Researchers Collaborate on Stem Cell Therapy for Lou Gehrig’s Disease (ALS)

Stephen Hawking

A research team made up of scientists from the University of Utah is collaborating on a stem cell therapy to fight amyotrophic lateral sclerosis, more commonly known as Lou Gehrig’s disease. Together with John Hopkins University School of Medicine, and Q therapeutics Inc, and the help of a $5 million dollars in funding from the National Institutes of Health, these researchers have teamed up to bring the cell-based therapy to the point of human clinical trials to treat this ALS. ALS is a progressive neurodegenerative disease that destroys certain nerve cells in the brain and spinal cord. As these cells degenerate, they lose the ability to send impulses that control muscle movement for speech, breathing, limb movement, and other functions, with death from respiratory failure typically occurring from two to five years after diagnosis. ALS affects roughly 30,000 people in this country. Famous sufferers of the disease include baseball legend Lou Gehrig and scientist Dr. Stephen Hawking.
The four-year National Institutes of Health grant will allow for the necessary manufacturing and testing requirements to gain U.S. Food and Drug Administration approval for human clinical trials.
Linda Kelley, Ph.D., director of the University of Utah’s Cell Therapy Facility, said the project is a collaboration in the truest sense of the word.
“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. We are pleased to help bring this groundbreaking therapy toward human use,” Kelley said.

“Our collaboration is a terrific example of how public-private partnerships can make innovative therapeutic products a reality.”
The researchers at Johns Hopkins recently published results of their work in Lou Gehrig’s Disese (ALS) in Nature Neuroscience, showing that a specific type of brain stem cell therapy can be effective in an animal model of ALS.

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