Minocycline is an antibiotic that has been used for years to treat bacterial infections. In recent years, scientists have explored its potential to improve nerve function in children with Angelman syndrome, or AS.

Medications such as minocycline that already have a proven safety record are attractive to researchers trying to find ways to treat people with rare diseases. Clinical trials of yet to be approved therapies may put people at risk because researchers have yet to learn about any adverse effects of the drugs.

How Minocycline works

AS symptoms, such as poor language skills, poor movement function, seizures, developmental delays, and behavioral issues, occur because of faulty communication between nerve cells in the brain. The genetic defect that causes AS affects the shape of dendrites, branches at the end of nerve cells that receive messages from other nerve cells.

It is a small molecule that is able to pass through the blood brain barrier, a membrane that protects the brain from harmful substances. Research in mice has shown that minocycline changes the shape of dendrites in the brain in a way that improves communication between nerve cells. Minocycline may also have other properties that protect nerve cells and improve nerve function.

Minocycline in clinical trials

Two clinical trials conducted in 2014 explored the idea that minocycline might benefit people with AS. The first was an open-label pilot study (NCT01531582) involving 25 children with the disorder, ages 4 to 12. Each child received minocycline by mouth for eight weeks.

Investigators used changes in scores of measures of children’s development, language, and behavior to assess minocycline’s effectiveness.

The study, published in the journal BMC Neurology in December 2014, showed that minocycline led to significant improvements in children’s communication, listening, fine movement skills, and ability to change behavior. The children tolerated the medication well and did not experience treatment-related adverse events.

The second trial (NCT02056665) was a randomized, double-blind, placebo-controlled study. Participants included both children and adults, ages 6 to 30.

They were assigned daily doses based on their body weight, with larger doses for those weighing more. They received minocycline in the morning and evening for eight weeks. The placebo group received tablets that looked identical to minocycline tablets at similar intervals. The study was completed in November 2014, but results have not been published.

Additional information

Studies have also explored minocycline as a treatment for other neurologic disorders that affect brain function. A study published in BMC Neurology in October 2010 reported that eight weeks of minocycline led to improvements in the behavior of children with Fragile X syndrome.

Other studies found that minocycline reduced the severity and progression of symptoms in animal models of Alzheimer’s, Parkinson’s, and ALS, or Lou Gehrig’s disease.

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