Parkinson’s Disease Research

The mission of the National Institute of Neurological Disorders and Stroke (NINDS) is to seek fundamental knowledge about the brain and nervous system and to use the knowledge to reduce the burden of neurological disease.  The NINDS is a component of the National Institutes of Health (NIH), the leading supporter of biomedical research in the world.  The NINDS conducts and supports three types of research:  basic—scientific discoveries in the lab, clinical—developing and studying therapeutic approaches to Parkinson’s disease, and translational—focused on tools and resources that speed the development of therapeutics into practice.  The goals of NINDS-supported research are to better understand and diagnose PD, develop new treatments, and ultimately, prevent PD.  NINDS also supports training for the next generation of PD researchers and clinicians, and serves as an important source of information for people with PD and their families.

A long-term NINDS research and planning strategy led to the NINDS-hosted January 2014 conference, “Parkinson’s Disease 2014: Advancing Research, Improving Lives,” at which neuroscientists, physicians, public and private organization representatives, and people with Parkinson’s disease discussed the highest research priorities, ranging from lab discoveries to developing new therapies for PD 

The Parkinson’s Disease Biomarkers Programs (PDBP), a major NINDS initiative, is aimed at discovering ways to identify individuals at risk for developing PD and to track the progression of the disease.  Identifying biomarkers (signs that may indicate risk of a disease and improve diagnosis) will speed the development of novel therapeutics for PD.  Projects are actively recruiting volunteers at sites across the U.S.  The NINDS also collaborates with the Michael J. Fox Foundation for Parkinson’s Research (MJFF) on BioFIND, a project collecting biological samples and clinical data from healthy volunteers and those with PD.

The NINDS Morris K. Udall Centers of Excellence for Parkinson’s Disease Research program—a central component of NINDS PD research—was established in 1997 to find the fundamental causes of PD and better diagnose and treat people with PD.  The NINDS currently funds 10 Udall Centers across the country, where researchers are examining PD disease mechanisms, the genetic contributions to PD, and potential therapeutic targets and treatment strategies.

Parkinson’s Disease Clinical Studies offer an opportunity to help researchers find better ways to safely detect, treat, or prevent PD and therefore hope for individuals now and in the future.  NINDS conducts clinical studies on Parkinson’s disease at the NIH research campus in Bethesda, Maryland, and supports PD studies at medical research centers throughout the United States.  But studies can be completed only if people volunteer to participate.  By participating in a clinical study, healthy individuals and people living with Parkinson’s disease can greatly benefit the lives of those affected by this disorder.  Talk with your doctor about clinical studies and help to make the difference in improving the quality of life for all people with Parkinson’s disease. Current studies include genetics and PD, search for PD biomarkers, experimental therapies and other treatment options, diagnostic imaging, brain control and movement disorders, DBS, and exercise and PD.

Animal models: These are valuable tools for scientists studying disease mechanisms to develop new treatments for people with PD.  For example, a study of the drug isradipine—which had been shown in animal models to have a protective effect on dopaminergic neurons—is being tested for a similar neuroprotective effect in humans.

Cognition and Dementia:  Mild cognitive impairment is common in PD, sometimes in its early stages, and some people develop dementia in the disease’s later stages.  The NINDS has funded research using neuroimaging to predict which individuals with PD might develop cognitive impairment.

Deep Brain Stimulation (DBS):  NINDS has been a pioneer in the study and development of DBS, which is now considered a standard treatment option for some people living with PD whose symptoms no longer respond to PD medications.  While NIH supported research on brain circuitry was critical to the development of DBS, NINDS research continues to fine-tune the optimal site within the brain to implant the DBS electrode to help even more people with PD regain function.

Researchers are continuing to study DBS and to develop ways of improving it. A two-part study funded by the NINDS and the Department of Veterans Affairs first compared bilateral DBS to best medical therapy, including medication adjustment and physical therapy.  Bilateral DBS showed overall superiority to best medical therapy at improving motor symptoms and quality of life.  The second part of the study, involving nearly 300 patients, compared subthalamic nucleus (STN) DBS to globus pallidus interna (GPI) DBS.  The two groups reported similar improvements in motor control and quality of life in scores on the Unified Parkinson’s Disease Rating Scale.  On a variety of neuropsychological tests, there were no significant differences between the two groups.  However, the STN DBS group experienced a greater decline on a test of visuomotor processing speed, which measures how quickly someone thinks and acts on information. Also, the STN DBS group had slight worsening on a standard assessment of depression, while the GPI DBS group had slight improvement on the same test.

Other clinical studies hope to determine the best part of the brain to receive stimulation and to determine the long-term effects of this therapy.  In addition, NINDS-supported researchers are developing and testing improved implantable pulse generators and conducting studies to better understand the therapeutic effect of neurostimulation on the brain.

Environmental studies:  Risk factors such as repeated occupational exposure to certain pesticides and chemical solvents may influence who develops PD.  A NINDS-funded research consortium is hunting for environmental risk factors that increase susceptibility to developing PD before age 50.  For example, NINDS-funded researchers analyzed the occupational histories of twins in which one of the pair developed PD.  Based on estimates of exposure to six chemicals previously linked to PD, the researchers concluded that two of the common solvents were significantly linked to development of PD.  

Exercise:  Exercise routines are often recommended to help individuals with PD maintain movement and balance necessary for everyday living.  A recent NINDS-funded study evaluated three different forms of exercise—resistance training, stretching, and tai chi—and found that tai chi led to the greatest overall improvements in balance and stability for people with mild to moderate PD.  A current trial is studying the effects of two levels of exercise in people who have been recently diagnosed with PD.  

Genetic studies:  A better understanding of genetic risk factors is playing a critical role in elucidating PD disease mechanisms.  A 2011 NINDS workshop led to an analysis of data from PD genome-wide studies around the world, to correlate genetic variants and common traits among people with PD.  The workshop contributed to the development of NeuroX, the first DNA chip that can identify genetic changes in persons at risk for a number of late-onset neurodegenerative diseases, including PD.  Another NINDS collaborative, the Consortium On Risk for Early-onset Parkinson's Disease (CORE PD), hopes to identify the genetic factors that contribute to the development of early-onset PD.  Current clinical studies include the genetic connection to memory and motor behavior, the search for genes that may increase the risk of PD and related neurodegenerative disorders, and identifying biomarkers for PD.

Mitochondria:  These cellular energy factories may play a central role in PD.  NINDS-funded scientists have found that hundreds of genes involved in mitochondrial function are less active in people with PD.  Drugs that target genes involved in mitochondrial function could perhaps slow progression of the disease.

Motor complications:  Involuntary movement, including dyskinesia (difficulty controlling intended muscle movement), as well as tremor, dystonia (involuntary muscle contractions), freezing of gait (inability to start walking), and other motor complications become evident as PD progresses; these symptoms are often difficult to treat.  NINDS scientists have studied the safety and effectiveness of drugs and interventions in alleviating motor symptoms in persons with PD.  For example, basic research using adenosine found it could improve motor complications associated with PD.  A current NINDS clinical study of motor complications is testing an at-home device to evaluate PD movement symptoms while performing different tasks.  

Nerve growth factors:  Growth factors are proteins involved in nervous system formation and are of interest to researchers studying neurodegenerative diseases.  One small clinical trial will assess the safety, tolerability, and potential clinical effects of gene therapy with Glial Derived Neurotrophic Factor (GDNF)—a protein that may help protect dopamine-producing nerve cells.  This trial for individuals with advanced PD is based on NINDS-sponsored research showing that an advanced viral technique for delivery of the GDNF gene into the brain improves the health and function of the dopamine neurons in animal models of PD.

Neuroprotective Drugs:  NINDS supports basic, clinical, and translational research aimed at protecting nerve cells from the damage caused by PD.  The NINDS-funded NeuroNext Network is designed to test new therapies and to validate biomarkers in a number of neurological disorders, including Parkinson’s disease.

Stem cells:   Scientists are exploring various types of cells, including induced pluripotent stem cells (iPSCs), as opportunities for PD drug discovery.  iPSC technology is used to define disease mechanisms and discover the most promising treatments for sporadic PD.  To pursue this area of research, NINDS established a PD cell research consortium in 2009 in collaboration with the Michael J. Fox Foundation and the Parkinson’s Disease Foundation.


Reference: National Institute of Neurological Diseases and Stroke

Last updated May 5, 2017

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