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Research made easy: Ketamine Clinical Trial

by Rachael Bloom

Ketamine? Isn’t that a tranquiliser?

Ketamine is a sedative which is most often used as an anaesthetic in operations. However, at much lower doses it has been shown to significantly improve symptoms in disorders such as major depression and obsessive-compulsive disorder and to be effective in pain management.

So what’s that got to do with Rett Syndrome?

Professor David Katz at Case Western Reserve University School of Medicine in Ohio, USA, has studied the impact of Ketamine on Rett mice.

Rett mice show imbalances in nerve cell activity in their brain. In some areas of the brain there is too much activity and in some, there is not enough. 

Professor Katz gave Ketamine to Rett mice in an attempt to correct these imbalances. Even at low doses, this imbalance in brain activity was corrected.  There were also significant improvements in neurological function, including breathing and reactions to sensory stimuli.

What will happen next?

The Case Western Reserve and Cleveland Clinic researchers will soon be launching a two-year clinical trial using low doses of Ketamine in up to 35 individuals with Rett Syndrome.

The trial will focus primarily on measuring changes in breathing patterns and overall severity of the patients’ condition.

Each participant will receive placebo* as well as different doses of Ketamine in a random order.

Comparing these results will enable researchers to work out if ketamine is effective as a treatment for Rett Syndrome and if so, what dose is best.

If the trial is successful, the next step will be to test whether the drug can be given on an on-going basis, rather than just a few doses.

Read the full Case Western press release about the Ketamine clinical trial here.

This work has been funded by the Rett Syndrome Research Trust.

Please note that no information is currently available re recruiting for this trial. You can find further information about current clinical trials for Rett Syndrome here.

*A placebo is a substance which has no effect, which is used to test drugs because sometimes a patient’s condition can improve or can be reported to improve, just because of the expectation that it will. This is known as the ‘placebo effect.’