Multiple Sclerosis Drug Passes Safety Trial

An investigational drug that researchers hope will reverse nerve damage in patients with multiple sclerosis (MS) has been found to be safe and well tolerated in early trials. The big question is whether it will work, researchers said.

An investigational drug that researchers hope will reverse nerve damage in patients with multiple sclerosis (MS) has been found to be safe and well tolerated in early trials.

The big question is whether it will work, wrote Diego Cadavid, MD, in a report on phase I trials of the drug published online Aug. 27 in Neurology Neuroimmunology & Neuroinflammation.

Cadavid is a senior director at Biogen Idec, the Cambridge, MA, company that developed the drug, known as anti-Lingo-1 or BIIB033.

Biogen paid for the trial, which was conducted in the US and the Netherlands.

“With these results we have been able to start phase II trials to see whether this drug can actually repair the lost myelin in humans and have any effect on restoring physical and cognitive function and improving disability,” Cadavid said in a statement.

The drug has worked in mice, but when it comes to humans “the jury is still out,” he said.

According to the US National Institutes of Health, an estimated 250,000 to 350,000 people in the US currently have MS and about 200 new cases are diagnosed weekly.

The prevalence of the disease is also rising, and it affects twice as many women as men.

Though there have been recent pharmaceutical advances in treating MS the new drugs tend to slow the disease progression, but not reverse it.

MS is an autoimmune disorder in which patients’ immune systems attack myelin—a natural nerve insulator. When myelin is destroyed, patients’ brains and spinal cords cannot get signals from the nerves, a condition that can ultimately lead to paralysis and death.

The researchers hope that their new drug candidate could be the first to repair the damage by blocking a proteinblocking a central nervous system protein that prevents myelin from sheathing nerve cells as it does in healthy people.

Another challenge is seeing whether existing methods can accurately measure whether or how much remyelinization is occurring in the patients in the trial. One promising technique may be a type of MRI using magnetization transfer ratio, the researchers reported.

In the safety trial, 72 healthy people without MS and 47 MS patients got either the drug or a placebo. There were no serious side effects in either group, the researchers reported.

Phase II trials involving 396 subjects began in August 2013 and in them researchers are giving participants the drug in combination with interferon beta, in the belief that the interferon will protect any newly formed myelin from being destroyed by MS patients’ immune systems.