Breaking News
February 17, 2019 - MUSC researchers discover new mechanism for a class of anti-cancer drugs
February 17, 2019 - HPV misconceptions are causing women to miss smear tests
February 17, 2019 - Sanofi and Regeneron Offer Praluent (alirocumab) at a New Reduced U.S. List Price
February 17, 2019 - Researchers say auditory testing can identify children for autism screening
February 17, 2019 - New method analyzes how single biological cells react to stressful situations
February 17, 2019 - WVU gynecologic oncologist investigates novel treatment for cervical and vaginal cancers
February 17, 2019 - ADHD diagnoses poorly documented
February 17, 2019 - Majority of gender minority youth do not identify with traditional sexual identity labels
February 17, 2019 - AbbVie, Teneobio enter into strategic transaction to develop potential treatment for multiple myeloma
February 17, 2019 - Lower Birth Weight May Up Risk for Psychiatric Disorders
February 17, 2019 - Scientists identify reversible molecular defect underlying rheumatoid arthritis
February 17, 2019 - Moffitt researchers shed light on how CAR T cells function mechanistically
February 16, 2019 - Female Anatomy May Play Big Role in Sperm’s Success
February 16, 2019 - BMI may mediate inverse link between fiber intake, knee OA
February 16, 2019 - Movement impairments in autism can be reversed through behavioral training
February 16, 2019 - Studies address racial disparities in postpartum period and cardiovascular health
February 16, 2019 - Scientists implicate hidden genes in the severity of autism symptoms
February 16, 2019 - Decreased deep sleep linked to early signs of Alzheimer’s disease
February 16, 2019 - Neuroscientists show how the brain responds to texture
February 16, 2019 - Gilead Announces Topline Data From Phase 3 STELLAR-4 Study of Selonsertib in Compensated Cirrhosis (F4) Due to Nonalcoholic Steatohepatitis (NASH)
February 16, 2019 - What Can I Do About Sweating? (for Teens)
February 16, 2019 - Companies navigate dementia conversations with older workers
February 16, 2019 - Newly developed stem cell technologies show promise for treating PD patients
February 16, 2019 - Collaborative material research could advance self-assembling nanomaterials
February 16, 2019 - Researchers take major step in creating technology that mimics the human brain
February 16, 2019 - Erasing memories associated with cocaine use reduces drug seeking behavior
February 16, 2019 - Artificial intelligence can accurately predict prognosis of ovarian cancer patients
February 16, 2019 - Racial disparities in cancer deaths on the decline for America
February 16, 2019 - FDA authorizes new interoperable insulin pump for children, adults with diabetes
February 16, 2019 - Coexisting Medical Conditions, Smoking Explain PTSD-CVD Link
February 16, 2019 - Skin Cancer Screening: MedlinePlus Lab Test Information
February 16, 2019 - ‘Happiness’ exercises can boost mood in those recovering from substance use disorder
February 16, 2019 - Cell manipulation could soon halt or reverse aging
February 16, 2019 - Pumped Breast Milk Falls Short of Breastfed Version
February 16, 2019 - Men’s porn habits could fuel partners’ eating disorders, study suggests
February 16, 2019 - Rapid progression of age-related diseases may result from formation of vicious cycles
February 16, 2019 - Immune checkpoint molecule protects against future development of cancer
February 16, 2019 - New method produces hydrogels that have properties similar to cells’ environment
February 16, 2019 - $4.1 million funding for heart research on Valentine’s Day
February 16, 2019 - General anesthesia in early infancy unlikely to have lasting effects on developing brains
February 16, 2019 - New breakthroughs for muscular dystrophy research
February 16, 2019 - First Opinion: Embryo editing for higher IQ is a fantasy. Embryo profiling for it is almost here
February 16, 2019 - Vapers develop cancer-related gene deregulation as cigarette smokers
February 16, 2019 - Bringing Antimicrobial Susceptibility Testing (AST) to the Community
February 16, 2019 - Decolonization protocol after hospital discharge can prevent dangerous infections
February 16, 2019 - Children with ASD more likely to face maltreatment, study finds
February 16, 2019 - Study finds genetic vulnerability to use of menthol cigarettes
February 16, 2019 - H-RT should be the standard of care for men with low risk prostate cancer, study shows
February 16, 2019 - New technique using patients’ own modified cells could help treat Crohn’s disease
February 16, 2019 - Therapeutic endoscopy has an expanding role in the treatment of IBD
February 16, 2019 - Blood clot discovery could lead to development of better treatments for blood diseases
February 16, 2019 - Intervention can increase exclusive breastfeeding rates
February 16, 2019 - New project explores how gaming technologies can help cancer patients communicate better
February 16, 2019 - Catalyst Biosciences Presents Updated Data from Its Phase 2/3 Trial of Subcutaneous Marzeptacog Alfa (Activated) in Individuals with Hemophilia A or B with Inhibitors at the 12th Annual EAHAD Congress
February 16, 2019 - Rerouting nerves during amputation reduces phantom limb pain before it starts
February 16, 2019 - A Hormone Produced When We Exercise Might Help Fight Alzheimer’s
February 16, 2019 - Millions of British people breathe toxic air travelling to GPs
February 16, 2019 - Conformance of genetic characteristics found to be crucial for longer preservation of kidney graft
February 16, 2019 - Researchers use optogenetic tool to control, visualize receptor signals in neural cells
February 16, 2019 - New reversible antiplatelet therapy could reduce risk of blood clots, prevent cancer metastasis
February 16, 2019 - Testosterone is not the only hormone needed for penis development
February 16, 2019 - FDA Advisory Committee Recommends Approval of Spravato (esketamine) Nasal Spray for Adults with Treatment-Resistant Depression
February 15, 2019 - Heart surgery technology developed at Baptist Health debuts after years of secrecy
February 15, 2019 - Prescription Opioids Double Risk of Triggering Fatal Car Crash
February 15, 2019 - New study helps doctors better understand high blood pressure in pregnant women
February 15, 2019 - Beta wave control in Parkinson’s diseased brain could be a potential therapy
February 15, 2019 - Media representations of love may justify gender-based violence in young people
February 15, 2019 - Yoga May Help With Rheumatoid Arthritis Symptoms, Severity
February 15, 2019 - Obstructive sleep apnea linked to inflammation, organ dysfunction
February 15, 2019 - Master your mind: A challenge from WELL for Life
February 15, 2019 - Why Some Brain Tumors Respond to Immunotherapy
February 15, 2019 - Must-Reads Of The Week From Brianna Labuskes
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
Gene therapy for Parkinson’s symptoms shows promise

Gene therapy for Parkinson’s symptoms shows promise

image_pdfDownload PDFimage_print

(HealthDay)—A new gene therapy might help improve motor symptoms in people with Parkinson’s disease who aren’t responding to other therapies, an early study has found.

“This is not a cure of Parkinson’s disease,” said James Beck, chief scientific officer of the Parkinson’s Foundation. “This is a potentially good treatment for symptom control. It provides an additional way of providing dopamine to the brain, but it doesn’t stop the progression of Parkinson’s disease.”

The new treatment uses a virus to deliver gene therapy to a targeted area of the brain. The gene therapy affects an enzyme called AADC. This enzyme transforms levodopa into dopamine in the brain.

Cells that make the neurotransmitter dopamine—a chemical messenger in the brain—die off in Parkinson’s disease, according to the U.S. National Institute on Aging. A loss of dopamine causes the symptoms of Parkinson’s disease, such as tremor and slow movements.

Standard treatments attempt to replace the lost dopamine. For example, one current medication is levodopa, but the cells that transform levodopa into dopamine have to be functioning for this treatment to work. As the dopamine-producing cells die off, it becomes harder and harder for the brain to respond to medications like this, Beck explained.

And, that’s where the new gene treatment may help.

Led by Dr. Chadwick Christine, at the University of California, San Francisco, researchers used MRI scanning to locate the right area of the brain. Then they infused the new gene therapy into a targeted area of the brain called the putamen. The study team chose this area because these brain cells aren’t destroyed by Parkinson’s disease.

The phase 1 trial included 15 people who were no longer responding to other Parkinson’s treatments. They all received one infusion of the gene therapy—known as VY-AADC.

After the treatment, researchers followed the patients’ health for up to 36 months and found that the treatment was well-tolerated. The most serious side effects—a blood clot and an irregular heart rhythm caused by the blood clot—were related to the surgery used to deliver the treatment, and not the treatment itself, the researchers said.

The therapy also showed a meaningful improvement in the time people spent without movement symptoms each day. And the effect appeared to be lasting, with some of the patients followed for as long as three years.

Dr. Alessandro Di Rocco, director of the Movement Disorders Program at Northwell Health in Great Neck, N.Y., reviewed the findings.

“This study seems to be a step forward in perfecting the [gene therapy] vector and they were able to give it safely,” he said.

Di Rocco added that the decrease in symptoms “is a real effect.”

But, like Beck, he cautioned that this is an early study with only a small number of people in the trial. Di Rocco also noted that the trial wasn’t “blinded,” so it’s possible there was a placebo effect for some.

Both Di Rocco and Beck also expressed concern about the possible cost of such therapy.

The University of California, San Francisco, and University of Pittsburgh Medical Center researchers aren’t the only team working on gene therapy for Parkinson’s symptoms. A group of British researchers is actively recruiting Parkinson’s patients to take part in a gene therapy trial that’s also aimed at increasing the availability of dopamine in the brain.

Their trial will look at up to 30 patients receiving treatment in London or Paris. For this study, researchers are targeting a part of the brain called the striatum.

Results of the American study were presented Sunday at the American Neurological Association meeting, in Atlanta. Findings presented at meetings should be viewed as preliminary until they’ve been published in a peer-reviewed journal.


Explore further:
Innovative gene therapy trial for Parkinson’s disease

More information:
James Beck, Ph.D., chief scientific officer, Parkinson’s Foundation; Alessandro Di Rocco, M.D., director, Movement Disorders Program, Northwell Health, Great Neck, N.Y., and professor of neurology, Zucker School of Medicine at Hofstra University; Oct. 21, 2018, presentation, American Neurological Association meeting, Atlanta

Learn more about Parkinson’s disease treatments from the Parkinson’s Foundation.

Tagged with:

About author

Related Articles