A critical pilot grant from the Southern California Clinical and Translational Science Institute (SC CTSI) was the catalyst for national recognition of Justin Ichida, PhD, as a biomedical inventor. Ichida was recently named a senior member of the National Academy of Inventors, a nonprofit member organization that encourages inventors in higher education. Senior members are rising stars with success in patents, licensing and commercialization, producing technologies that have helped or aspire to help society.

“It's a privilege to join the list of inventors who have made very significant inventions and impacts on human health through their discoveries,” said Ichida, PhD, John Douglas French Alzheimer’s Foundation Associate Professor of Stem Cell Biology and Regenerative Medicine at the Keck School at USC.

His research focuses on developing treatments for amyotrophic lateral sclerosis (ALS), a rare, deadly neurodegenerative disorder that occurs during middle age. As neurons responsible for movement begin to die, muscle control is progressively lost. There are different types of ALS, some driven by multiple genetic processes and some not genetically driven, and their mechanisms have not been well understood.

In the lab, Ichida’s team transforms blood cells drawn from patients with different types of ALS into stem cells. Next, the researchers biochemically reconfigure the stem cells into nerve cells.

“Our technology of making panels of nerve cells allows us to develop a ‘virtual biopsy’ of many ALS patients at once,” Ichida said.

Ichida’s research found that nerve cells from different ALS patients show the same characteristic defects that drive the disease.

“We see aggregations of a specific protein among nerve cells,” he said.

The proteins accumulate and prevent the nerve cells from functioning, and then the cells die. Ichida and his team are targeting this pathway of destructive protein accumulation.

“We are developing molecules that work therapeutically not only for one type of ALS but for all types of ALS,” said Ichida.

A 2018 Multidisciplinary Research Project Pilot grant from SC CTSI allowed Ichida to take a crucial first step toward discovery.

“Our Pilot program at SC CTSI prides itself on fueling groundbreaking innovations,” said Dr. Katie Page, Director of the Research Development core at the CTSI. “Dr. Ichida's exceptional contributions embody the transformative, forward-thinking work we champion and highlight the remarkable achievements we aim to support and celebrate.”

The Pilot program provides funding to support clinical and community-based research projects. This infusion of funds early on can help researchers create proof of concept and apply for larger funding grants later on.

“The SC CTSI funding enabled us to make the first panel of nerve cells from ALS patients and to learn that the cells did mimic disease processes in the dish,” he said. “One question at first was whether our lab’s nerve cells, which were a couple of weeks old, would manifest the same disease processes seen in ALS patients' post-mortem tissue or other ways of looking at their cells. We found that our process preserved and elicited whatever nerve cell defects occurred in actual patients. That told us we could potentially use this process in the laboratory to find some new therapeutics.”

The first potential treatment from Ichida's research is approaching clinical development following a license agreement between the pharmaceutical giant Takeda and AcuraStem, a startup co-founded by Ichida in 2016.

The recognition from the National Academy of Inventors, Ichida noted, was made possible by the supportive USC research environment, which includes clinicians who provide patient samples and technology transfer personnel and federal grantors, nonprofit funding agencies and the professionals focusing on commercializing his innovations.

He pointed to the grant from SC CTSI that initiated the research success.

“Many grants require that you have more proof that your approach is going to pay off before they award funding,” he said. “SC CTSI is different. They provide pilot funding for exciting projects that are still at a risky stage when there are questions about your approach. This relatively modest investment has turned into a promising set of work that has led to millions of dollars of additional funding.”