Rett Syndrome Research Trust Awards $1.3 Million For Ketamine Clinical Trial

By John Lavitt 03/20/15

Researchers are hoping to learn if low doses of ketamine can reverse the debilitating disease.

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The Rett Syndrome Research Trust (RSRT) awarded a Cleveland-based research team $1.3 million for a controlled clinical trial of low-dose ketamine for the treatment of Rett syndrome.

Rett syndrome (RTT) is caused by mutations in the MECP2 gene that result in a cascade of devastating physical symptoms that worsen over time. The neurodevelopmental disorder affects females almost exclusively. It is characterized by the loss of purposeful use of the hands, slowed brain and head growth, problems with walking, seizures, and intellectual disability. The disorder was identified by Dr. Andreas Rett, an Austrian physician who first described it in a journal article in 1966.

As an NMDA receptor antagonist, ketamine is a medication used mainly for starting and maintaining anesthesia. The drug induces a trancelike state while providing pain relief and sedation. Being led by David Katz, Ph.D., Professor of Neurosciences and Psychiatry at Case Western Reserve University School of Medicine and Dr. Daniel I. Sessler of the Cleveland Clinic, the clinical trial will test the effect of single doses of ketamine on breathing abnormalities and overall clinical severity, as well as EEG abnormalities, visually evoked potentials, and repetitive behaviors.

Past studies undertaken by Dr. Katz have shown that low-dose ketamine can reverse deficits in brain activity in mouse models of RTT in conjunction with significant improvements in neurological function, including breathing.

“This trial evolved as a dynamic collaboration among basic scientists, clinicians, and clinical trialists including expert advisers recruited by RSRT,” Katz and Sessler said in a joint statement. “We are grateful to RSRT for fostering this collaborative spirit and providing the support necessary to make this trial a reality.”

Since 2008, the Rett Syndrome Research Trust has awarded $25 million to research into the disorder. Monica Coenraads, RSRT Executive Director and mother of an 18 year-old daughter with RTT, explained how “[e]very dollar donated to RSRT can be tracked back to a family of an affected child and this family’s network of relatives, friends and colleagues."

"Our supporters around the world make our work possible and they have my immense appreciation," Coenraads said. "If you love a child with Rett or MECP2 Duplication Syndrome and find their current status quo unacceptable then I invite you to join us in our effort to end their immense suffering.”

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Growing up in Manhattan as a stutterer, John Lavitt discovered that writing was the best way to express himself when the words would not come. After graduating with honors from Brown University, he lived on the Greek island of Patmos, studying with his mentor, the late American poet Robert Lax. As a writer, John’s published work includes three articles in Chicken Soup For The Soul volumes and poems in multiple poetry journals and compilations. Active in recovery, John has been the Treatment Professional News Editor for The Fix. Since 2015, he has published over 500 articles on the addiction and recovery news website. Today, he lives in Los Angeles, trying his best to be happy and creative. Find John on Facebook, Twitter, and LinkedIn.