Ketamine as a Treatment for Rett Syndrome

Case Western Reserve University Background
Rett Syndrome (RTT) is one of a number of pervasive developmental disorders characterized by delays in the development of multiple basic functions including socialization and communication. Patients with RTT, after a period of apparently normal early postnatal development, develop a spectrum of symptoms that generally includes loss of acquired speech, head growth deceleration, autistic behaviors, motor, respiratory and autonomic dysfunction and increased risk of seizures.
RTT is caused by loss-of-function mutations in the gene encoding the methyl-CpG binding protein 2 (MeCP2), a transcriptional regulatory protein. Remarkably, mouse models of RTT with reversible inactivation of the MeCP2 gene have shown that RTT symptoms can be largely reversed upon re-activation of MeCP2. One effect of MeCP2 inactivation is the dysregulation of the N-methyl-D-aspartate receptor (NMDAR), and it has been shown that NMDAR antagonists have the potential to ameliorate the symptoms of RTT patients.
Technology Overview
Researchers at Case Western Reserve University (CWRU) have discovered a treatment regimen for RTT that utilizes ketamine, an NMDAR antagonist commonly used as an anesthetic. The doses of ketamine in this regimen are very low and sub-anesthetic, therefore potentially mitigating the negative side effects of the drug. This regimen involves intermittent, single-dose treatments of ketamine to an RTT patient for a period of weeks, until symptoms are alleviated.
In a mouse model of RTT, administration of low-dose ketamine eliminated apneic breathing, a core feature of RTT, for 24 hours (). The persistence of apnea reversal for 24 hours is highly significant because the half-life of ketamine is extremely short (less than 1 hour). This suggests that use of this intermittent dosing regimen results in sustained alteration in the underlying substrate of the disease rather than simply symptomatic relief, all while utilizing ketamine doses low enough to avoid negative side effects.

Treats underlying cause of RTT, not only symptom mitigation
Low doses of ketamine potentially mitigate negative side effects


RTT therapy
Pervasive developmental disorders therapy

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