Scientists at Vanderbilt University Medical Center (VUMC) and Myriad Genetics Inc. present a two-in-one drug for Parkinsons disease that remains safe and well-tolerated in mouse models.

Low doses of 3-beta-methyl-5-aminopropyl-1-propanolamine (MPAP) were given daily to mice with PD. In six of the 10 mice the mice had no disease one had mild clinical progression and one was euthanised. Mice treated with MPAP alone had significantly less neurological damage than those treated with either placebo or vehicle alone. The two-in-one medication also induced significant improvement over vehicle in clinical signs of post-synaptic -synuclein pathology in the hippocampus and cognitive impairment in the animals that received placebo or vehicle.

This study underscores the value of ongoing preclinical testing of novel therapies in animal models to improve the clinical outcomes of people suffering from both major forms of Parkinsons disease said Jane Milena M. D. M. C. professor of Cell and Developmental Biology at VUMC and senior investigator on the study.

There is no widely available treatment for Parkinsons disease a neurodegenerative disorder that affects the brains ability to transmit electrical impulses to muscle and other body parts. While medications known as tyrosine kinase inhibitors (TKI) are widely used for the treatment of certain forms of Parkinsons disease there is no approved drug to treat PD. The WHO estimates that 30 million people worldwide are affected by Parkinsons disease.

Vanderbilt scientists performed the study in collaboration with Myriad Genetics. One pilot study involving 570 mice with PD was published in Cell Reports. Milenas laboratory conducted the second pilot study involving 903 mice with PD and achieved serviceable efficacy by human-infection immuno-oxidant and transplantation studies. This study included mice derived from humans with neuropathological features and significantly improved in clinical signs of post-synaptic -synuclein pathology.

The study focuses on the acetylcholine-transporters the main type of transporter involved in the transport of messenger RNA from neurons to muscles. Milenas lab along with collaborators at Vanderbilt have proven that this glutamate-transport RNA transporter is more effective as a bridge to the brain and a powerful therapeutic target for Parkinsons disease.

We have built a library of molecules that have given us a ground-breaking new approach to drug development that potentially could treat Parkinsons disease in a novel way. This approach can work in tandem with the current therapy approaches to treat some forms of psychiatric and neurological disorders including post-traumatic stress and autism Milena said.

Vanderbilt scientists believe the novel compounds are one step further from the traditional 1-star-offense approach to drug development. They are highly targeted resilient and safe and well-tolerated in the hard-to-treat acute form of PD. The novel compounds were also promising candidates for potential clinical freezing to enhance clinical uptake.