Liposome Drug Delivery Vector Targeting the Blood Brain Barrier
In terms of drug delivery, the BBB is a formidable barrier. Current (pre-clinical) state-of-the-art drug delivery systems, designed to specifically target the BBB, maximally deliver <0.5% of the total injected drug dose to the brain. As such, prognoses for diseases of the brain (eg. Alzheimer’s disease, glioblastoma) remain notoriously poor.
Researchers at Leiden University have developed a novel lipid, which when mixed with a naturally occurring phospholipid and formulated into 150 nm liposomes, results in a drug delivery vehicle with >10-fold selectivity for the brain endothelium (ie. the BBB) over the systemic endothelium. Encapsulation of both small molecule drugs (doxorubicin) and inorganic nanoparticles (gold nanoparticles) has been successfully demonstrated, resulting in the selective delivery of these reagents to the BBB (following intravenous injection). The novel lipid, required for BBB targeting, is synthesised in a single synthetic step (plus purification) using readily available reagents (<10 Euro/g, Sigma).
Details and State of Development:
The researchers have also demonstrated proof-of-principle of successful encapsulation of small molecule drugs as well as larger cargoes in the newly developed nanocarrier.
1. Drugs specifically targeting the brain and/or the BBB, such as treatments for strokes, cancer and neurodegenerative diseases (e.g. Alzheimer’s, Parkinson’s, Huntington’s).
2. Enhancement of brain and/or BBB (theranostic) imaging.
All results have so far been confirmed in the embryonic zebrafish. Validation of this is technology in rodent models is currently ongoing. The researchers are looking for partners from industry/academia/SME to take this invention to the next stage: in vivo testing in mammalian models for brain-related diseas
Keywords: pharmaceutical industry, biotech/medical companies, neuropharma, drug delivery platform
- Selective drug delivery to the brain and/or BBB would represent a major breakthrough in the diagnosis and/or treatment of many brain diseases.
- The selective delivery of high concentrations of drugs to the BBB will increase the therapeutic efficacy of drugs to treat brain disease and minimise potential off-target drug toxicity in other organs.
- The liposomal formulation itself is qualitatively non-toxic (ie. embryo develops normally and no abnormalities are observed).
Luris reference numberINV-0221.0974
A patent application is currently being drafted.