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USC scientist screens potential therapies for Lou Gehrig’s disease

Justin Ichida tests drug-like compounds on motor neurons formed by reprogramming skin cells from ALS patients

Justin Ichida, stem cells, regenerative medicine, high-throughput screening, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC,
Ichida has developed a way to pre-screen drug-like compounds on cells from patients with the most common form of ALS. (USC Photo/Cristy Lytal)

Justin Ichida started young on his path to science. The USC researcher was in seventh grade when he discovered his passion for genetics through the pages of Michael Crichton’s Jurassic Park.

“It’s even the same type of biology that I do now,” said Ichida, an assistant professor at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC. “They were basically reprogramming DNA to make it into the whole animal, and we’re basically just reprogramming cells from one cell to another.”

The son of a lawyer and a real estate agent, he attended ‘Iolani School, Barack Obama’s crosstown rival in Honolulu.

 As an undergraduate at UCLA, he studied microbiology and molecular genetics.

And as a genetics Ph.D. candidate at Harvard Medical School, he found himself studying something even more ancient than dinosaurs in the lab of Nobel laureate Jack Szostak.

“My mentor in graduate school had outlined the biggest question possible in science: What is the origin of life?” Ichida said.

He worked on his recipe for primordial soup and cooked up what was likely a key ingredient: a proto-DNA called Threose Nucleic Acid or TNA.

For his postdoc, Ichida decided to focus his energies on something further along the evolutionary timescale: human cells.

In 2007, he helped identify ways to make induced pluripotent stem cells by using chemicals instead of the transgenic viruses that had been previously used. Transgenic viruses tend to introduce genes that aren’t normally expressed in a cell, which can alter its behavior. Using chemicals avoids this problem.

Motor neurons and more

His next project was to directly reprogram skin cells into motor neurons.

This technology forms the foundation of his current research at USC: testing drug-like compounds on motor neurons formed by directly reprogramming skin cells from patients with amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. Because these motor neurons have the same DNA as the individual ALS patients, they exhibit the disease’s signature degeneration, which causes progressive paralysis usually resulting in fatal respiratory failure within three to five years of diagnosis. The hope is that one of these compounds will keep the motor neurons alive in the petri dishes — and eventually in the patients.

I just want to do something that really changes people’s lives.

Justin Ichida

The principal investigator conducts this research in his own lab and at USC’s Choi Family Therapeutic Screening Facility. He spearheads this state-of-the-art facility, which will be used by researchers from USC and beyond to conduct important screens that could lead to the discovery and development of new drugs and therapies for patients.

“I just want to do something that really changes people’s lives,” Ichida said. “At this point, I think it would be coming up with therapies that can help people manage ALS. If we could do that, I would be happy. Aside from that, anything else is just a bonus.”

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USC scientist screens potential therapies for Lou Gehrig’s disease

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