Researchers in Barcelona have developed a shuttle to transport drugs into the brain, running through the blood-brain barrier.
If there is an irreplaceable organ, for the moment, that’s the brain. The gray matter is not only protected by the skull, but also by a barrier of cells that tightly regulates the transport of substances into this organ in order to prevent infection. The essential protective function of this barrier is also a red light for 98% of drug candidates for the treatment of the central nervous system. However, scientists at the Institute for Research in Biomedicine (IRB Barcelona) have presented a shuttle able to cross the blood-brain barrier and transport various substances into the brain.
The blood-brain barrier is not totally hermetic as the brain constantly requires oxygen, iron, insulin, etc. So there are transport mechanisms through “doors” which open and close continuously. Taking advantage of the receptors through which the brain receives iron, the shuttle-ambulance crosses the barrier without disrupting nutrient flow or altering the protective function of the barrier. Open sesame.
The therapeutic value of the shuttle lays precisely in these two properties, which make it unique, namely its small size, comprising only 12 amino acids, and its permanence in blood from between 12 and 24 hours thanks to its protease-resistant profile. In preliminary experiments with mice, the study also demonstrated the absence of an immune response and low toxicity.
Meritxell Teixidó, researcher associate at IRB Barcelona and leader of this line of investigation, explains “It is estimated that 20% of humans at some time will need a treatment that targets the brain. For many diseases, there are some good candidate drugs but none of them 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”.
The work has been carried out in IRB Barcelona’s Peptides and Proteins laboratory. Directed by Ernest Giralt, also senior professor of the University of Barcelona, this lab is one of the few leading labs worldwide devoted to these kinds of developments. The team is now studying its application for specific medical conditions. Together with clinical researchers, they are preparing treatments for glioblastoma (the most aggressive brain cancer in adults), Friedreich’s Ataxia (a hereditary neurodegenerative disease), and a type of paediatric brain cancer.