Scientists from Denali Therapeutics found that a new drug for Parkinson’s disease is safe in humans.
They found that an LRRK2 inhibitor called DNL201 showed no ill effects on volunteers in a clinical trial.
The research is published in Science Translational Medicine and was conducted by Patrick Lewis et al.
Parkinson’s disease is a disease that results from the destruction of neurons in the brain that produces dopamine, which is critical for motor function.
Prior research has suggested it comes about most often due to environmental factors in people with a genetic risk for it.
Prior research has also shown that mutated versions of a certain gene lead to overproduction of an enzyme called LRRK2, which leads to inflammation and other problems.
Currently, there are no therapies available to slow its progression.
One of the hallmarks of Parkinson’s is elevated levels of the LRRK2 enzyme which damages lysosomes—organelles responsible for removing toxins from cells in the brain. This leads to a buildup of toxins and cell death.
Previously, the researchers had developed a drug called DNL201 that has shown promise. It has been tested in animals, where it has effectively reduced LRRK2 enzyme levels.
In this new study, the team wanted to determine if DNL201 was safe for use in humans.
In the trial, 150 people were given the drug for 28 days. Of those, 122 were healthy adults and 28 had Parkinson’s.
The researchers found that administration of the drug brought down levels of LRRK2 enzymes and also showed no signs of the side effects that had been seen in some other animals.
The researchers suggest their results showed that DNL201 is ready for more clinical trials, including some focused on determining it if can slow Parkinson’s in humans.
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