Clinical trial for guanabenz as treatment for Multiple Sclerosis begins enrollment

Clinical trial for guanabenz as treatment for Multiple Sclerosis begins enrollment

April 30, 2015

Patients are now being enrolled in a clinical trial conducted to study guanabenz, an FDA-approved drug to treat high blood pressure, as a potential therapeutic to reduce loss of myelin in multiple sclerosis (MS) patients.

The trial, a collaboration between the Myelin Repair Foundation (MRF) and the National Institute of Neurological Disorders and Stroke at the National Institutes of Health (NIH) Clinical Center, is being led by NIH Investigators.

If successful, guanabenz could be the first MS treatment to focus on protecting myelin from damage, which is the hallmark of MS, rather than on suppressing the immune system – as all currently available MS treatments do.

In a Nature Communications paper published on March 13, 2015, an MRF-funded research team led by Brian Popko, PhD, Jack Miller Professor of Neurological Disorders at the University of Chicago and a member of the MRF's Research Consortium, reported that guanabenz prevents myelin loss and alleviates clinical symptoms of MS in animal models by prolonging an innate mechanism that is activated in response to stressors such as inflammation.

When this protective response is disrupted or overloaded – by the chronic inflammation seen in MS, for example – oligodendrocyte cell death and demyelination are significantly enhanced. Treatment with guanabenz strengthens this stress-response mechanism and helps protect oligodendrocytes from cell death.

"Guanabenz appears to enhance the cell's own protective machinery to diminish the loss of myelin," Popko said. "While there have been many efforts to stimulate remyelination, this now represents a unique protective approach. You don't have to repair the myelin if you don't lose it in the first place."

The MRF's clinical advisory board reviewed the preclinical data and encouraged advancement of guanabenz into clinical testing. The drug's protective efficacy and ability to alleviate myelin loss, coupled with its existing FDA approval and good safety profile, makes the clinical implications promising for MS patients, the advisory board concluded.

"We are very pleased that guanabenz is now moving into studies in MS patients," said Tassie Collins, PhD, Vice President of Translational Medicine at the MRF.

For more information on guanabenz, the clinical trial and enrollment, visit: