Jeffrey Rothstein, professor of neurology and neuroscience at
Johns Hopkins University in Baltimore, Md., was awarded an MDA
research grant totaling $392,706 over a period of three years to
study the most common genetic cause of amyotrophic lateral
sclerosis (ALS), using cells derived from patient skin samples.
“Understanding the pathophysiology of and developing new
therapeutics for ALS and dementias such as Alzheimer’s disease
and frontotemporal dementia has been an enormous challenge,”
Rothstein says. Among the biggest roadblocks has been the
difficulty of developing appropriate models of the disease —
ones that closely parallel the human condition. A new technique
to overcome this barrier is the use of induced pluripotent stem
cells (iPS cells). These cells can be taken from the skin of an
individual with ALS and treated with growth factors to return
them to a less differentiated state. From this stem-cell-like
condition, they can then be induced to develop into any cell
type, including muscle-controlling