Millions of people took the plunge when it came to joining the fight against amyotrophic lateral sclerosis. The international Ice Bucket Challenge—a social media phenomenon where people videoed themselves dumping cold water over their heads while spurring others to do the same—helped raise $100 million for research into the incurable neurological disease.
Justin Ichida, a researcher at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC, appreciated the increased awareness of the disease and support for research because he has spent more than a decade working to find a cure for ALS. “I’ve noticed that non-scientists I talk to are almost always aware of the disease now, whereas most were not before,” Ichida said. ALS, which first came to national attention in 1939 when it ended the career of baseball player Lou Gehrig, causes the degeneration of motor neurons that support brain and spinal cord function. Over time, patients lose the ability to control muscles and often suffer paralysis and respiratory failure. The cause of ALS is unknown for most people—at least 20 to 30 different genes contribute to the disease. But Ichida saw that no matter the mutation, all patients suffered a similar end result—their nerve cells stopped working properly.
“It’s a major point of mechanistic convergence in the disease,” Ichida said. “No matter the form of ALS, it results in that same problem. So if we target that correctly, we could hopefully treat a significant portion of the patients.”
Ichida pioneered a method to test thousands of potential drug treatments on cells taken from ALS patients to determine which are most likely to be effective at stemming the neurodegenerative disease. Already, Ichida has found several FDA-approved drugs that appear to prevent ALS patients’ cells from degenerating, at least in a petri dish. He is now working to find a compound that works well enough to take to clinical trial—where his method could ultimately be tested in patients to see if the effects of ALS can be reversed.
Working up to 14 hours a day in his lab, Ichida tests various chemical compounds—thousands so far—to see if anything creates a positive response in the cells and reverses neural degeneration. Because many neurodegenerative diseases appear to hamper cell function in the same way as ALS, Ichida hopes that his research will also shed light on Huntington’s Disease, Parkinson’s, Alzheimer’s and dementia.
how long most patients survive after their ALS diagnosis
The long hours don’t bother Ichida, who relishes the process of discovery and has wanted to be a scientist since the seventh grade, when he read “Jurassic Park.” And his passion for the subject shows little sign of waning.
“I’m excited to see what we know 25 years from now,” Ichida said. “I think we are beginning to see a lot of advances, but it’s still too early to say. We need at least 25 years.”