Case, Cleveland Clinic researchers receive $1.3 million to explore Rett Syndrome treatment

By TIMOTHY MAGAW
Originally Published: February 03, 2015 12:55 PM Modified: February 03, 2015 4:00 PM

Researchers at Case Western Reserve University and Cleveland Clinic have received $1.3 million to explore a treatment for Rett Syndrome — a disease that manifests itself in early childhood and leaves sufferers dependent on 24-hour-a-day care for the rest of their lives.

The funding, which comes from the Rett Syndrome Research Trust, will support a Phase 2 clinical trial involving low-doses of ketamine, which is a drug that has historically been used for sedation and anesthesia.

Research from David Katz, a Case Western Reserve University professor and one of the leaders of the study, has shown that low-dose ketamine can reverse deficits in brain activity in mice, which have also shown improvements in neurological function, including breathing. Dr. Daniel Sessler, chair of the department of outcomes research at the Clinic, also is one of the study’s leaders.

"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."

Co-investigators on the project include Tom Frazier, director of the Cleveland Clinic Center for Autism; Dr. Sumit Parikh, director of the Cleveland Clinic Neurogenetics, Metabolic & Mitochondrial Disease Program; and Edward J. Mascha, senior biostatistician in the Department of Outcomes Research at the Cleveland Clinic.

The award is part of $5.8 million in funding awarded last year by the Rett Syndrome Research Trust. Since 2008, it has awarded $25 million to research into the disorder, which is caused by mutations in a gene called MECP2 that result in a series of symptoms that worsen over time.