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Archive for the ‘Research’ Category

We Are Making Progress

Thursday, October 16th, 2014

By Carol Walton, CEO, The Parkinson Alliance

PUW2014IMG_4838

Carol Walton speaking about research at the 2014 Parkinson’s Unity Walk

Taking a step back to see how much Parkinson’s disease research has progressed

Every few years I believe that you need to step back and look at how Parkinson’s disease (PD) research has progressed.  Research takes so long and costs so much that sometimes it gets discouraging—but in the past few months I have read about some wonderful projects that tell me every penny we have spent on research has been well worth it!

Margaret Tuchman, President of The Parkinson Alliance, had Deep Brain Stimulation (DBS) almost 15 years ago.  The difference between what she went through over a decade ago to have this surgery and the practices and technology that are available today is amazing.  The Parkinson’s Unity Walk is funding research today that will potentially advance DBS even further, where if successful the outcome will be customized DBS in the future.  Many of you may have dogs that have a little chip put under their skin in case he/she gets lost for identification.  Technology is progressing towards that someday we will have a chip inserted into the DBS systems that will sense when your body needs electrical stimulation—what part of your body needs it and how much you need!

The National Institutes of Health (NIH) just funded a multi-center consortium collecting and analyzing thousands of pieces of data to develop a “global” view of neuromuscular diseases.  Dr. Clive Svendsen, professor and director of the Board of Governors Regenerative Medicine Institute, compared this shift in perspective to the way meteorologists began predicting weather years ago—viewing global trends and collecting vast amounts of data to create a forecast for a specific place and time.

As many of you know, falling and freezing of gait (FOG) are two of the major challenges in Parkinson’s disease.  In some cases, that one fall is enough to cause problems for many years.  We are currently funding a fascinating project entitled: What Patients Look at When Walking.  The focus of this project is an eye tracking machine that allows patients to walk freely, and records a video of what is in front of the patient and then indicates exactly what the eyes are focused on.  Hopefully, the knowledge we gain from this project will help prevent some of the falls and FOG for people with Parkinson’s and that will greatly enhance quality of life.

Sinemet, (carbidopa-levodopa) is still the gold standard of medication for Parkinson’s.  Today we know of at least four new delivery systems for this miracle drug.  Some medications will be able to provide 24/7 continuous delivery resulting in less “off” time for those living with PD and others will act as a rescue drug for people with Parkinson’s.  Drugs that are already approved by the Food and Drug Administration (FDA) are being tested to determine which ones might help people with PD.  The following are some examples:

  • Cynapsus is developing APL-130277, an easy-to-administer, fast-acting reformulation of apomorphine, in a sublingual (oral) thin film strip for the acute rescue of “off” motor symptoms of Parkinson’s disease.
  • Civitas Therapeutics is currently testing an inhaled version of Levodopa, called CVT-301, which reduces tremors and reduced movement caused by Parkinson’s disease and can be used as a “rescue” drug.
  • Impax Pharmaceuticals has submitted a New Drug Application with the FDA for Rytary, an investigational extended-release capsule formulation of carbidopa-levodopa for the treatment of idiopathic Parkinson’s disease.
  • NeuroDerm’s ND0612L and ND0612H for treatment of moderate and severe PD respectively, are both in clinical trials.  The delivery systems administer Levodopa subcutaneously through a small belt-worn pump similar to the administration devices used to deliver insulin to diabetic patients.  A second generation product, using a patch pump to deliver LD/CD, is under development.

Progress is being made and although we all want to know the cause and cure, we have many therapies to help with quality of life until we have the ultimate answer.  Research is so critical and we must keep up the fight because we are making progress.

This guest post was written by Carol Walton who is the CEO of The Parkinson Alliance, as well as the Executive Director of the Parkinson’s Unity Walk. 

Donation Dollars are at Work—A Highlight of 2014 Unity Walk Research Grants That are Underway

Thursday, October 16th, 2014
100% of donations to the Parkinson's Unity Walk goes to seven major U.S. Parkinson's disease foundations

100% of donations to the Parkinson’s Unity Walk goes to seven major U.S. Parkinson’s disease foundations

The fall is an exciting time of year at the Parkinson’s Unity Walk. The foundations benefiting from the funds raised at the Unity Walk submit their research proposals for the current year as well as provide updates on research projects conducted for the past two years. Proposals and updates are posted here on our website and available to all.

Research funded by the 2014 Unity Walk focuses on a wide range of topics. We continue to fund research that will take us closer to finding a cure for Parkinson’s disease and at the same time, fund research that will improve the quality of life of those who are living with Parkinson’s.

Here are just a few examples:

Levodopa-induced dyskinesia is one of the most debilitating symptoms associated with Parkinson’s disease. The Parkinson’s Disease Foundation is funding a research project to determine if a specific calcium channel in the brain contributes to Levodopa-induced dyskinesia, and whether silencing, or blocking this channel can alleviate these symptoms. If the research supports their hypothesis, it will provide a critical scientific rationale for design of clinical trials of current and new drugs to block this channel.

Research has proven that exercise improves the quality of life for those living with Parkinson’s disease but what kind of exercise will improve cognitive functioning, balance deficits, and aid in prevention of falls and injuries in PD? These are the specifics that the National Parkinson Foundation will study to compare the effects of skill-based vs. aerobic exercise vs. a control group. Post exercise changes in cardiovascular fitness, as well as executive functioning and cognition will be measured. Their hypothesis is that skill-based exercise will have the greatest improvement in executive functioning and lead to modification of the brain. We are interested to see what the research finds.

Currently, changes to Deep Brain Stimulation (DBS) only take place during a clinical appointment or within small ranges by a patient at home. What if research can help develop an adaptive or “intelligent” DBS that would record brain activity in real time and adjust the stimulation that responds to real time recordings of a patient’s movement and brain? The Michael J. Fox Foundation for Parkinson’s Research is using their funds to study this question; they will use a research tool to establish a connection between the implanted neuro-stimulator and an external computer that will allow for recording of brain activity in real time. The results of this research will help create the next generation of embedded adaptive neuro-stimulators used in DBS treatment.

Learn more about additional research projects funded by the 2014 Parkinson’s Unity Walk grants. It is your donations that make this research possible. Thank you for your continued support!

By Helaine Isaacs
PUW Event Director

Five Reasons I Advocate on Behalf of Clinical Trial Participation

Thursday, October 2nd, 2014

A Guest Post by Jean Burnsjean.burns.200

These are the reasons I have advocated on behalf of clinical trials for the past 12 years.

1.  Without clinical trial participation, there will be no new treatments.
2.  If people with Parkinson’s disease (PD) in the past had not participated, we would not have the treatments that we do.
3.  So future family members will not have to go through this.
4.  To help all people with PD everywhere.
5.  Because it might help you.

The first trial I joined was a phase 2/3 trial of a new drug that did slow progression or even stop it – in primates. This trial became notorious. The sponsor posted its premature ending on its website declaring the trial had failed and was over. FINISHED. They told none of the stake-holders. That experience was unsettling but it did not keep me from continuing to participate in future trials.

As a result of my participation in multiple trials over the years, I (or my data) are part of significant bodies of research. Coriell Cell Repositories, NIH’s Parkinson’s Disease Data Organizing Center, PDF’s Longitudinal and Biomarker Study in PD and NINDS’ Human Genetics Repository are just a few examples of the research that have benefited from my participation.

I am now patient #3 in a clinical trial funded by the National Institute of Neurological Disorders and Stroke – A Phase 1 Open-Label Dose Escalation Safety Study of Convection Enhanced Delivery (CED) of Adeno-Associated Virus Encoding Glial Cell Line-Derived Neurotrophic Factor (AAV2-GDNF) in Subjects with Advanced Parkinson’s Disease.

This Phase I clinical trial involving gene therapy and brain surgery is why my local family doctor calls me a “risk-taker.” Even though I don’t see myself in that way, I realize I have taken risks in participating in some Phase I clinical trials. As I tell my friends and families, it’s the one after the monkey trials end. This clinical trial involves risks to its human trial participants and requires a huge commitment of time and energy.

I am committed to participating for the next 5 years, and if my math is correct, during that time I will undergo the following:

  • 3 PET scans
  • 7 MRIs
  • at least 150 vials of blood
  • 3 lumbar procedures
  • 5 psych exams
    and
  • 60+ days traveling and in clinic days at the NIH (5 hour plane rides to and from Arizona)

You may have noticed that I use the phrase “human trial participant” in this blog. I do so because I believe we should never forget that it is human beings who volunteer for these trials. We often take great risks to our health, and we do so receiving no financial remuneration. We are lucky if the costs of being part of a clinical trial are fully reimbursed, and we are even luckier if the clinical trial sponsor makes the commitment to care for human trial participants who may be injured during their clinical trial.

So far, I have not personally benefited from my participation in these clinical trials. Yet, I am certain that my participation has furthered current research and will benefit those living with PD as well as those in the future who are at risk for being diagnosed with Parkinson’s disease.

This guest post was written by Jean Burns, Recipient of the 2011 Alan Bonander Humanitarian Award and Independent Parkinson’s Advocate & Activist (IPAA). For more information about Jean Burns’ work in the Parkinson’s community, please visit the following websites www.pdplan4life.com and www.pdblogger.com.

Take Action – Find a Clinical Trial That is a Match for You

Wednesday, October 1st, 2014

Two of our walkers share their experience after finding clinical trials that were a fit for them

Each fall, PUW’s focus is on research. This is the time of year when the seven leading foundations who receive funding from the donations raised at the Unity Walk submit their research grants for the coming year and update the grants that have been funded for the past two years. When we survey our walkers, the #1 reason they participate in the Unity Walk is their desire to raise funds for Parkinson’s research — which can progress to clinical trials. This month, our featured team and one of our guest bloggers are strong advocates for clinical trials as one way to take action.

Cyndy Gilbertson is this month’s Parkinson’s CHAMP. She has been living with Parkinson’s disease for over 27 years. Cyndy’s first clinical trial was for Azilect, now an approved medication for the treatment of Parkinson’s disease (PD). She has just undergone surgery at the National Institute for Health as part of a trial studying gene therapy that may retard and possibly reverse the disease process.

Cyndy Gilbertson

Cyndy Gilbertson

I’ve asked Jean Burns, the 2011 recipient of the Alan Bonander Humanitarian Award and a longstanding participant in clinical trials, to share her experience by preparing a guest post entitled, Five Reasons to Advocate on Behalf of Clinical Trial Participation. Jean has been participating in clinical trials for the past 12 years. Most recently, she has undergone brain surgery as part of a five year gene therapy trial conducted by the National Institute of Neurological Disorders and Stroke.

Jean Burns

Jean Burns

When I first decided to highlight the active participation of these two longstanding supporters of the Unity Walk who are also strong advocates for clinical trials, I had no idea they were currently participating in the same NIH clinical trial. Jean was #3 in the trial and Cyndy is #6. These women are courageous and their willingness to undergo surgery is extraordinary. Participation in most clinical trials do not require this level of commitment.

As research progresses into clinical trials, participants are needed to assess the effectiveness of these new potential treatments. Many trials face delays because of a lack of volunteers. We encourage those who are living with Parkinson’s disease and their loved ones to participate in clinical trials. The easiest way to find out what clinical trials you might be eligible for is to register with Fox Trial Finder. This resource was created by The Michael J. Fox Foundation for Parkinson’s Research to help increase the flow of willing participants—both people with Parkinson’s and control participants who do not have Parkinson’s—into the clinical trials that need them, accelerating the Parkinson’s drug development process. Fox Trial Finder will not only list ongoing PD clinical trials and research studies, but will match registrants to the trials that need them and are best-suited to their specific traits.

There are so many ways to get involved in advocating for the Parkinson’s community and participating in a clinical trial is one of them. If you find a clinical trial that is a match for you, please consider taking action in this way, and join members of the Parkinson’s community who are committed to doing all they can to be a part of finding a cure and new treatments for people living with Parkinson’s disease.

By Helaine Isaacs
PUW Event Director

Advocacy through PDF’s Parkinson’s Advocates in Research Program

Tuesday, March 11th, 2014

Karen Smith PUW for blogReceiving a diagnosis of Parkinson’s disease was not what I was expecting when at the age of 42, I sat in the office of a neurologist. That day, I took the first step on a long unknown journey. My life was not over but life as I knew it was. For the first few years, I only shared the diagnosis with close friends and family. I didn’t want to be perceived as handicapped or “less than” in anyone eyes. I didn’t want sympathy so I erected walls around the Parkinson’s piece of me. If I didn’t take PD out of those walls, I didn’t have to examine it or contemplate what effect the disease would have.

Over the course of the last thirteen years, I’ve experienced many physical and cognitive changes. Muscles refuse to cooperate and my body’s movements grow difficult. My view of myself has changed also.  I don’t want to become my illness. I tell myself every day “You are still you.”

I’ve had to redefine my life and what brings meaning to it. I made up my mind to live the best life possible with Parkinson’s disease. I determined that a part of that life would include advocacy. I not only want a better future for myself but for all those living with Parkinson’s disease. I start with hope and fuel that hope with action.

Living in a mid-sized Midwestern town made my advocacy efforts somewhat difficult. My city did not have movement disorder specialists, research clinics or any conferences or symposiums. Then I heard about a program offered through the Parkinson’s Disease Foundation (PDF). The program is called Parkinson’s Advocates in Research or PAIR. The PAIR program works through patients to bring about better treatments at a faster pace. This is accomplished by ensuring that people with Parkinson’s and care partners are primary partners in research alongside scientists, industry and government. They are bringing together the people who live with Parkinson’s and the people who are developing new treatments.

Through in-person trainings and an online course, the PAIR program provides people touched by Parkinson’s with the knowledge and skills needed to pair up with scientists and healthcare professionals. By collaborating with research institutions, the PAIR program facilitates partnerships between Research Advocates and professionals at the frontlines of research.

The support PDF provides doesn’t end with the training. PDF staff works closely with advocates to identify advocacy opportunities, participate in ongoing education (webinars, conference calls), share resources, and network. Only people with Parkinson’s and care partners know what it’s like to live with Parkinson’s disease. It’s important that researchers hear what they have to say. For example, PDF Research Advocates can raise issues that are all too often overlooked, and help to identify and solve barriers to Parkinson’s research. Most importantly, the presence of Research Advocates reminds scientists of the urgency to find new treatments and a cure for Parkinson’s as soon as possible. With the training I received through PDF I was ready to begin my role as a research advocate.

To learn more about the PAIR program, visit the PDF website http://www.pdf.org/pair. With hope and action we CAN all make a difference!

Karen Smith
Team Member, PDF Pacers