Le Bonheur researchers develop new treatment for TSC lesions

TSC is a neurocutaneous disorder characterized by excess cell growth and proliferation, resulting in multi-organ hamartomatosis. Skin lesions occur in more than 90 percent of TSC patients and are more common in late childhood or adolescence.

Historically, the skin lesions have been resistant to medical and surgical treatments. Oral rapamycin has been used in TSC patients, but the side effects prevent its routine use in patients without significant internal involvement.

Neuroscience Institute Co-director James Wheless, MD, collaborated with Hassan Almoazen, PhD, to create a novel rapamycin cream. The cream is easy to compound and apply, does not cause local or systemic side effects, and results in a dramatic improvement of facial angiofibromas.

Le Bonheur Children’s Hospital is one of the nation’s best children’s hospitals, featuring expert physicians and staff, state-of-the-art technology and family-friendly resources.