Friday, March 30, 2012

PDF Champions Friday!


Surprise… Happy 60th Birthday to Gary Chard from Wilmington, DE!  There is no better way to celebrate your birthday than with a surprise party thrown by family and friends - - while fundraising for PDF at the same time!

At the beginning of the year, Gary’s wife Beth Ann came to PDF with the fantastic idea of turning Gary’s special ‘secret’ bash into a PDF Champions fundraising event Gary was diagnosed with Parkinson’s disease in 2008 at the age of 56. Together Gary and Beth Ann have been extremely involved in the Parkinson’s community. Gary presents educational topics about Parkinson’s to support groups in NJ, PA, MD and DE and Beth Ann has become a true care partner and a PDF Research Advocate

I am so pleased to announce that Gary’s party raised over $2,500 for Parkinson’s research! Applause all around!

Remember: The Cure Begins with YOU!

Thursday, March 29, 2012

Thursday's Tip: PDF Champions

Youngsters can be the best fundraisers!

Who needs a fundraising tip or two? Here are a few unique ideas to get your children or grandchildren involved in the fight against Parkinson’s disease…

School Read-A-Thon: First request the support of your local school (s) or PTA organization. Once you have the go ahead, students can ask family, friends and neighbors to sponsor them for every book they read. As an incentive, students can win small prizes (either bought or donated) depending on how many books they have completed! This is a great way to get children involved; encouraging them to read and raise awareness for Parkinson’s the same time.

Wii Love to Party:  Have your children invite their friends over to participate in a video game party/competition. Set up an ‘entry fee’ and all proceeds go towards your PDF fundraising goal

Remember: The Cure Begins with YOU!

Wednesday, March 28, 2012

Celebrate Spring: PDF Champions

Come Celebrate Spring with PDF!

The Parkinson's Disease Foundation (PDF) and the Young New Yorkers for the Fight Against Parkinson’s committee invite you to Celebrate Spring, an evening of cocktails, dancing, live auction, hors d'oeuvres and dessert to benefit PDF’s research programs.

Here are all the details:

Wednesday, April 4

8:00 PM – 12:00 AM
LAVO, 39 East 58th Street, New York, NY

Entertainment: Music by DJ Brenda Black

New to this year’s event is a live auction hosted by Kathleen Guzman from Heritage Auction. Items include: 4 days/3 nights in Budapest, Hungary; Fishers Island Club Golf & Lunch; Autographed copy of LIFE by Keith Richards; and Sunday brunch for two at LAVO.

Founders: G. Pennington Egbert III, Georgina B. Schaeffer and Missy Egbert Sheehan

Co-Chairs: G. Pennington Egbert III, Jonathan E. Romero, Jr., Gregory and Barbara Romero, Georgina B. Schaeffer, Missy Egbert Sheehan and William B. Sheehan

History: Celebrate Spring was first conceived five years ago by G.Pennington Egbert III, his sister Missy Egbert Sheehan and their close friend Georgina B. Schaeffer.  The three, whose fathers both lived with Parkinson’s disease, joined together to create the Young New Yorkers for the Fight Against Parkinson’s committee, a group of nearly 100 New Yorkers dedicated to advancing a cure for Parkinson’s.  Each year the committee hosts Celebrate Spring, which has raised more than $300,000 since its inception. 

Proceeds will benefit PDF’s Côté Clinical Genetics Initiative, which supports research to identify and understand the role of genetic markers in Parkinson’s disease. 

If you are interested in becoming a PDF Champion and starting your own fundraising event, please visit

Remember: The Cure Begins With YOU!

Tuesday, March 27, 2012

Tuesday's Tip: PDF Champions

Coffee Anyone?

Fundraising for Parkinson's disease can be as simple as using your everyday contacts for support.

Just like myself, I know you frequent your local coffee shop more than you would like to admit. Be sure to take advantage of this special relationship in your efforts. Ask the shop manager or head barista if you can set up a donation container to collect spare change for your PDF Champions fundraiser or if you can display your event signage in the shop. Decorate it with green and yellow (PDF’s colors), quotes or pictures to really catch people’s attention.

You can even take this concept one step further. If you have a close relationship with any store owner ask if they would be willing to set up a point of purchase donation program. For example, if customers donate $1 at the register they receive 10 percent off their next purchase at the store or receive a free gift.

What has worked for you? If you are a past PDF Champion or fundraiser send me your successful ideas.

Remember: The Cure Begins with YOU!

Monday, March 26, 2012

Run for PDF!

Are you a runner looking for your next big race? Interested in completed a 26.2 to support Parkinson’s disease research? Well, you are in luck! PDF has six valuable entries into the 2012 Marine Corps Marathon on Sunday, day, October 28 in Arlington, VA! To make you aware of just how valuable these six entries are … last week’s registration marked the fastest sellout in the history of marathon events, the Marine Corps Marathon sold out 30,000 available online entries in 2 hours, 41 minutes, beating the previous registration record of eight hours for the 2011 Boston Marathon.

So, the only way to be part of this incredible race is through a charity partner. If you are interested in joining the PDF Marathon Team please contact me at (800) 457- 6676 or

Registration Details:

  • $50 (non-refundable) registration fee. With the registration fee, you will receive PDF gear (including a hat, banner, wristbands, racing t-shirt and your own personal PDF Champions fundraising webpage) as well as fundraising support from yours truly.

Fundraising Commitment:

  • By joining the team, you are committing to raising $2,000 for PDF to continue to improve the lives and futures of people touched by Parkinson’s. This fundraising amount is nothing to fear. With the support of family, friends and PDF you have the ability to reach and even surpass the $2,000 goal!

Remember: The Cure Begins with YOU!

Notes from a Day Spent Listening to Scientists Judging PDF Research Applications

Last Friday, March 16, was the occasion for reviewing applications for PDF’s International Research Grants program and Research Fellowships programs. Before us were some 30 proposals from some of the best young (and not-so-young) scientific investigators in the world.

As I sat there in the meeting as an observer, listening to the members of our scientific review committee as they made their comments and pronounced their judgments, I found myself scribbling notes on what seemed to be the principles on which they were basing their decisions. What they were saying, it seemed to me, said a lot about how we go about choosing the projects we will fund. Here are some of the ideas I picked up:

Relevance to Parkinson’s Disease. Every proposal that we fund – without exception -- must show promise in its potential to advance our understanding of PD, or charting the path to its cure.

New Ideas, New Investigators. To maximize the leverage of the program, successful applications will be one of two kinds. One is the innovative pilot project that shows promise for leading to a larger-scale endeavor that will be eligible for funding from the NIH (drawing on PDF’s “leverage” function). The other is the Parkinson’s-related application from an exemplary scientist whose past contributions have been largely in areas other than Parkinson’s – and who could be inspired by means of the PDF grant to turn his or her attention to PD.

Demonstrating Results.
The renewal of an earlier award to the same scientist depends on the investigator’s ability to show “significant progress” since the first grant – that is, you don’t get a second grant if you can’t show that you used the first grant well.

Establishing Credibility. To make it through the grant review process, good ideas aren’t enough; the applicant needs to be able to demonstrate – both in his/her personal accomplishments and in the reputation of the lab in which the work will be done – a stellar track record in producing innovative and useful science.

Including Advocates in the Research Process.
The meeting included three members of Parkinson’s Advocates in Research (PAIR), a PDF program in which lay leaders in the PD community are prepared to take on a variety of roles advocating for clinical research. An example of their contribution came early in the day, when one of these advocates raised a question about the validity of animal models in Parkinson’s research, which generated a spirited exchange among the scientist members of the committee.

I hope you are as impressed as I am with this little vignette of PDF’s research culture!

I conclude with a quotation from a memo on the mission of the program from Dr. James Beck, who is our Director of Research Programs at PDF and staffs the grants review committee. In thanking the scientists in the room, he said, in effect: “The two groups that will benefit most from your decisions today are not even here. One is the world of talented young scientists whose work will be made possible through your efforts. The other is the community of almost one million in the U.S. who live with Parkinson’s. In behalf of these two communities, PDF thanks you!”

Amen, James.

Friday, March 16, 2012

Grant Review at PDF

Today is a big day for many at PDF — it is our annual grant review. Of course, those most affected by what goes on in the big conference room will not be there. There are two groups who depend on tomorrow's outcome: the scientists who have given us their best ideas for our scientific advisors to judge and the people who live every day with Parkinson's and are counting on these great ideas to make a difference in their disease and their lives. I am confident that the team of scientists helping — members of our scientific advisory board and other experts from around the country — will select only the best science today.

All in all, nearly 25 reviewers have pitched in their time to help whittle down the 200 letters of intent to 63 full grant proposals to, now, only the top 20 or so grants. Because of time, it is these top 20 applications that will actually get discussed.

And for the second time, PDF is doing what no other PD organization in America is doing—making certain those people living with PD have a literal seat at the table and participate in the decision making process. These people, PDF's Research Advocates, part of the Parkinson's Advocates in Research program, insure what we will fund is not just the best for science's sake but for the sake of people too. Something in which we, as an organization, take great pride and you as a community should too.

Tomorrow would be difficult to organize without the obvious help of my colleagues at PDF. But equally important is the help from everyone who will not be joining me in the room — you, the Parkinson’s Community. Your generous support of allows us to do what we do every day: support research and ideas that will improve the lives and futures of people touched by Parkinson’s. Our research program would simply not be without you. Thank you.

Friday, March 2, 2012

Understanding the FDA Committee’s Decision to Recommend Approval for Neurogenic Orthostatic Hypotension Drug

From James Beck, Ph.D., Director of Research Programs

You may have read the recent news about the small pharmaceutical company,Chelsea Therapeutics, and their drug Northera™ (droxidopa). Chelsea recently received the recommendation of a FDA advisory committee to approve droxidopa as a treatment for neurogenic orthostatic hypotension or NOH for short. This recommendation was a big surprise since FDA documents released ahead of the committee meeting showed that agency staff members were clearly against approval; nevertheless, this approval recommendation will strongly influence the final decision expected to be made later this month.

What is NOH?
Normally, blood pressure increases as you move from a lying down to a standing position. This increase is necessary in order to maintain adequate blood flow to the brain. Not enough blood (or the oxygen it carries) results in a host of unpleasant issues including light headedness, dizziness, fainting and falls. Your nervous system automatically senses when you stand and then causes this pressure change. For some, the neural response fails to work and they develop precipitously low blood pressure when they stand, hence the name neurogenic (neural origin) orthostatic (standing) hypotension (low blood pressure). There are reports that about 40 percent of people living with Parkinson’s disease have some degree of orthostatic hypotension that is almost always neurogenic in origin.

How might droxidopa treat NOH?
Just like levodopa is the precursor of dopamine for Parkinson’s disease, droxidopa is the precursor of the norepinephrine neurotransmitter for NOH. Norepinephrine is used by the peripheral nervous system to help regulate blood pressure, generally more norepinephrine leads to a higher blood pressure. These levels fluctuate as you move about your day. Therefore, the concept behind droxidopa is that the peripheral nerve cells that regulate blood pressure are able to convert droxidopa into norepinephrine and provide an increased blood pressure. Several studies done in people with multiple system atrophy and peripheral autonomic failure (two diseases that can lead to NOH) have shown promising results. Currently, Chelsea Therapeutics is also conducting a fairly large (200 participants) study of droxidopa in PD. Preliminary results from that have been encouraging as well.

The problem of current therapies
There is currently only one FDA approved drug, midodrine, that is used to treat orthostatic hypotension. Because of the rare nature of NOH, midodrine was approved as an orphan drug before large, key studies showing efficacy were performed. (Because orphan drugs are therapies that target rare diseases, they get special treatment from FDA.) However, for midodrine, those studies were not done as required. Now the FDA is pressuring the manufacturer of midodrine to do the studies or pull the drug off the market.

Why would the FDA be against droxidopa?
Even though droxidopa has shown evidence of helping with NOH, the clinical studies to demonstrate the efficacy of this drug are far from clear-cut. The issues are outlined in the FDA Advisory Committee materials. For instance, in one study, the droxidopa demonstrated a strong, initial increase in blood pressure, but the effect slowly declined over a period of weeks. The therapeutic effect only returned after people were told they were on the drug and advised to stay on it during an open label part of the trial. Despite the variable effect of droxidopa on blood pressure, participants in the study kept diaries that consistently indicated a benefit of the drug. Besides issues of efficacy, there are concerns that long-term use may cause problems because of drug toxicity and that contaminants in the manufacturing process may also be dangerous.

So why did the committee recommend droxidopa?

Others who observed the hearings have suggested that, ultimately, NOH is a serious unmet medical need worthy of its orphan drug status. Moreover, several people who are affected by NOH made compelling remarks that provided a human face and story to the effects of this condition. Even though those people were reimbursed by Chelsea for their travel, I think it helps demonstrate the importance of people living with a disease being able to speak up for their needs. And all the more reason that PDF is behind its Parkinson's Advocates in Research program to help empower people to do just that.

Droxidopa is still far from being approved, but I think the recent vote demonstrates the importance of having people living with a disease, whether it be NOH or Parkinson's, involved in all stages of the clinical research process to insure that their voice is heard.