Magazine article The Exceptional Parent

Breaking Barriers Clinical Trial Program: Hope for the Hunter Syndrome Community

Magazine article The Exceptional Parent

Breaking Barriers Clinical Trial Program: Hope for the Hunter Syndrome Community

Article excerpt

A clinical trial program is now enrolling patients for its second cohort in a Phase 1/2a study in Hunter syndrome, a rare, hereditary, lysosomal storage disease. The clinical trial is called Breaking Barriers, and explores AGT-182, an investigational enzyme replacement therapy (ERT) that utilizes the body's natural system for transporting products across the blood-brain barrier (BBB) by targeting the receptor that delivers insulin to all cells of the body, including the brain. Its ability to cross the BBB makes AGT-182 unique among potential treatments for Hunter syndrome.

In people with Hunter syndrome, also known as mucopolysaccharidosis type II, or MPS II, an enzyme called iduronate-2sulfatase (IDS) is missing or does not work properly, leading to an abnormal buildup of complex sugars in tissues throughout the body, including the skeleton, joints, brain, spinal cord, heart, spleen and liver.

Hunter syndrome primarily affects males and is often severe and always progressive in the approximately 7,600 people it impacts worldwide, of which 25% reside in the United States.

Symptoms of Hunter syndrome include growth delay, joint stiffness and coarsening of facial features. In severe cases, patients experience respiratory and cardiac problems, enlargement of the liver and spleen, and neurological deficits that can lead to premature death. Hunter syndrome is almost always severe, progressive and life-limiting. Available treatments for Hunter syndrome are not expected to cross the BBB in clinically relevant amounts and therefore do not address the progressive neurological complications of the disease. …

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