University of Cincinnati Launches Phase 2 Trial of Novel Endocannabinoid-Enhancing Drug for Multiple Sclerosis Spasticity
Key Insights
The University of Cincinnati Gardner Neuroscience Institute is participating in an international Phase 2 trial testing a novel oral drug that enhances endocannabinoids (search) to treat muscle spasticity (search) in multiple sclerosis (search) patients.
The investigational therapy works by inhibiting two enzymes (MAGL and FAAH) that break down naturally occurring endocannabinoids (search), allowing these molecules to better regulate overly reactive spastic muscles.
The multicenter trial will randomize approximately 200 MS patients across multiple sites to receive one of three drug doses or placebo daily for six weeks, with an optional additional six-week extension period.
The University of Cincinnati Gardner Neuroscience Institute has joined an international Phase 2 clinical trial testing a novel oral drug designed to enhance the body's natural endocannabinoid system for treating muscle spasticity (search) in multiple sclerosis (search) patients. The trial represents the first potential breakthrough in oral spasticity treatments in decades, addressing a significant unmet medical need for MS patients who experience painful muscle stiffness and reduced mobility.
Targeting the Endocannabinoid System
The investigational therapy takes a unique approach by enhancing naturally occurring molecules rather than introducing foreign compounds. "The body naturally produces molecules called endocannabinoids (search) that help muscles relax and play a role in other functions, including regulating mood, pain and sleep," explained Dr. Shahla Hosseini, adjunct associate professor of Physical Medicine and Rehabilitation in UC's College of Medicine and a UC Health physician.
The trial drug works by inhibiting two specific enzymes - monoacylglycerol lipase (search) (MAGL) and fatty acid amide hydrolase (search) (FAAH) - that normally break down endocannabinoids (search). By blocking these enzymes, the therapy allows endocannabinoids to accumulate and better regulate overly reactive spastic muscles.
"Some endocannabinoid levels have been found to be lower in people with MS," Hosseini noted. "This finding makes the drug's mechanism even potentially more relevant to this group if it is able to help normalize their levels of endocannabinoids (search)."
Addressing Treatment Limitations
The trial comes at a critical time when treatment options for MS-related spasticity (search) remain severely limited. "Physicians who treat muscle spasticity have a limited number of medications from which to choose. In the past 20 to 30 years, we haven't seen development of new effective oral medications to treat spasticity in the United States," Hosseini said.
Current oral medications present significant challenges for patients. "The majority of the currently available oral medications have side effects like drowsiness, which limits their usefulness during the daytime, and so we really need a new effective treatment for spasticity (search) that's better tolerated by patients," she explained.
Many MS patients experience spasticity (search), which causes muscles to resist being stretched, leading to pain, impaired function, decreased mobility and poorer quality of life. While spasticity is typically managed with various treatments including oral medications, injections, stretching, yoga, botulinum toxin injections, and physical and occupational therapy, there is currently no true cure for the condition.
Trial Design and Scope
The international, multicenter Phase 2 trial will enroll approximately 200 people with MS across multiple sites. Participants will be randomized to receive one of three doses of the investigational drug or a placebo daily for six weeks. The study includes an optional additional six-week extension period that will compare effects of only the three different drug doses on treating spasticity (search), ensuring that participants who initially received placebo will have guaranteed access to the trial drug during this extended phase.
Broader Therapeutic Potential
The implications of this research may extend beyond multiple sclerosis (search). Hosseini noted that spasticity (search) also affects people with other neurological diseases involving the brain and spinal cord. If the trial drug proves effective in MS participants, it may show promise for wider use as an additional therapeutic tool for clinicians treating various neurological conditions that cause muscle spasticity.