Johns Hopkins scientists have been awarded a $3.7 million grant from the National Institutes of Health (NIH) to learn more about the nerve and muscle-wasting disease amyotrophic lateral sclerosis (ALS) using stem cells developed from ALS patients' skin. The award, given over a two-year span, will be shared with three other laboratories, including one at Harvard University and two at Columbia University.
The Johns Hopkins team, led by neurologist Jeffrey Rothstein, M.D., Ph.D., will collaborate with San Francisco-based biopharmaceutical company iPierian, Inc., which specializes in working with the novel stem cells to be used in this project.
The stem cells, known as induced pluripotent stem or iPS cells, can develop into cells that populate many organs, but are derived from non-stem cells.
The scientists plan to grow iPS cells from ALS patients' skin, then steer them to develop into motor neurons and astrocytes, the two types of nerve cells that 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. Importantly, this will serve as a scientifically rich national resource for human ALS cell lines, " says Rothstein.
ALS, still sometimes known as Lou Gehrig's disease, is characterized by a gradual loss of muscle strength and coordination. The disease is fatal, with only about 20 percent of patients living more than five years beyond diagnosis.
The funding for the Hopkins work comes from an NIH Grand Opportunities (GO) grant, part of the American Recovery and Reinvestment Act of 2009. According to the NIH, the grants in this program "provide investigators and institutions with the opportunity to engage in new avenues of research with a high likelihood of significant impact on growth and investment in biomedical or behavioral research and development, public health and health care delivery."
Patient-derived organoids: Transforming cancer research and personalized medicine