Unlocking the Blood-Brain-Barrier: A new simple and innovative way of delivering therapeutics to brain

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In a groundbreaking study published October 11 in Nature Communications, an international team of scientists from Newcastle University and Denver-based S M Discovery Group (SMDG) have designed a carrier system that delivers medicines to the brain through intravenous injection.

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Our breakthrough allows for minimally invasive and combination drug delivery through an intravenous injection to the brain

Professor Moein Moghimi, who led the research team, said, “Crossing the blood-brain-barrier has been a major impediment to effectively addressing central nervous system diseases, including brain tumors, and many neurological diseases like Parkinson’s, Alzheimer’s and Huntington’s. This breakthrough, based on more than 10 years of research, has significant implications for crossing the blood-brain-barrier and other biological barriers that have created challenges for drug delivery.”

The blood-brain-barrier

Scientists have traditionally faced the problem that blood capillaries in the brain are not permeable to many drugs. In fact, the majority of drugs are excluded from the brain by a protective barrier, called the blood-brain-barrier (BBB), and current treatment options are risky and challenging.

Some viruses, however, have found ways to bypass the BBB and enter the brain. For example, currently, one form of delivery to the brain is to use modified viruses to bypass the BBB and deliver medication to the brain.

Breakthrough technology

To this end, Prof. Moghimi and his team engineered small particles using a peptide that can behave like a carrier (like viruses) to the brain and can be packed with drugs for intravenous injection. This allows for minimally invasive and combination drug delivery to the brain.

At present, treatment of neurological disorders involves difficulties in ‘packaging’ viruses safely or delivering medication by injection into the cerebrospinal fluid, which are not without risks.

The new technological breakthrough for delivering genetic materials (as well as other therapeutics) to cells in the brain uses a modified peptide identified from a virus that targeted the brain capillaries. The peptide was synthesized and following chemical modifications in solution spontaneously formed a ‘small, hairy particle’.

The team of scientists found that when the developed particles were injected into a laboratory mouse, the particles targeted the brain, crossed the BBB, and reached neurons and microglia cells in the brain.

Safety and versatility

Through the mouse model, Professor Moghimi’s team has ascertained their technique is safe. Further research will test the technology in animal models of human diseases in preparation for clinical trials.

“We are very excited by our research – our technology platform is versatile and can carry different types of medicine; so, in principle, we can address shortfalls in drug delivery to the brain through intravenous injection,” concludes Professor Moghimi. “While we have a long way to go, we are confident that our technology platform will open up many opportunities to address neurodegenerative diseases with modern therapeutics and genetic drugs.”

Gary Leo, SMDG’s SVP of Corporate Development and former national president and CEO of the ALS Association, USA, said: “Bearing in mind the fact that neurological disorders are growing in incidence faster than any other disease class worldwide, society faces a huge economic burden and increasing demands for treatment and support services for patients suffering from these diseases.”

Dr. Shadi Farhangrazi, SMDG CEO and co-founder, added that new innovative and safe therapies are needed to develop effective treatment strategies. She stated, “our new technology platform reported in Nature Communications is a promising achievement to fulfill these goals.”

About S.M. Discovery Group (https://www.smdiscovery.com/)
S.M. Discovery Group (SMDG) is a biotechnology company focusing on utilizing an innovative patent – protected, proprietary technology platform for targeted delivery of therapeutic and diagnostic agents. SMDG’s platform technology is based on "safe-by-design" and "simple-by-design" concepts that actively deliver therapeutic molecules to the target cells and exert pharmacological activity. The platform technology allows for minimally invasive and combination delivery of various drugs, peptides, and nucleic acid therapeutics to the brain and other organs.

About Professor Moein Moghimi
Moein Moghimi is the Professor and Chair of Pharmaceutics at Newcastle University, UK and Adjoint Faculty at the Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Denver, Colorado, USA

Nature Communication 10: 4635 (2019), doi: 10.1038/s41467-019-12554-2

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