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Participation of Early Parkinson’s Patients in Clinical Trials Crucial to Finding Cure, Expert Says

The time between diagnosis and implementation of symptomatic treatment is critical in the effort to find a cure for Parkinson’s disease. However, many early Parkinson’s patients wait too long before seeking medical attention, leaving researchers with a small group of candidates for clinical trials, says Robert A. Hauser, MD, the director of the Parkinson’s & Movement Disorder Center at the University of South Florida.

In his commentary, “Help cure Parkinson’s disease: please don’t waste the Golden Year,” published in the journal npj Parkinson’s disease, Hauser stresses the importance of early Parkinson’s patients participating in clinical trials before they start taking symptomatic medications.

The discovery that Parkinson’s is associated with aggregation of misfolded alpha-synuclein proteins in the central nervous system suggests there are many potential targets for therapeutic intervention.
Researchers are enthusiastic about the idea that the disruption of this process, or removing toxic aggregates, can slow or stop disease progression. Unfortunately, there are still no validated biomarkers, such as a simple blood test, that can help monitor disease progression or test promising therapies in Parkinson’s patients.

So far, the most common way to test a therapeutic candidate is to assess its capacity to slow the progression of clinical signs and symptoms compared with placebo over time in patients with early Parkinson’s who are not yet receiving symptomatic Parkinson’s medications — such as levodopa, dopamine agonists, and MAO-B inhibitors.

However, this can prove to be a challenge, since patients with early Parkinson’s disease can only be followed, without the use of symptomatic medication, for about six to 12 months, what Hauser calls the “Golden Year.” After this, many patients will need medication to relieve symptoms.

“The critical time of about one year from when the patient can be diagnosed with early PD [Parkinson’s disease] based on mild classic motor features until they truly require symptomatic therapy can be considered the Golden Year,” Hauser said in a press release. “It is during this early, untreated phase, that progression of clinical symptoms reflects the progression of the underlying disease.”

Interference from symptomatic medications makes it difficult for researchers to tell if the potential treatment being tested is slowing disease progression or if they are just seeing effects from those other therapies.
However, patients with early Parkinson’s who are available to enroll in a clinical trial and whose symptoms are mild enough are in short supply.

Therefore, to test promising potential disease-modifying therapies, patients with early Parkinson’s have to be identified and referred to clinical trials before they go on symptomatic medications. Unfortunately, this is often not the case.
“If the time period over which we test the intervention is short, we reduce our ability to identify a difference between the intervention and placebo. If the time period over which we attempt to test the medication is too long, a substantial proportion of patients may require institution of symptomatic therapy and we lose our ability to monitor clinical disease progression during the observation period,” Hauser wrote.

Source: Parkinson’s News Today

Pre-clinical research may slow effects of Parkinson’s

by: ABC 33/40

ABC 33/40′ spoke with two people who live with the disease about new findings from Purdue University researchers.

Published in the scientific journal Molecular and Cellular Neuroscience, the researchers say they have pinpointed an important compound in the development of Parkinson’s disease in the brain.

Their work could be significant because it may impact how we treat the disease in several ways: like better therapies, new drugs and earlier diagnoses or preventative care.

Two men gave reporter Patrick Thomas a glimpse into what new treatments in their lives could do.

Whether he stands or sits, with shaky hands and trembling feet, Parkinson’s is life for Wayne Cook for the last ten years. “The things that you, used to take for granted,” Cook says as he tries to raise his left hand to his chest but can’t hold it still. “The fine motor skills are difficult.”

The same goes for Ken Cater over the last nearly 13 years of his life. “But you know it’s tough. Right now I’m dealing with a progressive disease,” says Cater.

Which Purdue University researchers say when they located the compound called Acrolein, it may lead to discoveries that alleviate symptoms of Parkinson’s. Cook tries to explain how it affects his speech. “You get something here (points to his brain) and then you know what you want to say, but it just stops right here. It won’t come out all the way.”

Cater explains what he thinks most fighting the disease want right now. Cater says, “The Holy Grail that we are looking for right now is something that can slow or stop the progression of the disease.”

Right now the research includes work that has been tested in animal models and cells, not humans.

Cook says he is even willing to be a part of any research that may eventually include human testing. “I’m anxious for it to happen. If they have got a study and they need volunteers I raise my hand,” says Cook.

But through the research, scientists say they lessened and reversed effects of Parkinson’s using the drug Hydralazine

Since it’s still only pre-clinical research Cater says he will wait and see. “I’m hopeful for a cure in the near future if not at least better treatments,” he says.

If you have questions about living with Parkinson’s as a patient or caretaker, the Parkinson Association of Alabama may be able to help.

Source: Pre-clinical research may slow or reverse effects of Parkinson’s, patients weigh in

Parkinson’s Foundation Enrolls 10,000th Patient in Largest Clinical Trial of the Disease Ever

By Carolina Henriques

The Parkinson’s Foundation has enrolled the 10,000th patient in the largest clinical trial of the disease yet to be conducted.

Among the critical discoveries so far, the research has shown that regular visits to neurologists, more exercise, and more attention to mental health could help improve patients’ wellbeing.

The Parkinson’s Outcomes Project is evaluating a broad range of factors associated with the disease, including medications, treatments, movement symptoms, cognition, anxiety and depression, and the disorder’s burden on caregivers.

Launched in 2009, the project has become a comprehensive platform for studying the lives of Parkinson’s patients. And it has led to the formation of a consortium of 29 experts in five countries.

The study includes over 100 people who have lived with Parkinson’s for more than 30 years and 83 who learned about their diagnosis before they were 30 years old. Its records include 25,000 visits to doctors and information from almost 9,000 caregivers.

Key conclusions drawn from the study include:

Regular visits to neurologists should be a priority for patients and caregivers because it could save thousands of lives a year.

Recent research has listed regular visits to a neurologist as an important step in Parkinson’s management. However, in a 2011 study, only 58 percent of 138,000 Parkinson’s-related difficulties led to neurologist care. Race was a significant demographic predictor of neurologist treatment, with non-whites being less likely to receive care.

Doctors should give patients’ physical activity more attention because studies have shown that increasing exercise and movement to at least 2 1/2 hours a week can slow the decline in patients’ quality of life.

Researchers have found that, in Parkinson’s, it’s not the type of exercise a patient engages in, but the frequency of the workout that’s important. Physical therapists recommend exercises whose goals include improving balance and coordination, flexibility, endurance, and strength.

Patient’s mental health should be a priority because researchers have found that depression and anxiety are leading factors in patients’ overall health.

Depression is one of the most common non-movement symptoms of the disease, with up to 60 percent of patients affected at one time or another.

Finally, doctors should do a better job of addressing gender differences between patients. A key reason is that many men can rely on wives and other family members for daily support and doctor visits. Women are less likely to have family caregiver support and be more frequent users of formal, paid caregiver services.

This discovery is supported by recent findings that confirm these gender disparities, such as a study in Neurology in 2017.

“We have obtained a wealth of information in what now represents the broadest and most inclusive patient population ever assembled in a clinical study of Parkinson’s,” Peter Schmidt, the senior vice president of the Parkinson’s Foundation, said in a press release. He has been directing the study.

“This project is truly innovative in that it not only follows thousands of patients over time, but that it studies everyone with Parkinson’s, from the newly diagnosed to people who have lived with the disease for 30 years or more,” added Thomas Davis, the study’s co-chair.

Researchers have been using the Parkinson’s Foundation’s Centers of Excellence network of 42 medical centers to enroll patients in the study.

In addition, “we are studying the quality of Parkinson’s care delivered at our Centers of Excellence to help patients who aren’t being seen at one,” said Fernando Cubillos, who oversees the study’s operations. “Our goal is to help identify the best care and disseminate that information widely.”

The post Parkinson’s Foundation Enrolls 10,000th Patient in Largest Clinical Trial of the Disease Ever appeared first on Parkinson’s News Today.

Source:: Parkinson’s Today

Record Pace of Recruitment Speeds Isradipine Trial

Great Clinical Trial Insight from the Michael J Fox Foundation By Allyse Falce.

Clinical trial news update: A recent MedPage Today article, “Quick Enrollment for STEADY-PD III Trial,” highlighted the accelerated enrollment of participants in the Safety, Tolerability and Efficacy Assessment of Dynacirc for PD (STEADY-PD) III trial, thanks in part to MJFF’s online trial matching tool, Fox Trial Finder.

A few months ago, we interviewed Kevin Biglan, MD, MPH, associate chair of clinical research for the Department of Neurology at the University of Rochester and co-principal investigator of STEADY-PD. Read more below about the trial and the remarkable efforts that led to the study’s successful recruitment:


Phase III testing for the compound isradipine is progressing after a remarkably short recruitment period; 336 participants enrolled in less than one year. Slow recruitment is a significant roadblock to testing of potential treatments and slows the pace of bringing new drugs to market. This success advances the pace of this study and may serve as a model for other programs.

Isradipine is a calcium channel blocker currently prescribed to treat high blood pressure. It came to the attention of Parkinson’s researchers when data from large studies showed lower risk of Parkinson’s disease (PD) among people who took the drug for hypertension. Scientists believe isradipine works to prevent the death of dopamine-producing cells and therefore may slow the progression of PD. The Michael J. Fox Foundation (MJFF) funded pre-clinical work to make that connection, as well as the Phase II trial. In 2014, isradipine researchers received a $23 million grant from the National Institutes of Health to move the Safety, Tolerability and Efficacy Assessment of Dynacirc® for PD (STEADY-PD) study into Phase III efficacy testing. Dynacirc® is the commercial name of the isradipine hypertension drug.

Kevin Biglan, MD, MPH, associate chair of clinical research for the Department of Neurology at the University of Rochester, is co-principal investigator of STEADY-PD. Dr. Biglan spoke to MJFF about the study’s successful recruitment period, and answered some commonly asked questions about Parkinson’s disease and calcium.

MJFF: Congratulations on completing study recruitment. What kind of participants were you looking for and how did you find them?

KB: We were looking for newly diagnosed individuals who had not yet started treatment for PD. Traditionally, this is a very difficult population to recruit. These individuals are just getting the news that they have Parkinson’s disease, and they’re not necessarily thinking about participating in research. And a lot of newly diagnosed people need treatment right away, so that eliminates many potential volunteers.

About 60 percent of people enrolled directly through the 55 study sites. The second largest group came through the MJFF online trial matching tool Fox Trial Finder. One hundred people were prescreened through the site, and about half of them ended up enrolling. And another subset of participants were referred by neurologists outside of the study sites.

Our timeline was 18 months; we were six months ahead of schedule. We worked with MJFF on a recruitment plan, and we think our methods of communication with the study sites and with volunteers who came through other sources may be of use to the Parkinson’s research community. We’re planning to write an article and share those tactics soon.

MJFF: How does accelerated trial recruitment speed drug development?

KB: The biggest barrier to drug development is enrolling an adequate number of individuals into a study. A lot of the time and costs of trials are associated with this delay in recruitment. The longer it takes to get people into a study, the longer it takes for us to find the results.

MJFF: When might isradipine be approved to treat PD?

KB: The last person will be out of the study in November 2018. After that, it’ll probably be about three to six months before we have final results. That would put us into the beginning of 2019. If the results look promising, because it’s a readily available drug, it may be prescribed for Parkinson’s soon after.

MJFF: Many Parkinson’s patients who don’t have hypertension have asked if they should begin taking isradipine. Is this a good idea?

KB: At this point, we still don’t know that isradipine has beneficial effects on Parkinson’s disease, so we recommend that people don’t start taking this medication until we have more information. Also, low blood pressure is a symptom of PD, and if you don’t have hypertension, this medication may exacerbate that condition. There are other side effects, mainly dizziness and swelling, associated with isradipine, too. Certainly, before you start any medication you should talk to your physician about it. There could be something specific to you that might put you at higher risk of developing problems, so it’s not something people should start without some discussion.

MJFF: If a patient is currently taking another calcium channel blocker, should they switch to isradipine?

KB: If a person with PD needs to be on a calcium channel blocker, for whatever reason, high blood pressure or otherwise, and their cardiologist or primary care doctor thinks isradipine is a reasonable alternative choice, then there’s likely no harm in switching between calcium channel blockers. But again, that’s a discussion that needs to occur between the patient and physician.

MJFF: Since this is a calcium blocker, should people stop taking calcium supplements? Or cut out calcium-rich foods?

KB: There’s no reason to worry about calcium supplements or calcium-rich foods. With isradipine, it’s targeting a specific calcium channel in the brain that we think may play a role in the cause of Parkinson’s disease. Calcium itself is highly regulated in the bloodstream. You don’t need to stop taking calcium supplements or avoid calcium-rich foods; there’s no evidence that those things have any negative effect on Parkinson’s disease.

MJFF: Thanks for speaking with us, Dr. Biglan. Anything else you’d like to add?

KB: We’re incredibly grateful to the Parkinson’s community for their partnership in this study. It’s going to allow us to answer a very important question about whether this treatment can slow progression of Parkinson’s disease sooner than we would have been able to without the assistance of patients, their families, and advocacy and research organizations.

Source:: Fox Feed Blog