The last three years have seen exciting findings happening with the Blood Brain Barrier (BBB), with 2013’s being published by Dr. Dao Pan and her fellow researchers working at Cincinnati Children’s Hospital here in Ohio.
Sierra Blankenship of Lima remembers how happy she was to hear of last year’s creation of the BBB. “I had forgotten about it,” she said, laughing. “But only off and on because I would wonder how far along they had gotten with it.”
This year’s researchers were able to merge a protein with an enzyme (apoE with a-L –idurondase IDUA) that allows the modified treatment to penetrate the BBB and enter brain neurons and astrocytes. In such a way that is controlled by the doctors, making it a dose-dependent approach.
The fatty apoE binds to the receptors on endothelial cells, a thin layer of cells that line the interior surface of blood vessels, of the BBB. The researchers found they were able to cross through those cells by tagging parts of the IDUA enzyme with part of the apoE, creating a way to reach brain tissues.
Past determinations show reduced drug efficacy due to having to bind therapies to receptors to get those therapies across the BBB and, of course, potency has been a huge problem.
Gene therapy of mouse models were used for five months, showing not only the ability to cross the BBB, but the models exhibited normal levels of glycosaminoglycans and lysosomal enzyme beta-hexosaminidase. Studies show lysosomal enzyme beta-hexosaminidase can help control mycobacteria infection.
The display of safety along with a positive demonstration of effectiveness will allow these scientists to continue, although the paper does caution, “that results in laboratory mice may face additional challenges when translating to clinical application in humans.”
Although they were working on a way to treat the disease mucopolysaccharidosis type I (MPS I), they believe, according to Dr. Pan, “Our findings will allow the development of drugs that can be tested for other brain diseases like Parkinson’s and Alzheimer’s.”
This can only mean one thing for those with multiple sclerosis: scientists will be able to use this study for those suffering from multiple sclerosis (MS) too and jump start research geared more towards that disease.
Time is all that is needed.
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Sources: http://www.cincinnatichildrens.org/research/cincinnati/life-of-researcher/default/; http://www.cincinnatichildrens.org/bio/p/dao-pan/; Cincinnati Children's Hospital Medical Center. "Experimental therapy crosses blood-brain barrier to treat neurological disease." ScienceDaily, 4 Feb. 2013. Web. 5 Feb. 2013.; http://www.ncbi.nlm.nih.gov/pubmed/18180457