Lou Gehrig’s disease, or amyotrophic lateral sclerosis (ALS), is an adult-onset, degenerative motor neuron disease. NYSCF undertakes advanced stem cell research to identify the causes, understand the progression of, and ultimately find a cure for ALS.
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hESC derived motor neurons and glia cells Francesco Paolo Di Giorgio, PhD, 2007 NYSCF-Druckenmiller Fellow |
ALS begins slowly with muscle weakness and progresses into paralysis, threatening key vital functions such as speech, movement, and breathing. Motor neurons, which transmit key signals from the brain to muscles, die off through a largely unknown process.
Its risk factors, too, remain poorly understood; only 5-10% of cases are familial and the remaining sporadic. ALS, blind to ethnicity, race, and socioeconomics, can strike anyone. Each year, nearly 5,600 Americans are diagnosed, and most survive only four years thereafter.
Current strategies to treat ALS are aimed to lessen symptoms and slow progression. The FDA has approved only one drug for this disease, which prolongs life in some patients by three to five months, on average.
With NYSCF support, scientists have made progress in understanding the causes of and discovering therapeutic candidates for ALS.
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NYSCF Chief Scientific Officer Dr. Kevin Eggan speaks at the 2010 NYSCF Conference |
How can stem cell research help us find better treatments and cures for ALS?
Stem cells provide a living window onto ALS. NYSCF scientists derive the actual motor neuron cells implicated in this disease from ALS patients’ skin samples. These cells mature, get sick, and die off in a dish as in a patient. We can, for the first time, scrutinize what goes wrong on a cellular level that leads to what goes wrong in a patient.
In 2008, NYSCF funded research for ALS that both Time and Science magazines named the “#1 Scientfic Breakthrough of the Year” for replicating the actual human cells affected by ALS. NYSCF Chief Scientific Officer Kevin Eggan, PhD, Principal Faculty member at the Harvard Stem Cell Institute, with colleagues discovered that glial cells, which are normally supportive of motor neurons, are toxic to them in ALS. Armed with this increased understanding, they have identified potential chemical compounds that rescue motor neurons.
Reported in a landmark Cell study in April 2013, NYSCF Scientific Advisor Lee Rubin, PhD, Principal Faculty member at the Harvard Stem Cell Institute, uncovered a drug-like compound through a large-scale stem cell-based screen that protected ALS motor neurons better.
The NYSCF-supported work involved generating motor neurons, through advanced stem cell techniques, from patients with the genetic form of ALS. After testing 5,000 compounds, this one compound emerged as especially potent in protecting motor neurons under various stressful conditions.
Phase II of the NYSCF ALS Research Initiative will support the discovery of other drug-like compounds to feed into preclinical trials. By capitalizing on The NYSCF Global Stem Cell Array, a fully robotic automated platform that generates thousands of standardized induced pluripotent stem (iPS) cell lines from patients’ skin samples, researchers will test compounds on a diverse panel of ALS patients with the familial and sporadic forms. For the first time, efficacy and potential toxicity will be determined in advance of clinical trials.
Coupled with the medical histories of ALS patients, these cells serve as an incredibly powerful tool to increase our understanding, to uncover treatments, and to find a cure.
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