The researchers will use iPS cells from patients to grow the two types of nerve cells affected by the disease.

Johns Hopkins University scientists have been awarded a $3.7 million grant from the NIH to learn more about amyotrophic lateral sclerosis (ALS) using stem cells developed from ALS patients’ skin. The two-year award will be shared with three other laboratories: one at Harvard University and two at Columbia University. The Johns Hopkins team will collaborate with San Francisco-based biopharmaceutical company iPierian, which specializes in working with the stem cells to be used in this project. 

The scientists plan to grow induced pluripotent stem (iPS) cells from ALS patients’ skin, then steer them to develop into motor neurons and astrocytes. These two types of nerve cells are affected in ALS. They will then use these laboratory-grown cells to study the biology and chemistry involved in the development and progression of the disease and to test drugs to intervene in the process. When the two-year program is complete, the cells generated will be available nationwide to other researchers.  

“We believe that the ability to work with the two types of cells most relevant for ALS, developed directly from ALS patients, will give us a tremendous boost toward understanding more about this disease,” says neurologist Jeffrey Rothstein, M.D., Ph.D., the Johns Hopkins team leader. “Importantly, this will serve as a scientifically rich national resource for human ALS cell lines.”

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