Will this Gene Therapy Really Ease Parkinson’s Disease Symptoms? 

Introduction

The battle for people with Parkinson's Disease may have just gotten bearable, according to the March 9th press release. This groundbreaking therapy study released showed that even those who suffered from advanced Parkinson's disease needed less levodopa, which promotes dopamine, than normal, and were seen to have less side effects than commonly noticed with this type of standard treatment. Those who received the therapy reported longer periods of time with not only lessoned debilitating problems but an overall more manageable life.

By using gene therapy to boost dopamine levels in the brain it could soothe the symptoms for Parkinson's Disease dramatically.

Parkinson's disease, also referred to as PD, is a neurodegenerative disorder that affects dopamine-producing neurons in a specific area of the brain called the substantia nigra. Each individual suffering from Parkinson's Disease experiences different symptoms which continuously progress over the years. The most common include: tremor, balance problems, and rigid or stiff muscles. Although scientists have not yet developed a cure for this disease, there are medications available and treatments that have shown some help. But, until now, treatment options have been risky procedures and according to The Centers for Disease Control and Prevention, complications during PD surgeries are the 14th cause of death this year in the United States alone.

Voyager Therapeutics of Cambridge, Massachusetts, shared data from an ongoing Phase 1b trial that is testing VY-AADC, a treatment designed to boost the amount of orally given levodopa, which like i said produces more dopamine. People with advanced Parkinson's disease who received the therapy have been seen to need less levodopa, and have shown fewer side effects which are commonly related with this standard therapy. They also reported longer amounts of time without debilitating movement problems, leading to an overall improvement of life. The findings suggest that the gene therapy might maximize the benefits of having muscle control, and reduce side effects commonly shown from forms of levodopa treatment.

More extensive trials will be needed in order to ultimately prove this as the most effective therapy but with these results, it seems it is going on in that direction.

In the phase 1b trial, 15 participants in three separate units were injected with VYAADC, which is an adeno-associated virus containing the AADC gene. After the injection, 14 out of 15 patients left the hospital within just two days, showing great progress and little to no symptoms. Those who participated were able to move much easier, for longer amounts of time. With the amount of success shown from this therapy, a phase ? trial is scheduled for later this year.

“The results from this trial are remarkable, with participants showing more hours of controlled movement, lessening their immobilization,” said Dr. Phillips, director of the Center for Parkinson's Therapy. “With the ongoing research and time put in by the most prestigious doctors and scientists in the world I believe that possibly in the near future, people living with Parkinson's will be able to have very few occasions of immobilization. I feel like this is one of the most amazing discoveries and will change thousands of lives for the better.”

The spread of Parkinson disease

Parkinson's disease affects roughly 200,000 people per year in the United States, mostly appearing in the late 40s. Besides increasing exercise or taking large amounts of medication each day, this form of therapy treatment will be the most reliable and will soon be the largest hope of people to live better, normal lives.

According to Brett Steinback, the scientist behind this study at the Steinbeck Institute, roughly one year after patients received the injection, they were shown to have newly developed brain connections that weren’t seen in previous scannings before the therapy. Shutting down the disease-infected pathways between the subthalamic nucleus, which controls pathway, and the brain’s motor regions, encouraged new, uninfected passageways to develop in their place. The newly developed passageways found in the brains of those participating are not even found in those not affected by Parkinson's. This shows us that the start of this progressing gene therapy allows those suffering from Parkinson's to form redeeming brain circuits to control movement.

Other treatment options are available, such as a treatment called “deep brain stimulation” which was found to not have the same miraculous rewiring effects as this one.     

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