Conner’s Historic Gene Therapy Treatment Gives His Family New Hope

On March 16, 2023, six-year-old Conner Hess became the first patient worldwide to receive SKYSONA for childhood cerebral adrenoleukodystrophy (CCALD) outside a clinical trial. For his parents, Richelle and Adam, the milestone offered a chance to protect their son’s future.

Early Screening Reveals a Serious Risk

Newborn screening identified Conner’s adrenoleukodystrophy, prompting regular MRI scans to watch for changes in his brain.

In September 2022, a scan revealed early signs of cerebral disease. CCALD damages the protective covering around nerve fibers in the brain and can cause severe neurological decline.

His parents began searching for treatment, but no suitable stem cell donor was available. A referral to Boston Children’s Hospital introduced them to another possibility: gene therapy using Conner’s own cells.

How SKYSONA Works

SKYSONA is made from a patient’s own blood-forming stem cells. In a laboratory, functional copies of the ABCD1 gene are added to help the cells produce a protein involved in breaking down certain fatty molecules.

Before receiving the modified cells, the patient undergoes chemotherapy to prepare the bone marrow. The cells are then returned through an intravenous infusion, with the goal of slowing the progression of neurological damage.

Conner’s stem cells were collected in January 2023. He returned in March for chemotherapy and his historic infusion.

Treatment Requires Lifelong Follow-Up

SKYSONA does not guarantee that the disease will stop progressing. It also carries serious risks, including blood cancers such as myelodysplastic syndrome and acute myeloid leukemia.

Updated prescribing information calls for lifelong monitoring for blood cancer, along with continued checks for worsening cerebral disease. Follow-up care is therefore an essential part of the treatment journey.

A Chance to Keep Growing

For Conner’s family, treatment brought hope after months of uncertainty. Their wish was for him to have more time to grow, learn, and enjoy the everyday childhood moments they wanted to protect.