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RESEARCH STRATEGIES

1. Improve ALS Drug Screening
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Project ALS opened the Jenifer Estess Stem Cell Laboratory, the world’s first privately funded laboratory to focus exclusively on the study of stem cells in ALS.

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The lab recruited stem cell experts from leading institutions to work together to create better stem cell models of ALS.

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The Project ALS team developed iPS technology—or ALS in a Dish—the first ever laboratory model of human ALS.

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Scientists around the world are now using ALS in a Dish to screen for drugs that might slow or stop ALS. The Project ALS team is working to improve ALS in a Dish to be more accurate and reliable.

Learn more: Background and Research Progress (52.62 KB)
 
2. REDUCE INFLAMMATION
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We may be able to slow down ALS by reducing neuro-inflammation.

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Project ALS has identified new therapeutic targets for neuro-inflammation…

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and is partnering with leading pharmaceutical companies to test anti-inflammatory drugs originally designed to treat cancers and other illnesses.

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The Project ALS team is focused on getting these drugs across the brain-blood-barrier.

Learn more: Background and Research Progress (60.32 KB)
 
3. STRENGTHEN THE BRAIN
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ALS destroys brain cells called motor neurons, which control most of our voluntary muscle movements.

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Yet, motor neurons that control eye movements are resistant to the disease.

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Project ALS has identified genetic differences between affected and unaffected motor neurons in ALS.

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…The goal now is to make all motor neurons as resistant to ALS as motor neurons controlling eye movements.

Learn more: Background and Research Progress (52.72 KB)
 
4. IDENTIFY BIOMARKERS
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From a patient blood sample…

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...Project ALS researchers are able to screen for lipids and other possible markers of ALS…

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…that may lead to earlier, more accurate diagnosis—and, ultimately, earlier therapeutic intervention.

Learn more: Background and Research Progress (56.85 KB)
 
5. FIX ALS GENES
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Researchers have identified more than 30 genes involved in ALS.

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The Project ALS team is developing technologies to edit, even silence ALS genes.

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We have developed a technique called gene silencing that is nearing human trial.

Learn more: Background and Research Progress (46.34 KB)
 
6. STUDY THE WHOLE BRAIN
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Because ALS is considered a disease of brain cells called motor neurons, historically, research has focused on this one specific cell type.

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Project ALS researches motor neurons unto themselves, but also in the context of related cell types, neural circuits, and the whole brain.

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For example, what is the relationship between motor neurons, and brain cells called glia and interneurons?

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Project ALS has invested in a deeper understanding of brain development. The more we learn about the developing brain, the better equipped we will be to intervene when something goes wrong.

Learn more: Background and Research Progress (61.33 KB)