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 16, 2012
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 ProgramsYou 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.
Tuesday, February 7, 2012
What Can Alzheimer’s Tell us About Parkinson’s?
From James Beck, Ph.D., Director of Research ProgramsUnderstanding how Parkinson’s disease starts and how it progresses remain two of the key unanswered questions about the disease. This is why a recent story in the New York Times caught my eye.
The article described how two independent groups of scientists found evidence that Alzheimer’s disease may spread from brain cell to brain cell much like a virus. These scientists engineered mice to make a human form of a protein called tau in only one small part of the brain. Clumps of the tau protein are a hallmark of Alzheimer’s disease. Like the rubble of demolished buildings, these clumps are all that are left of brains cells that have slowly died because of Alzheimer’s.
Proteins and Clumps
Proteins in the body are folded into special shapes … almost like pieces of origami. Some proteins, if they are not folded correctly to begin with or become “crumpled” due to some cellular problem, will stick together in clumps. Similar to how clumps of the protein tau are a hallmark of Alzheimer’s, clumps of a protein called alpha-synuclein are the hallmark by which Parkinson’s disease is diagnosed. These alpha-synuclein clumps are called Lewy bodies.
Parkinson’s and the Contagion Theory
Parkinson’s researchers have long noted what appears to be the spread of Lewy bodies from one brain region to another. Researchers observed possible clinical evidence that Parkinson’s could “spread” from one cell to another, when fetal cells were transplanted into the brains of people with Parkinson’s disease. The transplants subsequently developed Lewy bodies. That is, something about a Parkinson’s brain made genetically unrelated cells that were only a few years old develop the signs of Parkinson’s disease.
What came out of these observations was the controversial idea that Parkinson’s, once started, might be transmissible from brain cell to brain cell.
Several recent scientific papers are beginning to lend credence to this idea:
- Last year, Dr. Patrik Brundin’s research team in Lund, Sweden published a paper demonstrating that mis-folded alpha-synuclein (the basis of Lewy bodies) can spread from cell to cell in culture and could also spread in the brain of a mouse.
- More recently, Dr. Virginia Lee's team from the University of Pennsylvania published similar findings this past November.
- At a recent New York Academy of Sciences meeting on Parkinson’s disease, unpublished data was presented that also demonstrated how a single injection of alpha-synuclein clumps into a mouse brain could lead to the widespread formation of Lewy bodies in many different parts of the brain.
What Does It Mean?
Well it brings hope that a Parkinson’s therapy might be found in one of two ways: using special antibodies that target alpha-synuclein or a vaccine which primes the immune system to also target alpha-synuclein.
Either approach may not only halt the spread of Parkinson’s disease but maybe could also prevent it.
Your Feedback
What do you think about the recent Alzheimer’s study and how it relates to Parkinson’s? As always, please share your comments and I will do my best to respond.
Friday, January 20, 2012
Bruce and Bob
“It’s incredible how far we have come in 12 years!”
The venue for this quotation was the cavernous Paramount Theater in Asbury Park, New Jersey at 2 AM last Sunday morning, at the conclusion of a six-hour rock concert before a capacity crowd.
The event was the latest production of Light of Day Foundation, the brainchild of Bob Benjamin, a former music publicist and producer. Light of Day’s purpose is to raise money for scientific research on Parkinson’s disease (PD) and other neurological disorders through fundraising concerts – initially on the Jersey Shore, and more recently through tours in Europe and elsewhere.
And the speaker was Bruce Springsteen.
The legendary rock musician’s loyal support of Light of Day since the beginning stems from his long-time friendship with Bob, who has lived with PD bravely for many years.
Bruce is not the only musician to work with Light of Day; he is just the most famous among many. In all, more than a dozen musicians – singers, guitarists, drummers and others – have served on Bob’s board of directors, performed at his concerts, raised money for PDF and his other causes … and even performed with Bob in a just-released documentary film on the organization and its inspiration. One of these – Joe Grushecky, a guitarist – was up on stage throughout Springsteen’s marathon (two-hour!) final set.
They love their work. They adore Bob, their inspiration. And they are passionate about raising money to understand Parkinson’s, and stop the disease in its tracks.
To this writer, in his 16th year running PDF, the experience of working with Light of Day and its gifted, creative leadership is a labor of love, but it is more than this. It is symbolic of four characteristics that drive the success of a cause like Parkinson’s research.
One is the leadership of a celebrity individual – a person like Springsteen or Muhammad Ali – who attracts attention, wins hearts and fills halls.
The second is the existence of a powerful message – supporting science of the highest caliber, to understand a disease and chart the path to its cure.
The third is the presence of an organization like PDF that can parlay money and celebrity into solid work and accomplishment.
And the fourth – perhaps the most important – is the passion and authenticity of one or a few ordinary men or women who just happen to have an intimate connection with the disease and the quiet compulsion to do something about it. On the street or in a train, you might pass by a guy like Bob Benjamin without a second look. On the stage, as he chats with Bruce Springsteen and as you look around you at the evidence of his incredible accomplishments, you would never forget him.
The moral of this is simple: each one of us – like Bob – can make a difference.
The venue for this quotation was the cavernous Paramount Theater in Asbury Park, New Jersey at 2 AM last Sunday morning, at the conclusion of a six-hour rock concert before a capacity crowd.
The event was the latest production of Light of Day Foundation, the brainchild of Bob Benjamin, a former music publicist and producer. Light of Day’s purpose is to raise money for scientific research on Parkinson’s disease (PD) and other neurological disorders through fundraising concerts – initially on the Jersey Shore, and more recently through tours in Europe and elsewhere.
And the speaker was Bruce Springsteen.
The legendary rock musician’s loyal support of Light of Day since the beginning stems from his long-time friendship with Bob, who has lived with PD bravely for many years.
Bruce is not the only musician to work with Light of Day; he is just the most famous among many. In all, more than a dozen musicians – singers, guitarists, drummers and others – have served on Bob’s board of directors, performed at his concerts, raised money for PDF and his other causes … and even performed with Bob in a just-released documentary film on the organization and its inspiration. One of these – Joe Grushecky, a guitarist – was up on stage throughout Springsteen’s marathon (two-hour!) final set.
They love their work. They adore Bob, their inspiration. And they are passionate about raising money to understand Parkinson’s, and stop the disease in its tracks.
To this writer, in his 16th year running PDF, the experience of working with Light of Day and its gifted, creative leadership is a labor of love, but it is more than this. It is symbolic of four characteristics that drive the success of a cause like Parkinson’s research.
One is the leadership of a celebrity individual – a person like Springsteen or Muhammad Ali – who attracts attention, wins hearts and fills halls.
The second is the existence of a powerful message – supporting science of the highest caliber, to understand a disease and chart the path to its cure.
The third is the presence of an organization like PDF that can parlay money and celebrity into solid work and accomplishment.
And the fourth – perhaps the most important – is the passion and authenticity of one or a few ordinary men or women who just happen to have an intimate connection with the disease and the quiet compulsion to do something about it. On the street or in a train, you might pass by a guy like Bob Benjamin without a second look. On the stage, as he chats with Bruce Springsteen and as you look around you at the evidence of his incredible accomplishments, you would never forget him.
The moral of this is simple: each one of us – like Bob – can make a difference.
Wednesday, August 17, 2011
A Real Pro: Lenny Zwick’s Pro-Am Raises $55,000
From the sounds of laughter and the jokes among golfers at the Fifth Annual Zwick Pro-Am last Wednesday, it became immediately apparent to any observer that Lenny Zwick had left behind an inspiring legacy. Held at the Farms Country Club in Wallingford, CT, the Pro-Am benefited PDF’s research programs with more than $55,000 and honored the late Mr. Zwick, who lived with Parkinson’s for 25 years.
Photo credit: http://farmsccsuperintendent.blogspot.com
Photo credit: http://farmsccsuperintendent.blogspot.comThe crowd included more than one hundred of the friends, family and fellow club members who knew him well. Club pro and close family friend, Jim Hanlon served as the master of ceremonies and evoked Mr. Zwick’s spirit to bring the crowd to its feet more than a few times. Mr. Zwick’s family, including daughter Stacey, wife Susan and granddaughter Bianca, were glowing with pride and gratitude during the Pro-Am.
Mrs. Zwick reminded everyone of just the kind of man her husband was, “Lenny was a fighter. Even when the Parkinson’s was really taking hold and he played a bad game he would come home and say 'Ah! It was that [darn] shot.' It was never ‘that [darn] Parkinson’s!'"
Mr. Hanlon acknowledged the dedicated team that helps put this successful Pro-Am together each year, including the Farms Country Club and Dave Melillo, remarking, “The most amazing thing has been that we only had to ask for help once and that was the first year…ever since then every year I get a call asking, ‘What can I do?’”
At PDF, we are grateful for this dedication. We often say that our Champions – grassroots fundraisers like this group of Zwick family members and friends – keep us inspired daily. There was no exception to this rule at the Pro-Am, which has now raised over $250,000 over the course of five years to support PDF’s research programs.
Mrs. Zwick reminded everyone of just the kind of man her husband was, “Lenny was a fighter. Even when the Parkinson’s was really taking hold and he played a bad game he would come home and say 'Ah! It was that [darn] shot.' It was never ‘that [darn] Parkinson’s!'"
Mr. Hanlon acknowledged the dedicated team that helps put this successful Pro-Am together each year, including the Farms Country Club and Dave Melillo, remarking, “The most amazing thing has been that we only had to ask for help once and that was the first year…ever since then every year I get a call asking, ‘What can I do?’”
At PDF, we are grateful for this dedication. We often say that our Champions – grassroots fundraisers like this group of Zwick family members and friends – keep us inspired daily. There was no exception to this rule at the Pro-Am, which has now raised over $250,000 over the course of five years to support PDF’s research programs.
Read about other inspiring PDF Champions on our website. To find out how you can help by organizing your own Champions event, contact us at info@pdf.org.
Wednesday, June 29, 2011
Thank You, Brilliant Challenge Raises $400,000
The Parkinson's Disease Foundation (PDF) is very excited to announce that it has raised $400,000 through the Irving G. Brilliant Parkinson’s Research Challenge, all of which will be directed towards Parkinson’s research programs.
It was only because of your support - our donors and friends to whom the fight against Parkinson's is a priority - that we were able to surpass our original goal. How did we do it? Here's our story behind the challenge.
March, The Challenge Begins!
As you may remember, on March 30, PDF announced the launch of the challenge, by Mr. Brilliant, a businessman from Brooklyn, New York who lives Parkinson’s. Mr. Brilliant offered to match all gifts made during April, for up to $100,000.
April, The Challenge Doubles
On April 28, we announced with excitement that due to a generous gift by The Light of Day Foundation and Mr. Bob Benjamin, PDF far surpassed the original challenge goal.
Upon hearing about Mr. Benjamin's gift, Mr. Brilliant DOUBLED his challenge, promising to match any gifts for up to $200,000 made by Thursday, June 30.
May and June, More Than 1,000 Supporters
The community responded with enthusiasm to the doubling of the challenge. To date, more than 1,000 people have donated well over $200,000.
Mr. Brilliant is matching the $200,000 with his own generous donation, meaning a total of $400,000 will be directed towards Parkinson's research.
$400,000 for Parkinson’s Research
Thank you all for your generosity. As my colleague Eddie Pelto discussed several months ago, the fact that many donations were of modest size shows the impact of our grassroots community.
Stay tuned to our website next week, when we’ll announce our fiscal year 2012 research grants, so you can see the research your funds will support. As always this research is designed to bring us closer to understanding the cause(s) of and a cure for Parkinson's.
Sincerely,
Robin Elliott
It was only because of your support - our donors and friends to whom the fight against Parkinson's is a priority - that we were able to surpass our original goal. How did we do it? Here's our story behind the challenge.
March, The Challenge Begins!
As you may remember, on March 30, PDF announced the launch of the challenge, by Mr. Brilliant, a businessman from Brooklyn, New York who lives Parkinson’s. Mr. Brilliant offered to match all gifts made during April, for up to $100,000.
April, The Challenge Doubles
On April 28, we announced with excitement that due to a generous gift by The Light of Day Foundation and Mr. Bob Benjamin, PDF far surpassed the original challenge goal.
Upon hearing about Mr. Benjamin's gift, Mr. Brilliant DOUBLED his challenge, promising to match any gifts for up to $200,000 made by Thursday, June 30.
May and June, More Than 1,000 Supporters
The community responded with enthusiasm to the doubling of the challenge. To date, more than 1,000 people have donated well over $200,000.
Mr. Brilliant is matching the $200,000 with his own generous donation, meaning a total of $400,000 will be directed towards Parkinson's research.
$400,000 for Parkinson’s Research
Thank you all for your generosity. As my colleague Eddie Pelto discussed several months ago, the fact that many donations were of modest size shows the impact of our grassroots community.
Stay tuned to our website next week, when we’ll announce our fiscal year 2012 research grants, so you can see the research your funds will support. As always this research is designed to bring us closer to understanding the cause(s) of and a cure for Parkinson's.
Sincerely,
Robin Elliott
Thursday, April 28, 2011
Light of Day Foundation Donates $60,000, Irving G. Brilliant Doubles Challenge
Bob Benjamin, a leader in Parkinson’s fundraising and head of the Light of Day Foundation, contacted PDF this week to let us know that the foundation is donating $60,000 this month – raised from its Light of Day Concerts – to the Irving G. Brilliant Parkinson's Research Challenge.
While this brought PDF well past its initial goal of $100,000 (all of which Mr. Brilliant promised to match), the news got even better.
Upon hearing about Mr. Benjamin's gift, Mr. Brilliant DOUBLED his challenge. If PDF reaches $200,000 by Thursday, June 30, Mr. Brilliant will match every single gift to benefit Parkinson’s research.
Stories of Our Donors, NBC Nightly News Clip
Irving G. Brilliant, J.D.
Mr. Brilliant is a long-time resident of Brooklyn, NY, who was diagnosed with Parkinson's in 2003. A Harvard-trained attorney, Mr. Brilliant spent most of his career on Wall Street. At a young age, he sat on the US legal team at the Nuremberg trials prosecuting leaders of Nazi Germany. With his challenge gift, he is hoping to spur philanthropic interest in scientific research seeking the cause(s) of and a cure for the disease.
The Light of Day Foundation and Bob Benjamin
Mr. Benjamin was diagnosed with Parkinson’s in 1996 at the age of 38, and shortly thereafter, founded Light of Day Foundation, which has held concerts all over the world to raise funds for Parkinson’s. Performers have included Bruce Springsteen. The concerts have raised over $1 million to benefit Parkinson’s organizations. Last year, PDF presented Mr. Benjamin and The Light of Day Foundation with the Page and William Black Humanitarian Award.
Mr. Benjamin was recently profiled on NBC Nightly News for his work in the Parkinson’s community, a clip you can view below. In his interview, Mr. Benjamin states,
“I don’t look at what I can’t do, I look at what I can do.”
Certainly, he has given much to the Parkinson’s community.
We are grateful for not only his support, but for yours in helping us to reach the challenge goal to fund much needed Parkinson’s research. The efforts made this April by Mr. Benjamin, Mr. Brilliant and all of you around the country are truly inspirational.
We encourage you to watch this video below and invite your feedback on the fundraising challenge, the gifts by Mr. Benjamin and Mr. Brilliant and your experiences this April in raising awareness.
While this brought PDF well past its initial goal of $100,000 (all of which Mr. Brilliant promised to match), the news got even better.
Upon hearing about Mr. Benjamin's gift, Mr. Brilliant DOUBLED his challenge. If PDF reaches $200,000 by Thursday, June 30, Mr. Brilliant will match every single gift to benefit Parkinson’s research.
Stories of Our Donors, NBC Nightly News Clip
Irving G. Brilliant, J.D.
Mr. Brilliant is a long-time resident of Brooklyn, NY, who was diagnosed with Parkinson's in 2003. A Harvard-trained attorney, Mr. Brilliant spent most of his career on Wall Street. At a young age, he sat on the US legal team at the Nuremberg trials prosecuting leaders of Nazi Germany. With his challenge gift, he is hoping to spur philanthropic interest in scientific research seeking the cause(s) of and a cure for the disease.
The Light of Day Foundation and Bob Benjamin
Mr. Benjamin was diagnosed with Parkinson’s in 1996 at the age of 38, and shortly thereafter, founded Light of Day Foundation, which has held concerts all over the world to raise funds for Parkinson’s. Performers have included Bruce Springsteen. The concerts have raised over $1 million to benefit Parkinson’s organizations. Last year, PDF presented Mr. Benjamin and The Light of Day Foundation with the Page and William Black Humanitarian Award.
Mr. Benjamin was recently profiled on NBC Nightly News for his work in the Parkinson’s community, a clip you can view below. In his interview, Mr. Benjamin states,
“I don’t look at what I can’t do, I look at what I can do.”
Certainly, he has given much to the Parkinson’s community.
We are grateful for not only his support, but for yours in helping us to reach the challenge goal to fund much needed Parkinson’s research. The efforts made this April by Mr. Benjamin, Mr. Brilliant and all of you around the country are truly inspirational.
We encourage you to watch this video below and invite your feedback on the fundraising challenge, the gifts by Mr. Benjamin and Mr. Brilliant and your experiences this April in raising awareness.
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