A Charlotte neuropathologist is part of a consortium of researchers who hope that a class of drugs yet to be tried on patients with ALS, or Lou Gehrig’s disease, may lead to progress in treatment.
Amyotrophic lateral sclerosis is a neurodegenerative disorder that many people call by the name of the Hall-of-Fame baseball player whose all-star status brought it into the public consciousness.
Dr. Benjamin Brooks, director at Carolinas Neuromuscular/ALS-MDA Center in Charlotte, believes the drug class – not yet released by name because of pending patents – may help clear neural pathways that are clogged up by abnormal proteins in the brain.
“I don’t want to announce it right now, other than to say this emerging collaboration between UNC, Wake (Forest University), Duke University, and (Carolinas HealthCare) System is developing these drugs,” said Brooks.
By midsummer, Brooks hopes to begin a treatment study in Charlotte with a new drug that’s part of the class.
Research and treatment breakthroughs for ALS have been minuscule during much of the past half-century.
The fatal disease, which the Centers for Disease Control and Prevention estimates affects 30,000 Americans, attacks nerve cells in the brain and spinal cord, leading to paralysis of the muscles responsible for breathing, among others. Those who receive a diagnosis are usually given three to five years to live.
“Glass coffin: That’s ALS in a nutshell,” wrote Ron Schaffer, 67, an ALS patient who communicates through a speech app on an iPad. “You have a perfect mind, but can’t communicate.”
But recent discoveries within the past few years have given hope that treatment, a cure, or even a way to stave off the disorder may be around the corner.
In 2011, Brooks served as lead researcher on a team that discovered a malfunctioning protein recycling system in neurons was responsible for triggering ALS in patients.
Environmental toxins, especially heavy metals, are suspected of playing a role in the disease, too.
A national registry has been set up to help researchers track other similarities among ALS cases.
“There have been several paradigm shifts in the disease and how we approach it,” said Brooks. “And I believe there’ll be more within the next two years.”
But some are hoping for a quicker shortcut from the normal path from research in labs, to clinical trials, and finally to use by all ALS patients.
“Terminal patients should be allowed any drug physicians and researchers deem suitable for FDA trial,” ALS patient Schaffer wrote.
Schaffer – who lives in Inman, S.C., but is a patient at the ALS center in Charlotte where Brooks is director – is one of the rare cases. Diagnosed 11 years ago, he has suffered with the disease longer than most.
“It started with slurred speech. One morning I woke up slurring. Thought I had a stroke,” Schaffer wrote. “They gave me one to three years to live and get things in order.”
Schaffer spends most of his days resting on a bench outside by his pool. His wife, he said, cuts his food. He struggles to brush his teeth or shave. Recently, he has felt the muscles in his back growing weaker, causing him to start bending at the waist.
He worries he’ll run out of time before helpful new drugs to treat the disease will be readily available.
“The anticipation of feeling your own body slowly be paralyzed can’t be described,” he wrote.