Adeline Vanderver, MD, Program Director of the Leukodystrophy Center in the Division of Neurology at Children’s Hospital of Philadelphia (CHOP), provides an overview of the different types of clinical trials that are underway to treat individuals with leukodystrophies.
Leukodystrophies are a group of rare, progressive, metabolic, genetic diseases that affect the central and peripheral nervous system. Each type is due to a specific genetic abnormality that results in reduced myelin sheaths along neural axons. Symptoms vary with each type as well, but can dramatically limit movement, sensory abilities, and overall development. Although there are currently no approved clinical treatments for leukodystrophies, several clinical trials are currently underway. More are expected to be launched in the near future.
The existing leukodystrophy clinical trials fall into several broad categories: Those that are designed to address major symptoms, such as inflammatory processes, and those that target the underlying genetic mutations that lead to leukodystrophies.
Dr. Vanderver acknowledges that the concept of myelin restoration is still in the early stages of development. However, she remains hopeful that this approach will eventually lead to viable treatment options for affected individuals.
To learn more about leukodystrophies, visit theglia.org.
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