Breaking News
February 15, 2019 - Researchers uncover novel mechanism and potential new therapeutic target for Alzheimer’s
February 15, 2019 - Genetic variations in a fourth gene associated with higher ALL risk in Hispanic children
February 15, 2019 - Disruptive behavioral problems in kindergarten linked with lower employment earnings in adulthood
February 15, 2019 - New bioengineered device enhances the production of T-cells
February 15, 2019 - HDL proteome behaves like a tiny Velcro ball that is rolling on surfaces
February 15, 2019 - Puerto Rican children more likely to have poor or decreasing use of asthma inhalers
February 15, 2019 - Quality of patient care does not improve after physician-hospital integration
February 15, 2019 - Synopsys release new software for implant design and patient-specific planning
February 15, 2019 - 6 out of 10 hip replacements last 25 years or longer
February 15, 2019 - Health Tip: What You Should Know About Antibiotics
February 15, 2019 - New research challenges medical consensus that adenoids and tonsils significantly shrink during teenage years
February 15, 2019 - Discovery of weakness in a rare cancer could be exploited with drugs
February 15, 2019 - UVA scientists find potential explanation for mysterious cell death in Alzheimer’s, Parkinson’s
February 15, 2019 - New rules requiring female athletes to lower testosterone levels are based on flawed data
February 15, 2019 - Researchers comprehensively sequence the human immune system
February 15, 2019 - Researchers study animal venoms to identify new medicines for treating diseases
February 15, 2019 - Movement of wrist bones revealed by MRI and computer modeling
February 15, 2019 - Philips introduces new premium digital X-ray room to help shorten patient wait times
February 15, 2019 - Women fare worse than men following aortic heart surgery, study finds
February 15, 2019 - High-protein and low-calorie diet helps older adults lose weight safely, shows study
February 15, 2019 - Drug microdosing effects may not measure up to big expectations
February 15, 2019 - Discharged, Dismissed: ERs Often Miss Chance To Set Overdose Survivors On ‘Better Path’
February 15, 2019 - A digitized lab environment to be showcased at smartLAB 2019
February 15, 2019 - Scientists uncover main mechanisms of fluconazole drug resistance
February 15, 2019 - New study seeks to understand how colibactin causes cancer
February 15, 2019 - Photoacoustic imaging accurately measures the temperature of deep tissues
February 15, 2019 - Large study finds no association between phthalate exposure and breast cancer risk
February 15, 2019 - New research explains presence of ‘natural’ magnetism in human cells
February 15, 2019 - Bio-Rad launches new digital PCR system and kit for monitoring treatment response in CML patients
February 15, 2019 - Scientists shed light on damaging cell effects linked to aging
February 15, 2019 - High intensity exercise may improve health by increasing gut microbiota diversity
February 15, 2019 - Apellis’ APL-2 Receives Orphan Drug Designation from the FDA for the Treatment of Autoimmune Hemolytic Anemia
February 15, 2019 - Couples creating art or playing board games release ‘love hormone’
February 15, 2019 - Glimpsing The Future At Gargantuan Health Tech Showcase
February 15, 2019 - Common herbicide found to increase the risk of lymphoma
February 15, 2019 - Over-abundance of energy to cells could increase cancer risk
February 15, 2019 - Oxford Genetics appoints Jocelyne Bath as new Chief Operating Officer
February 15, 2019 - Castration-resistant metastatic prostate cancer responds to combination of immune checkpoint inhibitors
February 15, 2019 - Large-scale clinical trial begins to study liver transplantation between people with HIV
February 15, 2019 - Cannabis use among adolescents linked with increased risk of depression in adulthood
February 15, 2019 - Fractures, head injuries common in electric scooter accidents, UCLA study finds
February 15, 2019 - Prenatal maternal depression has important consequences for infant temperament, study shows
February 15, 2019 - Stereotactic body radiotherapy effective in treating men with low- or intermediate-risk prostate cancer
February 15, 2019 - Zogenix Submits New Drug Application to U.S. Food & Drug Administration for Fintepla for the Treatment of Dravet Syndrome
February 15, 2019 - Certain birthmarks warrant quick treatment, pediatricians say
February 15, 2019 - New machine learning method predicts if atypical ductal hyperplasia will turn cancerous
February 15, 2019 - Whole-genome sequencing and sharing real-time data could limit spread of foodborne bacteria
February 15, 2019 - FDA warns doctor for illegally marketing unapproved implantable device
February 15, 2019 - New injury documentation tool may provide better evidence for elder abuse cases
February 15, 2019 - Physiological age is a better predictor of survival than chronological age, shows study
February 15, 2019 - New study reveals high success rate for hip and knee replacements
February 15, 2019 - Prenatal exposures to BPA may pose threat to human ovarian function
February 15, 2019 - Suspicious spots on the lungs of children with rhabdomyosarcoma do not behave like metastases
February 15, 2019 - Diet drinks daily could raise stroke risk says study
February 15, 2019 - Many Systematic Reviews Do Not Fully Report Adverse Events
February 15, 2019 - Seven tips to protect your child from burns
February 15, 2019 - Keynote speakers announced for CBD Expo MIDWEST
February 15, 2019 - New DNA methylation GrimAge tool allows you to predict lifespan and healthspan
February 15, 2019 - New AI-driven platform analyze how pathogens infect human cells
February 15, 2019 - Increased activity of EHMT2 gene deficient neurons could cause autism in humans
February 15, 2019 - Recurring UTIs may mask symptoms of bladder or kidney cancer
February 15, 2019 - Researchers conduct extensive comparison of drugs used in treating neuroendocrine tumors
February 15, 2019 - Depression prevention for pregnant women and new mothers – new recommendations
February 15, 2019 - AHA News: Are There Health Benefits From Chocolate?
February 15, 2019 - The involvement of the gut in Parkinson’s disease: hype or hope?
February 15, 2019 - New PET imaging agent may help measure efficacy or failure of hormone therapy for breast cancer
February 15, 2019 - Preventing infections could help combat antimicrobial resistance
February 15, 2019 - Study investigates the role of estrogen in controlling glucose homeostasis
February 15, 2019 - Exposure to chemical in weedkiller Roundup raises risk of some cancers, study finds
February 15, 2019 - Smoking and drinking during pregnancy – stigma drives women to secrecy
February 15, 2019 - Low FODMAP diet reduces stomach issues caused by exercise
February 15, 2019 - Novel approach uses small amounts of tissue to quantify PD-L1 expression levels in tumors
February 15, 2019 - Breast pumps could be transmitting asthma-causing bacteria in babies, finds study
February 15, 2019 - The Pistoia Alliance Launches Next Phase of Blockchain Project to Develop Life Science R&D Use Cases
February 15, 2019 - The search for environmental causes of Parkinson’s disease moves forward
February 15, 2019 - Women scientists inhibited by funding methods that favor men, researchers say
February 14, 2019 - Few primary care physicians lack enough knowledge of cancer treatment options
February 14, 2019 - Prime real estate is determined by previous owner in the squirrel world
February 14, 2019 - Discovery of a ‘master switch’ within the immune system
February 14, 2019 - Health officers with surgical training are a safe alternative for performing C-sections
Parkinson’s Gene Therapy Wires New Brain Circuits

Parkinson’s Gene Therapy Wires New Brain Circuits

image_pdfDownload PDFimage_print

WEDNESDAY, Nov. 28, 2018 — An experimental gene therapy for Parkinson’s disease seems to work by rewiring key areas of the brain, a new study finds.

The researchers focused on 15 Parkinson’s patients who, in an earlier trial, had received so-called GAD gene therapy. GAD is an enzyme that spurs the production of a brain chemical involved in movement control.

In the previous trial, patients had shown improvements in their movement problems after receiving infusions of the GAD gene into the brain.

What hadn’t been clear was precisely why, said researcher Dr. David Eidelberg, who directs the Center for Neurosciences at the Feinstein Institute for Medical Research, in Manhasset, N.Y.

So for the new study, Eidelberg’s team examined specialized brain scans from 15 of the trial patients. The investigators found an unexpected answer: The gene therapy did not change the abnormal brain circuitry that marks Parkinson’s disease.

Instead, it essentially rewired a small area of the brain, to partially compensate for the faulty circuitry.

“It created its own set of circuits,” Eidelberg explained. “The disease circuitry continues — so this is not a cure.”

Still, he said, the gene therapy seems to spur new brain connections that can benefit people with Parkinson’s.

The study findings were published online Nov. 28 in Science Translational Medicine.

Parkinson’s disease affects nearly 1 million people in the United States alone, according to the Parkinson’s Foundation.

The root cause is unclear, but as the disease progresses, the brain loses cells that produce dopamine — a chemical that regulates movement. As a result, people suffer symptoms like tremors, stiff limbs, and balance and coordination problems that gradually worsen over time.

There are treatments to lessen those symptoms, including medications that boost dopamine levels or mimic the actions of dopamine. Another option for some patients is deep brain stimulation (DBS), where electrodes are implanted in a specific brain area to deliver continuous electrical pulses. It’s thought to help by suppressing abnormal electrical activity.

GAD gene therapy is done by inserting the gene into an inactivated cold virus. That viral “vector” is infused into a specific brain area called the subthalamic nucleus — which is one of the brain regions targeted in DBS treatment.

Originally, Eidelberg added, researchers thought the gene therapy would work in a “DBS-like” way.

But based on the new findings, that’s not the case.

Dr. Michael Okun is medical director of the Parkinson’s Foundation. He called the study “fascinating.”

“It showed that GAD gene therapy, unlike subthalamic nucleus DBS, did not change the expected Parkinson’s disease brain network,” Okun said. “Instead, it co-opted adjacent non-motor pathways.”

Why does that matter? One reason, according to Okun, is that it offers an “important lesson” for the gene therapy field going forward.

Researchers cannot make assumptions about a therapy’s “mechanism of action,” he said, based on the brain area it targets.

Eidelberg made another point: In future studies, researchers could use brain imaging to be sure that patients’ symptom improvements are due to a true effect of the gene therapy — rather than a “placebo effect.”

In the original trial, which involved a few dozen Parkinson’s patients, some were randomly assigned to receive GAD gene infusions. The rest underwent a “sham” procedure for comparison.

Over six months, both groups showed improvements in movement symptoms like stiffness and tremor. But the gene therapy group saw greater gains.

“It wasn’t a slam dunk,” Eidelberg stressed. “But they were doing better. And that persisted to the one-year mark.”

With any such therapy, there is a theoretical concern that the infused gene will have unintended effects.

“What we’ve seen is that this gene stays put,” Eidelberg said. “It doesn’t percolate all over the brain.”

In the original trial, there were no red flags, according to the researchers. The most common side effects were temporary headaches and nausea.

Various research teams are looking at different approaches to gene therapy for Parkinson’s. The hope, Eidelberg said, is to develop additional options that work for at least some patients — possibly freeing them from taking daily medications.

At this point, he noted, there is “a lot of interest” in doing a larger, later-stage trial of the GAD therapy. But none has begun yet.

The current study was funded by Neurologix Inc., the company that developed the gene therapy.

More information

The Parkinson’s Foundation has more on Parkinson’s treatment.

© 2018 HealthDay. All rights reserved.

Posted: November 2018

Tagged with:

About author

Related Articles