Parkinson's disease clinical research
Encyclopedia
Parkinson's disease Clinical Research (also known as clinical trials, medical research, research studies, or clinical studies) is any study intended to help answer questions about new treatments by studying their effects on human subjects. Clinical trials are designed and conducted by scientists and medical experts, who invite participants to undergo testing new vaccines, therapies, or treatments.
Only a small fraction of patients with Parkinson's disease
(PD) participate in clinical trials. When clinical trials lack participation, it causes a significant delay in the development of new drugs and treatments.
Though there are many types of clinical research, the two most common are interventional and observational. For example, researchers trying to identify causes of PD may conduct an observational study to examine genetic or environmental factors that may have triggered the disease in an individual. Natural history studies that evaluate how Parkinson’s affects different people and how it changes over time are another example of observational research.
Researchers conducting interventional studies (also known as clinical trial
s) test the impact of treatments. These can include changing behavior, taking medications, or performing surgery. Interventional and observational research are equally important in helping to answer questions, develop new treatments, and ultimately find a cure for Parkinson’s. Clinical trials are conducted in a series of phases.
Among the interventional and observational studies for Parkinson's disease, research is ongoing in a number of specific areas.
research investigates the function that therapy (both physical and occupational), and exercise have in furthering quality of life for people with Parkinson's disease. For example, a study may evaluate the effectiveness of a short-term occupational therapy program, or the therapeutic effects of a certain type of exercise on balance and fall prevention for people with Parkinson’s. Other quality of life studies are testing different types of treatments, such as an ongoing clinical study exploring Vitamin D
as a possible therapy to improve balance and decrease the risk of falling in people with Parkinson's
is the term that describes treatment that may slow down, stop, or reverse the progression of Parkinson’s. Researchers are attempting to develop neuroprotective agents for Parkinson's disease, as well as other neurodegenerative brain disorders.
Several molecules have been proposed as potential neuroprotective treatments. However none of them has been conclusively demonstrated to reduce degeneration in clinical trials. Agents currently under investigation as neuroprotective agents include anti-apoptotic
drugs (omigapil, CEP-1347), antiglutamatergic agents, monoamine oxidase
inhibitors (selegiline
, rasagiline
), promitochondrial drugs (coenzyme Q10, creatine
), calcium channel blocker
s (isradipine
) and growth factor
s (GDNF).
Researchers are also investigating vaccines for Parkinson's disease that produce cells that change the way the body's immune system responds to the loss of dopamine
. This treatment has shown success in reversing Parkinson's in mice, and researchers are investigating the viability of clinical studies in people.
gene as a suspected cause of early-onset Parkinson's.
(DBS) is a surgical technique whereby a tiny electrode is inserted deep in the brain. The electrode
is connected to a battery pack that implanted under the collarbone via a subcutaneous wire. DBS is effective in suppressing symptoms of PD, especially tremor. A recent clinical study led to recommendations on identifying which Parkinson's patients are most likely to benefit from DBS.
-producing cells, thus helping to slow or stop the progression of the disease.
(PDF), PDtrials
is a collaborative initiative of Parkinson’s organizations that is committed to increasing knowledge and awareness about clinical research and Parkinson’s studies. The primary aim is to provide information and resources that help educate people with Parkinson’s and empower them to make informed decisions about clinical trials participation.
PDtrials lists Parkinson’s clinical trials that are active and seeking participants throughout the United States and Canada. The PDtrials.org website provides thorough, up-to-date information about studies. Searches can be performed for distinct Parkinson’s clinical trials using various criteria such as location, trial type, and symptom.
PDtrials also offers educational resources on clinical research and PD. These resources include a biannual bulletin that lists trials, a brochure about participation, guides for getting involved, and information about what questions to ask about studies.
is an annual multi-day training preparing people with PD to become advocates within the clinical research process.
The program focuses on education via training sessions, clinical researcher led workshops, as well as interaction with study coordinators and representatives from both the government and the industry. Former Learning Institute graduates are also often part of the sessions. Through this annual training, the Learning Institute prepares participants for designated activities that include, but are not limited to, increasing knowledge of clinical research and study participation, giving insight to research sponsors about trial design, and serving as representatives on clinical research reviews and advisory boards. After the conclusion of the Learning Institutes, graduates strive to ensure that the patient perspective is a continuing component in the process of treatment development.
The Michael J. Fox Foundation
aims to develop a cure for Parkinson's disease. It is the major Parkinson's fundraiser in the US with 140 million dollars in research between 2001 and 2008. In 2010, the Fox foundation launched the first large-scale clinical study on evolution biomarkers of the disease with a cost of 40 million dollars in 5 years.
Only a small fraction of patients with Parkinson's disease
Parkinson's disease
Parkinson's disease is a degenerative disorder of the central nervous system...
(PD) participate in clinical trials. When clinical trials lack participation, it causes a significant delay in the development of new drugs and treatments.
Research directions
The purpose of clinical research is usually to test the safety and efficacy of new treatments. Clinical research may also be conducted to learn other things about medical treatments or procedures, such as how the treatment interacts with other drugs.Though there are many types of clinical research, the two most common are interventional and observational. For example, researchers trying to identify causes of PD may conduct an observational study to examine genetic or environmental factors that may have triggered the disease in an individual. Natural history studies that evaluate how Parkinson’s affects different people and how it changes over time are another example of observational research.
Researchers conducting interventional studies (also known as clinical trial
Clinical trial
Clinical trials are a set of procedures in medical research and drug development that are conducted to allow safety and efficacy data to be collected for health interventions...
s) test the impact of treatments. These can include changing behavior, taking medications, or performing surgery. Interventional and observational research are equally important in helping to answer questions, develop new treatments, and ultimately find a cure for Parkinson’s. Clinical trials are conducted in a series of phases.
Among the interventional and observational studies for Parkinson's disease, research is ongoing in a number of specific areas.
Quality of life
Quality of lifeQuality of life
The term quality of life is used to evaluate the general well-being of individuals and societies. The term is used in a wide range of contexts, including the fields of international development, healthcare, and politics. Quality of life should not be confused with the concept of standard of...
research investigates the function that therapy (both physical and occupational), and exercise have in furthering quality of life for people with Parkinson's disease. For example, a study may evaluate the effectiveness of a short-term occupational therapy program, or the therapeutic effects of a certain type of exercise on balance and fall prevention for people with Parkinson’s. Other quality of life studies are testing different types of treatments, such as an ongoing clinical study exploring Vitamin D
Vitamin D
Vitamin D is a group of fat-soluble secosteroids. In humans, vitamin D is unique both because it functions as a prohormone and because the body can synthesize it when sun exposure is adequate ....
as a possible therapy to improve balance and decrease the risk of falling in people with Parkinson's
Neuroprotection
NeuroprotectionNeuroprotection
Neuroprotection within the nervous system protects neurons from apoptosis or degeneration, for example following a brain injury or as a result of chronic neurodegenerative diseases....
is the term that describes treatment that may slow down, stop, or reverse the progression of Parkinson’s. Researchers are attempting to develop neuroprotective agents for Parkinson's disease, as well as other neurodegenerative brain disorders.
Several molecules have been proposed as potential neuroprotective treatments. However none of them has been conclusively demonstrated to reduce degeneration in clinical trials. Agents currently under investigation as neuroprotective agents include anti-apoptotic
Apoptosis
Apoptosis is the process of programmed cell death that may occur in multicellular organisms. Biochemical events lead to characteristic cell changes and death. These changes include blebbing, cell shrinkage, nuclear fragmentation, chromatin condensation, and chromosomal DNA fragmentation...
drugs (omigapil, CEP-1347), antiglutamatergic agents, monoamine oxidase
Monoamine oxidase
L-Monoamine oxidases are a family of enzymes that catalyze the oxidation of monoamines. They are found bound to the outer membrane of mitochondria in most cell types in the body. The enzyme was originally discovered by Mary Bernheim in the liver and was named tyramine oxidase...
inhibitors (selegiline
Selegiline
Selegiline is a drug used for the treatment of early-stage Parkinson's disease, depression and senile dementia. In normal clinical doses it is a selective irreversible MAO-B inhibitor, however in larger doses it loses its specificity and also inhibits MAO-A...
, rasagiline
Rasagiline
Rasagiline is an irreversible inhibitor of monoamine oxidase used as a monotherapy in early Parkinson's disease or as an adjunct therapy in more advanced cases. It is selective for MAO type B over type A by a factor of fourteen....
), promitochondrial drugs (coenzyme Q10, creatine
Creatine
Creatine is a nitrogenous organic acid that occurs naturally in vertebrates and helps to supply energy to all cells in the body, primarily muscle. This is achieved by increasing the formation of Adenosine triphosphate...
), calcium channel blocker
Calcium channel blocker
A calcium channel blocker is a chemical that disrupts the movement of calcium through calcium channels.CCB drugs devised to target neurons are used as antiepileptics. However, the most widespread clinical usage of calcium channel blockers is to decrease blood pressure in patients with...
s (isradipine
Isradipine
Isradipine is a calcium channel blocker of the dihydropyridine class. It is usually prescribed for the treatment of high blood pressure in order to reduce the risk of stroke and heart attack...
) and growth factor
Growth factor
A growth factor is a naturally occurring substance capable of stimulating cellular growth, proliferation and cellular differentiation. Usually it is a protein or a steroid hormone. Growth factors are important for regulating a variety of cellular processes....
s (GDNF).
Researchers are also investigating vaccines for Parkinson's disease that produce cells that change the way the body's immune system responds to the loss of dopamine
Dopamine
Dopamine is a catecholamine neurotransmitter present in a wide variety of animals, including both vertebrates and invertebrates. In the brain, this substituted phenethylamine functions as a neurotransmitter, activating the five known types of dopamine receptors—D1, D2, D3, D4, and D5—and their...
. This treatment has shown success in reversing Parkinson's in mice, and researchers are investigating the viability of clinical studies in people.
Genetics
Of those people with PD, it is only a small percentage that inherits the disease. However, the study of genetic forms of Parkinson’s can assist scientists in learning more about the non-inherited forms. Several current studies are examining the genetic factors of Parkinson's disease. An example of genetic research is a recent study that investigated the GBAGlucocerebrosidase
β-Glucocerebrosidase is an enzyme with glucosylceramidase activity that is needed to cleave, by hydrolysis, the beta-glucosidic linkage of the chemical glucocerebroside, an intermediate in glycolipid metabolism...
gene as a suspected cause of early-onset Parkinson's.
Surgery
Advances in surgical procedures and neuroimaging techniques have ensured that surgical approaches can be as effective as medication at relieving some PD symptoms. Deep brain stimulationDeep brain stimulation
Deep brain stimulation is a surgical treatment involving the implantation of a medical device called a brain pacemaker, which sends electrical impulses to specific parts of the brain...
(DBS) is a surgical technique whereby a tiny electrode is inserted deep in the brain. The electrode
Electrode
An electrode is an electrical conductor used to make contact with a nonmetallic part of a circuit...
is connected to a battery pack that implanted under the collarbone via a subcutaneous wire. DBS is effective in suppressing symptoms of PD, especially tremor. A recent clinical study led to recommendations on identifying which Parkinson's patients are most likely to benefit from DBS.
Participant groups
Parkinson’s clinical research studies need volunteers at all stages of the disease to help solve the unanswered questions about Parkinson’s and to develop new treatments. Some studies seek to enroll specific groups of people.Newly diagnosed
A number of Parkinson's disease clinical research studies seek to enroll people newly diagnosed with PD that are not currently undergoing any treatment. These trials vary in scope, some focusing on neuroprotection in which researchers seek to determine whether a certain compound might offer protection to dopamineDopamine
Dopamine is a catecholamine neurotransmitter present in a wide variety of animals, including both vertebrates and invertebrates. In the brain, this substituted phenethylamine functions as a neurotransmitter, activating the five known types of dopamine receptors—D1, D2, D3, D4, and D5—and their...
-producing cells, thus helping to slow or stop the progression of the disease.
Healthy controls
In addition to patients with PD, healthy controls, including friends and family members of those with Parkinson’s, are also needed for clinical trials. Family members may participate in genetic studies, and healthy people can participate in trials that require a control group of participants without PD. Control groups are necessary as a means of testing the research being studied.Benefits
People with PD, their friends, and their family members all have many reasons to consider participating in clinical research. Many participants believe that their involvement benefits themselves and the future of other people with the disease. Without clinical research participants, many of the advances in treating PD would not have happened. In addition to furthering the scientific community’s knowledge of Parkinson’s, clinical trial participation may offer access to leading healthcare professionals and potentially useful new drugs and therapies. This care is often provided free of charge in exchange for participation in the study. Finally, by participating in clinical studies, those whose lives are impacted by PD may increase knowledge and understanding of the disease.How to participate
It can be a challenge to find the right clinical trial. People with PD may consult their doctors, discuss with their family members, and speak to other clinical trials participants about their experiences.Clinical research resources
People with Parkinson's disease who are considering participating in clinical research have resources available to help them navigate the clinical research process.PDtrials
Led by the Parkinson's disease FoundationParkinson's Disease Foundation
The Parkinson's Disease Foundation is a leading national presence in the United States in Parkinson's disease research, education and public advocacy. PDF funds scientific research to find the causes of and a cure for Parkinson's...
(PDF), PDtrials
PDtrials
PDtrials is an initiative to increase education and awareness about Parkinson's disease clinical research. The initiative is led by the Parkinson's Disease Foundation, and involves a collaboration between a number of Parkinson's organizations in the U.S...
is a collaborative initiative of Parkinson’s organizations that is committed to increasing knowledge and awareness about clinical research and Parkinson’s studies. The primary aim is to provide information and resources that help educate people with Parkinson’s and empower them to make informed decisions about clinical trials participation.
PDtrials lists Parkinson’s clinical trials that are active and seeking participants throughout the United States and Canada. The PDtrials.org website provides thorough, up-to-date information about studies. Searches can be performed for distinct Parkinson’s clinical trials using various criteria such as location, trial type, and symptom.
PDtrials also offers educational resources on clinical research and PD. These resources include a biannual bulletin that lists trials, a brochure about participation, guides for getting involved, and information about what questions to ask about studies.
Clinical Research Learning Institute
The Parkinson's disease Foundation’s Clinical Research Learning InstituteClinical Research Learning Institute
The Clinical Research Learning Institute is an initiative of the Parkinson's Disease Foundation designed to prepare people with Parkinson's disease to serve as advocates within the clinical research process...
is an annual multi-day training preparing people with PD to become advocates within the clinical research process.
The program focuses on education via training sessions, clinical researcher led workshops, as well as interaction with study coordinators and representatives from both the government and the industry. Former Learning Institute graduates are also often part of the sessions. Through this annual training, the Learning Institute prepares participants for designated activities that include, but are not limited to, increasing knowledge of clinical research and study participation, giving insight to research sponsors about trial design, and serving as representatives on clinical research reviews and advisory boards. After the conclusion of the Learning Institutes, graduates strive to ensure that the patient perspective is a continuing component in the process of treatment development.
Research organizations
The Parkinson's disease Foundation’s is a leading national presence in the United States in Parkinson's disease research, education and public advocacy. PDF works on behalf of people who live with Parkinson's disease by funding promising clinical research to find treatments and cures for Parkinson's. PDF was founded in 1957, and since then has invested over $90 million on scientific research.The Michael J. Fox Foundation
The Michael J. Fox Foundation
The Michael J. Fox Foundation for Parkinson's Research is dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson's today. Established by actor Michael J...
aims to develop a cure for Parkinson's disease. It is the major Parkinson's fundraiser in the US with 140 million dollars in research between 2001 and 2008. In 2010, the Fox foundation launched the first large-scale clinical study on evolution biomarkers of the disease with a cost of 40 million dollars in 5 years.