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However, the GDNF protein requires complex surgery to deliver the treatment to the brain because it’s a large molecule that cannot cross the blood-brain barrier - a protective barrier that prevents some drugs from getting into the brain.
BT13, a smaller molecule, is able to cross the blood-brain barrier - and therefore could be more easily administered as a treatment, if shown to be beneficial in further clinical trials.
Professor David Dexter, Deputy Director of Research at Parkinson’s UK, said:
People with Parkinson’s desperately need a new treatment that can stop the condition in its tracks, instead of just masking the symptoms.
One of the biggest challenges for Parkinson’s research is how to get drugs past the blood-brain barrier, so the exciting discovery of BT13 has opened up a new avenue for research to explore, and the molecule holds great promise as a way to slow or stop Parkinson’s.
More research is needed to turn BT13 into a treatment to be tested in clinical trials, to see if it really could transform the lives of people living with Parkinson’s.”
Dr. Yulia Sidorova, lead researcher on the study, said:
We are constantly working on improving the effectiveness of BT13. We are now testing a series of similar BT13 compounds, which were predicted by a computer program to have even better characteristics.
Our ultimate goal is to progress these compounds to clinical trials in a few coming years." Source:
Parkinson's UK Journal reference:
Mahato, A.K., et al. (2019) Glial cell line–derived neurotrophic factor receptor Rearranged during transfection agonist supports dopamine neurons in Vitro and enhances dopamine release In Vivo. Movement Disorders . doi.org/10.1002/mds.27943 .
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