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ABOUT THE PACKARD CENTER

Packard Center for ALS Research at Johns Hopkins

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    ONGOING ALS RESEARCH PROJECTS

Causes of ALS: Unnaturally Aggregated Proteins

Unnatural clumping of proteins in cells is a common feature of Alzheimer’s, Parkinson’s and other neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. And it’s another possible source of toxicity. 

Insoluble aggregates of the SOD1 protein typical of familial ALS, for example, are found in the motor neurons of model ALS mice. Especially interesting is that the aggregates appear just before or at the same time that ALS symptoms begin.

Packard researchers found that clumped, or aggregated, protein appears early on and accumulates as the disease progresses, suggesting that aggregation may be an early event in ALS’s pathology.

Recently, aggregates of two other proteins, TDP-43 and FUS, were isolated in ALS patients – mostly those with the familial disease, though some with sporadic. The fact that they appear in both types of patients is important. More studies will show if the proteins are part of the toxic insult that triggers ALS. Also, there is, in principle, the possibility of a prime drug target – one that lets us stop or slow the disease early on.

Learn more about the research projects funded and coordinated by the Packard Center for ALS Research at Johns Hopkins, and targeted at finding the causes of ALS and a cure. Subscribe to ALS Alert to stay informed about ALS research and clinical trials.

From Our Experts

Current ALS Clinical Trials

  • Skin biopsies to generate cell lines for study of ALS

  • A Longitudinal Study of Cognition and Behavior in ALS

  • A Multicenter Study for the Validation of ALS Biomarkers

  • Electrical impedance myography as an outcome measure in ALS clinical trials

  • Quality of Life and Caregiver Burden in ALS

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