BACKGROUND:  Essential tremor (ET) affects at least 4 million people in the United States, about half of whom seek medical treatment. Primidone and propranolol are the first-line therapies; however, they produce satisfactory results in less than half of patients. Thus, there is active interest in new therapeutics for this condition.

In the past, an animal model using harmaline to produce tremors in rodents has been used to study new therapeutics. Harmaline-induced tremor has been shown to respond to drugs that suppress ET. Previously studied compounds have suffered from a lack of chemical stability and poor selectivity for the desired target.

INNOVATION:  Researchers at UCLA have identified two compounds which suppress tremor in the harmaline rodent model of ET. The first compound is a derivative of mibefranil, a previously marketed compound that was withdrawn from the market due to a lack of specificity. Our newly identified compound demonstrates greater stability, greater specificity, and similar potency when compared to mibefranil. The second compound has a similar profile of strong potency and high selectivity. The data suggests that these compounds or their analogs will be effective for clinical essential tremor.

POTENTIAL APPLICATIONS 

• Treatment for essential tremor

ADVANTAGES

• The identified compounds have improved specificity when compared to previous compounds effective in an animal model for the treatment of essential tremor

DEVELOPMENT-TO-DATE:  In vivo data utilizing the harmaline rodent model of essential tremor has demonstrated efficacy of the two compounds.

Reference: UCLA Case Nos. 2009-384, 2009-408