Breaking through the blood-brain barrier
A small peptide capable of crossing the blood-brain barrier has been developed by a European research collaboration. The “shuttle” protein may be able to take large drug molecules with it, giving hope to people with difficult-to-treat brain disorders and cancers.
The blood-brain barrier separates the brain’s extracellular fluid from the body’s circulating blood flow. Essential small molecules like oxygen, iron and some hormones can diffuse across the barrier. Larger molecules, like toxins and therapeutic drugs, can’t penetrate the barrier.
“It is estimated that 20% of humans at some time will need a treatment that targets the brain and for many diseases there are some good candidate drugs but none have the capacity to reach their target and thus there is a subsequent loss of potential. Our shuttle offers a solution to an urgent clinical need,” says Dr Meritxell Teixidó of the Institute for Research in Biomedicine, Barcelona, in a press release.
While the idea of a “shuttle” in the bloodstream conjures images from Fantastic Voyage, the reality is that the shuttle is a 12-amino-acid-long peptide. It opens a “door” through the barrier via iron receptors, allowing the shuttle protein to travel through the barrier while leaving it intact. Another feature of the peptide is that it is relatively long lasting in the blood – up to 24 hours.
“Peptides have a half-life in blood of very few minutes … nobody wants to take a medicine every 5 minutes,” explains Teixidó. He added that their peptide was protease resistant and easy to produce on a large scale.
The group is now looking at using the shuttle protein to deliver therapeutic drugs and antibodies to the brain, with particular focus on glioblastoma, an aggressive brain cancer, and Friedreich’s Ataxia, a hereditary neurodegenerative disease.
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