While the search for disease modifying treatments of FTD remains elusive, on a day-to-day basis clinicians are challenged to manage the severe behavioral and psychiatric symptoms. Unfortunately, current medication options have limited efficacy. In addition, commonly used treatments such as antipsychotics carry a significant burden of side-effects, including falls, and a small increase in the risk of stroke and mortality. There is a clear need for new treatments using novel mechanisms for psychiatric symptoms of FTD. One promising candidate is nabilone, a synthetic cannabinoid (i.e., a medication acting the brain system that reacts to cannabis) that has shown benefit for agitation in Alzheimer’s disease (AD) and has potentially beneficial properties on inflammation in neurodegenerative diseases (mechanisms that have been related to FTD). This medication is already approved by the FDA and readily available as a generic drug.

We propose to conduct the first open-label clinical trial of nabilone for agitation in FTD to obtain data on effectiveness, optimal dose and tolerability. There is a need to obtain data on effectiveness on a wide variety of symptoms in FTD, while also ensuring the safety of the medication. We further need data on dosing and tolerability in this population, which is younger in average than AD subjects from previous studies. If successful, results from this study would lead to practice recommendations and a larger confirmatory randomized clinical trial.