April 03, 2013
By Andy Hyland
|ALS patient Dena Highberger and Richard Barohn, M.D.|
Researchers looking for a new treatment for amyotrophic lateral sclerosis (ALS), a progressive neurological disorder commonly known as Lou Gehrig's disease, are turning to a drug already used in another neurologic disorder to see if it can help patients in the fight against the devastating disease.
Richard J. Barohn, MD, distinguished professor and chairman of the University of Kansas Medical Center's Department of Neurology, has treated patients with ALS for decades. He's now leading a new study funded by a $1.6 million grant from the Food and Drug Administration's Office of Orphan Products Development.
Researchers at 10 different sites will soon enroll patients in a study that will examine the effect of the drug rasagiline — already approved for use in Parkinson's disease patients — in patients with ALS.
Today, 50 percent of ALS patients die within three to five years of being diagnosed. As many as 30,000 patients have the disease in the U.S. at any given time.
"It's never a good diagnosis to have to make," Barohn says.
Treatment options are limited, too. A single drug is available to patients today, and it can delay the progression of the disease by only about three to six months.
"We have to do better than that," Barohn says. "If we can double the length of the effect of the drug, even that would be a huge breakthrough for patients with this disease."
Zachary Macchi, a second-year medical student at KU Medical Center from Topeka, is also contributing to the research study. He was connected to it through a KU Medical Center program for students interested in learning more about research opportunities, and has been heavily involved in data analysis and has presented parts of the research at conferences.
"Time is a big factor in all of this," Macchi says. "The faster we can get all of this figured out, the better off patients will be."
Friends and family members of Bob Willson know the effects of ALS all too well. The 44-year-old Kansas City resident died from the disease in May 2010 — less than three years after his diagnosis — but not before inspiring many others to continue the fight against the disease in his name.
"He was larger than life, both in his personality and his stature," says Rich Ehlers, a friend who is now the director of Project 5 for ALS, a fundraising group that is helping to fund ALS research at KU Medical Center and other sites.
Wilson was known as a "do-er," Ehlers says. Willson personally participated in several different clinical trials in the hopes of finding a cure. The efforts are continuing with help of the Project 5 group (named for Willson's rugby jersey number).
"A lot of research is done in silos," Ehlers says. "The answer is probably going to be found by working with a number of other neurological disorders. They're so similar that a breakthrough in one will probably lead to a breakthrough in others."
Barohn, who was Willson's doctor, still wears a bracelet on his arm in support of the Project 5 group.
"As a physician, you don't come across people like Bob very often," he says. "He was just one of those magnetic personalities, and he had so many friends."
So far, data from a preliminary pilot study seem promising, but Barohn says the drug must still be further studied before it can be finally determined if it's effective. He says he knows the chance that this drug becomes the answer for ALS is low, but even if the research can find new indicators that can show how the disease is progressing, it can help lead to new advances down the line.
"We want to find something that works, not just for Bob, but for the thousands and thousands of patients who die from ALS each year," Barohn says.