ALS (Lou Gehrig's Disease)

Wednesday, August 6th, 2014

NYSCF-supported ALS research identifies promising new biological pathway


Researchers at the Harvard Stem Cell Institute, led by NYSCF Chief Scientific Officer Dr. Kevin Eggan, discovered a new, promising treatment for ALS, or Lou Gehrig's disease. The research leading to this discovery began eight years ago and was initially and continuously supported by NYSCF.

Published in Science Translational Medicine, the scientists showed that genetically intervening in a biological pathway associated with ALS resulted in an increased survival rate of 5-10% in an animal model.

Critically, the scientists confirmed that this biological pathway is identical in animal models and in the human disease cells in a dish, forewshadowing the potential future elimination of animal model testing for ALS and other diseases.

Read more in the Harvard Gazette >>
Read more from the Harvard Stem Cell Institute >>
Read more from IFL Science >>


Friday, April 4th, 2014

NYSCF-supported ALS research headed to clinical trials


ALS research that NYSCF has supported for 8 years is now progressing to clinical trials for patients with this devastating disease. NYSCF began supporting Dr. Kevin Eggan's laboratory in 2007 when he was unable to obtain funds to make ALS diseased cells in a dish. NYSCF has been supporting his laboratory since then and the continued success of this research has culminated with two related papers that were published today on a potential ALS treatment that will now go to clinical trials.

The two papers, published today in Cell Stem Cell and Cell Reports, outline a discovery in which an epilepsy medicine may be useful in the treatment of ALS or Lou Gehrig's disease. First author on both papers was Evangelos Kiskinis, PhD, a NYSCF - Druckenmiller Fellow alumnus, working in the laboratory of Dr. Eggan at the Harvard Stem Cell Institute.

While a great deal more research needs to be done before determining the drug's sucess, the researchers are currently designing an initial clinical trial to test the safety of the drug in ALS patients with clinicians at Massachusetts General Hospital.

Read the press release on EurekAlert >>
Read more in the Harvard Gazette >>
Read the paper in Cell Reports >>
Read the paper in Cell Stem Cell >>

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.

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. 

Learn more about stem cells:

Stem Cells 101 | Videos | Stem Cell News


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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