. The Jefferson researchers are collaborating with specialists at Children's Hospital of Philadelphia to monitor myelin and enzyme levels using magnetic resonance spectroscopy and magnetic resonance imaging. "We'll also look at behavioral aspects of the children--a whole battery of tests before and after gene therapy," Dr. Freese points out.

"We anticipate expanding our gene therapy approach to other inherited metabolic disorders, such as Krabbe disease," he notes, together with David Wenger, Ph.D., professor of medicine at Jefferson Medical College, who leads the Lysosomal Disease Testing Laboratory. "We hope to set up a clinical trial

Contact: Steve Benowitz
Thomas Jefferson University

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