GPS pill can overcome the blood-brain barrier

Microscopic, synthetically manufactured protein capsules to deliver drugs to brain cells

March 28, 2012

Scientists working at the Life Science Inkubator (LSI) in Bonn have developed a “drugs ferry” which conveys drugs directly from the bloodstream to their specific destination in the brain. Animal studies show that this transport system can overcome the natural “blood-brain barrier” which usually shields the brain from drug-based treatment. The process opens up new prospects for the treatment of human brain disorders, like brain tumours and multiple sclerosis. It is based on microscopic, synthetically manufactured protein capsules which, based on their molecular make-up, are programmed to deliver drugs to brain cells. As planned, a start-up company will develop the technology further on completion of the incubation process. This pioneering project is visible proof of the successful implementation of the LSI concept, which is unique in Germany. The LSI was established by Max Planck Innovation, the Max Planck Society’s technology transfer organisation, with the aim of facilitating the establishment of spin-offs in the field of the life sciences and is funded by the Federal Ministry of Education and Research and the German federal state of North Rhine-Westphalia.

Neurodegenerative diseases like multiple sclerosis, Alzheimer’s, Parkinson’s and brain tumours cause the gradual deterioration of the brain. Exactly what causes the diseases is still partly unclear, as their symptoms are varied, and can range from paralysis to memory loss and dementia. Standard treatment methods are limited in their effect. This is due particularly to the fact that the brain cannot simply be treated using drugs. The reason for this is the natural filter mechanism in the wall of the blood vessels which supply the brain: this “blood-brain barrier” protects the brain tissue against pathogens; however, it also prevents many drugs from reaching the brain from the bloodstream. Scientists from the LSI have now developed a “drugs ferry” that can overcome this barrier. Its effectiveness has been confirmed for the first time in tests on mice. A tracer was introduced into the animals’ bloodstream using the new process and was later detected in their brains. This offers an innovative method of treating diseases of the central nervous system.

“Some substances can pass through the blood-brain barrier without problem. Our ‘ferry’ is perceived by the brain as something that is absolutely essential to it. This enables it to slip through the blood-brain barrier,” says project leader Heiko Manninga. “This means that we can now transport a wide range of substances into the brain via the bloodstream. We were surprised ourselves that the process worked so well.” The tests also showed that the drug not only arrives in the cells but that it can still take effect there. “The package is delivered unscathed. This is a vital condition for its potential use in treatment,” says Manninga.

Drug container on a nanoscale

The process is based on artificially manufactured protein capsules known as “virus-like-particles” (VLPs). The research group developed spherical “engineered protein capsules” which are just millionths of a millimetre in size and can be loaded with active drug substances.

The capsule proteins act like a kind of navigation system which causes the tiny transporters to deliver their drug load to the brain cells. “Our process delivers drugs to the precise locations where they are intended to take effect. It’s like a kind of pill with a built-in GPS system,” explains the project leader. VLPs have already been used in immune research for some years now and they are also used for practical vaccination. “However, the use of these protein capsules for the transportation of active substances is a new approach. Particularly in relation to the treatment of brain diseases,” adds Manninga.

His project group will continue their research in the context of a start-up company. The researchers have set themselves the initial development objective of treating glioblastoma multiforme (GBM), a particularly aggressive form of brain tumour. “We will continue the studies on mice and test different drugs for the treatment of brain tumours. We hope to be able to start working on the first patient studies within the next two to three years,” he predicts.

Manninga also sees potential for the application of this method in the treatment of other neurodegenerative diseases, particularly multiple sclerosis. Around 120,000 people in Germany and approximately 2.5 million worldwide are affected by this inflammation of the nervous system.

First spin-off from the LSI

The LSI, which is funded by a public-private partnership, aims to develop marketable technologies from scientific discoveries and inventions. Various research projects from the fields of biotechnology, pharmaceuticals and medical technology have been funded by the LSI since 2009. “Innovative ideas need entrepreneurs who can introduce these ideas to the market. At the LSI, we support this exact combination of expertise, a concept that is unique in Germany,” says Jörg Fregien, CEO of the LSI. Based on the results of the VLP project, the establishment of the LSI’s first spin-off is now underway. The start-up company is financed by an investment company associated with the LSI and by private investors. This initial investment will be followed by additional funding. In addition, strategic cooperation with an industrial partner is also planned. The future head of the company, Heiko Manninga, plans to create around 30 jobs by 2016.


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