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ALS Ice Bucket Challenge Progress

Scientific Focus Areas


The number of genes identified to cause familial ALS has multiplied since the discovery of SOD1. Efforts such as whole genome sequencing are underway with the aim to identify more ALS genes and target them for therapy.

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Drug development

The Lawrence and Isabel Barnett Drug Development Program supports studies of all drug development areas from compound screening all the way to entry into clinical trials.

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Disease mechanisms

Understanding how ALS disease works on many biological levels is necessary to identify potential therapeutic targets.

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Stem cells

Stem cell technology has allowed patients to donate samples (such as blood or skin) that are turned into motor neurons. This approach has emerged as a key research tool, a potential way of delivering therapies, as well as a vehicle for drug screening.

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Studies conducted at a very small scale including at an atomic and molecular level will provide new ways to evaluate ALS disease.

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Disease models

Animals, from worms to mice, that mimic ALS disease give an important avenue to explore both disease mechanisms and to investigate outcome measures of potential therapeutics from a cellular to a behavioral level before testing in humans.

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Clinical studies

Establishing and advancing standards in care for both people living with ALS and their caregivers is necessary to improve and enrich their lives now. Studies in this category include topics in clinical management grants and clinical trials.

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Cognitive studies

There is a large body of evidence demonstrating that cognitive impairment can be involved in ALS, including evidence of overlap with other disorders like Frontotemporal Dementia (FTD).

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Assistive technology

Improving communication systems through developing accessible, portable devices for people living with ALS is paramount to maintaining a high quality of life.

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A measurable substance, like chemical changes in the blood or structural changes in the brain, are desperately needed to improve diagnosis, follow disease progression and track response to therapy.

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Environmental factors

Features in a person’s environment alone or in combination with genetic predisposition can contribute to the development of ALS.

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Precision medicine

Effective treatments will target an individual patient’s disease process and take into account individual variability in genes, environment and lifestyle for each person.

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Natural history studies

Projects that follows people living with ALS over time to track disease will help identify demographic, genetic and environmental factors that correlate with disease, along with informing drug development and patient care, among other factors.

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