Encouraging preliminary result in treating Fragile X syndrome

Fragile X syndrome (FXS) is a genetic disorder characterized by several symptoms in common with autism, as well as by learning and cognitive deficits, ADHD and epilepsy. As discussed in Chap. 9, several different therapies have shown positive results in mouse models of FXS, and so a number of clinical trials of those tehrapies are underway. One trial not discussed in the book involves minocyline, which is a medication used originally as an antibiotic, but also has anti-inflammatory activity. It is prescribed for adolescent acne and is being tested in a variety of neurodegenerative disorders. In the first clinical trial for FXS (Paribello et al., 2010), 20 patients (ages 13-22) were treated for 8 weeks in an open label paradigm, that is, they knew they were being given the medication. This of course raises the possibility of a placebo effect. The results of this trial revealed significant improvement in a number of different tests, including irritability, speech, attention deficits, self-injurious behavior, anxiety, social avoidance and repetitive behavior. No serious side effects were observed, although a potential risk for auto-immunity was found in 2 patients. This is a worry, since minocycline can cause auto-immunity in children. Nonetheless, minocycline is approved for long-term use in treating acne. Its antibiotic activity is also a worry, particularly in light of likely effects on intestinal microbiota. Another noteworthy p0int is that several preliminary studies (including double blind studies) on schizophrenia subjects suggest that minocycline can augment the positive effects of anti-psychotic medications. Similar results have been obtained in preliminary trials with non-steroidal anti-inflammatory drugs, altho the efficacy may depend on the severity of the symptoms and/or where in the time course of the disease the drugs are administered (see 2011 review by Urs Meyer).

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