USC Stem Cell researcher Justin Ichida has marshaled the expertise of pharmaceutical company Sanofi and startup DRVision Technologies, along with $1.5 million in federal funding, to find new drugs in the fight against amyotrophic lateral sclerosis, or Lou Gehrig’s disease.
ALS patients suffer from the death of the cells that transmit signals from the brain to the muscles, called motor neurons, leading to progressive paralysis and usually resulting in fatal respiratory failure within three to five years of diagnosis.
The three-year grant comes from the Department of Defense. Each year, the DoD funds two ALS Therapeutic Development Awards because military veterans are more likely than civilians to suffer from this fatal disease for reasons that are not yet understood.
“The awards are intended to either identify a new drug target or candidate for ALS, or to take an existing one further into the clinic,” said Ichida, an assistant professor in the Department of Stem Cell Biology and Regenerative Medicine and director of the Choi Family Therapeutic Screening Facility at the Keck School of Medicine of USC (…)
DRVision Technologies will design software to analyze the resulting microscopic images for signs of improved motor neuron survival — a task that postdocs could handle for 800 compounds, but not for 42,000.
“In the preliminary data, Justin’s lab successfully used our software to track these motor neurons,” said Sam Alworth, director of marketing and sales for DRVision. “So my understanding is that it’ll be an issue of scaling that up. This collaboration with USC falls into the category of applied research and development where we use and expand our tools in collaboration with a leading scientist to solve a particular problem.”
If the funded study reveals viable “hits,” expert chemists at Sanofi may develop these compounds into safe, effective drugs that are stable in the blood and possess other key prerequisites to testing in a human clinical trial.
“There’s basically almost 100 percent certainty that the gene expansion happened once, and everybody’s a descendant of that person,” Ichida said. “This happened several hundred years ago in Scandinavia. So in Northern Europe, 50 percent of familial ALS or more is from this one mutation, and in North America, it’s still pretty common” (…)
“The importance is that it’s going to be one of the first examples of taking a patient-specific disease model from stem cells and using it to discover drugs at a pharmaceutical scale,” he said. “And specifically for ALS, the fact that Sanofi’s involved means that if they do find something, they’re going to want to develop it. So I think it has a really high chance of getting to patients, if we find something interesting.”