Clinical Trial to Test Ketamine to Treat Rett Syndrome

Ketamine vials (Licensed under Public Domain via Wikimedia Commons)

23 March 2015. A clinical trial is planned to test an anesthetic used in surgery as a treatment for Rett syndrome, a rare developmental disorder affecting girls. The trial testing the anesthetic ketamine will be conducted by Case Western Reserve University medical school in Cleveland, funded by a $1.3 million grant from Rett Syndrome Research Trust.

Rett syndrome is a genetic disorder affecting 1 in 10,000 to 15,000 female births, and while it is caused by a mutation in the MECP2 gene, the disease is not inherited. Symptoms of Rett syndrome are similar to autism, reflected in problems with communication, learning, and coordination, as well as breathing difficulties such as hyperventilation, beginning at about 6 months of age. Treatments for Rett syndrome generally involve management of symptoms and occupational skill therapy.

The clinical trial will test the ability of ketamine to reverse symptoms of Rett syndrome. Neuroscience professor David Katz at Case Western Reserve tested ketamine as a therapy for Rett syndrome in lab mice genetically engineered to exhibit the disease. Katz reports that low doses of ketamine were able to rebalance characteristic neurological activity of Rett syndrome in the mice and show improved neurological functions.

While ketamine is widely used a sedative and anesthetic in surgery, it also has a checkered history. Ketamine was a 1990s-era party drug known on the street as Special K, but is showing promise recently as a treatment for depression, working as quickly as 6 hours and with the capability of reducing suicidal thoughts. Due to ketamine’s potential for abuse and ability to cause adverse effects, such as nausea and hallucinations, clinicians are moving ahead cautiously with the drug.

“Because ketamine was initially developed for use in surgical anesthesia, it has never been used to treat a chronic illness,” says Katz in a university statement. “So one of the next questions will be whether we can develop a chronic dosing paradigm with this or similar drugs that would be safe and effective for patients with Rett syndrome.”

The trial led by Katz, and with colleagues at the Cleveland Clinic, plans answer those questions by enrolling 35 individuals with Rett  syndrome. Participants will receive ketamine in various low doses or a placebo, in random order, in multiple 2-day sessions, with each session about a month apart. The researchers will look particularly at changes in breathing and behavioral symptoms.

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