Clinical Trials within U.S.: Batten Disease

BATTEN DISEASE IS an inherited disorder of the nervous system that begins in childhood. Children with Batten disease suffer seizures and progressive loss of motor skills, sight, and mental capacity, eventually becoming blind, bedridden, and unable to communicate. Batten disease is often fatal by the late teens or 20s. As yet, no specific treatment is known that can halt or reverse the symptoms of Batten disease. However, seizures can sometimes be reduced or controlled with anticonvulsant drugs, and other medical problems can be treated appropriately as they arise. Physical therapy and occupational therapy also may help patients retain functioning as long as possible.

Recently, a company (StemCells, Inc. of Palo Alto, California) has initiated a phase 1 clinical trial to investigate the safety and preliminary efficacy ...