A New Study Shows Small-molecule Drug through Blood-Brain Barrier

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Mayo clinic researchers demonstrated within a mouse model that a synthetic peptide carrier works to deliver brain cancer chemotherapy drugs, as well as neurological medications. Neuroscientist Gobinda Sarkar, Ph.D, of the Mayo Clinic and corresponding author of the study discussed that they were able to transport eight different molecules, and much less disruptively through the brain, as they followed the normal physiological process of the brain. The findings were published in PLOS ONE, showing that the researchers could transport the needed drugs without having to modify any of the molecules. Currently, the blood-brain barrier works to protect the brain from invasive or foreign chemicals that circulate through the body, but it also blocks the brain from treating tumors and other conditions. Usually, the needed drugs are large in mass and volume to effectively pass through the barrier, and be useless in treatments.

            For the study, the synthetic peptide K16 ApoE was injected into a vein, and would then bind to proteins in the blood which then creates entities passing off as near-normal ligands for receptors on the blood-brain barrier. The researchers thought that the interaction between the ligand and receptor creates pores that are transient in the barrier that allow molecules transport to the brain. The research team has been able to transport thus far have been Ciaplatin, Moethotrexate, Cetuximub, three different dyes, and synthetic peptides Y8 and I-125. Previous studies have delivered antibodies that targeted amyloid plaques in the brains of mice with Alzheimer’s disease, and the team thought that this means would be the least complicated and expansive, but the most versatile method for administering drugs to the brain.

 Some agents could kill brain tumor cells outside of the brain, but the blood-brain barrier blocked the tumors in the brain. Senior author Robert Jenkins, M.D., Ph.D, discussed that the use of the peptide carrier could allow the agents access to the brain to kill the tumor cells. The overall method could be suitable for therapy under the conditions that it is feasible as a repeatable procedure; it would be easy to introduce into modern medical practice, and it could be versatile in sizes and locations within the brain.


1)    Mayo Clinic. (2014, June 4). Small-molecule drugs moved through blood-brain barrier in new study. ScienceDaily. Retrieved June 5, 2014 from www.sciencedaily.com/releases/2014/06/140604105317.htm

By Lauren Horne

The Roskamp Institute is a 501(c)3 research facility dedicated to translating the efforts of its qualified research staff into real-world results for those suffering from neurological diseases. To learn more about our programs and to get information about donating, visit www.rfdn.org.