The Lysosome – A Crucial Cellular Orchestrator.

The Lysosome is an Underexploited Cellular Orchestrator

The Lysosome is an Underexploited Cellular Orchestrator

  • Often called the “stomach of the cell”, lysosomes digest and recycle internal and external molecules, like waster minerals and cellular debris.
  • 1955: Lysosome described by Christian de Duve, terming them “suicide bags” for their role in autophagy and apoptosis.
  • 1974: de Duve awarded Nobel Prize for his pioneering work describing lysosomes
  • Subsequent research implicates lysosomes in multiple pathological conditions and should be evaluated for not just lysosomal storage diseases, but for cancer, neurodegeneration, infection and autoimmunity.

The lysosome is one of the most poorly understood cellular mechanisms. Traditionally – and somewhat dismissively – referred to as the “stomach” or “trash bin” of the cell, lysosomes contain enzymes to break down and recycle internal and external macromolecules, like waste materials and cellular debris.

Research in the last two decades in particular has expanded our understanding of the lysosome’s role to include much broader functions such as membrane repair and amino acid sensing. The organelle also emerges as a signaling hub for mTOR to maintain energy homeostasis

With significant implication in various pathological conditions, lysosomal pathways are evaluated as a pharmacological target for lysosomal storage diseases, cancer, neurodegeneration as well as playing some role in immune function with implications both for infectious diseases and autoimmune processes

Lysosomes also involve the delivery of other biologicals and antibody drug conjugates. In addition, many drugs are found to accumulate inside lysosomes, allowing them to contribute to our understanding of pharmacokinetics, drug-drug interactions, and toxicity profiling.

Lysosome-Disease Correlation

The overarching mechanisms of cellular regulation and autophagy are at the frontier of our understanding of cellular function and disease and the lysosome is at the core of this story. Regulatory dysfunction — including autophagic dysfunction and dysfunction around programmed cell death — lay at the heart of dozens of diseases as disparate as Tay-Sachs and Ebola. That the lysosome can be involved in so many different disease and functional pathways, signals at its underlying role as a cellular regulator and orchestrator.

Bexion believes that lysosome native therapies are an important new tool to fight diseases including cancer. Inherent in our approach is the belief that these therapies will not only be more effective but have a much more attractive toxicity profile – perhaps none at all.

The Lysosome is Intrinsic to a Multitude of Diseases

The Lysosome is Intrinsic to a Multitude of Diseases

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