BT13 and Parkinson’s Disease Treatment

By Israh Ghobbar

Neurons in Parkinson’s Disease

Recent research has proven that a new molecule, BT13, could improve neurochemical mechanisms to help treat Parkinson’s disease.

Parkinson’s disease is a progressive and degenerative nervous system disorder with a usual onset after the age of 60. Following Alzheimer’s, a disease causing memory loss and a decline in cognitive function, Parkinson’s disease is the second most common neurodegenerative disease. Symptoms include tremors, slowed movement, rigid muscles, and impaired posture and balance. Men are 1.5 times more likely to have Parkinson’s than women and it is estimated that 7 to 10 million people have the condition, which has no cure as of yet.

Diagram showing the symptoms of Parkinson’s Disease

People with Parkinson’s disease typically experience a loss of 70-80% of dopamine cells, a hormone responsible for mental health. Dopamine also controls movement, explaining why the loss of dopamine cells causes difficulties in motor function. As dopamine levels fall, new symptoms may appear such as a loss of automatic movement (actions performed without conscious thought e.g. arm swinging and blinking). As a result, research is being conducted with a focus on promoting dopamine production in the brain.

Previous research specialised in a molecule called glial cell line-derived neurotrophic factor (GDNF), which was meant to “heal” damaged dopamine-producing cells in the brain, restoring their function. This treatment is ineffective as it requires a difficult surgical procedure which involves delivering the molecule straight into the brain. This is because the large molecule is unable, on its own, to penetrate the blood-brain barrier, a semi- permeable membrane that stops potentially harmful substances and a large number of drugs from entering the brain. This means GDNF treatment is not able to pass clinical trials.

Researchers are continuously testing newly developed chemical compounds in hopes of formulating effective therapy procedures. Professor David Dexter, deputy director of research at Parkinson’s UK, a research and support charity, emphasizes the need for “a new treatment that can stop the condition in its tracks, instead of just masking the symptoms.”

The University of Helsinki has analysed a molecule, BT13, believed to help treat Parkinson’s disease. Research on this molecule was conducted on mice in vitro (outside the organism) and in vivo meaning BT13 was injected directly into the mice.

BT13 was able to boost dopamine production in mice and protect dopamine-producing brain cells from dying by activating a specific receptor. BT13 binds the signalling receptor (RET receptor), but unlike GDNF, BT13 is able to bypass the blood-brain barrier as it is a small molecule, making it more easily administered. When BT13 was given to mice through an injection to the brain, the molecule activated cell survival and growth pathways, increasing the release of dopamine from their striatum, the motor control brain area primarily affected by Parkinson’s.

Although treatment with BT13 is promising, more research needs to be conducted before this treatment can be applied to humans in clinical trials. Dr. Yulia Sidorova, a co-leading researcher at Parkinson’s UK, stated, “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.”


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