New molecule shows promise for the treatment of Parkinson

Recent mouse model research suggests that a new molecule could target key neurochemical pathways and help treat Parkinson’s disease.

Work into therapies for the Parkinson's disease is moving ahead, testing the promise of a newly discovered drug.
Work into therapies for the Parkinson’s disease is moving ahead, testing the promise of a newly discovered Compound.

Disease of Parkinson is a progressive, degenerative condition which affects the central nervous system.

Its most prominent symptoms usually involve movement— they can include involuntary limb tremors, stiffness, and slow motion.

According to estimates in 2016, around 6.1 million people worldwide live with this condition which has not yet been cured.

Specialists remain on the lookout for potential drugs, to which end they constantly test newly developed chemical compounds.

In a new study, Finnish-based researchers at the University of Helsinki resided on a molecule called BT13, which they believe has potential as a therapeutic for Parkinson’s disease.

In a review paper published in the journal Movement Disorders, the team presents the findings of their work— carried out in vitro and in vivo, in mouse models.

“People with Parkinson’s urgently need a new treatment that can halt the disease in its tracks, rather than just masking the symptoms,” stresses Prof. David Dexter, Deputy Director of Research at Parkinson’s UK, a UK-based research and charity-based organisation.

The charity has granted a grant to partly fund the researchers ‘ efforts to investigate the potential of BT13 in Parkinson’s therapy.

The reason behind the research

Previous research has shown that people with Parkinson’s disease typically experience significant cell loss that releases dopamine, a hormone and chemical messenger that plays a critical role in the mental and brain health.

Dopamine also helps to regulate movement, which explains why a key aspect of Parkinson’s disease is the loss of dopamine-producing cells.

For this purpose, as a more personalized cure for Parkinson’s, many experts have been experimenting with ways to improve dopamine production within the brain.

Until now, many studies had focused on the potential of a specialized molecule — called glial cell line-derived neurotrophic factor (GDNF) — that may be able to “heal” damaged dopamine-producing cells in the brain, restoring their function.

However, GDNF treatment has a significant flaw: it requires a challenging surgical procedure involving direct delivery of the molecule to the brain.

This is because the molecule can not penetrate the blood-brain barrier on its own, a protective boundary which prevents potentially harmful substances and micro-organisms from entering the brain. A vast number of drugs can’t cross that.

Moreover, recent reports have shown that GDNF therapy has not passed clinical trials because it was not effective enough to treat the condition.

The first step of many

Additionally, scientists have been looking at alternative ways to stimulate the development of dopamine.

The University of Helsinki team conducted experiments on cell lines and mouse models to see if a newly discovered, GDNF-like molecule could be more successful.

We found that the molecule— BT13 — really could boost dopamine in mice’s brains. It also tended to prevent the brain cells that were charged with generating dopamine from dying off and, unlike GDNF, could bypass the blood-brain barrier.

“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 science to explore, and the molecule holds great promise as a way to slow down or stop Parkinson’s,” commented Professor Dexter, who was not involved in current research.

However, the professional associated with Parkinson’s UK points out, the researchers have a lot of work ahead of them before they can conclude that the new approach works in humans.

“There is a need for more research to turn BT13 into a drug to be tested in clinical trials, to see if it could potentially change the lives of people living with Parkinson’s,” he says.

Yulia Sidorova, Ph.D.— the co-lead researcher of the study— agrees, noting she and her colleagues are already working hard toward this end.

“We are continually working to make BT13 more successful. We are now testing a series of similar BT13 compounds which a computer program predicted would have even better features,” she says.

“Our ultimate goal is to progress these compounds to clinical trials in a few coming years.”

– Yulia Sidorova, Ph.D.

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