Receptor-Ligand Combination for Specific Drug Delivery to the Brain

Technology #15383

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Researchers
Nicholas Muzyczka Ph.D.
Hector Ruben Mendez Gomez
Managed By
April Kilburn
Assistant Director 352-392-8929
Patent Protection
US Patent Pending

Molecule Crosses the Blood-Cerebrospinal Fluid Barrier to Deliver Therapy into Targeted Cells

This engineered receptor-ligand combination targets and crosses the brain barriers of mammals to deliver drugs and therapeutics to the brain. About 12 percent of all deaths worldwide are caused by neurological disorders such as Alzheimer’s, Parkinson’s and epilepsy. Neurological disorders represent a large area of unmet medical need due to the limited efficiency of treatments available. As one of the most delicate organs of the body, the brain is protected against potentially toxic substances by the blood-brain barrier and the blood-cerebrospinal fluid barrier. As a result, treating brain disorders is limited by the inability of therapeutic drugs to cross either of these barriers. Available methods either allow potentially unwanted molecules to enter the brain along with the therapy or rely on drug delivery systems that limit specificity and so deliver to other organs along with the brain. Researchers at the University of Florida have developed an artificial-receptor/ligand combination that allows for specific, targeted drug delivery to the brain. The engineered receptor-ligand combination can be used as a tool to cross the highly selective barrier of the brain to deliver therapeutic and diagnostic agents with maximum specificity, reducing off-target effects during drug delivery.

Application

Engineered receptor-ligand combination that targets drug delivery to the brain

Advantages

  • Mimics large molecules that can cross brain barriers, achieving access to the brain
  • Uses an artificial receptor and ligand combination, resulting in specific, targeted drug delivery
  • Utilizes non-mammalian proteins, increasing the specificity and delivery of the agent to the targeted tissue and lowering toxicity to undesired tissue

Technology

Available drug delivery systems to the brain either allow unwanted molecules to break through the protective brain barriers along with the desired therapeutic or lack specificity and deliver agents to other tissues in addition to the brain, potentially causing toxicity. University of Florida Researchers have developed an engineered receptor-ligand combination that crosses the blood-cerebrospinal fluid barrier and allows for maximum specificity with reduced off-target effect when delivering therapeutic or diagnostic agents to the brain. In this proprietary combination, the cells in the brain barriers would express the engineered-receptor; the intravenous injection of ligand would recognize only that receptor, thereby creating a high-affinity and potentially specific mammalian delivery system for therapeutic molecules in the brain. This receptor/ligand combination did not interact with other receptors in the mouse model and has the potential to increase efficiency and accuracy in drug delivery to the brain.