Utah researchers collaborate on stem cell therapy for ALS

A team of Utah-based researchers are collaborating on a stem cell therapy to fight amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

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SALT LAKE CITY, Utah—A team of Utah-based researchers are collaborating on a stem cell therapy to fight amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

Armed with $5 million from the National Institutes of Health (NIH), Dr. Linda Kelley, director of the University of Utah's Cell Therapy Facility, Dr. James Campanelli of University of Utah spin-out Q Therapeutics Inc., and Utah native Dr. Nicholas Maragakis of the Johns Hopkins University School of Medicine, have teamed up to bring the cell-based therapy to the point of human clinical trials to treat ALS. 

According to Arvino Singh, product development manager at the University of Utah, the four-year NIH grant will enable critical manufacturing and testing requirements necessary to gain U.S. Food and Drug Administration approval for human clinical trials.

Kelley, who will serve as principal investigator on the grant and professor of internal medicine at the University of Utah School of Medicine, says the project is a collaboration in the truest sense.

"While the university will be home to the grant, the stem-cell technology that Q Therapeutics brings to the table and the clinical expertise of Dr. Maragakis are essential to the project," Kelley says. "Our collaboration is a terrific example of how public-private partnerships can make innovative therapeutic products a reality."

Singh says the award validates the approach being taken toward emerging technologies, such as regenerative medicine.
"This kind of collaboration between the University and its commercial spin-out companies is a great opportunity," Singh says. "We have all of the infrastructure and the clean rooms to produce these cells."

ALS is a progressive, neurodegenerative disease that kills certain nerve cells in the brain and spinal cord. More than 5,000 people in the United States are diagnosed every year with the neurodegenerative disease that causes sporadic limb movement, and most patients die within a few years from respiratory failure.

The race to find new and effective treatments for ALS is the perfect platform for stem cell researchers, Singh says.

"The objective of the collaboration is to take the research from the bench  to the bedside," he notes.

Campanelli, senior director of research and development for Q Therapeutics, says the collaboration provides an exceptional opportunity for his researchers as well.

"The close proximity of our two groups has allowed us to readily address manufacturing and processing issues that would have been a challenge to overcome had we needed to go outside Utah," he says.

Maragakis adds that the collaboration is a key milestone in the development of therapeutics to treat those who suffer with ALS.

"Given the lack of good treatment alternatives for this fatal disease, this project could lead to a first-in-class therapy that significantly alters the course of disease for many ALS patients," Maragakis notes.

Maragakis and his team of researchers at Johns Hopkins recently published results of their work in ALS in Nature Neuroscience, showing that a specific type of brain stem cell therapy can be effective in an animal model of ALS.

In ALS, motor nerve cells in the spinal cord and brain that control voluntary muscle movement slowly degenerate. Maragakis produced the technique of transplanting diseased cells that surround nerve cells with healthy ones, slowing the progression of the disease.

In animal models using rats, the healthy cells staved off inevitable death by a few weeks, which could translate into a longer period of time for humans, notes Campanelli.

"It was a statistically significant increase," Campanelli said. "It's not a cure per se, but with a patient alive for just a few years, living for another year or two would be beneficial."

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